The US Food and Drug Administration (FDA) granted orphan drug designation for a new induced pluripotent stem cell therapeutics for Duchenne muscular dystrophy (DMD). 

The therapeutic called GIVI-MPC could create new skeletal muscles expressing full-length dystrophin protein, the missing protein in DMD. 

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Rauf Ashraf, the chief executive officer of IPS HEART, the developer of the therapeutic, said in a press release, “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.”

The biggest challenge in the treatment of DMD is the inability of any gene therapy to deliver the full-length DMD gene to the body. Moreover, none of the available therapies can create new skeletal muscles to replace the atrophied ones in DMD. 

GIVI-MPC was able to create new human skeletal muscles expressing full-length human dystrophin protein both in a mouse and pig model of the disease. The therapeutic uses the small molecule givinostat to reprogram human induced pluripotent stem cells into new skeletal muscles.

Orphan drug designation provides many benefits for an investigational drug, including tax credits for certain clinical trials, exemption from user fees, and 7-year exclusivity following marketing approval.

DMD is a rare genetic disease caused by a mutation in the DMD gene, the largest in the human genome, which leads to the complete absence of dystrophin protein. 

Dystrophin acts as a shock absorber during muscle contraction so when it is not present, muscle cells get damage at each contraction and deteriorate to be eventually replaced by scar tissue. 

DMD first causes the skeletal muscles to degenerate rendering patients unable to walk before eventually affecting the diaphragm and heart muscles and being fatal. 

Reference

FDA grants IPS HEART orphan drug designation for GIVI-MPC for treatment of Duchenne muscular dystrophy. News release. IPS HEART. February 22, 2023.

Neurologists