CARLSBAD, Calif. — Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation to TYRA-300, an oral...
Latest News
MINNEAPOLIS/ST. PAUL — Focal segmental glomerulosclerosis (FSGS) is a rare kidney disorder that affects children and adults, and can lead to kidney failure. New findings from a team led by the University of Minnesota Medical School show patients with FSGS who were treated with the medication sparsentan experienced improved kidney function—making...
Fayetteville, Arkansas – The U.S. Department of Defense awarded just under $514,000 to an interdisciplinary team of researchers at the University of Arkansas to study the efficacy of “self-delivering” gene editors in the treatment of Duchenne Muscular Dystrophy, or DMD. DMD results from a mutation in the dystrophin gene and...
PHILADELPHIA – Amicus Therapeutics (Nasdaq: FOLD) announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat for AT-GAA, the Company’s investigational two-component therapy for the treatment of Pompe disease. Pompe disease...
Tick-borne encephalitis (TBE) is a viral infection, endemic to parts of Europe and Asia If approved, the vaccine may help reduce the risk of TBE for people traveling to endemic areas, potentially including military personnel serving in these locations NEW YORK – Pfizer Inc. (NYSE:PFE) today announced that the U.S....
BURLINGTON, Mass. – PharmaEssentia Corporation (TPEx: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, announced that the U.S. Food and Drug Administration (FDA) has approved BESREMi® (ropeginterferon alfa-2b-njft) for the treatment of adults with polycythemia...
Ingelheim am Rhein, Germany and Ridgefield, Connecticut – Boehringer Ingelheim’s JASCAYD® (nerandomilast) tablets has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of progressive pulmonary fibrosis (PPF) in adults.5 This represents a new treatment option in the U.S. for this debilitating lung condition, with JASCAYD being the...
SANTA MONICA, Calif. – Kite, a Gilead Company (Nasdaq: GILD), announced the U.S. Food and Drug Administration (FDA) has granted approval for Tecartus® (brexucabtagene autoleucel) for the treatment of adult patients (18 years and older) with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). Following FDA Breakthrough Therapy Designation...
FOSTER CITY, Calif. – Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leader in rare liver disease, announced that the U.S. Food and Drug Administration (FDA) has approved LIVMARLI™ (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older. LIVMARLI, a...
VANCOUVER, Canada – BirchBioMed Inc., a leading clinical stage biopharmaceutical company, expert in the development of products for scarring and other fibrosis-related disorders and autoimmune therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to FS2 (kynurenic acid) for the investigational treatment of idiopathic pulmonary...