The FDA has granted accelerated approval to YUVIWEL (navepegritide), developed by Ascendis Pharma, as the first and only once-weekly treatment for children aged 2 and older with achondroplasia. The drug provides continuous systemic exposure to CNP over the weekly dosing interval and is expected to be commercially available in early Q2 2026. The approval was based on improved annualized growth velocity data from three randomized clinical trials.

BridgeBio Pharma announced positive Phase 3 trial results for infigratinib in treating achondroplasia (dwarfism) in children, showing superior annualized height velocity compared to placebo. The company plans to submit FDA and regulatory applications in the second half of 2026. The stock surged 6.92% to $78.41, nearing its 52-week high.

Ascendis Pharma announced strong 2025 financial results with YORVIPATH generating €477 million and SKYTROFA generating €206 million in revenue. The company expects TransCon CNP approval in the U.S. by February 2026 and outlined expansion plans across multiple product candidates and geographic markets. Strategic collaborations with Novo Nordisk, Eyconis, VISEN Pharmaceuticals, and Teijin Limited are advancing, with a planned $120 million share repurchase program for 2026.

Ascendis Pharma received notification from the FDA extending the PDUFA target action date for TransCon CNP by three months to February 28, 2026, after submitting additional information for their New Drug Application for treating children with achondroplasia.

BioMarin Pharmaceutical reported strong Q4 earnings, beating consensus estimates for both EPS and revenue. The company provided upbeat 2025 guidance, expecting sales of $3.1-$3.2 billion and EPS of $4.20-$4.40.

Ascendis Pharma granted Novo Nordisk an exclusive global license to develop, manufacture, and commercialize Novo Nordisk's proprietary products for metabolic and cardiovascular diseases using Ascendis' TransCon technology platform. The deal includes upfront, milestone, and sales-based payments for Ascendis.

BioMarin reaffirms its $4 billion revenue target for 2027 despite competition from Ascendis Pharma's recent trial data. The company is implementing a $500 million cost transformation program and expects to reach adjusted operating margins of 40% by 2026. Goldman Sachs analyst remains bullish on BioMarin's growth prospects.

Bausch & Lomb and Edgewise Therapeutics were among the top 7 mid-cap stock gainers last week, with Edgewise Therapeutics shares rocketing 50.19% after announcing positive topline data from a clinical trial, and Bausch & Lomb's parent company Bausch Health considering a sale.

Ascendis Pharma announced the pricing of a $300 million public offering of 2 million American Depositary Shares at $150 per share. The company plans to use the proceeds to support commercial activities, clinical development, and general corporate purposes.

Eyconis, Inc., a biopharmaceutical company focused on developing novel ophthalmology therapeutics, has appointed Dr. Mark J. Bachleda as its new CEO and Board member. Dr. Bachleda brings extensive industry experience and will lead the company's efforts to advance its lead drug candidate for age-related macular degeneration (AMD) towards an Investigational New Drug (IND) filing.

Ascendis Pharma's investigational drug TransCon CNP for achondroplasia, a rare form of dwarfism, demonstrated superior annualized growth velocity compared to placebo in a clinical trial. The company plans to submit marketing applications to the FDA and European regulators in 2025.

Ascendis Pharma's TransCon CNP demonstrated superior annualized growth velocity (AGV) compared to placebo in a pivotal trial for children with achondroplasia, a type of dwarfism. The treatment was generally well-tolerated and Ascendis plans to submit regulatory filings in the U.S. and Europe.