FDA grants IPS HEART Orphan Drug Designation for GIVI-MPC for Treatment of Duchenne Muscular Dystrophy
FDA grants IPS HEART Orphan Drug Designation for GIVI-MPC for Treatment of Duchenne Muscular Dystrophy
HOUSTON--(BUSINESS WIRE)--IPS HEART has been granted orphan drug designation by the FDA for GIVI-MPC, a first-in-class stem cell therapeutic to create new skeletal muscle with 100% full length dystrophin for the treatment of patients with Duchenne Muscular Dystrophy(DMD).
The FDA has granted an orphan drug designation (ODD) to GIVI-MPC, a first-in-class IPS stem cell therapeutic for the treatment of patients with Duchenne Muscular Dystrophy. Current treatments for DMD have limited therapeutic potential as unfortunately none can create any new skeletal muscle or can deliver 100% full length human dystrophin. GIVI-MPC has succeeded in creating new human skeletal muscle with 100% full length human dystrophin in both mdx mice and dystrophic pigs. While there is a lot of hope with newer DMD gene therapy approaches, unfortunately to date there has been no reversal of the underlying disease as gene therapy can only deliver a fraction of the dystrophin gene (~30%) and cannot create any new skeletal muscle.
IPS HEART, founded in 2014, around cardiac and rare muscle diseases is on track to be the first IPS stem cell company to advance its first in class disease modifying therapy into human clinical trials. “Given our successful pre-IND meeting with the FDA and our ongoing developmental efforts including GMP manufacturing, we believe that we will be the first IPS stem cell company with a disease modifying therapy to advance both drugs into human clinical trials whereby all current drugs largely only provide symptomatic relief,” said Rauf Ashraf, CEO of IPS HEART. While heart attacks still kill more annually then all forms of cancer combined, IPS HEART’s first drug candidate ISX9-CPC has created significant new heart regeneration with new functional human heart muscle 90 days after heart attack with its patented human IPS stem cell platform.
About the FDA Orphan Drug Designation
Orphan Drug Designation provides various development benefits for an investigational drug, including seven-year exclusivity after marketing approval is received.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a rare, fatal neuromuscular genetic disease with patients usually succumbing to the disease in their twenties.
About IPS HEART/GIVI-MPC
GIVI-MPC is IPS HEART’s second drug candidate that uses the small molecule Givinostat to reprogram human iPSCs into new skeletal muscle tissue while also delivering 100% full-length human dystrophin for the treatment of Duchenne Muscular Dystrophy.
