FDA Action Alert: Key Decisions Await Merck, BMS, Sarepta, and More
The upcoming week is critical for the pharmaceutical industry, as the U.S. Food and Drug Administration (FDA) is set to announce its decisions on several major drug applications. These decisions will impact treatments for diseases ranging from pneumococcal infections to chronic inflammatory demyelinating polyneuropathy (CIDP), colorectal cancer (CRC), narcolepsy, endometrial carcinoma, and Duchenne muscular dystrophy (DMD).
Merck Awaits Verdict on Pneumococcal Vaccine
Merck’s investigational 21-valent vaccine, V116, designed to prevent invasive pneumococcal disease and pneumococcal pneumonia in adults, is poised for the FDA’s review. The decision is expected on June 17, backed by data from the Phase III STRIDE-3 trial, which demonstrated that V116 induced non-inferior immune responses compared to a 20-valent comparator vaccine. Additional studies, including STRIDE-4, STRIDE-5, and STRIDE-6, will also support Merck’s application. If approved, V116 would be the first pneumococcal conjugate vaccine specifically for adults.
Argenx Seeks Approval for Vyvgart Hytrulo in CIDP
Argenx is seeking FDA approval to use its antibody fragment Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) to treat chronic inflammatory demyelinating polyneuropathy (CIDP), a rare autoimmune disease. The decision is anticipated on June 21. The supplemental Biologics License Application (sBLA) is supported by the Phase II ADHERE study, which showed a 61% reduction in the risk of relapse with Vyvgart Hytrulo compared to placebo. Additionally, 67% of patients in the open-label phase demonstrated clinical improvement. Vyvgart Hytrulo, a subcutaneous formulation of efgartigimod alfa combined with recombinant human hyaluronidase PH20, is already approved for treating generalized myasthenia gravis.
BMS Targets Krazati Expansion into Colorectal Cancer
Bristol Myers Squibb (BMS) is awaiting the FDA’s decision on the supplemental New Drug Application (sNDA) for its KRAS inhibitor Krazati (adagrasib), in combination with cetuximab, for treating locally advanced or metastatic colorectal cancer (CRC) with KRAS G12C mutations. The decision is due on June 21. The application is supported by data from the KRYSTAL-1 study, which reported a 34% objective response rate and median overall survival of 15.9 months. Krazati, an oral small molecule inhibitor, received accelerated approval in December 2022 and was originally developed by Mirati Therapeutics, which BMS acquired for $4.8 billion in October 2023.
Harmony Aims for Wakix Expansion into Pediatric Narcolepsy
Harmony Biosciences is seeking FDA approval for the use of Wakix (pitolisant) to treat excessive daytime sleepiness in pediatric narcolepsy patients aged six years and older. The decision is expected on June 21. The application includes data from a Phase III study demonstrating Wakix’s efficacy and safety in narcolepsy patients aged six to seventeen. Wakix, a histamine 3 receptor antagonist, was initially approved in August 2019 for treating excessive daytime sleepiness in adult narcolepsy.
Merck Targets Addition to Keytruda Indications
Merck is awaiting the FDA’s decision on its sBLA to use Keytruda, its blockbuster PD-1 inhibitor, in combination with chemotherapy for treating primary advanced or recurrent endometrial carcinoma. The decision is due on June 21. Supported by data from the Phase III NRG-GY018 study, Keytruda’s proposed regimen reduced the risk of disease progression or death by 46% in mismatch repair proficient patients and by 70% in mismatch repair deficient patients. If approved, Keytruda would become the first immunotherapy indicated for frontline treatment of advanced endometrial cancer regardless of mismatch repair status.
Sarepta Seeks Full Approval for DMD Gene Therapy
Sarepta Therapeutics is seeking to convert the accelerated approval of its gene therapy Elevidys (delandistrogene moxeparvovec-rokl) into traditional approval and expand its label to include all Duchenne muscular dystrophy (DMD) patients with confirmed DMD gene mutations. The decision is expected on June 21. Elevidys, currently indicated for ambulatory pediatric patients aged four to five years, received accelerated approval in June 2023. However, the therapy failed its primary efficacy endpoint in the SRP-9001-102 study and the Phase III EMBARK study, raising concerns. Despite these setbacks, Sarepta remains hopeful for full approval.
As the FDA prepares to make these pivotal decisions, the outcomes will significantly impact the pharmaceutical landscape and offer potential new treatments for various serious conditions.