BioMarin Pharmaceutical Inc. develops and commercializes medicines for rare genetic diseases, with recurring news centered on commercial portfolio performance, regulatory approvals, clinical data and business development. Company updates commonly reference therapies for achondroplasia, phenylketonuria, hemophilia, mucopolysaccharidosis and lysosomal storage diseases, including VOXZOGO, PALYNZIQ, GALAFOLD and POMBILITI + OPFOLDA.
BioMarin news also covers financial results, guidance, acquisition integration, debt financing and governance changes. Clinical and regulatory announcements focus on genetically defined conditions, skeletal disorders, enzyme-based therapies and investigational rare-disease programs such as DMX-200 for focal segmental glomerulosclerosis.
BioMarin Pharmaceutical (BMRN) entered an exclusive license agreement with GC Biopharma and Hanmi Pharm for investigational Fabry disease therapy HM15421/GC1134A.
The agreement grants rights worldwide excluding Korea, with a $70 million upfront payment, up to an additional $405 million in development and commercial milestone payments, and tiered royalties on net sales. The long-acting enzyme replacement therapy, administered under the skin, is in a Phase 1/2 trial. After that study, BioMarin will assume sole responsibility for subsequent clinical development, manufacturing and commercialization outside Korea; GC and Hanmi retain Korean commercialization rights. The candidate received FDA Orphan Drug Designation in 2024.
BioMarin Pharmaceutical (NASDAQ: BMRN) will host its third quarter 2026 financial results call and webcast on November 2, 2026.
The call begins at 4:30 p.m. ET. President and Chief Executive Officer Alexander Hardy will discuss the results and provide a general business update. A live audio webcast will be available on BioMarin's investor website, with a replay archived for one week following the call.
BioMarin Pharmaceutical (Nasdaq: BMRN) will present data from 13 studies on skeletal conditions at the ASBMR annual meeting in Boston. A post-hoc analysis, performed after the trial, examines VOXZOGO's effect on growth of the foramen magnum and craniocervical junction, regions at the skull base, in children with achondroplasia, including infants. BioMarin describes the imaging findings as encouraging; numerical results are not provided.
BioMarin submitted a supplemental FDA application for hypochondroplasia and reports European and other regional submissions remain on track, with a potential 2027 launch if approved. Presentations also cover Phase 3 hypochondroplasia results, preclinical BMN 333 research and 52-week Phase 3 BMN 401 results in children with ENPP1 deficiency. A separate VOXZOGO application covering long-term outcomes is under FDA review, with a Feb. 28, 2027 target action date.
BioMarin Pharmaceutical (BMRN) named Cara Bauer Executive Vice President and Chief People Officer, effective Oct. 5, 2026, succeeding retiring Amy Wireman. Bauer will oversee the company's Human Resources organization. She joins from Kyverna Therapeutics, where she served as Chief Human Resources Officer, and brings more than 25 years of experience across the biopharmaceutical and technology industries.
Bauer previously served as global head of human resources at Kite, a Gilead company, and held human resources leadership roles at Riot Games, Netflix, Amgen and Novo Nordisk. Wireman will remain at BioMarin in an advisory capacity to support the transition.
BioMarin (BMRN) presented long-term Phase 3 extension results for POMBILITI + OPFOLDA in late-onset Pompe disease at the World Muscle Society congress.
The analysis covered 82 participants receiving continuous treatment from PROPEL baseline. Mean change in predicted six-minute walking distance was 0.7% in previously treated patients through year five and 10.5% in previously untreated patients through year 4.5. Mean change in predicted forced vital capacity, a lung-function measure, was -2.8% and -4.4%, respectively. No new safety signals emerged, but 51.2% experienced treatment-related adverse events. Five participants stopped treatment for safety reasons, including two with serious treatment-related events.
BioMarin also reported that investigational nivudirsen produced near full-length dystrophin expression and was generally well tolerated in an ongoing Phase 1/2 Duchenne muscular dystrophy study.
BioMarin Pharmaceutical (BMRN) has appointed Robert Plenge, M.D., Ph.D., Executive Vice President and Chief Research Officer of Bristol Myers Squibb, to its Board of Directors, effective September 10, 2026.
Plenge leads Bristol Myers Squibb’s research across nine global sites and joined the company in 2019 through its acquisition of Celgene, where he was vice president of the immunology and inflammation portfolio in research and early development. His prior roles include vice president and head of translational medicine at Merck, assistant professor of medicine at Harvard Medical School, and associate member of the Broad Institute. He also serves on the boards of Alltrna and the PhRMA Foundation. BioMarin’s CEO said Plenge’s expertise in human genetics and drug discovery will support the company’s efforts to expand its pipeline for rare genetic diseases.
BioMarin (BMRN) reported that Phase 3 CANOPY‑HCH‑3 data for VOXZOGO (vosoritide) in children with hypochondroplasia were published in NEJM Evidence and presented at the 2026 ESPE meeting.
Treatment with VOXZOGO met the study’s primary endpoint, showing a statistically significant improvement in annualized growth velocity versus placebo after 52 weeks, with an LS mean difference of 2.33 cm/year (p<0.0001). VOXZOGO also improved standing height (LS mean difference 2.35 cm; p<0.0001), height Z‑score (LS mean difference 0.39 standard deviation score; p<0.0001) and arm span (LS mean difference 1.03 cm; p=0.0082). Quality‑of‑life measures showed numerical improvements and longer‑term follow‑up is ongoing.
The safety profile was consistent with prior VOXZOGO studies in achondroplasia, with most adverse events mild and no treatment‑related serious events identified. BioMarin has submitted an sNDA to the FDA, is preparing submissions to EMA and other regulators, and targets a potential 2027 launch if approved.
BioMarin (BMRN) will present at several healthcare investor conferences in September 2026, including events hosted by Wells Fargo, Cantor, Citi and Morgan Stanley. Presentations take place between September 9 and 14, 2026, at specified times in Eastern Time.
Live audio webcasts and limited-time archives of BioMarin’s remarks will be accessible through the company’s investor relations website at https://investors.biomarin.com/. The company describes itself as a global rare disease biotechnology firm with nine commercial therapies and an active clinical and preclinical pipeline.
BioMarin (Nasdaq: BMRN) announced the closing of its previously disclosed acquisition of Alesta Therapeutics, making Alesta a wholly owned subsidiary as of September 1, 2026. The transaction follows a definitive agreement signed on August 18, under which BioMarin sought to acquire Alesta’s lead clinical-stage asset, ALE1.
The deal adds ALE1, an oral small molecule in an ongoing Phase 1/2a trial for hypophosphatasia (HPP), to BioMarin’s clinical pipeline and skeletal conditions portfolio. The study is evaluating safety, tolerability and pharmacokinetics/pharmacodynamics in healthy volunteers and adults with HPP, a rare genetic bone disease linked to mutations in the ALPL gene. Before closing, Alesta spun out all non‑ALE1 assets into a new entity, so BioMarin’s acquisition focuses on the HPP program. According to BioMarin, this move aligns with its strategy in genetically defined rare diseases, where it already markets nine therapies and maintains a broad clinical and preclinical pipeline.
BioMarin (Nasdaq: BMRN) entered into binding global settlement terms with Ascendis Pharma A/S, resolving all pending patent-related and ancillary disputes concerning Ascendis's drug Yuviwel, including a Section 337 investigation before the U.S. International Trade Commission and litigation in multiple countries.
Ascendis will pay BioMarin a 20% royalty on net Yuviwel sales in the U.S., retroactive to the first commercial sale, and 18% on net sales in the EU, Brazil and South Korea through May 2030. The settlement includes a license to BioMarin patents related to Yuviwel for all current and potential indications, as well as combination use, and recognizes the value of BioMarin’s CNP technology and VOXZOGO.