The FDA is approaching several key decision dates this month for therapies targeting a range of conditions, including gastrointestinal disorders, rare diseases, psychiatric illnesses, and respiratory ailments.
Vanda's Tradipitant for Gastroparesis
Vanda Pharmaceuticals anticipates an FDA decision by September 18 regarding its new drug application for tradipitant, an investigational NK-1R antagonist for the treatment of gastroparesis. This condition, affecting approximately 6 million people in the U.S., involves delayed stomach emptying, leading to symptoms such as nausea, bloating, vomiting, and abdominal pain. Tradipitant, a small molecule blocker of neurokinin (NK)-1 receptors found in the gastrointestinal tract and brain regions associated with the vomit reflex, has the potential to suppress these symptoms. The application is supported by clinical efficacy studies and preclinical toxicology data.
Zevra's Arimoclomol for Niemann-Pick Disease Type C
Zevra Therapeutics is awaiting a verdict by September 21 on arimoclomol, an oral drug candidate for Niemann-Pick disease type C (NPC). NPC is a rare neurodegenerative lysosomal storage disease characterized by impaired cholesterol and lipid transport, leading to toxic buildup in organs, including the brain. Patients experience speech, swallowing, and movement problems, with the disease often leading to death within months. 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 seemingly small magnitude.
Heron Therapeutics' Extended-Release Needle for Zynrelef
Heron Therapeutics is seeking approval for a new extended-release solution vial access needle (VAN) for its local anesthetic Zynrelef (bupivacaine and meloxicam), with a decision expected by September 23. The VAN is designed to simplify Zynrelef preparation, reducing withdrawal time from up to three minutes to 20-45 seconds. Zynrelef, a dual-acting local anesthetic combining bupivacaine and meloxicam, is currently approved for post-surgical pain relief after procedures like total knee arthroplasty, open inguinal herniorrhaphy, and bunionectomy. Phase III studies have demonstrated significant pain reduction and decreased opioid use with Zynrelef, though it carries a boxed warning for serious cardiovascular and gastrointestinal events.
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, affecting the lining of the chest and lungs. The supplemental Biologics License Application (sBLA) is supported by data from the Phase II/III KEYNOTE-483 study, which 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 an FDA decision by September 26 for KarXT, an investigational muscarinic antipsychotic for schizophrenia. KarXT uniquely activates M1 and M4 muscarinic receptors without directly blocking dopamine receptors, potentially easing positive, negative, and cognitive symptoms. If approved, KarXT would represent a novel pharmacological approach to schizophrenia treatment. The FDA is reviewing data from the EMERGENT clinical development program, including EMERGENT-1, EMERGENT-2, EMERGENT-3, and EMERGENT-4 studies. While EMERGENT-3 missed a key secondary endpoint and raised cardiovascular risk concerns, EMERGENT-4 demonstrated sustained efficacy, with over 75% of patients showing at least a 30% symptomatic improvement at 52 weeks.
Sanofi and Regeneron's Dupixent for COPD
Sanofi and Regeneron anticipate an FDA decision by September 27 on their bid to expand the label of Dupixent (dupilumab) to include chronic obstructive pulmonary disease (COPD). Dupixent, a monoclonal antibody that blocks the IL-4 and IL-13 pathways, has already been approved for atopic dermatitis, asthma, and chronic rhinosinusitis with nasal polyps. The COPD application is supported by data from the Phase III BOREAS and NOTUS trials. In BOREAS, Dupixent added to standard of care reduced moderate or severe exacerbations by 30% over 52 weeks, while also improving lung function and quality of life. NOTUS data showed a 34% reduction in moderate or severe exacerbations. The FDA requested additional efficacy analyses from both trials, extending the review period by three months, but emphasized that it had no concerns regarding the approvability of Dupixent for COPD.
