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...
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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...
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...
MADRID, Spain — Amadix, a Spanish biotech company, announced today that PreveCol®, its colorectal cancer screening blood test, has received Breakthrough Device Designation from the U.S. Food and Drug Administration (FDA). With this milestone, Amadix becomes the first European company to announce this recognition for early detection of colorectal cancer....
CAMBRIDGE, Mass. & BEIJING – BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that BRUKINSA® (zanubrutinib) has received accelerated approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory...
CAMBRIDGE, Mass. & BEIJING – BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that BRUKINSA® (zanubrutinib) has received approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with Waldenström’s macroglobulinemia (WM). “We...
HYOGO, Japan — JCR Pharmaceuticals Co., Ltd. Chairman and President Shin Ashida announced that the U.S. Food and Drug Administration (FDA) granted orphan drug designation (ODD) to JR-441, an investigational drug for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, or Sanfilippo syndrome type A). JR-441 is a blood-brain barrier...
SAN RAFAEL, Calif. – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the U.S Food and Drug Administration (FDA) placed a clinical hold on the BMN 307 Phearless Phase 1/2 study. The Phearless study is evaluating BMN 307, an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy, in adults with phenylketonuria (PKU). ...