BioMarin Pharmaceutical Inc.

BioMarin Enters Into Exclusive Worldwide License Agreement With GC Biopharma and Hanmi Pharm for Investigational Next-Generation Enzyme Replacement Therapy, Adding a Second Potential Treatment for Fabry Disease

BioMarin licensed worldwide rights (excluding Korea) to HM15421/GC1134A, a long-acting subcutaneous enzyme replacement therapy for Fabry disease, from GC Biopharma and Hanmi Pharm. BioMarin will pay $70 million upfront with up to $405 million more in development and commercial milestones plus tiered royalties; the candidate is in a Phase 1/2 trial and holds FDA Orphan Drug Designation. The deal adds a second potential Fabry franchise asset alongside GALAFOLD, extending BioMarin's rare-disease pipeline following its earlier Amicus acquisition.

•3d ago

BioMarin Presents New Data Advancing Understanding of Achondroplasia and Hypochondroplasia Treatment at the American Society for Bone and Mineral Research Annual Meeting

BioMarin will present data from 13 studies at the ASBMR 2026 Annual Meeting, headlined by new imaging results showing VOXZOGO's effect on foramen magnum and craniocervical junction growth in children with achondroplasia, including infants. The company recently submitted an sNDA to the FDA for VOXZOGO in hypochondroplasia; if approved, it would be the first targeted therapy for that condition, with a potential 2027 launch. BioMarin estimates roughly 14,000 treatable children with hypochondroplasia in its global footprint. Data on long-acting follow-on BMN 333 and the Phase 3 ENERGY 3 study of BMN 401 in ENPP1 deficiency round out the pipeline showcase. The release is scientific-reputation building ahead of the Feb. 28, 2027 PDUFA date for VOXZOGO's full-approval sNDA rather than a regulatory decision.

•23d ago

BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress

BioMarin presented five-year Phase 3 open-label extension data for POMBILITI + OPFOLDA in late-onset Pompe disease at the World Muscle Society Congress in Hiroshima. The 82-participant analysis showed durable motor function and relative stabilization of pulmonary function, with ERT-naive patients gaining 10.5% in percent-predicted six-minute walk distance and no new safety signals. BioMarin also shared Phase 1/2 data for nivudirsen (BMN 351) in Duchenne muscular dystrophy, showing near full-length dystrophin expression and favorable tolerability. The data are supportive of the commercial franchise but incremental, as POMBILITI + OPFOLDA is already approved and marketed.

•0mo ago

BioMarin Announces Positive Results of Phase 3 VOXZOGO® (vosoritide) Study in Hypochondroplasia Published in NEJM Evidence

BioMarin's Phase 3 CANOPY-HCH-3 study of VOXZOGO (vosoritide) in children with hypochondroplasia met its primary endpoint, showing a statistically significant improvement in annualized growth velocity versus placebo after 52 weeks (LS mean difference of 2.33 cm/year, p<0.0001). Secondary endpoints also reached significance: standing height improved by 2.35 cm, height Z-score by 0.39 SD, and arm span by 1.03 cm, with a safety profile consistent with the drug's established use in achondroplasia and no treatment-related serious adverse events. The data, published in NEJM Evidence and presented at ESPE 2026, support a recently filed sNDA to expand VOXZOGO's label to hypochondroplasia — a population BioMarin estimates at roughly 14,000 eligible children in its global footprint; approval would make it the first targeted therapy for the condition, with a potential 2027 launch. Separately, an sNDA for full approval of VOXZOGO in achondroplasia is under FDA review with a PDUFA target action date of Feb. 28, 2027.

•1mo ago

BioMarin Announces Close of the Alesta Therapeutics Acquisition

BioMarin completed its acquisition of Alesta Therapeutics, adding the oral small molecule ALE1 to its clinical pipeline for the treatment of hypophosphatasia (HPP). ALE1 is a potential first-in-class therapy currently in a Phase 1/2a trial assessing safety and tolerability in healthy volunteers and adults with HPP. Prior to the close, Alesta spun out all non-ALE1 assets to a new entity, leaving BioMarin with the skeletal condition asset as a wholly owned subsidiary.

•2mo ago

BioMarin to Acquire Alesta Therapeutics to Gain ALE1, a Potential First Oral Therapy for Hypophosphatasia, Adding an Important Clinical Program to BioMarin's Pipeline

BioMarin announced a definitive agreement to acquire Alesta Therapeutics for $275 million upfront, plus up to $215 million in milestone payments, to secure ALE1, a Phase 1/2a oral therapy for hypophosphatasia (HPP). The acquisition is expected to close this quarter and will be funded using cash on hand, with Alesta spinning out non-ALE1 assets and no employees transferring to BioMarin. ALE1 targets a central disease metabolite to treat the rare genetic bone disease HPP and fits strategically within BioMarin's Skeletal Conditions Business Unit.

•2mo ago

BioMarin Reports Second Quarter 2026 Financial and Operating Results

BioMarin reported Q2 2026 total revenue of $990 million, a 20% increase year-over-year, driven by strong demand for VOXZOGO and the initial contributions from the acquired Amicus portfolio including GALAFOLD and POMBILITI + OPFOLDA. The company raised its full-year 2026 guidance, now projecting total revenues between $3.875 billion and $3.925 billion and Non-GAAP diluted EPS between $4.90 and $5.10, up from prior ranges. VOXZOGO revenue guidance was specifically increased to at least $1 billion, while management identified approximately $280 million in GAAP cost synergies from the Amicus integration expected to be fully realized by 2028.

•2mo ago

BioMarin and n-Lorem Foundation Enter Early Research Collaboration to Develop Potential First-in-Disease Medicine for Newly Identified ReNU Syndrome

BioMarin and n-Lorem Foundation entered a strategic collaboration to develop a first-in-disease antisense oligonucleotide medicine for ReNU syndrome, a rare neurodevelopmental condition. The disease is estimated to affect approximately 100,000 people globally and currently has no approved treatments addressing the underlying genetic cause. The partnership combines BioMarin's genetic medicine leadership with n-Lorem's antisense expertise, with BioMarin leading development for the wider community following preclinical studies.

•3mo ago

U.S. Food and Drug Administration Accepts BioMarin's Supplemental New Drug Application for Full Approval of VOXZOGO® (vosoritide) for Children With Achondroplasia

BioMarin announced that the FDA has accepted its supplemental New Drug Application for the full approval of VOXZOGO in children with achondroplasia. The FDA set a PDUFA target action date of Feb. 28, 2027, with the submission supported by long-term safety and efficacy data from three ongoing studies. Converting from accelerated to traditional approval would solidify VOXZOGO's market position by verifying clinical benefits such as adult height outcomes and body proportionality.

•3mo ago

BioMarin Announces New Three-Year VOXZOGO® (vosoritide) Data in Hypochondroplasia and BMN 333 Early Results at ENDO 2026

BioMarin presented three-year data showing sustained growth improvements and a favorable safety profile for VOXZOGO in children with hypochondroplasia, supporting a planned supplemental New Drug Application in the third quarter of 2026. In the Phase 2 extension study, mean height standard deviation score improved by 0.72 SD and annualized growth velocity increased significantly from baseline to year one. Early Phase 1 data for investigational BMN 333 demonstrated sustained exposure supporting a weekly dosing schedule for achondroplasia, with exposure levels increasing more than 13 times compared to other long-acting agents.

•4mo ago

BioMarin Announces Positive Phase 3 Pivotal Study Results for VOXZOGO® (vosoritide) in Children with Hypochondroplasia

BioMarin announced positive results from the pivotal Phase 3 CANOPY-HCH-3 study for VOXZOGO in children with hypochondroplasia. The study met its primary endpoint, showing a statistically significant annualized growth velocity improvement of 2.33 cm/yr versus placebo (p<0.0001), along with significant gains in standing height, height Z-score, and arm span. Safety findings were consistent with the established profile in achondroplasia with no new signals. BioMarin plans to submit a supplemental New Drug Application (sNDA) to the FDA in the third quarter of 2026, followed by submissions to the EMA and other regional authorities.

•5mo ago

BioMarin Reports First Quarter 2026 Financial and Operating Results

BioMarin reported Q1 2026 total revenue of $766 million, up 3% year-over-year, with Non-GAAP diluted EPS of $0.76. The company significantly raised full-year 2026 revenue guidance to a range of $3.825 billion to $3.925 billion, reflecting a 20% growth rate at the midpoint driven by the addition of GALAFOLD and POMBILITI + OPFOLDA from the Amicus acquisition. While Non-GAAP EPS guidance was slightly lowered to $4.85 to $5.05 due to acquisition costs, the company generated $221 million in operating cash flow and secured $3.7 billion in financing.

•5mo ago

BioMarin Completes Acquisition of Amicus Therapeutics

BioMarin completed the acquisition of Amicus Therapeutics in an all-cash transaction valued at approximately $4.8 billion, or $14.50 per share. The deal adds Galafold for Fabry disease and Pombiliti plus Opfolda for Pompe disease to BioMarin's commercial portfolio, along with U.S. rights to the Phase 3 asset DMX-200. BioMarin stated it will provide updated FY 2026 guidance during its Q1 earnings call scheduled for May 4, 2026.

•6mo ago