As reported in Yahoo! Finance, the United States’ Food and Drug Administration (FDA) recently granted Orphan Drug designation to MP1032. This therapy, developed by clinical-stage biotech company MetrioPharm, is being developed for individuals living with Duchenne muscular dystrophy (DMD). 

Orphan Drug designation is granted to therapies designed to treat rare conditions; in the United States, rare conditions are those affecting fewer than 200,000 people across the country. This designation also comes with benefits for drug developers. For example, MetrioPharm will receive fee waivers, tax credits, increased regulatory assistance, and seven years of market exclusivity upon drug approval. Learn more about Orphan Drug designation. 

Currently, there are no cures for Duchenne muscular dystrophy. Treatments are designed to manage symptoms, such as occupational and physical therapy, nutritional supplements, and high-dose corticosteroids. MetrioPharm developed MP1032 to overcome current burdens associated with treatment, such as significant adverse reactions associated with long-term corticosteroid use. MP1032 is a small molecule ROS scavenger designed with anti-inflammatory properties. 

So far, preclinical research has found MPS1032 to be effective in improving muscle strength and function. MPS1032, used in conjunction with Prednisolone, also increases efficacy. Moving forward, MetrioPharm hopes to begin a Phase 2 study next year to further evaluate this therapy. 

Understanding Duchenne Muscular Dystrophy (DMD) 

There are nine forms of muscular dystrophy; Duchenne is one of these forms. An estimated 15,000 children in the United States, and 300,000 worldwide, are living with DMD. This rare muscular disorder predominantly impacts males, though it can occur—much more rarely—in females. DMD results from gene mutations that prevent the body from creating dystrophin in the muscles. Ultimately, this causes muscle weakness and wasting. This disorder most commonly manifests between three to six years old. Symptoms can, but do not always, include learning disabilities, frequent tripping and falling, progressive muscle weakness that often begins in the lower extremities, difficulty walking or moving positions, fatigue, a waddling gait, and cardiomyopathy.