Parvus Therapeutics Inc.'s PVT-201 has received orphan drug status from the U.S. FDA for the treatment of primary biliary cholangitis (PBC), an autoimmune disease of the liver.
The U.S. Department of Health and Human Services (HHS) and the FDA already are getting a glimpse of the post-Chevron world and how the Supreme Court’s June 28 unwinding of the 40-year-old Chevron deference could rein in agency policies that defy Congress’ expressed intent.
The FDA has granted orphan drug designation to Be Biopharma Inc.’s BE-101, a novel engineered B-cell medicine being developed for the treatment of hemophilia B.
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.
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