The FDA recently informed Muscular Dystrophy News that it has granted orphan drug designation to the experimental drug ISX9-CPC, a product of the IPS Heart company located in Houston, Texas. The designation awarded to the experimental drug is an incentive to accelerate the creation of treatments for rare diseases that affect under 200,000 people in the U.S.

IPS Heart’s stem cell therapy is designed to treat heart issues such as generating new heart tissue. Benefits, if the treatment is approved, include tax credits and market exclusivity for seven years.

ISX9-CPC was previously granted a rare drug designation to treat pediatric Duchenne Muscular Dystrophy (DMD). The status is awarded to therapies involving rare diseases that affect people aged seventeen or younger and are life-threatening.

About DMD

Mutations in the gene that codes for the dystrophin protein, which helps to protect muscles from being injured during movements are the fundamental cause of DMD. People diagnosed with DMD are found to have little or no dystrophin. The result is progressive muscle weakness. Becker muscular dystrophy (BMD) is also the result of extremely low amounts of dystrophin resulting in impaired activity.

ISX9-CPC cell therapy develops new heart tissue through a molecule that is called ISX9 which reprograms stem cells into cardiac muscle. Mature cells are collected and reprogramed into stem cells where they evolve into various cell types. One treatment of ISX9-CPC reduced scar tissue and improved heart function in a laboratory-staged heart attack.

The IPS Heart Company is in the process of developing a stem cell therapy that is designed to produce new skeletal muscles in people with DMD. The therapy was previously granted a rare pediatric drug and orphan drug status in the EU.