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, is being developed as a first-in-class intravenous protein therapeutic for the treatment of rare myopathies with an immune component.
Facioscapulohumeral Dystrophy (FSHD) is an inheritable muscle disease, characterized by the progressive weakening and loss of skeletal muscles. It is a severe, rare genetic myopathy with an immune component for which there are currently no approved treatments.
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 done to evaluate the safety, tolerability, pharmacokinetics and immunogenicity of multiple intravenous doses of Resolaris in adult patients with FSHD.
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.
Subscribe to PharmaTutor News Alerts by Email >>
