Dynacure Receives Orphan Drug Designation for DYN101 for the Treatment of Centronuclear Myopathies

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to DYN101, an investigational antisense medicine. The drug modulates the expression of dynamin 2 (DNM2) and is for the treatment of Centronuclear Myopathies (CNM).

Dynacure, which produces the therapy, is expected to initiate its first in-human Phase I/II study ‘Unite-CNM’ later this year. The trial will be a European multicentre, ascending dose study to evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of DYN101.

CNM is driven by mutations in multiple genes including MTM1, DNM2, and BIN1. Researchers at the pharmaceutical company discovered a link between an increase in DNM2 and the direct cause of the disease, allowing them to develop the regulatory drug.

Pre-clinical studies have demonstrated that DYN101 has the potential to be disease-modifying in CNM, with efficacy in treating animal models. Prevention and reversion of the disease were observed with a dose-dependent improvement in mice survival.

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