The Food and Drug Administration (FDA) has granted Orphan Drug designation to DYN101 (Dynacure) for the treatment of centronuclear myopathies (CNM).

Centronuclear and myotubular myopathies (CNM) are rare, life-threatening disorders characterized by muscle weakness and atrophy from birth. The disease is often caused by mutations in the dynamin 2 (DNM2), BIN1 and MTM1 genes. DYN101 is an investigational antisense oligonucleotide that works by modulating the expression of DNM2 to treat CNM.

Findings from preclinical studies have shown the potentially disease-modifying effects of DYN101 in CNM. In animal models, a dose-dependent improvement was observed that led to the prevention and reversion of the disease. 

The Company is expected to initiate the phase 1/2 Unite-CNM study in the second half of 2019 to evaluate the safety, tolerability and efficacy of DYN101 in patients aged ≥16 years with CNM.

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“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,” said Stephane van Rooijen (MD MBA), Chief Executive Officer of Dynacure.  “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.”

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