Experimental Duchenne Muscular Dystrophy Treatment Earns Rare Pediatric Disease Designation

According to a story from apnews.com, the biopharmaceutical company FibroGen, Inc., has recently announced that its investigational treatment pamrevlumab has earned Rare Pediatric Disease designation from the US Food and Drug Administration (FDA). The drug is being developed as a treatment for Duchenne muscular dystrophy, a rare genetic disorder. The treatment has also earned Fast Track designation from the agency.

About Duchenne Muscular Dystrophy (DMD)

Duchenne muscular dystrophy is a neuromuscular disease, and it is one of the more severe types of muscular dystrophy. It is characterized by progressive muscle weakness that usually begins around age four and worsens quickly. As an X-linked genetic disease, males are mostly affected, with females only occasionally displaying mild symptoms. The disease is caused by mutations of the dystrophin gene. Symptoms of Duchenne muscular dystrophy include falling, abnormal walking posture, eventual loss of walking ability, muscle fiber deformities, intellectual disability (not in all cases), enlargement of the tongue and calf muscles, skeletal deformities, muscle atrophy, heart abnormalities, and difficulty with breathing. Treatment includes a variety of medications and therapies that can help alleviate symptoms and slow disease progression. Lifespan is usually into the thirties with good care. Better treatments for this disease are urgently needed. To learn more about Duchenne muscular dystrophy, click here.

About Rare Pediatric Disease Designation

Rare Pediatric Disease designation is a special designation from the FDA that is typically granted for therapeutics being developed to treat a disease that is considered rare, which is defined as any disease that affects less than 200,000 people in the US. In addition, the disease must primarily affect people under age 18. If the FDA approves the drug’s Biologics License Application (BLA), the developing company could then be eligible for a Priority Review voucher that can be used on a future application or can be sold to another company.

About Pamrevlumab

Pamrevlumab is an antibody designed as a connective tissue growth factor inhibitor. This factor has been identified to play a mediating role in proliferative and fibrotic disorders. It is in the midst of being tested in two phase 3 clinical studies. Aside from its potential utility in Duchenne muscular dystrophy, pamrevlumab is also being developed for unresectable pancreatic cancer and idiopathic pulmonary fibrosis.

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