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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 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.
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.