Ongoing | Cancer Accelerated Approvals
This listing includes accelerated approvals (AAs) for malignant hematology and oncology indications that have postmarketing requirement(s) for ongoing clinical trial(s) to verify clinical benefit. Please refer to Drugs@FDA for the latest approvals and prescribing information for specific products. Visit the verified and withdrawn AA indication pages for more information and the Postmarket Requirements and Commitments page for the status of specific requirements.
Indications may remain on this page until FDA updates product labeling or publishes a Federal Register notice regarding a change in status.
| Drug Name | Accelerated Approval (AA) Indication | AA Date | AA Post-Marketing Requirement | Original Projected Completion 1 |
|---|---|---|---|---|
| Elrexfio (elranatamab-bcmm) | Adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody. | 8/14/2023 |
4476-1: Complete a randomized clinical trial in patients with relapsed or refractory multiple myeloma. Patients should be randomized to receive an elranatamab-based regimen compared to standard therapy for relapsed or refractory multiple myeloma. The primary endpoint should be progression-free survival and secondary endpoints should include overall survival and overall response rate. The trial should enroll sufficient numbers of older patients (ages 65-74 years and 75 years and above) to enable an evaluation of elranatamab in a study population that reflects the age of the U.S. population of patients with multiple myeloma. |
9/30/2026 |
| Talvey (talquetamab-tgvs) | Adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody | 8/9/2023 |
4473-1: Conduct a randomized clinical trial that evaluates the clinical efficacy and safety of talquetamab in patients with relapsed or refractory multiple myeloma. Patients should be randomized to receive a talquetamab-based regimen compared to standard therapy for relapsed or refractory multiple myeloma. The primary endpoint should be progression-free survival and secondary endpoints should include overall survival and overall response rate. The trial should enroll sufficient numbers of racial and ethnic minority patients and older patients (ages 65-74 years and 75 years and above) to enable an evaluation of talquetamab in a study population that reflects the U.S. population of patients with multiple myeloma. |
12/31/2026 |
| Columvi (glofitamab-gxbm) | Relapsed or refractory diffuse large B-cell lymphoma, not otherwise specified or large B-cell lymphoma arising from follicular lymphoma, after two or more lines of systemic therapy | 6/15/2023 | 4464-1 Complete a randomized clinical trial that evaluates the clinical benefit of glofitamab in patients with diffuse large B-cell lymphoma. The trial should compare glofitamab in combination with gemcitabine and oxaliplatin (GemOx) to rituximab in combination with GemOx for patients with relapsed or refractory diffuse large B-cell lymphoma. The primary endpoint should be overall survival with secondary endpoints that include progression-free survival and response rate. | 9/30/2024 |
| Epkinly (epcoritamab-bysp) | Adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from indolent lymphoma, and high-grade B-cell lymphoma after two or more lines of systemic therapy. | 5/19/2023 | 4435-1: Complete a randomized clinical trial in patients with relapsed or refractory large B-cell lymphoma. The trial should compare epcoritamab monotherapy to an investigator’s choice of standard therapies for patients with relapsed or refractory large B-cell lymphoma. The primary endpoint should be OS with secondary endpoints that include PFS and response rate. | 6/30/2025 |
| Keytruda (pembrolizumab) | In combination with enfortumab vedotin-ejfv for the treatment of adult patients with locally advanced or metastatic urothelial cancer who are not eligible for cisplatin-containing chemotherapy | 4/3/2023 | 4429-1: Conduct clinical trial EV-302, “Enfortumab Vedotin and Pembrolizumab vs. Chemotherapy Alone in Untreated Locally Advanced or Metastatic Urothelial Cancer” and submit the final OS, PFS, ORR and DoR results, intended to verify and describe the clinical benefit of enfortumab vedotin in combination with pembrolizumab in patients with untreated locally advanced or metastatic urothelial cancer. | 9/30/2025 |
| Padcev (enfortumab vedotin-efjv) | In combination with pembrolizumab for the treatment of adult patients with locally advanced or metastatic urothelial cancer who are not eligible for cisplatin-containing chemotherapy | 4/3/2023 | 4428-1: Conduct clinical trial EV-302, “Enfortumab Vedotin and Pembrolizumab vs. Chemotherapy Alone in Untreated Locally Advanced or Metastatic Urothelial Cancer” and submit the final OS, PFS, ORR and DoR results, intended to verify and describe the clinical benefit of enfortumab vedotin in combination with pembrolizumab in patients with untreated locally advanced or metastatic urothelial cancer. | 9/30/2025 |
| Zynyz (retifanlimab-dlwr) | Adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC). | 3/22/2023 | 4412-1: Conduct a multicenter clinical trial intended to confirm the clinical benefit of retifanlimab-dlwr in patients with metastatic or recurrent locally advanced MCC who have not received prior systemic therapies for metastatic or recurrent locally advanced MCC. The trial will enroll at least 100 patients to be followed for a minimum of 12 months to establish the ORR and characterize the DOR. Include an analysis of OS, when 70% of patients have died, or all patients have been followed for at least three years. | 3/31/2025 |
| Jaypirca (pirtobrutinib) | Adult patients with relapsed or refractory mantle cel lymphoma (MCL) after at least two lines of systemic therapy, including a BTK inhibitor. |
1/27/2023 | 4389-1: Complete a randomized clinical trial to obtain data on the clinical efficacy and safety of pirtobrutinib in patients with MCL. The trial should compare pirtobrutinib monotherapy to an investigator’s choice of approved BTK inhibitors in patients with MCL. The primary endpoint should be PFS as assessed by an independent review committee, with secondary endpoints that include OS and objective response rate. The trial should enroll a sufficiently representative study population to reflect the racial and ethnic diversity of the U.S. patient population with MCL and allow for interpretation of the results in these patient populations. | 12/31/2026 |
| Tukysa (tucatinib) | In combination with trastuzumab for the treatment of adult patients with RAS wild-type, HER2-positive, unresectable or metastatic colorectal cancer that has progressed following treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy | 1/19/2023 | 4388-1: Conduct a randomized clinical trial to obtain data on the clinical efficacy of tucatinib for patients with RAS wild type, HER2-positive, unresectable or metastatic colorectal carcinoma. The trial should compare tucatinib in combination with trastuzumab with the standard of care in patients with RAS wild type, HER2-positive, unresectable or metastatic colorectal carcinoma. The primary endpoint should be PFS per blinded assessment or OS. The trial should enroll a sufficiently representative study population to reflect the racial and ethnic diversity of the U.S. patient population with RAS wild type, HER2-positive, unresectable or metastatic colorectal carcinoma and allow for interpretation of the results across this representative study population. | 04/30/2026 |
| Lunsumio (mosunetuzumab-axgb) | Adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy. |
12/22/2022 | 4375-1: Conduct a randomized clinical trial in patients with relapsed or refractory follicular lymphoma (FL), with patients randomized to receive mosunetuzumab in combination with lenalidomide or rituximab in combination with lenalidomide. The primary endpoint should be PFS, with secondary endpoints that include RR and OS. The trial should enroll a sufficiently representative study population to reflect the racial and ethnic diversity of the U.S. patient population with FL and allow for interpretation of the results in these patient populations. | 12/31/2025 |
| Krazati (adagrasib) | Adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy. | 12/12/2022 | 4378-1: Conduct a randomized comparative clinical trial of adagrasib in adult patients with KRAS G12C mutated, locally advanced or metastatic NSCLC who have received at least one prior systemic therapy, to obtain OS, PFS, ORR, and DOR. This data may be obtained from the ongoing clinical trial entitled, “A Randomized Phase 3 Study of MRTX849 versus Docetaxel in Patients with Previously Treated Non-Small Cell Lung Cancer with KRAS G12C Mutation.” | 12/31/2025 |
| Elahere (mirvetuximab soravtansine-gynx) | Adult patients with folate receptor-alpha (FRα) positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens. Select patients for therapy based on an FDA-approved test | 11/14/2022 | 4347-1: Conduct the clinical trial IMGN853-0416 (MIRASOL) titled “A randomized, open-label, Phase 3 study of Mirvetuximab soravtansine vs. Investigator’s choice chemotherapy in platinum-resistant, advanced, high-grade epithelial ovarian, primary peritoneal, or fallopian tube cancers with high folate receptor-alpha expression” and provide the final PFS, ORR, and OS analyses to obtain data on the clinical efficacy of mirvetuximab soravtansine for patients with platinum-resistant ovarian cancer. | 12/31/2024 |
| Tecvayli (teclistamab-cqyv) | Adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody | 10/25/2022 | 4334-1: Conduct a randomized clinical trial in patients with relapsed or refractory multiple myeloma. The trial should enroll sufficient numbers of racial and ethnic minority patients and older patients (ages 65-74 and 75 and above) to enable an evaluation of teclistamab in a study population that better reflects the U.S. population of patients with multiple myeloma. Patients should be randomized to receive a teclistamab-based regimen compared to standard therapy for relapsed or refractory multiple myeloma. The primary endpoint should be PFS and secondary endpoints should include OS, ORR, and DOR. | 3/31/2026 |
| Lytgobi (futibatinib) | Adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements | 9/30/2022 | 4345-1: Conduct a randomized clinical trial comparing dosages of futibatinib 16 mg and 20 mg once daily to verify and describe the clinical benefit of futibatinib in patients with advanced or metastatic cholangiocarcinoma harboring an FGFR2 gene fusion or other rearrangement. The ORR and DOR should be assessed by a blinded independent review. The study should also evaluate other clinical outcomes that denote clinical benefit, such as PROs. This study should enroll a minimum of 120 patients and all responders should have a minimum of 6 months from the date of initial response (or until disease progression, whichever comes first). Ensure that racial and ethnic minorities are adequately represented in the trial population, at a minimum, proportional to the prevalence of FGFR2 alterations in these subgroups in the US population. | 10/31/2027 |
| Retevmo (selpercatinib) | Adult patients with locally advanced or metastatic solid tumors with a RET gene fusion that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options | 9/21/2022 | 4342-1: Complete clinical trial(s) to obtain data on the clinical efficacy of selpercatinib through more precise estimation of the ORR and mature response duration per independent review assessment, in at least 60 patients with locally advanced or metastatic RET-fusion positive solid tumors other than NSCLC and thyroid cancer, who have progressed on prior systemic treatment or have no satisfactory alternative treatment options. A sufficient number of patients with tumor types for which responses require additional characterization (e.g., colorectal cancer, esophagogastric cancer, and glioma) will be evaluated. ORR and DOR will be assessed by independent central review and all responding patients will be followed for at least 12 months following the onset of response or until disease progression or death or early treatment discontinuation, whichever comes first. Include available data regarding RET fusion partners and co-occurring genetic alterations for all patients. | 12/31/2025 |
| Enhertu (Fam-trastuzumab deruxtecan-nxki) | Adult patients with unresectable or metastatic NSCLC whose tumors have an activating HER2 (ERBB2) mutation, as detected by an FDA-approved test, and who have received a prior systemic therapy |
8/11/2022 |
4321-1: Complete a clinical trial to obtain data on the clinical efficacy of fam-trastuzumab deruxtecan-nxki for the treatment of patients with unresectable or metastatic NSCLC whose tumors have an activating HER2 (ERBB2) mutation and have previously received systemic therapy, to provide a more precise estimation of the BICR-assessed ORR and DOR. This report will contain data from patients with NSCLC harboring HER2 mutations and data from at least 102 patients who have received prior systemic therapy, after all responders have been followed for at least 6 months from the date of initial response (or until disease progression, whichever comes first). 4321-2: Conduct a multicenter, randomized clinical trial of fam-trastuzumab deruxtecan-nxki in patients with treatment-naïve, unresectable or metastatic NSCLC whose tumors have an activating HER2 (ERBB2) mutation. The final analysis should include the final PFS and OS results. |
3/31/2024 |
| Tafinlar (dabrafenib) | In combination with trametinib for adult and pediatric patients 6 years of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options. 2 | 6/22/2022 | 4298-1: Conduct a clinical trial(s) in at least 80 patients with solid tumors with a BRAF V600E mutation to verify and describe the clinical benefit of dabrafenib in combination with trametinib, through more precise estimation of the ORR and mature response duration. Include patients with unresectable or metastatic solid tumors with a BRAF V600E mutation from the ongoing trial and from a prospectively conducted trial (which will exclude patients with melanoma, non-small cell lung cancer, anaplastic thyroid cancer, biliary tract cancer, gliomas and colorectal cancer). Follow all patients for at least 6 months from the onset of response to characterize the response rate and duration. | 10/31/2028 |
| Mekinist (trametinib) | In combination with dabrafenib for adult and pediatric patients 6 years of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options 2 | 6/22/2022 | 4297-1: Conduct a clinical trial(s) in at least 80 patients with solid tumors with a BRAF V600E mutation to verify and describe the clinical benefit of trametinib in combination with dabrafenib, through more precise estimation of the ORR and mature response duration. Include patients with unresectable or metastatic solid tumors with a BRAF V600E mutation from the ongoing trial and from a prospectively conducted trial (which will exclude patients with melanoma, non-small cell lung cancer, anaplastic thyroid cancer, biliary tract cancer, gliomas and colorectal cancer). Follow all patients for at least 6 months from the onset of response to characterize the response rate and duration. | 10/31/2028 |
| Kymriah (tisagenlecleucel) | Treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy | 5/27/2022 | Conduct a randomized phase 3 trial in adult patients with r/r FL. Patients will be randomized to tisagenlecleucel or an investigator’s choice of regimens consistent with the standard of care. The primary endpoint will be PFS with secondary endpoints that include OS and ORR. | 9/30/2028 |
| Vijoice (alpelisib) | Treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy | 4/5/2022 | 4260-1: Conduct a multiregional clinical trial to verify and describe the clinical benefit of alpelisib film-coated tablets, through more precise estimation of confirmed ORR and mature response duration per blinded independent review, in adult and pediatric patients 2 years of age and older with PROS, including those with severe manifestations. Responding patients will be followed for at least 36 months from the onset of response, or until disease progression, whichever comes first. Evaluate a sufficient number of patients to characterize response rate and durability of response by PIK3CA mutation type, PROS subtype, and age. | 8/31/2027 |
| Pepaxto (melphalan flufenamide) | In combination with dexamethasone for the treatment of adult patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD-38 directed monoclonal antibody | 2/26/2021 | 4030-1: Submit the final study report and datasets from a randomized phase 3 clinical trial that verifies and describes the clinical benefit of melphalan flufenamide in patients with R/R MM. Patients should be randomized to receive melphalan flufenamide compared to standard therapy for R/R MM. The primary endpoint should be PFS assessed by an Independent Review Committee. The secondary endpoints should include overall response rate and OS. | 2/28/2022 |
| Gavreto (pralsetinib) | Treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate) | 12/1/2020 | 3959-2: Submit the final report of integrated studies and datasets, to verify and further characterize the clinical benefit of pralsetinib for the treatment of patients with RET fusion-positive thyroid cancer who have received radioactive iodine (if appropriate for their tumor histology) to provide a more precise estimation of the BICR-assessed ORR and DOR in at least 50 patients in a variety of histologies after all responding patients have been followed for 12 months following onset of response or until disease progression, whichever comes first. | 6/30/2025 |
| Tivdak (tisotumab vedotin-tftv) | Treatment of adult patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy | 9/20/2021 | 4131-1: Conduct the clinical trial innovaTV 301 titled, “Tisotumab Vedotin versus Chemotherapy in Recurrent or Metastatic Cervical Cancer” and provide the final OS and PFS analyses to describe and verify the clinical benefit of tisotumab vedotin in patients with recurrent or metastatic cervical cancer. | 11/30/2024 |
| Exkivity (mobocertinib) | Treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy | 9/15/2021 | 4148-1: Conduct a multicenter, randomized clinical trial and submit the final PFS results that verify and describe the clinical benefit of mobocertinib in patients with locally advanced or metastatic NSCLC whose tumors harbor EGFR exon 20 insertion mutations | 3/31/2024 |
| Brukinsa (zanubrutinib) | Treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen | 9/14/2021 | 4128-1: Conduct a randomized clinical trial that verifies and describes the clinical benefit of zanubrutinib in patients with relapsed or refractory MZL. The trial should include sufficient representation of racial and ethnic minorities to better reflect the U.S. patient population and allow for interpretation of the results in these patient populations. The primary endpoint should be PFS, with secondary endpoints that include ORR and OS. | 10/31/2028 |
| Jemperli (dostarlimab-gxly) | Treatment for adult patients with mismatch repair deficient (dMMR) recurrent or advanced solid tumors, as determined by an FDA-approved test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options | 8/17/2021 | 4124-1: Conduct a clinical trial evaluating ORR, and DOR, to verify and describe the clinical benefit of Jemperli in patients with dMMR, recurrent or advanced solid tumors, including at least 300 patients across all tumor types, and including a sufficient number of patients and representation of tumor types (other than endometrial and GI tumors). In order to characterize response rate and DOR, patients should be followed for at least 12 months from the onset of response. Submit the datasets with final report. | 10/31/2022 |
| Truseltiq (infigratinib) | Treatment of adult patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangement as detected by an FDA approved test | 5/28/2021 | 4067-1: Submit the final PFS (as assessed by blinded independent review) analysis and interim OS analysis at the time of final PFS analysis, including datasets from a randomized clinical trial comparing infigratinib to chemotherapy to verify and describe the clinical benefit of infigratinib in patients with advanced or metastatic cholangiocarcinoma harboring an FGFR2 gene fusion or other rearrangement. Ensure that racial and ethnic minority subjects are adequately represented in the trial population, at a minimum, proportional to the prevalence of FGFR2 alterations in these subgroups in the US population. | 6/30/2027 |
| Lumakras (sotorasib) | Treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy. | 5/28/2021 | 4071-1: Conduct a multicenter, randomized clinical trial and submit the final PFS results that verify and describe the clinical benefit of sotorasib in patients with locally advanced or metastatic NSCLC with a history of prior systemic therapy for advanced disease and whose tumors harbor KRAS G12C mutation. | 7/30/2022 |
| Rybrevant (amivantamab-vmjw) | Treatment of adult patients with locally advanced or metastatic non small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA approved test, whose disease has progressed on or after platinum based chemotherapy. | 5/21/2021 | 4070-1: Submit the final report, including datasets for PFS , DOR, and OS from a randomized clinical trial to verify and confirm the clinical benefit of amivantamab-vmjw for the treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations. This could be from the ongoing clinical trial entitled, “A Randomized, Open-label Phase 3 Study of Combination Amivantamab and Carboplatin- Pemetrexed Therapy, Compared With Carboplatin-Pemetrexed, in Patients With EGFR Exon 20ins Mutated Locally Advanced or Metastatic Non-Small Cell Lung Cancer.” | 2/28/2023 |
| Keytruda (pembrolizumab) | In combination with trastuzumab and fluoropyrimidine- and platinum-containing chemotherapy, for the first-line treatment of patients with locally advanced unresectable or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. | 5/5/2021 | 4033-1: Submit the final PFS and final OS analyses and datasets for the ongoing clinical trial KEYNOTE-811, “A Phase III, Randomized, Double-blind Trial Comparing Trastuzumab Plus Chemotherapy and Pembrolizumab With Trastuzumab Plus Chemotherapy and Placebo as First-line Treatment in Participants With HER2 Positive Advanced Gastric or Gastroesophageal Junction Adenocarcinoma” to verify and describe the clinical benefit of pembrolizumab with trastuzumab plus chemotherapy for patients with HER2-positive advanced or metastatic gastric or gastroesophageal adenocarcinoma. | 9/30/2024 |
| Zynlonta (loncastuximab tesirine-lpyl) | Treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, DLBCL arising from low grade lymphoma, and high-grade B-cell lymphoma. | 4/23/2021 | 4029-1: Conduct a randomized, phase 3 clinical trial to verify and describe the clinical benefit of loncastuximab tesirine-lpyl in patients with R/R large B-cell lymphoma. The trial should include sufficient numbers of racial and ethnic minority patients to better reflect the U.S. patient population and allow for interpretation of the results in these patient populations. Patients should be randomized to receive loncastuximab tesirine-lpyl plus immunotherapy or immunochemotherapy. The primary endpoint should be PFS , with secondary endpoints that include OS and ORR. | 12/31/2025 |
| Trodelvy (sacituzumab govitecan-hziy) | Treatment of adult patients with locally advanced or metastatic urothelial cancer (mUC) who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor | 4/13/2021 | 4044-1: Submit the final study report and datasets for OS and PFS from trial IMMU-132-13 titled “A Randomized Open-Label Phase III Study of Sacituzumab Govitecan Versus Treatment of Physician's Choice in Subjects With Metastatic or Locally Advanced Unresectable Urothelial Cancer (TROPiCS-04)”, to verify and confirm the clinical benefit of sacituzumab in the urothelial cancer patient population. | 4/30/2025 |
| Yescarta (axicabtagene ciloleucel) | Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy | 3/5/2021 | 1: A randomized phase 3 trial of axicabtagene ciloleucel in patients with relapsed or refractory FL. Patients will be randomized to axicabtagene ciloleucel or to an investigator’s choice of regimens consistent with the standard of care. The primary endpoint will be PFS, with secondary endpoints that include ORR and OS. | 9/30/2027 |
| Tepmetko (tepotinib) | Treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) harboring mesenchymal-epithelial transition (MET) exon 14 skipping alterations. | 2/3/2021 | 4013-1: Submit the final reports including datasets from clinical studies to confirm and further characterize the clinical benefit of tepotinib for the treatment of patients with NSCLC harboring MET exon 14 skipping alterations who are treatment-naïve and who have previously received systemic therapy, by providing a more precise estimation of the blinded independent central review-assessed ORR and DOR. This report will contain data from patients with NSCLC harboring MET exon 14 skipping alterations; data from at least 130 patients who are treatment naïve, after all responders have been followed for at least 12 months from the date of initial response (or until disease progression, whichever comes first); and from at least 143 patients who have been previously treated with systemic therapy, after all responders have been followed for at least 6 months from the date of initial response (or until disease progression, whichever comes first). | 4/30/2023 |
| Darzalex Faspro (daratumumab and hyaluronidase-fihj) | Treatment of patients with light chain (AL) amyloidosis in combination with bortezomib, cyclophosphamide and dexamethasone in newly diagnosed patients. | 1/15/2021 | 3951-1: Submit the final study report and datasets from a randomized clinical trial to verify and further characterize the clinical benefit and safety of daratumumab subcutaneous for the treatment of patients with AL amyloidosis. This submission should include the final analysis and datasets of PFS or OS results. | 6/30/2025 |
| Danyelza (naxitamab-gqgk) | In combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. | 11/25/2020 | 3950-1: Submit the final report, including datasets, from an ongoing multicenter clinical trial to verify and further characterize the clinical benefit of naxitamab for the treatment of patients with R/R neuroblastoma in combination with GM-CSF in bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy; and to provide a more precise estimation of ORR and DOR (according to the revised International Neuroblastoma Response Criteria by blinded independent review). Enroll a minimum of 80 patients with evaluable disease and systematically follow all responding patients for at least 12 months from the onset of response, or until disease progression, whichever comes first. Include subgroup analyses of ORR and DOR by prior anti-GD2 exposure, disease status (refractory vs. relapsed disease), anti-drug antibody (ADA) status (neutralizing antibody [nAb] positive versus nAb negative, ADA positive versus ADA negative), and area of disease involvement (bone marrow vs. bone) in the final report reflecting a sufficient number of patients in relevant subgroups. Derive the clinical data from a minimum of 6 study sites, with at least 5 patients enrolled per site in at least 5 sites. | 9/30/2027 |
| Monjuvi (tafasitamab-cxix) | In combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT). | 7/31/2020 | 3904-1: Submit the final report and datasets from a randomized, Phase 3 clinical trial to verify the clinical benefit of tafasitamab in patients with DLBCL. The trial should include sufficient numbers of racial and ethnic minority patients to better reflect the U.S. patient population and allow for the interpretation of the results in these patient populations. Patients should be randomized to receive immunotherapy and/or chemotherapy with or without tafasitamab and lenalidomide. The primary endpoint should be PFS, with secondary endpoints that include OS and ORR. | 12/31/2025 |
| Tecartus (brexucabtagene autoleucel) | Treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL). | 7/24/2020 | 1: Complete additional follow-up of all 68 subjects treated with brexucabtagene autoleucel in ZUMA-2 Cohort 1 to a minimum of 18 months from the time of first response. Data will continue to be collected according to the ZUMA-2 protocol’s established schedule of assessments. 2: Conduct a study of brexucabtagene autoleucel treatment of subjects with relapsed or refractory mantle cell lymphoma who have not been exposed to a Bruton tyrosine kinase (BTK) inhibitor. A cohort of subjects naïve to BTK inhibitor therapy will be added to the ongoing ZUMA-2 study to fulfill this requirement. Eighty-six subjects will be enrolled. The primary efficacy endpoint will be objective response rate with a supportive efficacy endpoint of duration of response based on a minimum follow-up of 18 months after first objective disease response. |
10/31/2025 |
| Xpovio (selinexor) | Treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma (FL), after at least 2 lines of systemic therapy. | 6/22/2020 | 3866-1: Submit the final report and datasets from a randomized, double-blind, placebo-controlled phase 3 trial that verifies and describes the clinical benefit of selinexor in patients with relapsed or refractory DLBCL. Patients should be randomized to receive chemoimmunotherapy with or without selinexor. The primary endpoint should be PFS , with secondary endpoints that include OS and ORR. | 4/30/2026 |
| Tazverik (tazemetostat) | Treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least 2 prior systemic therapies. Treatment of adult patients with R/R FL who have no satisfactory alternative treatment options. | 6/18/2020 | 3872-1: Submit the final report and datasets from a randomized, phase 3 clinical trial that verifies and describes the clinical benefit of tazemetostat in patients with R/R FL whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test. The trial should include sufficient numbers of racial and ethnic minority patients to better reflect the U.S. patient population and allow for the interpretation of the results in these patient populations. Patients should be randomized to receive immunotherapy with or without tazemetostat. The primary endpoint should be PFS, with secondary endpoints that include OS and ORR. 3872-2: Submit the final report and datasets from a randomized, phase 3 clinical trial that verifies and describes the clinical benefit of tazemetostat in patients with R/R FL whose tumors do not have an EZH2 mutation as detected by an FDA-approved test. The trial should include sufficient numbers of racial and ethnic minority patients to better reflect the U.S. patient population and allow for the interpretation of the results in these patient populations. Patients should be randomized to receive immunotherapy with or without tazemetostat. The primary endpoint should be PFS, with secondary endpoints that include OS and ORR. |
7/31/2025 |
| Keytruda (pembrolizumab) | Treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden high (TMB H) [=10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. | 6/16/2020 | 3871-1: Submit the final report and datasets from clinical trials evaluating ORR and DOR, to verify and describe the clinical benefit of pembrolizumab in adult and pediatric patients with unresectable or metastatic TMB-H [=10 mutations/megabase (mut/Mb)] solid tumors (as determined by an FDA-approved test) that have progressed following prior treatment and who have no satisfactory alternative treatment options. A sufficient number of patients and representation of tumor types (other than lung cancers, MSI-H or dMMR cancers, or melanoma; and including CNS tumors that were determined to be TMB-H based on testing of tissue obtained prior to initiation of temozolomide chemotherapy), and with cancers having a TMB of 10 to <13 mut/Mb, will be evaluated to characterize response and DOR. A minimum of 20 pediatric patients will be studied. ORR and DOR will be assessed by independent central review for patients with cancers having a TMB of =10 mut/Mb, =10 mut/Mb to <13 mut/Mb, and =13 mut/Mb. All responding patients will be followed for at least 12 months from the onset of response. | 12/31/2025 |
| Zepzelca (lurbinectedin) | Treatment of adult patients with metastatic small cell lung cancer (SCLC) with disease progression on or after prior platinum-based chemotherapy. | 6/15/2020 | 3831-1: Submit the final report and datasets for the OS and PFS analysis as determined by an Independent Review Committee from a clinical trial to confirm the clinical benefit of lurbinectedin in SCLC that may inform product labeling. This could be from the Study titled, “Phase III Randomized Clinical Trial of Lurbinectedin (PM01183)/Doxorubicin Versus Cyclophosphamide, Doxorubicin and Vincristine (CAV) or Topotecan as Treatment in Patients With SCLC Who Failed One Prior Platinumcontaining Line (ATLANTIS)”. | 2/28/2021 |
| Rubraca (rucaparib) | Treatment of adult patients with a deleterious BRCA mutation (germline and/or somatic)-associated metastatic castration-resistant prostate cancer (mCRPC) who have been treated with androgen receptor-directed therapy and a taxane-based chemotherapy | 5/15/2020 | 3833-1: Submit the PFS and OS analyses and datasets demonstrating clinical benefit of rucaparib from a phase 3 randomized trial in patients with mCRPC associated with homologous recombination deficiency who have been treated with androgen receptor directed therapy. The results may inform product labeling. | 10/31/2025 |
| Pomalyst (pomalidomide) | Treatment of patients with AIDS-related Kaposi’s Sarcoma after failure of highly active antiretroviral therapy (HAART). | 5/14/2020 | 3855-1: Submit the final report from a clinical trial evaluating ORR , DOR, and safety to verify and describe the clinical benefit of pomalidomide in patients with Kaposi sarcoma who are HIV positive (after failure of HAART) and HIV negative, that may inform product labeling. | 12/31/2027 |
| Pomalyst (pomalidomide) | Treatment of Kaposi’s Sarcoma in patients who are HIV-negative | 5/14/2020 | 3855-1: Submit the final report from a clinical trial evaluating ORR, DOR, and safety to verify and describe the clinical benefit of pomalidomide in patients with Kaposi sarcoma who are HIV positive (after failure of HAART) and HIV negative, that may inform product labeling. | 12/31/2027 |
| Retevmo (selpercatinib) | Adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy | 5/8/2020 | 3829-1: Submit the final report including datasets from a multi-center, randomized trial comparing selpercatinib to physician’s choice of approved therapies in patients with kinase inhibitor-naïve, progressive, advanced or metastatic RET-mutant MTC to confirm clinical benefit of selpercatinib with PFS as a key secondary end point as assessed by blinded independent central review. | 10/31/2026 |
| Retevmo (selpercatinib) | Adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). |
5/8/2020 | 3829-3: Submit a final report including datasets, to verify and further characterize the clinical benefit of selpercatinib for the treatment of patients with RET fusion-positive thyroid cancer who have received radioactive iodine (if appropriate for their tumor histology) to provide a more precise estimation of the blinded independent central review-assessed ORR and DOR in at least 50 patients after all responding patients have been followed for 12 months following onset of response or until disease progression, whichever comes first. | 12/31/2023 |
| Pemazyre (pemigatinib) | Treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. | 4/17/2020 | 3801-1: Submit the results of a randomized trial demonstrating improvement of PFS or OS in patients with unresectable locally advanced or metastatic cholangiocarcinoma with a FGFR2 gene fusion or rearrangement, to confirm clinical benefit of pemigatinib. | 12/31/2026 |
| Yervoy (ipilimumab) | In combination with nivolumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib | 3/10/2020 | 3823-1: Submit the final report demonstrating an improvement in OS from a multicenter, randomized trial to confirm the clinical benefit of nivolumab in combination with ipilimumab over standard therapy in patients with advanced HCC, that may inform product labeling. Submit the datasets with the final report. | 7/31/2024 |
| Opdivo (nivolumab) | In combination with ipilimumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib | 3/10/2020 | 3822-1: Submit the final report demonstrating an improvement in OS from a multicenter, randomized trial to confirm the clinical benefit of nivolumab in combination with ipilimumab over standard therapy in patients with advanced HCC, that may inform product labeling. Submit the datasets with the final report. | 7/31/2024 |
| Tazverik (tazemetostat) | Treatment of adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection. | 1/23/2020 | 3787-1: Submit the final results from a confirmatory randomized trial in patients with epithelioid sarcoma to confirm clinical benefit and provide additional efficacy data that may inform product labeling for tazemetostat. 3787-2: Submit the final report and datasets for the final analysis of ORR and DOR for clinical trial EZH-202 titled, “A Phase II, Multicenter Study of the EZH2 Inhibitor Tazemetostat in Adult Subjects With INI1-Negative Tumors or Relapsed/Refractory Synovial Sarcoma” to verify and confirm clinical benefit of tazemetostat, that may inform product labeling. An additional 25 patients from Cohort 6 beyond those included in the original NDA submission will be evaluated and all responding patients will be followed for at least 12 months from the onset of response. |
9/30/2029 |
| Brukinsa (zanubrutinib) | Treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. | 11/14/2019 | 3735-1: Complete and submit the final results of Trial BGB-3111-306 - the ongoing randomized, Phase 3 clinical trial of BRUKINSA in combination with rituximab versus bendamustine and rituximab in patients with previously untreated MCL. The primary endpoint is PFS as assessed by Independent Review Committee (IRC). OS is a key secondary endpoint. PFS and OS would be analyzed based on superiority testing. Enrollment of approximately 500 patients is expected | 2/28/2027 |
| Rozlytrek (entrectinib) | Treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy. | 8/15/2019 | 3689-2: Submit the final report, including datasets, from ongoing and proposed trials conducted to verify and describe the clinical benefit of entrectinib, through more precise estimation of the ORR and mature response duration per independent review assessment, in adult and pediatric patients 12 years of age and older with solid tumors with a NTRK gene fusion and without a known acquired resistance mutation; are metastatic or would require surgical resection that would result in severe morbidity; and have no satisfactory alternative treatment or that have progressed following treatment. A sufficient number of patients will be evaluated to more precisely characterize response and durability of response for each of the following tumor types: pediatric solid tumors, CRC, CNS cancers, GYN cancers, and melanoma. A minimum of 40 patients with cancers other than pediatric solid tumors, CRC, CNS cancers, GYN cancers, melanoma, STS, non-small cell adenocarcinoma lung cancer, mammary analogue secretory carcinoma, and secretory breast cancer will also be studied. ORR and DOR will be assessed by independent central review and all responding patients will be followed for at least 12 months from the onset of response. 3689-1: Submit the final report, including datasets, from the first 54 patients with NTRK-fusion solid tumors enrolled across the ALKA, STARTRK-1 [NCT02097810], and STARTRK-2 [NCT02568267] studies to verify and describe the clinical benefit and further characterize the DOR in patients who achieved a complete or partial response to entrectinib. All responding patients will be followed for at least 2 years from the onset of response or until disease progression, whichever comes first. DOR will be assessed by independent central review. |
3/31/2027 |
| Balversa (erdafitinib) |
|
4/12/2019 | 3561-1: Submit the analysis, and datasets with the final report demonstrating clinical benefit of erdafitinib in patients with locally advanced and mUC with susceptible FGFR 3 or FGFR 2 genetic alterations from clinical trial BLC3001 entitled; “A Phase 3 Study of Erdafitinib Compared With Vinflunine or Docetaxel or Pembrolizumab in Subjects with Advanced Urothelial Cancer and Selected FGFR Gene Aberrations.” | 10/31/2022 |
| Keytruda (pembrolizumab) | Treatment of adults and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC) | 12/19/2018 | 3546-1: Conduct and submit the results of a multicenter clinical trial to confirm the clinical benefit of pembrolizumab in patients with locally advanced or metastatic MCC who have not received prior systemic therapies for metastatic MCC. The trial will enroll at least 50 patients to be followed for a minimum of 12 months to establish the ORR and characterize the DOR. OS, which is a secondary endpoint, will be followed to maturity until at least 70% of patients have died, or for an additional 2 years beyond the primary data analysis cut-off, to characterize effects on survival. | 12/30/2032 |
| Vitrakvi (Capsules) (larotrectinib) | Treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation; are metastatic or where surgical resection is likely to result in severe morbidity; and have no satisfactory alternative treatments or that have progressed following treatment | 11/26/2018 | 3540-1: Submit the final report, including datasets, from ongoing and proposed trials conducted to verify and describe the clinical benefit of larotrectinib, through more precise estimation of the ORR and mature response duration per independent review assessment, in adult and pediatric patients with solid tumors with a NTRK gene fusion and without a known acquired resistance mutation; are metastatic or where surgical resection is likely to result in severe morbidity; and have no satisfactory alternative treatment or that have progressed following treatment. A sufficient number of patients will be evaluated to characterize response and durability of response for each of the following tumor types: CRC, NSCLC, CNS tumors, and melanoma. A minimum of 40 patients with cancers other than CRC, NSCLC, CNS tumors, melanoma, STS, thyroid cancer, infantile fibrosarcoma, and salivary cancers (e.g., BC, GI stromal tumors, cholangiocarcinoma, biliary tract cancers) will also be studied. ORR and DOR will be assessed by independent central review and all responding patients will be followed for at least 12 months from the onset of response. | 8/31/2025 |
| Keytruda (pembrolizumab) | Treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib | 11/9/2018 | 3492-1: Conduct and submit the results of one or more randomized trials to verify and describe the clinical benefit of pembrolizumab as compared to available therapy in patients with locally advanced, unresectable or metastatic HCC as demonstrated by an improvement in OS or a large improvement in PFS that is clinically meaningful. | 10/31/2019 |
| Opdivo (nivolumab) | In combination with ipilimumab, is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or DNA mismatch repair deficient (dMMR), metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan | 7/10/2018 | 3449-1: Submit the final report, including datasets, from a randomized trial conducted to verify and describe the clinical benefit of nivolumab, administered in combination with ipilimumab, in patients with MSI-H or dMMR metastatic colorectal cancer. The trial will be designed to demonstrate a clinically meaningful improvement in PFS in patients randomized to receive nivolumab and ipilimumab as compared to patients randomized to receive nivolumab alone. In addition, the trial should evaluate for differences in OS between arms based on a pre-specified analysis. The analysis plan should describe the power for the OS analysis, as well as all assumptions made in determining the power. | 7/31/2024 |
| Yervoy (ipilimumab) | In combination with nivolumab, is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or DNA mismatch repair deficient (dMMR), metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan | 7/10/2018 | 3450-1: Submit the final report, including datasets, from a randomized trial conducted to verify and describe the clinical benefit of ipilimumab, administered in combination with nivolumab, in patients with MSI-H or dMMR metastatic colorectal cancer. The trial will be designed to demonstrate a clinically meaningful improvement in PFS in patients randomized to receive ipilimumab and nivolumab as compared to patients randomized to receive nivolumab alone. In addition, the trial should evaluate for differences in OS between arms based on a pre-specified analysis. The analysis plan should describe the power for the OS analysis, as well as all assumptions made in determining the power. | 7/31/2024 |
| Calquence (acalabrutinib) | Treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. | 10/31/2017 | 3291-1: Submit the complete final report and datasets demonstrating clinical efficacy and safety from a randomized, double-blind, placebo-controlled, clinical trial of Calquence in combination with standard immunochemotherapy versus immunochemotherapy alone in patients with MCL. | 11/30/2024 |
| Aliqopa (copanlisib) | Treatment of adult patients with relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies | 9/14/2017 | 3273-1: In order to verify the clinical benefit of Aliqopa (copanlisib), submit the complete final clinical report and datasets from a randomized, double-blind, placebo-controlled trial of Aliqopa in combination with immunochemotherapy versus immunochemotherapy alone in patients with relapsed FL, MZL, SLL, or lymphoplasmacytic lymphoma/Waldenström’s macroglobulinemia. The primary endpoint is PFS. Enroll approximately 520 patients. | 9/30/2022 |
| Opdivo (nivolumab) | For the treatment of adult and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. | 7/31/2017 | 3243-1: Submit the final report, including datasets, from trials conducted to verify and describe the clinical benefit of nivolumab 240 mg intravenously every two weeks in patients with MSI-H or dMMR CRC who have progressed following treatment with fluoropyrimidine, oxaliplatin and irinotecan, including at least 150 patients enrolled in BMS-initiated trials. In order to characterize response rate and duration, patients will be followed for at least 12 months from the onset of response. | 9/30/2021 |
| Opdivo (nivolumab) |
Treatment of adult patients with classical Hodgkin lymphoma that has relapsed or progressed after: |
4/25/2017 | 3089-1: Conduct a randomized phase 3 clinical trial in classical Hodgkin lymphoma that verifies and isolates the clinical benefit of nivolumab for patients with classical Hodgkin lymphoma. The primary endpoint would be PFS as determined by an independent review committee. OS would be a key secondary endpoint. | 12/31/2026 |
| Opdivo (nivolumab) | For the treatment of classical Hodgkin Lymphoma that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and post-transplantation brentuximab vedotin. | 5/17/2016 | 3089-1: Conduct a randomized phase 3 clinical trial in classical Hodgkin lymphoma that verifies and isolates the clinical benefit of nivolumab for patients with classical Hodgkin lymphoma. The primary endpoint would be PFS as determined by an independent review committee. OS would be a key secondary endpoint. | 12/31/2026 |
| Beleodaq (belinostat) | Treatment of relapsed or refractory peripheral T-Cell Lymphoma (PTCL) | 7/3/2014 | 2178-2: Characterize the comparative efficacy and safety of belinostat when used in combination with a treatment regimen such as CHOP, versus the combination of pralatrexate plus CHOP, versus CHOP alone for the initial therapy of patients with PTCL. Perform a confirmatory, prospective randomized (1:1:1) trial of previously untreated patients with PTCL, with PFS as the primary efficacy endpoint. Enroll a sufficient number of patients to characterize the efficacy and safety of each drug added to CHOP, versus CHOP alone. The PFS endpoint should be determined by a blinded independent review committee. PFS analysis should be performed when the trial has experienced the planned number of events necessary for trial completion. Using the same data cutoff date as the PFS analysis, perform an interim analysis of OS. Submit a complete final report with all supporting datasets. 2178-1: Establish the optimal and safe dose of belinostat in combination with the CHOP regimen. Perform a Phase 1 dose finding trial of belinostat plus CHOP for the treatment of patients with PTCL. Enroll a sufficient number of patients to characterize the safety of belinostat in combination with the CHOP regimen. Submit a complete final report with all supporting datasets |
1/31/2021 |
| Folotyn (pralatrexate) | Treatment of relapsed or refractory peripheral T-Cell Lymphoma (PTCL) | 9/24/2009 | 2179-2: Characterize the comparative efficacy and safety of pralatrexate when used in combination with a treatment regimen such as CHOP, versus the combination of Beleodaq plus CHOP, versus CHOP alone for the initial therapy of patients with PTCL. Perform a confirmatory, prospective randomized (1:1:1) trial of previously untreated patients with PTCL, with PFS as the primary efficacy endpoint. Enroll a sufficient number of patients to characterize the efficacy and safety of each drug added to CHOP, versus CHOP alone. The PFS endpoint should be determined by a blinded independent review committee. PFS analysis should be performed when the trial has experienced the planned number of events necessary for trial completion. Using the same data cutoff date as the PFS analysis, perform an interim analysis of OS. Submit a complete final report with all supporting datasets. 2179-1 : Establish the optimal and safe dose of pralatrexate in combination with the CHOP regimen. Perform a Phase 1 dose-finding trial of pralatrexate plus CHOP for the treatment of patients with PTCL. Enroll a sufficient number of patients to characterize the safety of pralatrexate in combination with the CHOP regimen. Submit a complete final report with all supporting datasets. |
6/30/2017 |