The first new U.S. FDA-approved therapeutic option for PTSD in more than 20 years will have to wait. A supplemental NDA seeking approval of Rexulti (brexpiprazole) combined with sertraline, filed by one of Japan’s biggest pharmas, Otsuka Pharmaceutical Co. Ltd., received a complete response letter (CRL) from the agency.
Breaking with its long-held, oft-recited mantra that observational studies are great for generating hypotheses but not as evidence for approval, the U.S. FDA is initiating the approval of leucovorin calcium tablets for patients with cerebral folate deficiency, a neurological condition that affects folate transfer into the brain.
A failed July inspection of manufacturer Catalent Indiana LLC has delayed another U.S. FDA approval, the latest being that of Scholar Rock Inc.’s selective anti-latent myostatin antibody, apitegromab, which was expected to become the first therapy to enhance skeletal muscle in patients with spinal muscular atrophy.
After a long regulatory road that included a complete response letter in May, Stealth Biotherapeutics Inc. finally got its Barth syndrome drug across the finish line, with the U.S. FDA granting accelerated approval to Forzinity (elamipretide HCl) to improve muscle strength in those with the ultra-rare pediatric mitochondrial cardioskeletal disease.
The U.S. FDA approved Merck & Co. Inc.’s Keytruda Qlex (pembrolizumab and berahyaluronidase alfa-pmph) injection on Sept. 19, making it the first and only subcutaneously (SC)-administered immune checkpoint inhibitor that can be administered in about a minute.
NS Pharma Inc.’s NS-051 (NCNP-04) has been awarded orphan drug designation by the FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping.
The FDA has cleared HDT Bio Corp.’s IND application for HDT-321, a first-in-class prophylactic designed to protect against Crimean-Congo hemorrhagic fever (CCHF).
Medtronic plc received U.S. FDA approval for Altaviva, a minimally invasive implantable tibial neuromodulation device designed to treat urge urinary incontinence. Insertion near the ankle requires neither sedation nor imaging and patients walk out the clinic door with the device already activated.
Organox Ltd.’s normothermic machine perfusion device, Metra, has been cleared by the U.S. FDA for use during air transport. The greenlight from the regulatory body paves the way for the broader use of donor organs that might otherwise be discarded, as the Metra system circulates oxygen and nutrient rich perfusate through the organ at near-body temperature, extending preservation times.
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