Becoming the first type II RAF inhibitor for relapsed or refractory BRAF-altered pediatric low-grade glioma, Day One Biopharmaceuticals Inc.’s Ojemda (tovorafenib, DAY-101) gained U.S. FDA accelerated approval on April 23, a week earlier than its expected PDUFA date, bringing the Brisbane, Calif.-based company a rare pediatric disease priority review voucher.
The FDA has granted orphan drug designation to the active ingredient in Soligenix Inc.’s Marvax, a heat stable subunit protein vaccine of recombinantly expressed Marburg marburgvirus (MARV) glycoprotein, for the prevention and post-exposure prophylaxis against MARV infection.
BLR Bio LLC announced that the U.S. FDA has granted orphan drug designation to BLR-200, the company’s investigational therapy for the treatment of systemic sclerosis (SSc).
The FDA has granted orphan drug designation to the active ingredient in Soligenix Inc.’s Suvax, a subunit protein vaccine of recombinantly expressed Sudan ebolavirus (SUDV) glycoprotein, for the prevention and post-exposure prophylaxis against SUDV infection. SUDV is a type of ebolavirus for which there is no current treatment or vaccine.
Creative Medical Technology Holdings Inc.'s Immcelz (CELZ-101) has been awarded orphan drug designation by the FDA. Immcelz is aimed at preventing allograft rejection in patients undergoing pancreatic islet cell transplantation.
The EMA has awarded orphan drug designation to GC Biopharma Corp.’s intracerebroventricular enzyme replacement therapy (ERT) candidate, GC-1130A, for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A), developed in collaboration with Novel Pharma Inc.
Solid Biosciences Inc.’s SGT-003 has been granted orphan drug designation by the FDA. The company’s next-generation Duchenne muscular dystrophy gene therapy candidate was also granted fast track designation last month.
The FDA has awarded orphan drug designation to Ocelot Bio Inc.'s lead candidate OCE-205 for the treatment of ascites due to all etiologies except cancer. Ocelot Bio plans to initiate clinical studies of OCE-205 in refractory ascites next year.
Neuexcell Therapeutics Inc. has announced that NXL-004, an investigational AAV gene therapy product being developed for the treatment of malignant glioma, has been awarded orphan drug designation by the FDA.
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