FDA grants orphan drug status to ISX9-CPC for heart issues in DMD
Experimental stem cell therapy aims to generate new heart tissue
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS Heart’s experimental stem cell therapy for heart problems in Duchenne muscular dystrophy (DMD), the company announced in an email sent to Muscular Dystrophy News Today.
This FDA designation aims to accelerate the development of treatments for rare diseases, like DMD, that affect fewer than 200,000 individuals in the U.S. It provides certain benefits, including tax credits and seven years of market exclusivity if the treatment is ultimately approved.
ISX9-CPC is designed to treat two forms of heart problems that may appear in people with Duchenne: heart failure and cardiomyopathy, a disease of the heart muscle. The therapy was previously granted rare pediatric drug designation as a treatment for DMD. That status is given to therapies for rare diseases in which serious or life-threatening manifestations primarily affect children and adolescents ages 17 and younger.
In preclinical studies, therapy significantly reduced scar tissue
DMD is caused by mutations in the DMD gene that codes for the dystrophin protein, which helps protect muscles from damage during movements. People with Duchenne have little to no dystrophin, resulting in progressive muscle weakness and wasting. In Becker muscular dystrophy (BMD), which is also caused by mutations in DMD, dystrophin is produced at abnormally low amounts, or its activity is impaired.
ISX9-CPC is a single-course stem cell therapy that aims to generate new heart tissue. It uses a small molecule, called ISX9, to reprogram human induced pluripotent stem cells (iPSCs) into functional cardiac muscle.
These 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.
In preclinical studies, a single administration of ISX9-CPC significantly reduced scar tissue, generated cardiomyocytes — the muscle cells in the heart — and improved heart function in a mouse model of heart attack. In particular, the company noted that the treatment increased ejection fraction, which refers to the percentage of blood that is pumped out to the body with each heartbeat.
IPS Heart is also developing GIVI-MPC, another stem cell therapy designed to produce new skeletal muscle in people with DMD and BMD. This therapy has been designated as an orphan drug in the European Union for BMD, and was previously granted orphan drug and rare pediatric drug status for DMD.
The company now is focusing on advancing GIVI-MPC to a Phase 1/2 trial in people with Duchenne, while looking for possible partnerships to advance the clinical development in Becker.
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