Weekly Buzz: RARE, REGN Secure FDA Nod, BMRN Stikes Deal; HUMA, EYPT Face Trial Setbacks

This week’s biopharma roundup highlighted FDA decisions and deal activity, with regulatory wins in rare diseases but setbacks in areas such as wetAMD and lung cancer. Ultragenyx (RARE) received accelerated FDA approval for GENGLYCOS (DTX401), an AAV8 gene-transfer therapy for glycogen storage disease type Ia, in both adult and pediatric patients. Implantica (8IM1.F) secured U.S. Premarket Approval for RefluxStop, an implant-based treatment for gastroesophageal reflux disease, after five-year safety and efficacy data in nearly 1,800 patients in Europe. Regeneron (REGN) was cleared for Pasatru (garetosmab-grts) in fibrodysplasia ossificans progressiva to reduce new heterotopic ossification lesions and flare-ups. Separately, Lupin’s generic Pitolisant tablets gained FDA approval, and IDEAYA Biosciences signed a clinical collaboration with Genentech on pancreatic cancer.