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Dynacure’s centronuclear myopathies drug DYN101 gets FDA orphan drug designation

French pharma company Dynacure has secured orphan drug designation for its investigational antisense drug DYN101 from the US Food and Drug Administration (FDA) for the treatment of Centronuclear Myopathies (CNM).

DYN101, which is designed to modulate the expression of dynamin 2 (DNM2), will undergo its first human clinical trial in the form of the Unite-CNM phase 1 / 2 study in the second half of this year. The investigational antisense drug is being developed by Dynacure in collaboration with US biotech company Ionis Pharmaceuticals.

The Unite-CNM clinical trial will be held in Europea as an ascending dose study to assess the safety, tolerability, pharmacokinetics and preliminary efficacy of DYN101 in nearly 18 patients, aged over 16 years of age with X-linked recessive (XLCNM/ Myotubular Myopathy) and autosomal dominant (ADCNM).

Dynacure's centronuclear myopathies candidate DYN101 gets FDA orphan drug designation. Photo courtesy of Stuart Miles/

Dynacure’s centronuclear myopathies candidate DYN101 gets FDA orphan drug designation. Photo courtesy of Stuart Miles/

Centronuclear and myotubular myopathies, or in short Centronuclear Myopathies, are serious, rare, life-threatening disorders that affect skeletal muscles from the time of birth.

The disease is caused by mutations in multiple genes such as MTM1, DNM2 and BIN1 and Dynacure scientists discovering the connection between an increase in DNM2 and the direct cause of the disease.

Stephane van Rooijen – CEO of Dynacure said: “Orphan Drug Designation in the US is a critically important regulatory milestone in our global development plan for DYN101 to treat several forms of centronuclear and myotubular myopathies.

“The US designation complements our orphan drug designation in the EU and we look forward to enroll in our first-in-human clinical study with DYN101 later this year to treat this devastating rare disease.”

The FDA gives orphan drug designation to novel drugs that are intended for the treatment of a rare disease or condition. The FDA orphan drug designation gives seven years of market exclusivity if approved along with significant development incentives such as tax credits associated with clinical trial expenses, an exemption from the FDA-user fee, and also the regulator’s assistance in clinical trial design.

DYN101 has also been granted orphan drug designation by the European Medical Agency.

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