Rare Facial Disorder Drug Designated Orphan Drug Status

aTyr Pharma announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to Resolaris, a physiocrine-based product candidate for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

Resolaris is currently being studied in a Phase 1b/2, randomized, double-blind, placebo-controlled trial designed to evaluate the safety, tolerability, pharmacokinetics and immunogenicity of multiple intravenous doses of Resolaris in adult patients with FSHD. The study is ongoing at multiple sites within the European Union. FSHD is a severe, rare genetic myopathy with an immune component for which there are currently no approved treatments.

For more information visit aTyrPharma.com.

Want to read more?