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news.The European Commission (EC) has granted orphan drug designation for US-based biotherapeutics firm aTyr Pharma's investigational new drug Resolaris to treat facioscapulohumeral muscular dystrophy (FSHD).
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Resolaris is being developed as a first-in-class intravenous protein therapeutic to treat rare myopathies with an immune component.
The drug represents the company’s first Physiocrine-based product candidate in the clinic. Resolaris is begins developed as a first-in-class intravenous protein therapeutic to treat rare myopathies with an immune component.
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 recognized 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."
Earlier, the company reported that the first patient trial of Resolaris in adults with FSHD, a rare and severe genetic myopathy for which there are currently no approved treatments.
The double-blind, placebo-controlled, multiple ascending dose Phase Ib/II trial will be conducted in about 44 FSHD patients currently enrolling at multiple sites in the European Union (EU).