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) 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.
BioMarin (Nasdaq: BMRN) entered a definitive agreement to acquire Alesta Therapeutics to obtain ALE1, Alesta’s lead clinical-stage asset for hypophosphatasia (HPP). BioMarin will pay $275 million upfront and up to $215 million in development and regulatory milestone payments, funded with cash on hand.
Alesta will spin out all non‑ALE1 assets to a new entity, and all Alesta employees will transfer to that spinout, so no employees join BioMarin. ALE1, a potential first oral therapy for HPP, is in an ongoing Phase 1/2a trial. The deal, approved by both boards, is expected to close this quarter, subject to customary conditions, and is expected to be modestly dilutive to BioMarin’s 2026 results excluding upfront consideration.
BioMarin (NASDAQ: BMRN) will present at the Canaccord Genuity 46th Annual Growth Conference in Boston on Wednesday, August 12, 2026, at 8:00AM ET. A live audio webcast and a time-limited archived replay will be accessible via the company’s investor website.
BioMarin (NASDAQ:BMRN) reported second quarter 2026 total revenues of $990 million, up 20% year-over-year, driven mainly by newly acquired GALAFOLD and POMBILITI + OPFOLDA, VOXZOGO growth, and higher PALYNZIQ demand. Metabolic Conditions revenue rose 25% Y/Y to $695 million.
GAAP net income fell to $45 million from $241 million, and Non-GAAP income declined to $236 million from $282 million, reflecting Amicus acquisition-related costs, higher interest expense, and increased commercial and R&D spending. GAAP diluted EPS was $0.23 versus $1.23; Non-GAAP diluted EPS was $1.20 versus $1.44.
BioMarin raised full-year 2026 guidance, with total revenues now expected between $3.875–$3.925 billion, VOXZOGO revenues between $1.0–$1.05 billion, and Non-GAAP diluted EPS between $4.90–$5.10. The company identified about $280 million in GAAP cost reductions from the Amicus deal, targets gross leverage below 2.5x by mid-2027, and projects high-margin growth from GALAFOLD and POMBILITI + OPFOLDA.
Pipeline updates include positive Phase 3 hypochondroplasia data and an sNDA for VOXZOGO, Phase 1 data for BMN 333, addition of BMN 820, continued development of BMN 351, discontinuation of BMN 401, and an ASO collaboration with n-Lorem for ReNU syndrome.
BioMarin (NASDAQ: BMRN) announced that President and CEO Alexander Hardy will host a conference call and webcast on Thursday, August 6, 2026, at 4:30 p.m. ET to discuss second quarter 2026 financial results and provide a general business update.
Investors can join via phone using conference ID 3551298 or access a live audio webcast through the investor section of the BioMarin website. A replay will be available for one week.
BioMarin (Nasdaq: BMRN) and the nonprofit n-Lorem Foundation announced an early research collaboration and global exclusive license to develop a first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a rare neurodevelopmental disorder caused by variants in the RNU4-2 gene.
The partners will jointly perform preclinical studies and select a lead ASO candidate targeting the RNU4-2 (n.64_65insT) variant, which is estimated to represent about 75% of ReNU syndrome cases. According to BioMarin, the company will then lead development of the investigational medicine for the broader ReNU community, while n-Lorem will continue individualized trials for patients already accepted into its program.
ReNU syndrome, identified in 2024, is associated with cognitive, language and adaptive behavioral impairments and is projected to affect roughly 100,000 people globally. There are currently no approved medicines that address the underlying cause of this condition.