SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has approved the company’s supplemental Biologics License Application (sBLA) for PALYNZIQ® (pegvaliase-pqpz) to include pediatric patients 12 years of age and older with phenylketonuria (PKU). PALYNZIQ is the only enzyme substitution therapy approved to...
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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...
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...
NEW YORK, NY — Pfizer Inc. (NYSE: PFE) today announced that the U.S. Food and Drug Administration (FDA) has granted full approval to BRAFTOVI® (encorafenib) in combination with cetuximab (marketed as ERBITUX®) and fluorouracil-based chemotherapy for the treatment of adult patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation based on...
PARIS, FRANCE, 13 JANUARY 2026 – Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for IPN60340 in combination with venetoclax and azacitidine (Ven-Aza) in first line unfit acute myeloid leukemia, an aggressive blood cancer affecting older adults. IPN60340...