aTyr Pharma (NSDQ:LIFE) said today that its candidate Resolaris was granted fast track designation by the FDA for the treatment of facioscapulohumeral muscular dystrophy, making it the 1st therapeutic candidate to receive the designation for the rare form of muscular dystrophy.
Resolaris is derived from a naturally occurring protein released by human skeletal muscle cells. aTyr is exploring the use of Resolaris as a therapy for patients with rare myopathies with an immune component characterized by excessive immune cell involvement.
FSHD is a rare genetic myopathy that affects 19,000 people in the U.S., according to aTyr, and there are no approved treatments. Patients with FSHD suffer from skeletal muscle deterioration, fatigue, chronic pain and muscle weakness.
Resolaris is being studied in a Phase Ib/II clinical program as a treatment for FSHD, according to the San Diego-based company, and it expects to report data from 3 clinical trials in December.
The fast track designation is designed to expedite the review process for drug candidates that aim to treat rare and serious conditions. Product candidates may be eligible for priority review when the company submits a Biologic License Application. The company is also eligible for more frequent communication directly with the FDA.
“This fast track designation, which is granted to drug candidates addressing serious conditions and that demonstrate the potential to address unmet medical needs, represents another step forward for our rare muscle disease franchise,” CEO John Mendlein said in prepared remarks. “This designation highlights the significant need to develop and ultimately approve meaningful new therapeutics to treat patients with rare myopathies, such as FSHD.”
The company’s stock was trading at $3.05 apiece in late-afternoon trading activity, up 5.17%.
