FDA grants rare pediatric disease designation to Summit Therapeutics’ DMD drug

Having been granted fast track status recently, ezutromid has now been awarded rare pediatric disease designation.

Ezutromid is a utrophin modulator with a potential disease modifying treatment for all patients with the fatal muscle wasting disease DMD.

The FDA defines rare pediatric disease as a disease that primarily affects people aged from birth to 18 years and affects less than 200,000 individuals in the US.

Through the rare pediatric disease designation, a sponsor can receive a voucher that allows a priority review for another product.

The voucher may only be used once, but may be sold or transferred for several times.

Summit said it may not get a voucher as the program is set to expire next month although it notes the possibility it may be extended again by the US Congress.

Summit Therapeutics CEO Glyn Edwards said: "Rare Pediatric Disease designation builds upon the Fast Track and Orphan Drug designations which the FDA has already awarded to ezutromid, recognizing a significant unmet medical need in the treatment of DM.

"We plan to leverage these regulatory advantages in the continued clinical development of ezutromid, which is currently in a Phase 2 clinical trial called PhaseOut DMD, to bring ezutromid to patients in need as quickly as possible."

DMD is a fatal genetic disease that occurs in neraly 1 in 5,000 males with one third of cases arising in boys with no familial history of the disease.

The PhaseOut DMD trial intends to provide proof of concept for ezutromid and utrophin modulation by measuring muscle fat infiltration, as well as by measuring utrophin protein and muscle fibre regeneration in muscle biopsies.