FDA Action Alert: It's Decision Week for BMS's Mavacamten

FDA_Al Drago/CQ Roll Call

Al Drago/CQ Roll Call

The U.S. Food and Drug Administration is wrapping up its April calendar with a handful of PDUFA dates. Continue reading for details.

BMS's Mavacamten for Obstructive Hypertrophic Cardiomyopathy

Bristol Myers Squibb has a target action date of April 28 for its mavacamten for symptomatic obstructive hypertrophic cardiomyopathy (obstructive HCM). This progressive disorder thickens the heart walls, making it harder for the heart to expand normally and fill with blood. The drug is a selective cardiac myosin inhibitor.

The drug has demonstrated it can decrease cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation, which causes hypercontractility, left ventricular hypertrophy and reduced compliance. The regulatory application is based on results from the EXPLORER-HCM trial.

Supernus’ ADHD Drug for Adults

Supernus Pharmaceuticals has a target action date of April 29 for its supplemental New Drug Application (sNDA) for Qelbree (viloxazine extended-release capsules) for the treatment of attention-deficit hyperactivity disorder (ADHD) in adults. The drug was approved for ADHD in kids 6 to 17 years of age in April 2021. The sNDA is based on positive topline results from a Phase III trial of the drug in adults. A daily dose of up to 600mg hit the primary endpoint of “robust statistical significance compared to placebo in improving the symptoms of ADHD.”

Axsome’s AXS-07 for Acute Migraine

Axsome Therapeutics has a target action date of April 30 for its New Drug Application for AXS-07 for acute treatment of migraine. The drug (MoSEIC meloxicam-rizatriptan) is a novel, oral, rapidly absorbed therapeutic. The NDA is based on data from two Phase III trials, MOMENTUM and INTERCEPT, which demonstrated statistically significant elimination of migraine pain with the drug compared to placebo and active controls.

At the company’s fourth-quarter and full-year 2021 financial report on March 1, Dr. Herriot Tabuteau, MD, said, “2021 was a year of continued progress which has put us in a position to potentially launch two new investigational medicines for patients living with depression and migraine. Specifically, FDA review of our NDA for AXS-05 in depression is progressing, and the April 30 PDUFA data for our NDA for AXS-07 in the acute treatment of migraine is approaching.”

Coherus’ Toripalimab for Nasopharyngeal Carcinoma

Coherus BioSciences has a target action date of April 30 for its Biologics License Application (BLA) for toripalimab nasopharyngeal carcinoma. It is under priority review.

On April 8, Coherus and Shanghai Junshi Biosciences announced the results of the prespecified final progression-free survival (PFS) analysis and the interim OS analysis of the Phase III JUPITER-02 trial of toripalimab for the first-line treatment of recurrent or metastatic nasopharyngeal carcinoma.

In the final PFS analysis, JUPITER-02 provided statistically significant improvement in PFS compared to chemotherapy plus placebo, with an improvement in median PFS of 13.2 months. It also provided significant improvements in the secondary endpoints of PFS, objective response rate (ORR) and duration of response (DoR). Also, in a separate Phase III trial, the drug in combination with chemotherapy demonstrated a statistically significant overall survival benefit in the prespecified interim analysis in patients with non-small cell lung cancer.

Hutchmed’s Surufatinib for Pancreatic and Extra-Pancreatic Neuroendocrine Tumors

Hutchmed has a target action date of April 30 for its NDA surufatinib for pancreatic and extra-pancreatic neuroendocrine tumors. It received Fast Track Designation in April 2020 and Orphan Drug Designation in November 2019. The NDA is supported by data from two positive Phase III trials, SANET-p and SANET-ep in China and a study in the U.S.

Surufatinib is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptors (VEGFR) and fibroblast growth factor receptor (FGFR).

Incyte’s Parsaclisib for Relapsed or Refractory Non-Hodgkin Lymphomas

The target action date for Incyte's parsaclisib for marginal zone lymphoma (MZL) and mantle cell lymphoma (MCL) is April 30. The NDA submission was built on data from several Phase II trials. It has been granted Priority Review for adults with r/r MZL who have received at least one previous anti-CD20-based therapy and adults with MCL who have received at least one prior treatment. These were for Priority Review. The NDA for the drug in adults with relapsed or refractory follicular lymphoma (FL), who have received at least two previous systemic therapies, is under standard review with a PDUFA date of August 30, 2022.

Parsaclisib is a novel potent, highly selective, next-generation oral inhibitor of phosphatidylinositol 3-kinase delta.

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