Antisense Oligonucleotide Fast-Tracked for Myotubular and Centronuclear Myopathies

The Food and Drug Administration (FDA) has granted Fast Track designation to DYN101 for the treatment of myotubular and 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 antisense oligonucleotide designed to reduce the expression of the DNM2 protein, which at elevated levels, is toxic to muscle cells.

The Company is currently evaluating DYN101 in the phase 1/2 UNITE-CNM trial (ClinicalTrials.gov Identifier: NCT04033159) in patients 16 years of age and older with CNM caused by mutations in the MTM1 or DNM2 gene. Initial data are expected within the second half of 2022.

“CNM is a progressively debilitating life-threatening disease with no approved therapies to help,” said Leen Thielemans, Chief Development Officer of Dynacure. “Receiving Fast Track designation will provide us with greater access to FDA and guidance on regulatory pathways.”

The FDA previously granted Orphan Drug designation to DYN101 for this indication.

References

1. Dynacure receives Fast Track designation for DYN101, an investigational antisense oligonucleotide for the treatment of myotubular and centronuclear myopathies. News release. Dynacure. Accessed January 6, 2022. https://www.prnewswire.com/news-releases/dynacure-receives-fast-track-designation-for-dyn101-an-investigational-antisense-oligonucleotide-for-the-treatment-of-myotubular-and-centronuclear-myopathies-301455840.html. 
2. Dynacure: Pipeline. Dynacure. Accessed January 6, 2022. https://www.dynacure.com/pipeline/.

This article originally appeared on MPR