IPS HEART, a private cell therapy company developing treatments for Duchenne muscular dystrophy and heart failure, announced that it has received a third Rare Pediatric Drug Designation (RPDD) from the US Food and Drug Administration (FDA).

The designation is for their product candidate, ISX9-CPC, which is being developed to treat cardiomyopathy associated with Danon disease, a rare and fatal disorder. ISX9-CPC is a stem cell therapeutic candidate that aims to generate new functional human heart muscle and reduce cardiac fibrosis.

The FDA has previously awarded RPDDs for ISX9-CPC and another drug, GIVI-MPC, both for the treatment of Duchenne muscular dystrophy. Additionally, GIVI-MPC has received an Orphan Drug Designation from the FDA for the same condition.

Rauf Ashraf — IPS HEART CEO said: “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.

“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.”

Danon disease typically leads to sudden death or necessitates cardiac transplantation by the second or third decade of life, highlighting the need for effective treatments.

ISX9-CPC is the first patented drug candidate by IPS HEART that uses the small molecule ISX-9. This molecule works by reprogramming iPSCs, or induced pluripotent stem cells, into new, functional heart muscle. It is currently being developed to treat heart failure, Duchenne Cardiomyopathy, and now, with this new designation, Danon Cardiomyopathy.

  Danon disease, Duchenne muscular dystrophy, IPS HEART, ISX9-CPC, Rauf Ashraf, US FDA, US Food and Drug Administration, USA