Meta Pharmaceuticals Inc. announced that the FDA has granted rare pediatric disease designation to its investigational new drug META-001-PH for the treatment of primary hyperoxaluria (PH), an autosomal recessive metabolic disorder in which oxalate is overproduced and deposited in the body.
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.
Umoja Biopharma Inc. has received clearance from the FDA to begin a first-in-human trial of its CAR T product UB-VV111. The company expects to dose the first patient by the end of 2024.
Gigagen Inc., a subsidiary of Grifols SA, has received clearance from the FDA of its IND application to conduct a phase I trial of GIGA-2339 for the treatment of hepatitis B virus (HBV) infections.
Intellia Therapeutics Inc. has received clearance from the U.K. Medicine and Healthcare products Regulatory Agency (MHRA) to initiate a phase I/II study of NTLA-3001 for the treatment of α1-antitrypsin deficiency (AATD)-associated lung disease.
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.
Actinium Pharmaceuticals Inc. has received FDA clearance of an IND application to study Iomab-ACT for targeted conditioning prior to a bone marrow transplant (BMT) in patients with sickle cell disease.
Iaso Biotechnology Co. Ltd. has announced that China’s National Medical Products Administration (NMPA) has approved the IND application for IASO-782 injection for a new indication – systemic lupus erythematosus.
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