FDA grants rare paediatric drug status for IPS HEART’s stem cell drugs

18 Apr 2023
www.pharmaceutical-technology.com
Orphan DrugCell Therapy
IPS HEART’s GIVI-MPC and ISX9-CPC are indicated to treat patients with Duchenne muscular dystrophy. Credit: Dr. Edwin P. Ewing, Jr. / commons.wikimedia.org.
IPS HEARTod GIVI-MPC AdmiISX9-CPCon (FDA) has granted rare paediatric dDuchenne muscular dystrophyART’s stem cell therapeutics, GIVI-MPC and ISX9-CPC, to treat Duchenne muscular dystrophy (DMD) patients.
GIVI-MPFood and Drug Administration (FDA)al muscle with 100% full-length dystrophin, while ISX9-CPC is designed to create new fuGIVI-MPC cardISX9-CPCle for indiDuchenne muscular dystrophy (DMD)
GIVI-MPCe small molecule Givinostat, GIVI-MPC reprogrammes human induced dystrophint stem cISX9-CPCo new human skeletal muscle tissue, and delivers 100% full-length human dystrophin to treat DMD.
The drug candidate receivGivinostatanGIVI-MPCsignation (ODD) from the FDA in February 2023.DMD
ISX9-CPC is IPS HEART’s first patented drug candidate, and uses small FDAecule ISX-9 to reprogramme the iPSCs into new functional human heart muscle to treat heart failure and Duchenne cardiomyopathy.
ISX9-CPCed new human heart muscle with a greater than 50% improvement in ejection fraction and a 70% reduction in scar tissue 90 days after a heart attack.heart failureDuchenne cardiomyopathy
ISX9-CPC is expected to become the company’s first drug to be used clinically in humans.heart attack
ISX9-CPCT CEO Rauf Ashraf said: “Given our successful pre-IND meeting with the FDA and our ongoing developmental efforts on both drugs, we believe that we will be the first company with a disease-modifying therapy to advance both drugs into human clinical trials whereby all current drugs largely only provide symptomatic relief.
“We are in dialogue with large pharmaceutical companies and investors to partneFDAinvest with us to help rapidly advance both therapies to the clinic”.
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