EMA Orphan Drug status for aTyr’s Resolaris in FSHD

Privately-held US biotech firm aTyr Pharma says that the European Commission 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, chief executive 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," he noted.