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news.Biotherapeutics company aTyr Pharma has received orphan drug designation from the US Food and Drug Administration (FDA) for its new drug Resolaris for the treatment of facioscapulohumeral muscular dystrophy (FSHD).
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FSHD is a severe, rare genetic myopathy with an immune component for which there are currently no approved treatments.
Being developed as an intravenous protein therapeutic for the treatment of rare myopathies with an immune component, Resolaris represents aTyr’s first Physiocrine-based product candidate in the clinic.
aTyr Pharma CEO and executive chairman John Mendlein said: "FSHD is a debilitating, progressive disease for which there are currently no approved treatments, and the patient community is facing significant unmet medical needs.
"Receiving orphan designation from the FDA will support our efforts to bring an approved therapy to patients as quickly as possible."
Resolaris is being studied in a phase 1b/2, randomized, double-blind, placebo-controlled trial in adult patients with FSHD at multiple sites in the European Union.
The trial is claimed to be designed to evaluate the safety, tolerability, pharmacokinetics and immunogenicity of multiple intravenous doses of Resolaris in adult patients with FSHD.