A Look Back at the FDA News from April 2020

In April 2020, the FDA accelerated approval decisions in HER2-positive breast cancer, as well as triple-negative breast cancer. Overall, there were 11 approvals across multiple malignancies. The FDA also made strides in clinical cancer research by granting Fast Track and Breakthrough Therapy designations in metastatic urothelial cancer, metastatic cervical cancer, neuroendocrine tumors, metastatic pancreatic cancer, and EGFR exon 20-positive non–small cell lung cancer.

Two treatments were granted Priority Review in April, both of which have Prescription Drug Free User Act target action date in the summer.

Here’s a look back on the FDA happenings from the month of April 2020:

FDA Approves Luspatercept for Myelodysplastic Syndrome-Associated Anemia

On April 3, The FDA granted approval to luspatercept-aamt as treatment of adult patients with anemia failing an erythropoiesis-stimulating agent that requires 2 or more red blood cell units over 8 weeks, that is associated with very low- to intermediate-risk myelodysplastic syndromes, intermediate-risk myelodysplastic syndromes with ring sideroblasts or myelodysplastic/myeloproliferative neoplasm.

FDA Grants Pembrolizumab Priority Review for TMB-High Tumors

The FDA accepted and granted a priority review to a supplemental Biologics License Application, on April 7, for pembrolizumab for the treatment of adult and pediatric patients with unresectable or metastatic solid tumors with tissue tumor mutational burden-high who have progressed following prior treatment and who have no satisfactory alternative treatment options.

FDA Grants Fast Track Designation to Sacituzumab Govitecan in Locally Advanced mUC

On April 7, sacituzumab govitecan was granted Fast Track designation by the FDA for the treatment of patients with locally advanced or metastatic urothelial cancer who have previously received a programmed death receptor-1 or programmed death-ligand 1 inhibitor, and a platinum-containing chemotherapy in the neoadjuvant/adjuvant, including patients who are platinum ineligible.

FDA Grants Fast Track Designation to Balstilimab in Metastatic Cervical Cancer

The FDA has granted Fast Track designation to balstilimab, an investigational anti-PD-1 agent for the treatment of patients with cervical cancer on April 7, based on comprehensive data that suggest balstilimab can fill an unmet medical need in the space, Agenus, Inc announced in a press release. In light of the new designation, the company now plans to submit 2 Biologic License Applications in 2020, for this indication and the other for the combination of balstilimab and zalifrelimab in metastatic cervical cancer.

FDA Grants Priority Review to BLA for First-Line Nivolumab/Ipilimumab Plus Limited Chemo in NSCLC

On April 8, the FDA has accepted the Biologics License Application for and granted Priority Review to the combination of nivolumab plus ipilimumab with limited chemotherapy as a first-line treatment of patients with metastatic or recurrent non–small cell lung cancer who have no EGFR or ALK genomic tumor aberrations, according to a press release from Bristol Myers Squibb. The Prescription Drug Free User Act target action date is set as August 6, 2020, and the combination was granted Fast Track designation.

FDA Approves Encorafenib Plus Cetuximab in BRAF V600E+ mCRC

The FDA has approved the combination of encorafenib and cetuximab on April 9, as treatment of patients with metastatic colorectal cancer with a BRAF V600E mutation, as detected by an FDA-approved test, after prior therapy.

Avelumab Granted FDA Breakthrough Therapy Designation in Metastatic Urothelial Cancer

On April 9, a Biologics License Application for avelumab, a PD-L1 inhibitor, was submitted to the FDA for consideration as first-line treatment of patients with locally advanced or metastatic urothelial cancer and the agent was granted Breakthrough Therapy Designation.

FDA Approves Selumetinib for Pediatric Neurofibromatosis Type 1 Plexiform Neurofibromas

The FDA has approved selumetinib on April 13, for the treatment of pediatric patients, aged 2 years and above, with neurofibromatosis type 1 who have symptomatic, inoperable plexiform neurofibromas.

FDA Approves Mitomycin Gel For Low-Grade Upper Tract Urothelial Cancer

On April 15, The FDA has granted approval to mitomycin gel as treatment of adult patients with low-grade upper tract urothelial cancer.

FDA Approves Novel Diagnostic Assay to Accompany Encorafenib in Treatment of mCRC

The FDA has granted approval to the therascreen BRAF V600E Kit on April 16, as a companion diagnostic to encorafenib, a BRAF inhibitor indicated for the treatment of adult patients with metastatic colorectal cancer harboring a BRAF V600E mutation, as detected by an FDA approved test, in combination with cetuximab after prior therapy.

FDA Approves Tucatinib Plus Trastuzumab/Capecitabine in HER2-Positive Breast Cancer

On April 17, the FDA granted accelerated approval for tucatinib tablets in combination with trastuzumab and capecitabine as treatment of adult patients with advanced unresectable or metastatic HER2-positive breast cancer, including patients with brain metastases who have received one or more prior anti-HER2-based regimens in the metastatic setting.

FDA Grants Two Fast Track Designations to Surufatinib in Pancreatic and Extrapancreatic NETs

The FDA granted two Fast Track designations to surufatinib, on April 17, for the treatment of both advanced and progressive pancreatic neuroendocrine tumors and extra-pancreatic neuroendocrine tumors in patients who are not amenable for surgery. The agent was previously granted Orphan Drug Designation by the FDA for this indication.

FDA Approves Pemigatinib for FGFR2+ Cholangiocarcinoma

On the 18th of the month, the FDA has approved pemigatinib for the treatment of adult patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma harboring an FGFR2 fusion or rearrangement.

FDA Approves cobas HPV Test for Measurement of Cervical Cancer Risk in Women with High-Risk HPV

The FDA granted approval for the cobas high-risk human papillomavirus test, on April 21, which identifies women at risk for developing cervical cancer by exposing the presence of high-risk human papillomavirus DNA in cervical samples. Left untreated, human papillomavirus lesions can progress to cervical cancer.

FDA Approval Sought for Relugolix in Advanced Prostate Cancer

On April 21, a New Drug Application was been submitted to the FDA for once-daily, oral relugolix at a dose of 120 mg as treatment of male patients with advanced prostate cancer, based on results from the phase III HERO study.

FDA Approves Expanded Indication for Frontline Rituximab Plus Ibrutinib in CLL/SLL

The FDA granted approval to the combination of ibrutinib plus rituximab on April 21, for the frontline treatment of adult patients with chronic lymphocytic leukemia or small lymphocytic leukemia, based on data from the E1912 trial.

FDA Grants RMAT Designation to Tisagenlecleucel in R/R Follicular Lymphoma

On April 22, the FDA granted a Regenerative Medicine Advanced Therapy designation to the chimeric antigen receptor T-cell therapy, tisagenlecleucel, for an investigational new indication as treatment of patients with relapsed or refractory follicular lymphoma.

FDA Grants Accelerated Approval to Sacituzumab Govitecan in mTNBC

The FDA has accelerated approval to sacituzumab govitecan-hziy, on April 22, for the treatment of adult patients with metastatic triple-negative breast cancer who have received at least 2 prior therapies for metastatic disease.

Pembrolizumab sBLA Resubmitted to FDA With New Dosing Frequency

On April 23, a supplemental Biologics License Application for pembrolizumab has been resubmitted to the FDA to update the dosing frequency to include a 400 mg dose administered via infusion over 30 minutes, every 6 weeks. The resubmission of the sBLA is a solution to Complete Response Letter issued to Merck by the FDA in February 2020. If approved, this dose of pembrolizumab will be an expansion of the prior approval for the pembrolizumab dose of 200 mg every 3 weeks.

FDA Approves Pembrolizumab Companion Diagnostic for Frontline PD-L1+ NSCLC

The FDA granted approval for PD-L1 IHC 22C3 pharmDx on April 23, as a companion diagnostic as an identifier of patients with non–small cell lung cancer who are eligible for frontline treatment with pembrolizumab monotherapy. The CDx will identify these patients on the Dako Omnis platform for immunohistochemistry and in situ hybridization.

FDA Grants Breakthrough Designation to Mobocertinib for EGFR Exon 20+ NSCLC

Mobocertinib was granted a Breakthrough Therapy designation by the FDA on April 27, for the treatment of patients with metastatic non–small cell lung cancer harboring EGFR exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy.

FDA Approves Every-6-Week Pembrolizumab Dosage

On April 28, a new dosage for pembrolizumab of 400 mg administered every 6 weeks across all adult indications has been approved by the FDA, whether the PD-1 inhibitor is used as monotherapy or in a combination regimen.

FDA Grants Fast Track Designation to Second-Line Eryaspase for Pancreatic Cancer

A Fast Track designation for eryaspase was granted by the FDA on April 29 for the treatment of patients with metastatic pancreatic cancer in the second-line setting, underscoring the need for new treatment options for this patient population.

FDA Approves Niraparib As Frontline Maintenance in Advanced Ovarian Cancer

At the close of the month, the FDA granted approval to niraparib as first-line maintenance treatment of adult patients with advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in complete or partial response to first-line platinum-based chemotherapy.