Resolaris Granted Orphan Drug Status for Rare Genetic Myopathy
The Food and Drug Administration (FDA) has granted Orphan Drug designation to Resolaris (aTyr Pharma) for the treatment of limb girdle muscular dystrophy (LGMD).
The company is developing Resolaris as a potential treatment for LGMD sub-type (LGMD2B), a rare recessive genetic myopathy caused by a toxic loss of function in the dysferlin gene. Patients present with progressive debilitating muscle weakness and atrophy, as well as immune cell invasion in the skeletal muscle. Currently, there are no approved therapies for LGMD.
Resolaris is a first-in-class intravenous protein therapeutic derived from histidine aminoacyl tRNA synthetase (HARS), a naturally occurring protein released by human skeletal muscle cells.
For more information visit atyrpharma.com.