A BioMarin combination therapy helped adults with late-onset Pompe disease maintain walking distance over five years of continuous therapy. Respiratory function remained relatively stable, with no new safety signals identified.
Long-term data reinforce durable efficacy and safety of POMBILITI + OPFOLDA in late-onset Pompe disease SAN RAFAEL, Calif., Oct. 2, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data evaluating the efficacy and safety of POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) in late-onset Pompe disease (LOPD) were presented at the 31st Annual Congre...
EMERYVILLE, Calif.--(BUSINESS WIRE)---- $BMRN--Profluent, the frontier AI lab for biology, today announced a collaboration with BioMarin Pharmaceutical Inc. to design and optimize novel enzyme therapies for BioMarin's pipeline. Under the agreement, Profluent will apply its foundational AI models to generate novel potential drug candidates, and BioMarin will screen the results with the goal to e...
SAN RAFAEL, Calif., Sept. 14, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that the company has appointed Robert Plenge, M.D.
Detailed Phase 3 CANOPY-HCH-3 data in children living with hypochondroplasia also featured in a late-breaking oral presentation at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting VOXZOGO demonstrated statistically significant improvements across multiple measures of growth, including annualized growth velocity, standing height, height Z-score and arm span BioMarin r...
Acquisition adds ALE1, a potential first oral therapy for hypophosphatasia to clinical pipeline SAN RAFAEL, Calif., Sept. 1, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) said today that it completed the previously announced agreement to acquire Alesta Therapeutics.
Agreement Resolves all Pending Patent-Related Proceedings Between BioMarin and Ascendis SAN RAFAEL, Calif., Aug. 30, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced that it has entered into binding terms with Ascendis Pharma A/S, resolving the patent and ancillary disputes pending globally, including before the U.S. International Trade Commission (ITC) concernin...
BioMarin will acquire Alesta Therapeutics for $275 million upfront, and up to an additional $215 million based on milestones. Alesta Therapeutic's lead drug is a potential treatment for hypophosphatasia (HPP).
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