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Mkt Cap

$10.59B

Volume

1.96M

Next Earnings

October 26th

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BMRNBioMarin Pharmaceuticals Inc

BMRN

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Mkt Cap

$10.58B

Volume

1.96M

Next Earnings

October 26th

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FDA Grants Priority Review to BridgeBio's Dwarfism Drug FilingBridgeBio Pharma announced FDA acceptance of its regulatory filing for infigratinib, an oral drug to treat achondroplasia in children, with priority review and a decision expected by February 4, 2027. The drug demonstrated 2.1 cm/year greater height growth than placebo in Phase III trials. If approved, it would be the first oral therapy for achondroplasia, differentiating it from injectable competitors. BridgeBio also has two other near-term regulatory catalysts (BBP-418 and encaleret) that could diversify its revenue beyond its current marketed product Attruby.Zacks Investment Research · Oct 7, 2026

Enzyme Replacement Therapy Market to Grow to USD 27.90 Billion by 2035 at a CAGR of 8.59% - SNS InsiderThe global Enzyme Replacement Therapy (ERT) market is projected to grow from USD 12.24 billion in 2025 to USD 27.90 billion by 2035, with a CAGR of 8.59%. Growth is driven by early identification of rare lysosomal storage disorders, increased newborn screening, orphan drug incentives, and expanding healthcare access. North America leads with 38.76% market share, while Asia-Pacific shows the fastest growth at 9.43% CAGR. Imiglucerase dominates products, and Gaucher disease represents the largest therapeutic condition.GlobeNewswire Inc. · Sep 25, 2026

BioMarin Stops Voxzogo Development for Noonan SyndromeBioMarin Pharmaceutical announced it is discontinuing development of Voxzogo for Noonan syndrome due to study feasibility and treatment landscape concerns, though not due to safety or efficacy issues. Despite this setback, Voxzogo generated $472 million in first-half 2026 sales (up 9% YoY) and is on track to achieve blockbuster status with expected 2026 sales of $1.00-$1.05 billion. The drug faces increasing competition from Ascendis Pharma's Yuviwel and BridgeBio's infigratinib, though BioMarin secured a royalty-bearing settlement with Ascendis.Zacks Investment Research · Sep 16, 2026

How BioMarin Turned a Competitive Threat Into a Royalty-Bearing AssetBioMarin settled a patent dispute with Ascendis Pharma over the achondroplasia drug Yuviwel, converting a competitive threat into a royalty stream. Under the agreement, BioMarin will receive 20% royalties on U.S. Yuviwel sales and 18% in the EU, Brazil, and South Korea through May 2030. While this monetizes competition, BioMarin still faces market pressure from Yuviwel and potential future competitors like BridgeBio's infigratinib.Zacks Investment Research · Sep 3, 2026

Fabry Disease Treatment Market 2026-2031 - Growth Driven by Increasing Adoption of Enzyme Replacement TherapyThe global Fabry disease treatment market is projected to grow from USD 2.63 billion in 2025 to USD 4.09 billion by 2031, with a CAGR of 7.63%. Growth is driven by expanded screening identifying more patients, advances in gene and mRNA therapies, and sustained enzyme replacement therapy demand. However, high treatment costs remain a significant barrier to patient access, particularly in emerging markets. Asia-Pacific is expected to record the fastest growth at 8.28% CAGR.GlobeNewswire Inc. · Aug 25, 2026

Duchenne Muscular Dystrophy Clinical Trial Pipeline: DelveInsight Highlights Major Advances, Transformative Therapies, and 75+ Leading Players Wheeling the Therapeutics LandscapeDelveInsight's report highlights a robust DMD clinical trial pipeline with 70+ active players developing 75+ pipeline drugs. Key companies including Capricor, Solid Biosciences, Roche, Dyne Therapeutics, and BioMarin Pharmaceutical are advancing promising therapies targeting dystrophin expression and gene transfer. Approximately 6+ drugs are in late-stage development, with recent FDA designations granted to multiple candidates including AAV-SERCA2a and PBGENE-DMD, signaling strong momentum in addressing the high unmet need for disease-modifying DMD treatments.GlobeNewswire Inc. · May 26, 2026

BioMarin Reveals Dwarfism Study Data, Stock SoarsBioMarin Pharmaceutical's Phase 3 CANOPY-HCH-3 study for VOXZOGO demonstrated a statistically significant 2.33 cm/year increase in annualized growth velocity for hypochondroplasia treatment, meeting its primary endpoint. The company plans to submit a supplemental New Drug Application to the FDA in Q3 2026. The stock surged 8.05% to $54.24 on the positive results, with analysts maintaining a Buy rating and average price target of $87.47.Benzinga · May 21, 2026

CIBRA Capital Makes a Big Merger Arbitrage Bet On Amicus Therapeutics (FOLD)CIBRA Capital significantly increased its stake in Amicus Therapeutics by 1.48 million shares worth $21.17 million during Q1 2026, making it the fund's largest position at 11.78% of AUM. The investment is a merger arbitrage bet on BioMarin Pharmaceutical's $14.50 per share acquisition offer, with potential $0.20 per share profit if the deal completes as expected.The Motley Fool · Apr 25, 2026

Biotech Stock Up 372% Gets Sold as New Pick Rises 40% in 2026Boone Capital Management liquidated its entire $13.57 million stake in Cogent Biosciences (945,042 shares) in Q4 2026, despite the stock surging 372% over the past year. The fund reallocated capital to earlier-stage biotech opportunities like TYRA, which has gained 40% in 2026, suggesting a strategic shift toward higher-risk, higher-upside clinical-stage investments.The Motley Fool · Mar 20, 2026

This Investor Sold $104 Million of Nuvalent Stock Amid Cancer Drug Developer's Nearly 30% RallyVestal Point Capital sold 1.054 million shares of Nuvalent (NUVL) for approximately $103.93 million in Q4, reducing its position to less than 1% of assets under management. Despite the sale, Nuvalent shares have rallied nearly 30% over the past year and the company is well-positioned financially with $1.4 billion in cash, awaiting potential FDA decisions on its cancer drug candidates later in 2026.The Motley Fool · Mar 17, 2026

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