RARE Daily

FDA Grants Rare Pediatric Disease Designation to Sellas Therapy for Pediatric ALL

June 24, 2024

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Sellas Life Sciences’ SLS009, a highly selective CDK9 inhibitor, for the treatment of pediatric acute lymphoblastic leukemia.

Pediatric acute lymphoblastic leukemia (ALL), the most common cancer diagnosed in children, is a life-threatening disease with a high unmet medical need. Despite significant advances in the treatment of pediatric ALL, relapse continues to be the most common cause of treatment failure. There are patient subpopulations with high-risk and very high-risk features in need of less toxic therapies that would ultimately extend their long-term event-free survival (EFS) which remains around 50 percent for very high-risk groups. In clinical trials, SLS009 has demonstrated a very favorable safety profile with complete absence, to date, of any non-hematologic clinical higher-grade toxicities.

Rare Pediatric Disease designation is granted by the FDA for serious or life-threatening diseases that affect fewer than 200,000 people in the United States, and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If, in the future, a New Drug Application for SLS009 for the treatment of pediatric AML is approved by the FDA, Sellas might be eligible to receive a Priority Review Voucher (PRV) that could be redeemed to receive a priority review for any subsequent marketing application. PRVs may be used by the sponsor or sold to another sponsor for their use and have recently sold for approximately $100 million.

“This designation will significantly help expedite clinical development,” said Angelos Stergiou, president and CEO of Sellas.

Photo: Angelos Stergiou, president and CEO of Sellas


Stay Connected

Sign up for updates straight to your inbox.