RARE Daily

FDA Grants Rare Pediatric Drug Designation for IPS Heart’s Stem Cell Therapy to Treat Cardiomyopathy Due to Danon Disease

July 6, 2023

Rare Daily Staff

The U.S. Food and Drug Administration awarded a third Rare Pediatric Drug designation for IPS Heart candidate, ISX9-CPC, for treatment of cardiomyopathy associated with Danon disease.

Danon disease is a rare fatal disease leading to sudden death or the need for cardiac transplantation by the second or third decade of life.

IPS Heart’s ISX9-CPC stem cell therapeutic candidate is designed to create new functional human heart muscle along with reducing cardiac fibrosis.

“While the rare disease community is largely focused on gene therapy approaches, gene therapy, unfortunately, cannot create new heart muscle nor can it create any new skeletal muscle or deliver full length dystrophin,” said Rauf Ashraf, CEO of IPS Heart. “Large pharmaceutical companies are starting to show clinical success with IPS stem cell therapy as Vertex recently reported two diabetic patients drastically cut their A1c levels while freeing them from their insulin shots for a full year. We are hopeful similar advances will be seen in devastating rare diseases.”

The FDA previously awarded RPDDs for ISX9-CPC and GIVI-MPC for the treatment of Duchenne muscular dystrophy. Separately, the FDA granted Orphan Drug designation for its GIVI-MPC for the treatment of Duchenne muscular dystrophy.

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The Rare Pediatric Disease Priority Review Voucher program is intended to address the challenges that drug companies face when developing treatments for these unique patient populations. Under this program, a sponsor who receives an approval for a drug or biologic for a “rare pediatric disease” may be eligible for a voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product or sold to another sponsor for priority review of their marketing application. Recently, Sarepta Therapeutics sold its voucher for $102 million.

Photo: Rauf Ashraf, CEO of IPS Heart

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