GIVI-MPC earns FDA orphan drug designation to treat DMD

The first-in-class stem cell therapy is being developed by IPS HEART

Andrea Lobo avatar

by Andrea Lobo |

Share this article:

Share article via email
This illustration is an announcement that reads

GIVI-MPC, a stem cell therapy to create new muscle in Duchenne muscular dystrophy (DMD), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA).

Orphan drug status encourages the development of therapies for rare diseases — those affecting fewer than 200,000 people in the U.S. — by providing certain benefits, such as seven years of market exclusivity upon approval and exemption from FDA fees.

“Given our successful pre-[investigational new drug] meeting with the FDA and our ongoing developmental efforts … we believe that we will be the first [induced pluripotent] 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,” Rauf Ashraf, CEO of IPS HEART, the company developing GIVI-MPC, said in a company press release.

Recommended Reading
This illustration shows a closeup of a DNA strand against a background of red X's.

Study finds unique DMD disease mechanism, may help in diagnosis

DMD is the most common type of muscular dystrophy, caused by mutations in the DMD gene, which provides instructions to produce a protein called dystrophin. This protein works within a complex of other proteins, as a shock absorber to protect muscle cells from wear and tear during muscle contractions.

Most mutations that cause DMD result in a complete lack of production of functional dystrophin protein. This leads to progressive loss of muscle strength and function, affecting mobility and causing heart and respiratory complications.

According to IPS HEART, current DMD treatments have limited therapeutic potential as they cannot create new skeletal muscle or deliver functional full-length human dystrophin to patients. Also, while there is hope with gene therapy, no approach has been able to reverse the underlying DMD disease processes.

GIVI-MPC is a first-in-class stem cell therapy to create new skeletal muscle with full-length dystrophin. It uses the small molecule givinostat to reprogram human induced pluripotent stem cells (iPSCs) into new muscle tissue. iPSCs are produced by collecting mature cells, from the skin or blood, and reprogramming them back into a stem cell-like state where they can be programmed into virtually any cell type.

Of note, givinostat is being developed by Italfarmaco as a potential oral treatment for DMD and Becker muscular dystrophy.

Preclinical data in mouse and pig models of muscle dystrophy showed that GIVI-MPC treatment was able to create human skeletal muscle with full-length dystrophin. According to IPS HEART, givinostat is anti-inflammatory and promotes stem cell differentiation, among other properties.

The therapy is IPS-HEART’s second drug candidate. The company’s first treatment candidate — ISX9- CPC — is intended to create new heart muscle and restore heart function, with current indications including DMD.