Dynacure Receives Orphan Drug Designation from the FDA for DYN101

Dynacure, a clinical stage drug development company focused on improving the lives of patients with rare and orphan disorders, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2) for the treatment of Centronuclear Myopathies (CNM). Dynacure expects to initiate its first in human study, a Phase 1 / 2 study ‘Unite-CNM’, in the second half of 2019. DYN101 is being developed in collaboration with Ionis Pharmaceuticals, the leader in RNA-targeted drug discovery.

Centronuclear and myotubular myopathies (CNM), are serious, rare, life-threatening disorders that affect skeletal muscles from birth. CNMs derive their name based on the central location of the muscle fiber nucleus, which is an abnormal finding observed in muscle biopsies. The disease is driven by mutations in multiple genes including MTM1, DNM2 and BIN1 and Dynacure scientists have discovered the link between an increase in DNM2 and the direct cause of the disease (Cowling et al 2014 JCI). There are many genetic forms of CNM including X-linked recessive (XLCNM/ Myotubular Myopathy), autosomal dominant (ADCNM), and autosomal recessive (ARCNM), which are all associated with poor prognosis. Centronuclear Myopathies affect between 4,000 and 5, 000 patients in the EU, US, Japan and Australia1.

“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 (M.D. 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.”

The FDA grants Orphan Drug Designation to novel drugs that seek to treat a rare disease or condition and provides 7 years of market exclusivity if approved, plus significant development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.