aTyr Pharma, Inc. ("aTyr"), a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address rare diseases, announced today the European Commission (EC) has granted orphan drug designation to Resolaris™ for the treatment of facioscapulohumeral muscular dystrophy (FSHD). Resolaris™, an investigational new drug representing aTyr's first Physiocrine-based product candidate in the clinic, is being developed as a first-in-class intravenous protein therapeutic for the treatment of rare myopathies with an immune component.
"Our mission is to develop medicines that will make a meaningful difference to patients impacted by debilitating rare diseases. We are very pleased that the EMA has recognized the potential of Resolaris™ for patients suffering from FSHD," said John Mendlein, Ph.D., CEO and executive chairman of aTyr Pharma. "The Agency's decision is an important advancement for our promising Physiocrine-based medicines, and marks a key milestone in our strategy to deliver Resolaris™ to address the unmet needs of patients with this rare disease."
aTyr previously announced the first patient study of Resolaris™ in adults with FSHD, a rare and severe genetic myopathy for which there are currently no approved treatments. The Phase 1b/2 study is a double-blind, placebo-controlled, multiple ascending dose trial in up to 44 FSHD patients currently enrolling at multiple sites in the European Union (EU).