Skyline Therapeutics (Shanghai) Co. Ltd.’s SKG-1108, a novel one-time intravitreally delivered gene therapy, has been awarded U.S. orphan drug designation for the treatment of retinitis pigmentosa.
Invenra Inc.’s bispecific antibody, INV-724, developed for the treatment of neuroblastoma, has been awarded orphan drug and rare pediatric disease designations by the FDA.
Ractigen Therapeutics Co. Ltd.’s small activating RNA (saRNA) therapeutic, RAG-18, has been awarded U.S. orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy.
Actio Biosciences Inc.’s ABS-0871 has received both orphan drug designation and rare pediatric disease designation from the FDA. ABS-0871 is in preclinical development for the treatment of Charcot-Marie-Tooth disease subtype 2C (CMT2C).
Innorna Co. Ltd.’s IN-016 has received orphan drug status from the U.S. FDA for the treatment of progressive familial intrahepatic cholestasis (PFIC). Earlier in July, IN-016 had been granted Rare Pediatric Disease Designation. PFIC is a group of rare genetic disorders associated with defects in bile acid secretion or transport, resulting in unwanted bile accumulation within the liver.
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.
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