Resolaris Receives U.S. FDA Orphan Designation for Treatment of Facioscapulohumeral Muscular Dystrophy

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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aTyr Pharma (“aTyr”), a biotherapeutics company involved in the discovery and clinical development of pioneering medicines for patients with severe rare diseases using its knowledge of Physiocrine biology, a newly discovered set of physiological modulator, recently announced that Resolaris has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) as treatment for facioscapulohumeral muscular dystrophy (FSHD).

Facioscapulohumeral muscular dystrophy (FSHD) is a genetic muscle disorder in which the muscles of the face, shoulder blades and upper arms are among the most affected. FSHD usually begins before age 20, with weakness and atrophy of the muscles around the eyes and mouth, shoulders, upper arms and lower legs. Later, weakness can spread to abdominal muscles and sometimes hip muscles.

“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., CEO and executive chairman of aTyr Pharma.

ResolarisTM is a first-in-class intravenous protein therapeutic for the treatment of rare myopathies with an immune component, and it is aTyr’s first Physiocrine-based product candidate in the clinic. The medicine is being examined at multiple sites in the European Union in a double-blind, randomized, placebo-controlled phase 1b/2 trial in adult patients suffering with FSHD.

The trial is evaluating Resolaris pharmacokinetics, tolerability, safety and immunogenicity in multiple intravenous doses in adult FSHD patients.

FSHD is a rare genetic myopathy with an immune component, and at the moment there are no approved drugs aimed at treating this severe condition. The FDA’s Orphan Drug Designation program aims to advance the development of medicines which have potential to diagnosing or treating rare diseases affecting less than 200,000 people in the United States. Under this program, sponsors that develop orphan-designated drugs are eligible for incentives that include seven years of market exclusivity after FDA approval, waiver or partial payment of application fees, and certain tax credits.

Since 1983, the program has enabled the development and marketing of more than 400 drugs and biologic products for the treatment of rare diseases.