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Investing.com -- Shares of Ascendis Pharma A/S (NASDAQ:ASND) climbed 5.8% today after the company announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review its New Drug Application for TransCon CNP. The treatment, aimed at children with achondroplasia, has been granted a Prescription Drug User Fee Act (PDUFA) goal date of November 30, 2025.
The FDA’s decision to prioritize the review of TransCon CNP, an investigational prodrug of C-type natriuretic peptide (CNP), follows clinical trials demonstrating significant benefits in annualized growth velocity compared to placebo. These benefits extend beyond linear growth, encompassing improvements in lower limb alignment, spinal canal dimensions, muscle strength, and overall growth. The safety and tolerability profile of TransCon CNP was reported to be similar to that of a placebo, further bolstering the positive market response.
Ascendis Pharma’s clinical trials have marked the first instance of a treatment showing improvements beyond linear growth at 52 weeks when compared to a placebo. The company’s Executive Vice President of Endocrine & Rare Disease Medical (TASE:BLWV) Sciences and Chief Medical Officer, Dr. Aimee Shu, expressed the urgency felt by patients and physicians for a meaningful treatment option to address the complications of achondroplasia. With the FDA not currently planning to hold an advisory committee meeting on the application, Ascendis Pharma anticipates a streamlined review process.
The market’s reaction reflects optimism surrounding the potential approval of TransCon CNP, which, if sanctioned, could represent a significant advancement in the treatment of achondroplasia. The priority review designation by the FDA is reserved for medicines that could notably improve the safety or efficacy of treating, preventing, or diagnosing serious conditions.
Ascendis Pharma is now preparing to work closely with the FDA during the review process in hopes of making TransCon CNP available to patients as swiftly as possible. The company’s commitment to addressing profound medical needs in achondroplasia is echoed by the Founder of The Chandler Project, Chandler Crews, who highlighted the potential for therapies like TransCon CNP to improve health outcomes significantly.
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