aTyr Pharma, a biotherapeutics company engaged in the discovery and development of physiocrine-based therapeutics to address rare diseases, announced that Resolaris has been granted Orphan Drug designation by the US Food and Drug Administration (FDA) 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.
“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,” said John Mendlein, Ph.D., chief executive officer and executive chairman of aTyr Pharma.
Resolaris is currently being studied in a phase 1b/2, randomised, double-blind, placebo-controlled trial in adult patients with FSHD at multiple sites in the European Union. The trial is designed to evaluate the safety, tolerability, pharmacokinetics and immunogenicity of multiple intravenous doses of Resolaris in adult patients with FSHD.
FSHD is a severe, rare genetic myopathy with an immune component for which there are currently no approved treatments. The FDA’s Orphan Drug designation programme is intended to advance the development of products which demonstrate promise in diagnosing or treating rare conditions that affect fewer than 200,000 people in the US. Sponsors developing orphan-designated products are eligible for incentives under the programme, including seven years of market exclusivity following FDA approval, waiver or partial payment of application fees, and certain tax credits. The programme has successfully enabled the development and marketing of more than 400 drugs and biologic products for rare diseases since 1983.