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) 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.
BioMarin (Nasdaq:BMRN) reported top-line Phase 3 ENERGY 3 results for BMN 401 in children aged 1-12 with ENPP1 deficiency. One co-primary endpoint was met, with statistically significant increases in plasma PPi through week 52 versus control, but no corresponding improvement in RGI-C or secondary clinical endpoints.
BMN 401 was generally well-tolerated with no new safety signals, and BioMarin is evaluating next steps. Detailed data will be shared at a future medical meeting.
4D Path (BAX) announced the first close of a strategic financing to accelerate development of treatment‑predictive biomarkers for solid tumors and partnerships with biopharma. The company appointed Katherine Vega Stultz as Board Chair and added Robert J. Hombach to the board to strengthen clinical, commercial and financial leadership.
The release highlights pan‑cancer potential and prior clinical relevance across breast, ovarian, colorectal and skin cancers, and frames the board changes plus financing as catalysts for accelerated growth and partnership activity.
BioMarin (NASDAQ: BMRN) reported Q1 2026 total revenues of $766 million and raised full‑year 2026 revenue guidance to $3.825B–$3.925B, reflecting a midpoint growth rate of ~20% year‑over‑year following the Amicus acquisition and addition of GALAFOLD and POMBILITI + OPFOLDA.
GAAP net income fell to $106M and GAAP diluted EPS was $0.54; non‑GAAP income and EPS also declined. Company secured ~$3.7B financing and ended Q1 with ~$2B cash.
BioMarin (Nasdaq: BMRN) presented data at PES 2026 showing long-term VOXZOGO (vosoritide) treatment improved arm span Z-scores, maintained arm span-to-height proportionality, and preserved bone mineral density while increasing bone mineral content in children with achondroplasia.
Children starting treatment after age 5 had mean standing height gains of 10.60 cm at six years and 13.59 cm at eight years versus untreated cohorts; topline Phase 3 hypochondroplasia results are expected in 1H 2026.
BioMarin (Nasdaq: BMRN) completed its acquisition of Amicus Therapeutics on April 27, 2026, in an all-cash deal at $14.50 per share, valuing the transaction at approximately $4.8 billion. The deal adds commercial therapies Galafold (migalastat) and Pombiliti + Opfolda, plus U.S. rights to Phase 3 candidate DMX-200. BioMarin plans to provide updated FY 2026 guidance on its Q1 earnings call on May 4, 2026.
BioMarin (NASDAQ: BMRN) will host a conference call and webcast on Monday, May 4, 2026 at 4:30 p.m. ET to discuss first quarter 2026 financial results and provide a general business update. Alexander Hardy, president and CEO, will host the call.
Dial-in and replay numbers are provided and a live audio webcast and one-week replay will be available via the investor site: https://investors.biomarin.com/.
BioMarin (Nasdaq: BMRN) announced a Board leadership transition: Ian T. Clark was elected Chair, subject to his election at the Annual Meeting on June 2, 2026, and will succeed Richard A. Meier, who is retiring after two decades on the Board.
Mr. Clark joined the Board in 2025 and brings executive and board experience including prior service as Genentech CEO; the change becomes effective immediately following the Annual Meeting.
BioMarin (Nasdaq: BMRN) reported new data showing early treatment with VOXZOGO (vosoritide) improves proportionality, arm span and BMI in children with achondroplasia, with durable multi-year gains. Key findings include a 4.7 cm additional height gain after four years and ULBR improvement to −0.53 by year four. Real-world data cover >5,000 infants and children across 50+ countries and align with clinical-trial results supporting earlier treatment.
BioMarin (Nasdaq: BMRN) announced FDA approval of PALYNZIQ (pegvaliase-pqpz) for adolescents aged 12 and older with phenylketonuria (PKU).
Approval is based on Phase 3 PEGASUS data: mean blood Phe fell from 1025 to 567 µmol/L at Week 72 on PALYNZIQ versus 1029 to 973 µmol/L on diet alone, with a treatment difference of -409 µmol/L (95% CI -579, -240). Common adverse reactions (≥20%) included injection site reactions, arthralgia, headache and hypersensitivity reactions.