FDA Grants Myonexus Therapeutics Rare Pediatric Disease Designation

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Myonexus Therapeutics’ MYO-101, an experimental gene therapy for a form of limb girdle muscular dystrophy, a rare, genetic muscle wasting disease.

LGMDs are cause progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs. In LGMD type 2E, the disease usually manifests itself before age 10. It progresses to loss of the ability to walk in the teen years, and often leads to death before age 30. There is currently no treatment or cure for LGMD type 2E.

The gene therapy is one of five pioneered at Nationwide Children’s Hospital Center for Gene Therapy that Myonexus licensed. Myonexus recently entered into an exclusive partnership with Sarepta Therapeutics to develop multiple gene therapies to treat five distinct forms of limb girdle muscular dystrophies including LGMD type 2E.

“The FDA’s Rare Pediatric Disease designation for MYO-101 reflects the compelling data underlying the MYO-101 program and the potential to provide a first-ever treatment option for LGMD type 2E,” said Michael Triplett, CEO of Myonexus Therapeutics.

The FDA grants Rare Pediatric Disease designation for diseases that primarily affect children from birth to 18 years old and affect fewer than 200,000 persons in the United States. This program is intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases.

If MYO-101 is approved by the FDA for LGMD type 2E, the Rare Pediatric Disease designation may enable Myonexus to receive a priority review voucher. Priority review vouchers can be used by the sponsor to receive Priority Review for a future drug approval submission, which would reduce the FDA review time from twelve months to six months.

The vouchers are potentially lucrative because they are transferable. Most recently, Spark Therapeutics sold a priority review voucher at the end of April for $110 million.

May 16, 2018
Photo: Michael Triplett, CEO of Myonexus Therapeutics