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COPENHAGEN - Ascendis Pharma A/S (NASDAQ:ASND), a biopharmaceutical company with a market capitalization of $9.8 billion, announced today that the U.S. Food and Drug Administration (FDA) has accepted for priority review its New Drug Application (NDA) for TransCon CNP (navepegritide), a treatment for children with achondroplasia. The FDA has set a goal date of November 30, 2025, to complete its review of the drug. According to InvestingPro data, the company maintains an impressive 85.3% gross profit margin despite being in its growth phase.
TransCon CNP is a once-weekly administered prodrug designed to treat achondroplasia by providing continuous exposure of C-type natriuretic peptide (CNP) to receptors on tissues, including growth plates and skeletal muscle. Achondroplasia is a rare genetic condition that causes various complications beyond skeletal dysplasia, affecting over 250,000 individuals globally.
Clinical trials have shown TransCon CNP to significantly improve annualized growth velocity, the primary endpoint, over placebo. Additionally, it demonstrated multiple benefits such as better lower limb alignment, spinal canal dimensions, muscle strength, and growth. These results come with a safety and tolerability profile similar to placebo. The company’s revenue has grown by 12.06% over the last twelve months, reflecting its expanding pipeline potential. InvestingPro analysis reveals several additional insights about the company’s financial health and growth prospects, with 6 key ProTips available to subscribers.
The FDA’s priority review status is granted to drugs that may offer significant improvements in the treatment, prevention, or diagnosis of serious conditions. Ascendis Pharma has reported that the FDA is not planning to hold an advisory committee meeting to discuss the application at this time.
Janet Legare, M.D., Professor of Pediatrics at the University of Wisconsin School of Medicine and Public Health, expressed encouragement over the FDA’s decision, highlighting the potential for addressing the serious complications associated with achondroplasia.
Chandler Crews, Founder of The Chandler Project, echoed the sentiment, emphasizing the urgent need for meaningful treatment options for those living with the condition.
Ascendis Pharma is a global biopharmaceutical company that focuses on applying its TransCon technology to develop therapies for unmet medical needs. This announcement is based on a press release statement from Ascendis Pharma. Analysts maintain a strong buy consensus on the stock with a potential 31% upside from current levels. For comprehensive analysis and detailed financial metrics, investors can access the full Pro Research Report available on InvestingPro, which provides deep-dive analysis of this and 1,400+ other US stocks.
In other recent news, Ascendis Pharma has reported impressive first-quarter results, with revenue from Yorvipath™ reaching €44.7 million, which was approximately 50% higher than both JPMorgan’s estimates and consensus figures. Following these results, JPMorgan increased its price target for Ascendis Pharma from $200 to $245, maintaining an Overweight rating on the stock. The firm expressed confidence in Yorvipath™’s growth potential, revising its worldwide sales forecasts for the product upwards by 27-53% for the years 2025 to 2030. Additionally, Ascendis Pharma announced positive outcomes from its ApproaCH Trial, which focused on TransCon CNP for treating achondroplasia in children, showing significant improvements in growth and bone structure without serious adverse events. Stifel reiterated its Buy rating for Ascendis Pharma, with a price target of $212, citing optimism about the company’s progress with Yovipath and strategies to enhance patient access. Furthermore, the Phase 2 PaTH Forward Trial for TransCon PTH demonstrated sustained benefits in treating hypoparathyroidism, with 98% of patients maintaining normal serum calcium levels. Cantor Fitzgerald also maintained its Overweight rating with a $200 target, highlighting the potential impact of upcoming COACH trial results on Ascendis Pharma’s valuation. These developments reflect Ascendis Pharma’s ongoing efforts to advance its product pipeline and address unmet medical needs.
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