The FDA is approaching several key decision deadlines this month for drugs targeting a range of conditions, including gastrointestinal disorders, rare diseases, pain management, cancer, schizophrenia, and COPD.
Vanda's Tradipitant for Gastroparesis
Vanda Pharmaceuticals anticipates an FDA decision by September 18 regarding its new drug application (NDA) for tradipitant, an investigational NK-1R antagonist for gastroparesis. This condition affects approximately 6 million people in the U.S. and involves delayed stomach emptying, leading to symptoms such as nausea, bloating, and abdominal pain. Tradipitant, a small molecule blocker of neurokinin (NK)-1 receptors, aims to suppress nausea and vomiting associated with gastroparesis. The data package submitted to the FDA includes clinical efficacy studies and preclinical toxicology data.
Zevra's Arimoclomol for Niemann-Pick Disease Type C
Zevra Therapeutics is awaiting a potential approval for arimoclomol, an oral drug candidate for Niemann-Pick disease type C (NPC), with the FDA's decision expected by September 21. NPC is an ultrarare neurodegenerative lysosomal storage disease characterized by progressive physical and cognitive decline. Arimoclomol aims to address NPC by crossing the blood-brain barrier and increasing nerve resistance to destruction. An FDA advisory committee recently voted 11-5 to recommend arimoclomol's approval, acknowledging its potential benefits despite their perceived small magnitude.
Heron's Extended-Release Needle for Zynrelef
Heron Therapeutics is proposing a new extended-release solution vial access needle (VAN) for its local anesthetic Zynrelef (bupivacaine and meloxicam). The FDA is expected to release its verdict on or before September 23. VAN is designed to simplify the preparation of Zynrelef, reducing withdrawal time and potentially enhancing safety and uptake. Zynrelef, a dual-acting local anesthetic, is currently approved for post-surgical pain relief following procedures like total knee arthroplasty and bunionectomy. Phase III studies have demonstrated significant pain reduction and decreased opioid requirements with Zynrelef.
Merck's Keytruda for Pleural Mesothelioma
Merck is seeking to expand the label of its PD-1 inhibitor Keytruda (pembrolizumab) to include pleural mesothelioma, with an FDA decision due September 25. Pleural mesothelioma is a rare malignancy linked to asbestos exposure. Merck's supplemental Biologics License Application (sBLA) is supported by data from the Phase II/III KEYNOTE-483 study. Results from the study showed that Keytruda plus chemotherapy significantly improved overall survival (OS), reducing the risk of death by 21% compared to chemotherapy alone. Progression-free survival was also significantly better in the Keytruda plus chemotherapy arm.
BMS's KarXT for Schizophrenia
Bristol Myers Squibb (BMS) awaits the FDA's decision on KarXT, an investigational muscarinic antipsychotic for schizophrenia, by September 26. KarXT works via the dual activation of M1 and M4 muscarinic receptors, differing from current treatments that directly block dopamine receptors. This novel mechanism could potentially address positive, negative, and cognitive symptoms of schizophrenia. The FDA is reviewing data from the EMERGENT clinical development program, including EMERGENT-1, EMERGENT-2, EMERGENT-3, and EMERGENT-4 studies, which have demonstrated KarXT's efficacy in treating schizophrenia. Interim findings from the long-term EMERGENT-4 study showed that KarXT elicited at least a 30% symptomatic improvement at 52 weeks in more than 75% of treated patients.
Sanofi/Regeneron's Dupixent for COPD
Sanofi and Regeneron are nearing the FDA's decision on their bid to expand the label of Dupixent (dupilumab) into chronic obstructive pulmonary disease (COPD), with a verdict due September 27. Dupixent, a fully human monoclonal antibody, targets and blocks the IL-4 and IL-13 pathways, moderating the type 2 inflammatory response. The application is supported by data from the Phase III BOREAS and NOTUS trials. In BOREAS, Dupixent reduced the rate of moderate or severe exacerbations by 30% over 52 weeks, while also improving lung function and quality of life. Recent data from NOTUS put Dupixent's efficacy against moderate or severe exacerbations at 34%.
