Actuate Therapeutics Receives FDA Rare Paediatric Disease Designation for Elraglusib in Ewing Sarcoma Treatment
Wednesday, November 13, 2024
Actuate Therapeutics has been granted a rare paediatric disease designation by the U.S. Food and Drug Administration (FDA) for its investigational treatment,
Ewing sarcoma is a rare, aggressive cancer that typically affects children and adolescents, primarily in bones or surrounding soft tissue.
Elraglusib is currently being evaluated in a Phase 1/2 trial, named Actuate-1902 (NCT 04239092). The open-label, multicentre study is assessing the safety and effectiveness of elraglusib in children with relapsed or refractory cancers, specifically EWS and similar small round cell sarcomas.
This therapy, a novel inhibitor targeting glycogen synthase kinase-3 beta (GSK-3β), is being developed to treat Ewing sarcoma (EWS), a challenging cancer that predominantly affects children and adolescents.
This FDA designation highlights the need for innovative treatments in Ewing sarcoma, a highly metastatic cancer originating in bone tissue, with peak incidence occurring around the age of 15.
EWS is the second most common primary malignant bone tumour among young people, with approximately 25% of new cases presenting with metastatic disease at the time of diagnosis—a factor significantly linked to poor survival rates.
So far, eight patients with relapsed/refractory EWS have been enrolled, receiving a combination of elraglusib with topotecan and cyclophosphamide.
Rare Paediatric Disease Designation is awarded by the FDA for serious conditions affecting fewer than 200,000 individuals in the U.S., with most cases impacting those under 18.
Should elraglusib ultimately receive FDA approval for treating EWS, Actuate may qualify for a Priority Review Voucher, which the company could either utilise for its own treatments or potentially sell for use by another firm.
Source: actuatetherapeutics.com
