FDA Docket Packed with Key Decisions for December

The U.S. Food and Drug Administration (FDA) is set for a busy December, with over 15 decisions lined up for the coming weeks. Several major pharmaceutical companies are awaiting verdicts on their latest treatments, ranging from CAR T-cell therapies to antibiotics for gonorrhea.

BMS and Amgen Seek Expansions for Biologic Blockbusters

Bristol Myers Squibb (BMS) is anticipating a decision on December 5 for the potential expansion of its CAR T-cell therapy, Breyanzi, into marginal zone lymphoma (MZL). If approved, Breyanzi would become the first CAR T option for MZL. The therapy has shown promising results in the Phase II TRANSCEND FL study, with a 95.5% overall response rate and a 62.1% complete response rate.

Amgen is also looking to broaden the reach of its anti-CD19 antibody, Uplizna, with a target action date of December 14 for its application in generalized myasthenia gravis (gMG). The expansion bid is supported by data from the Phase III MINT study, which demonstrated significant improvements in patients' daily activities and disease severity at 26 weeks compared to placebo.

Antibiotic Advancements: GSK and Innoviva Target Gonorrhea

Two pharmaceutical companies are vying for FDA approval of new treatments for uncomplicated gonorrhea. GSK's oral antibiotic Blujepa is scheduled for a decision on December 11, while Innoviva's zoliflodacin awaits a verdict on December 15. Both drugs have shown promising results in Phase III trials, with GSK's Blujepa demonstrating a 92.6% treatment success rate and Innoviva's zoliflodacin achieving a 90.9% microbiological cure rate.

Other Notable Decisions on the Horizon

BioCryst Pharmaceuticals is expecting a decision by December 12 on the expansion of its hereditary angioedema drug Orladeyo to include children aged 2 to 11 years. This comes after a three-month delay requested by the FDA to review additional data submissions.

Milestone Pharmaceuticals is awaiting the FDA's response to its resubmission for Cardamyst, a nasal spray for tachycardia, following a rejection in March 2024. The decision is expected on December 13 and will address chemistry, manufacturing, and controls issues raised in the previous review.

As the pharmaceutical industry braces for these crucial decisions, the outcomes are set to shape treatment landscapes across multiple therapeutic areas, potentially offering new options for patients and expanding the reach of existing therapies.