FDA Grants Rare Pediatric Disease Designation to LGMD Type 2E

MAY 16, 2018
Krista Rossi
Today, Myonexus Therapeutics announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for MYO-101, which is an AAV-based gene therapy for the treatment of limb girdle muscular dystrophy (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," stated Michael Triplett, CEO of Myonexus Therapeutics in a recent statement.

LGMDs are genetic diseases characterized by progressive, debilitating weakness. Before muscle wasting progresses to muscles in the arms and legs, it begins in muscles around the hips and shoulders. In LGMD type 2E, the disease usually onsets before age 10, progresses to loss of ambulation in the teen years, and often leads to death before age 30. Currently, no treatments or cure for LGMD type 2E exists.

Within the lab of Louise Rodino-Klapac, PhD and under clinical guidance by Jerry Mendell, MD, at Nationwide Children's Hospital Center for Gene Therapy, Myonexus is innovating 5 licensed novel gene therapies. The MYO-101 program includes just one.

MYO-101 is engineered to transfect a gene that both codes for and restores beta-sarcoglycan protein; the goal is to restore the dystroglycan complex. It utilizes an AAVrh.74 vector system that has shown strong preclinical safety and efficacy data. This vector is the same one being used in the micro-dystrophin gene therapy program Sarepta is developing with Nationwide Children’s Hospital.

The phase 1/2a study of MYO-101 is expected to commence in mid-2018. A reported 60-day biopsy data in late-2018 or early 2019 is planned for by both companies. In regards to LGMD2L, MYO-102 for LGMD2D, MYO-103 for LGMD2C, MYO-201 for LGMD2B, and MYO-301 are also being advanced by Myonexus.

Louise Rodino-Klapac, PhD, the principal investigator for the Center for Gene Therapy at Nationwide Children’s Hospital and associate professor in the Department of Pediatrics at Ohio State University. He is also the inventor of the Myonexus portfolio of LGMD candidates and the chief scientific officer and co-founder of Myonexus, a spinout of Nationwide Children’s Hospital, Center for Gene Therapy.

"We look forward to initiating an i.v. systemic Phase 1/2a trial of MYO-101 later this year," added Bruce Halpryn, COO of Myonexus Therapeutics.

In addition, Myonexus and Sarepta Therapeutics, Inc recently announced they have entered an exclusive partnership to develop multiple gene therapies to treat 5 distinct forms of limb girdle muscular dystrophies (LGMDs) with LGMD type 2E among them.

The MYO-101 program was previously granted Orphan Drug Designation (ODD) by FDA.

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