GIVI-MPC named FDA orphan drug for treatment of Becker MD
IPS Heart stem cell therapy aims to be disease-modifying for BMD, DMD
The U.S. Food and Drug Administration (FDA) has granted IPS Heart’s GIVI-MPC — a stem cell therapy designed to make new muscle tissue in people with muscular dystrophy, or MD — orphan drug status for the treatment of Becker muscular dystrophy (BMD).
This designation aims to encourage the development of treatments for rare diseases, or those affecting fewer than 200,000 people in the U.S., by providing certain benefits, including tax credits and seven years of market exclusivity if the therapy is ultimately approved.
GIVI-MPC was previously awarded both orphan drug status and rare pediatric drug designation for Duchenne muscular dystrophy (DMD), another type of MD in which the hallmark symptoms of muscle weakness and wasting typically start at about ages 2 or 3. In Becker MD, meanwhile, symptom onset varies widely from person to person, and can begin at ages ranging from 5 to 60.
IPS Heart is also developing ISX9-CPC, another stem cell therapy meant to produce new cardiac muscle, to treat heart complications seen in DMD.
“With our successful FDA pre-IND [investigational new drug] meeting and our ongoing developmental efforts on both drugs, we believe that we will be the first company with bona fide disease-modifying therapies to advance both drugs into human clinical trials, unlike all current drugs that largely only provide symptomatic relief,” Rauf Ashraf, CEO of IPS Heart, said in a press release. A pre-IND meeting refers to interaction with the FDA before an application is filed to conduct clinical trials.
Per the release, the company is “committed to rapidly advancing both therapeutics to the clinic.”
Available treatments for Becker, Duchenne MD provide symptom relief
Becker MD is caused by mutations in the DMD gene that result in low levels of the protein dystrophin, or in a less functional version of the protein being produced. Dystrophin acts as a shock absorber to protect muscle cells from damage during muscle contractions; low levels or a faulty version lead to progressive muscle weakness and wasting, impairing mobility and causing heart and breathing symptoms.
In DMD, functional dystrophin is completely missing.
GIVI-MPC is a single-course stem cell therapy meant to generate new skeletal muscle tissue with full-length dystrophin. It works by reprogramming human induced pluripotent stem cells, or iPSCs, into new muscle tissue using givinostat.
iPSCs are obtained by collecting mature cells from the skin or blood and reprogramming them back into a stem cell-like state, where they can differentiate into other cell types. Givinostat is an oral therapy approved for the treatment of DMD in the U.S., under the brand name Duvyzat, that’s now in clinical testing for BMD.
The company noted that, while gene therapies deliver a smaller yet functional form of dystrophin, they cannot create new muscle to replace lost tissue or enable the production of full-length dystrophin.
According to Ashraf, IPS Heart is “pursuing ongoing partnerships with various large pharmaceutical companies … to help rapidly advance both [GIVI-MPC and ISX9-CPC] to the clinic.”
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