Welcome to our dedicated page for Biomarin Pharmaceutical news (Ticker: BMRN), a resource for investors and traders seeking the latest updates and insights on Biomarin Pharmaceutical stock.
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 (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.
BioMarin (Nasdaq:BMRN) announced that the U.S. FDA has accepted its supplemental New Drug Application (sNDA) seeking full approval of VOXZOGO (vosoritide) for children with achondroplasia, and set a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.
According to BioMarin, the sNDA is supported by long-term safety and efficacy data from three ongoing studies (111-205, 111-208, 111-302), including adult height and additional clinical outcomes such as body proportionality and arm span. The submission is intended to fulfill the postmarketing requirement for converting VOXZOGO’s 2021 accelerated approval to traditional approval, backed by what the company describes as the longest efficacy and safety data package for a medicine studied in achondroplasia.
BioMarin (Nasdaq:BMRN) reported new VOXZOGO (vosoritide) and BMN 333 data at ENDO 2026. A three-year Phase 2 extension in 13 children with hypochondroplasia showed sustained gains in annualized growth velocity and height SDS, with a favorable safety profile.
Phase 1 results for investigational BMN 333 in healthy adults supported weekly dosing for achondroplasia, with over 13-fold higher free CNP exposure than another long-acting CNP agent and no dose-limiting toxicities. BioMarin plans a VOXZOGO sNDA in Q3 2026 and has begun a registration-enabling Phase 2/3 BMN 333 trial.
BioMarin (Nasdaq:BMRN) reported positive Phase 3 CANOPY-HCH-3 results for VOXZOGO (vosoritide) in children with hypochondroplasia. The study met its primary endpoint, showing an LS mean difference in annualized growth velocity of +2.33 cm/year versus placebo at week 52 (p<0.0001).
VOXZOGO also led to statistically significant gains in standing height, height Z-score (both p<0.0001) and arm span (p=0.004). Safety was consistent with the established achondroplasia profile, with no new safety signals. A sNDA to the FDA is planned for Q3 2026, followed by EMA and other global filings.