Biomarin Pharmaceutical (BMRN) Stock News

BioMarin (NASDAQ: BMRN) and Veeva Systems (NYSE: VEEV) announced a long-term enterprise agreement on January 8, 2026 to expand their existing relationship.

The partnership aims to increase speed, agility and operational efficiency in developing and commercializing therapies by further leveraging and providing strategic input into Veeva's software, data, services, and consulting products, and to optimize the healthcare professional and patient experience. Company leaders highlighted the collaboration's role in accelerating access to transformative medicines globally.

BioMarin (Nasdaq: BMRN) agreed to acquire Amicus Therapeutics for $14.50 per share, valuing the deal at approximately $4.8 billion. The transaction, approved by both boards, is expected to close in Q2 2026 subject to customary approvals. The deal adds marketed therapies Galafold and Pombiliti + Opfolda that generated $599 million in net product revenue over the past four quarters and resolves U.S. Galafold patent litigation with U.S. exclusivity expected through Jan 2037. BioMarin expects the acquisition to be accretive to Non-GAAP diluted EPS within 12 months and substantially accretive in 2027 and plans to finance with cash plus about $3.7 billion of debt.

BioMarin (NASDAQ: BMRN) said its management team will present at the Jefferies Global Healthcare Conference on Tuesday, November 18, 2025 at 10:30 AM GMT in London, England. An audio webcast will be available live and an archived version of the remarks will be posted on the company investor website for a limited time.

Investors can access the live webcast and archive at https://investors.biomarin.com/.

BioMarin (Nasdaq: BMRN) announced the FDA has accepted for Priority Review its supplemental biologics license application to expand PALYNZIQ (pegvaliase-pqpz) for adolescents aged 12–17 with phenylketonuria (PKU).

The sBLA is based on positive Phase 3 PEGASUS results showing statistically significant reductions in blood phenylalanine (Phe) versus diet alone. The FDA set a PDUFA target action date of Feb. 28, 2026. Data were presented at the 15th ICIEM in Kyoto, and BioMarin plans to seek a similar label expansion with the European Medicines Agency. PALYNZIQ is currently the first and only enzyme substitution therapy approved for adults with PKU.

BioMarin (NASDAQ: BMRN) reported Q3 2025 results and a corporate update on October 27, 2025. Total revenues were $776M (+4% vs Q3 2024) and YTD revenues reached $2,347M (+11% Y/Y). Year-to-date VOXZOGO revenue rose 24% Y/Y and Q3 VOXZOGO sales were $218M; PALYNZIQ grew 20% in Q3. GAAP net loss was $(31)M in Q3, driven primarily by a $221M acquired IPR&D charge from the Inozyme acquisition. Non-GAAP income fell to $22M in Q3 from $178M a year earlier. Cash and investments were about $1.99B and operating cash flow was $369M in Q3 ($728M YTD). The company reaffirmed VOXZOGO full-year guidance ($900M–$935M) and updated 2025 guidance, raising mid-point revenue while lowering Non-GAAP EPS and margin guidance to reflect the IPR&D charge. BioMarin plans to pursue options to divest ROCTAVIAN.

BioMarin (NASDAQ: BMRN) announced a conference call and webcast with Alexander Hardy, president and CEO, on Monday, October 27, 2025 at 4:30 p.m. ET to discuss third quarter 2025 financial results and provide a general business update.

Dial-in numbers are provided for U.S./Canada and international participants, and a live audio webcast will be available via the investor site at https://investors.biomarin.com/. A replay will be archived on the site for one week following the call.

BioMarin Pharmaceutical (Nasdaq: BMRN) presented new data from 14 studies at the ASBMR 2025 Annual Meeting, highlighting significant advances in bone health treatments. The key findings focus on VOXZOGO® (vosoritide) treatment outcomes in children with achondroplasia.

The Phase 2 CANOPY study demonstrated that children receiving VOXZOGO showed improved spinal morphology, with 57% experiencing reduced thoracolumbar kyphosis compared to 33% in the placebo group. Additionally, Phase 3 data revealed substantial growth benefits in post-puberty patients, with treated young men gaining 7.55 cm more and young women gaining 8.07 cm more in height compared to untreated individuals.

The company also presented research on ENPP1 deficiency, advancing BMN 401 as a potential first-in-disease enzyme therapy, with pivotal data expected in early 2026 and potential launch in 2027.

BioMarin (NASDAQ:BMRN) announced positive Phase 3 PEGASUS trial results for PALYNZIQ® in treating adolescents with phenylketonuria (PKU). The study demonstrated a 49.7% decrease in mean blood phenylalanine (Phe) levels compared to just 0.3% with diet alone.

Key achievements include: 51.6% of PALYNZIQ-treated patients achieved blood Phe levels ≤600 µmol/L, and 45.2% of participants achieved ≥50% reduction in blood Phe from baseline. The safety profile aligned with adult studies, though 5.6% experienced serious adverse events (anaphylaxis) leading to discontinuation.

BioMarin plans to submit these results to global health authorities in the second half of 2025 to expand PALYNZIQ's indication to include adolescent treatment.

BioMarin Pharmaceutical (NASDAQ: BMRN) has announced its participation in three major healthcare investor conferences in September 2025:

• Cantor Global Healthcare Conference on September 3 at 8:00 AM EST
• Wells Fargo Healthcare Conference on September 4 at 8:45 AM EST
• Morgan Stanley Global Healthcare Conference on September 9 at 11:30 AM EST

The company will provide live audio webcasts of the presentations through their investor relations website, with archived versions available for limited time after each event.