A Look Back at FDA News from April 2019

In April 2019, several therapies were approved by the FDA in breast cancer, lung cancer, bladder cancer, and renal cell carcinoma (RCC). In addition, several applications were submitted for approval by the FDA while other agents recieved priority review or breakthrough designations.

Here's a look back on the FDA happenings for the month of April 2019:

MEK Inhibitor Granted Breakthrough Designation for NF1 Plexiform Neurofibromas

On April 1, 2019, the FDA granted selumetinib (AZD6244), a MEK inhibitor, a breakthrough therapy designation for the treatment of patients ≥3 years old with symptomatic and/or progressive, inoperable neurofibromatosis type 1—related plexiform neurofibromas.

Palbociclib Approved by FDA for Treatment of Male Patients With HR+/HER2- Breast Cancer

The FDA has expanded the approval of combination palbociclib (Ibrance) capsules plus endocrine therapy to include the treatment of male patients with hormone receptor (HR)-positive, HER2-negative advanced or metastatic breast cancer on April 4, 2019.

NDA Review Period Extended by FDA for Quizartinib in FLT3-ITS+ AML

On April 4, 2019, the FDA extended the review period of the new drug application (NDA) for quizartinib as a treatment for adult patients with relapsed/refractoryFLT3-ITD-positive acute myeloid leukemia (AML).

Luspatercept Submitted to FDA for Treatment of Anemias

A supplemental biologics license application (sBLA) was submitted to the FDA for the approval of luspatercept for the treatment of adult patients with very low- to imtermediate-risk myeodysplastic sundrome (MDS)-associated anemia with ring sideroblasts who require red blood cell (RBC) transfusions, and also for adult patients with beta-thalassemia—associated anemia who require such transfusions. The sBLA was submitted on April 5, 2019, based on data from 2 clinical trials.

Novel Immunotherapy Tebentafusp Granted Fast Track Designation for Metastatic Uveal Melanoma

On April 11, 2019, the FDA granted a fast track designation to tebentafusp (IMCgp100) for the treatment of patients with HLA-A*0201-positive previously untreated metastatic uveal melanoma based on findings from a phase I study. This agent demonstrated an overall survival (OS) rate of 74% at 1 year, and at a median follow-up of 15.9 months, the median OS has not yet been reached.

Pembrolizumab Approved by FDA for Lower PD-L1 Cutoff in NSCLC

The FDA expanded the indication of pembrolizumab on April 11, 2019, to include the frontline treatment of patients with stage III non—small cell lung cancer (NSCLC) who are ineligible for surgery or definitive chemoradiation, or metastatic NSCLC, with a PD-L1 expression (tumor proportion score) level of ≥1% and do not harbor EGFR or ALK aberrations.

Erdafitinib Granted FDA Approval in Bladder Cancer

FDA Grants Fast Track and Orphan Drug Designations to Novel Agent Targeting P53 in MDS

On April 12, 2019, the FDA approved the first targeted agent for metastatic bladder cancer. Erdafitinib (Balversa) received an accelerated approval for the treatment of adult patients with locally advanced or metastatic bladder cancer with anFGFR3orFGFR2alteration that has progressed on platinum-containing chemotherapy.

On April 19, 2019, the FDA granted a fast track designation and an orphan drug designation to APR-246 for the treatment of patients with MDS. APR-246 is a novel agent that targets p53.

FDA Approves Pembrolizumab With Axitinib for Frontline Treatment of RCC

The FDA granted pembrolizumab approval on April 22, 2019, for the treatment of patients with advanced RCC in combination with axitinib (Inlyta). This approval is based on results from the phase III KEYNOTE-426 trial in which this frontline combination statistically improved overall response rates, progression free survival, and OS compared to sunitinib (Sutent).

ODAC Meetings Scheduled for FDA for Pexidartinib in TGCT and Quizartinib in AML

The Oncologic Drugs Advisory Committee (ODAC) will meet on May 14, 2019 to discuss the NDA seeking approval of pexidartinib for the treatment of adult patients with symptomatic tenosynovial giant cell tumor, according to an announcement from the FDA on April 19, 2019. The ODAC has also scheduled a meeting to discuss an NDA for quizartinib tablets as a treatment of adult patients with relapsed/refractoryFLT3-ITD-positive AML.

PD-L1 IHC 22C3 pharmDX Assay Approval Expanded by FDA in NSCLC

On April 17 2019, the FDA expanded its approval of the PD-L1 22C3 pharmDX assay to be used as a companion diagnostic for identifying more patients with stage III or metastatic NSCLC who can undergo frontline treatment with pembrolizumab. This expansion coincides with the FDA’s approval of pembrolizumab as a frontline treatment for patients with stage III NSCLC who are not candidates for surgery or definitive chemoradiation, or metastatic NSCLC, with a PD-L1 expression (tumor proportion score [TPS]) level of ≥1% and do not harbor EGFR or ALK aberrations.

Darolutamide Granted Priority Review by FDA for Treatment of Nonmetastatic CRPC

A new drug application was submitted on April 29, 2019, for darolutamide as a treatment for patients with nonmetastatic castration-resistant prostate cancer. The NDA was submitted based on data from the phase III ARAMIS trial which demonstrated a 59% reduction in the risk of metastases or death with darolutamide compared to placebo.

Apalutamide Submitted to FDA for Treatment of Metastatic Castration-Sensitive Prostate Cancer