Biotherapeutics firm aTyr Pharma has received orphan drug designation from the European Commission (EC) for its Resolaris to treat facioscapulohumeral muscular dystrophy (FSHD).
Resolaris is a firm’s investigational new drug being developed as a first-in-class intravenous protein therapeutic to treat rare myopathies with an immune component.
It is derived from a naturally occurring protein released in-vitro by human skeletal muscle cells, noted aTyr Pharma.
aTyr Pharma CEO and executive chairman John Mendlein said: "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 recognised the potential of Resolaris for patients suffering from FSHD.
"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."
The company previously announced the first patient study of Resolaris in adults with FSHD, a rare and severe genetic myopathy, which there are currently no approved treatments for.
According to the company, the Phase Ib/II study is a double-blind, placebo-controlled and multiple ascending dose trial in up to 44 FSHD patients currently enrolling at multiple sites in the European Union (EU).
aTyr Pharma is involved in the development of Physiocrine-based therapeutics to provide solutions for rare diseases.