Core Insights - Longeveron Inc. supports the passing of the Mikaela Naylon Give Kids a Chance Act, which reauthorizes the Rare Pediatric Disease Priority Review Voucher Program, incentivizing the development of treatments for rare pediatric diseases [2][3]. Company Overview - Longeveron Inc. is a clinical stage biotechnology company focused on developing cellular therapies for life-threatening, rare pediatric and chronic aging-related conditions [2][9]. - The company's lead investigational product is laromestrocel (Lomecel-B™), an allogeneic mesenchymal stem cell therapy aimed at addressing unmet medical needs in various disease areas, including Hypoplastic Left Heart Syndrome (HLHS), Alzheimer's disease, and Pediatric Dilated Cardiomyopathy [9]. Legislative Impact - The Mikaela Naylon Give Kids a Chance Act allows companies to receive a priority review voucher upon FDA approval of a therapy for rare pediatric diseases, which can be used for expedited review of future therapies or sold [3][8]. - Since August 2024, these vouchers have been sold for $150-200 million each, with over 60 vouchers awarded for 40 pediatric diseases since the program's inception [3]. Clinical Development - Longeveron is currently conducting the ELPIS II Phase 2b clinical trial for laromestrocel in children with HLHS, enrolling 40 pediatric patients across twelve leading treatment institutions [5]. - Top-line results from the ELPIS II trial are expected in the third quarter of 2026, and if positive, the company plans to submit a Biological License Application (BLA) for full approval [5]. Unmet Medical Need - HLHS is a severe congenital heart defect with a high mortality rate, where only 50% of affected infants survive to adolescence despite current treatment options [4][6]. - There is a significant unmet medical need to improve right ventricular function in HLHS patients to enhance both short-term and long-term outcomes [6].

Atossa's Rare Pediatric Disease Designation for (Z)-endoxifen in neuromuscular diseases qualifies for a future PRV upon FDA approval About Rare Pediatric Disease Designation The FDA's Rare Pediatric Disease designation is reserved for serious or life-threatening diseases that primarily affect individuals from birth to 18 years old and that meet the definition of a rare disease or condition within the meaning of Section 526 of the Federal Food, Drug & Cosmetic Act ("FD&C Act"). Drugs granted RPD designation ...