FDA Grants Fast Track for Muscular Dystrophy Treatment

FDA grants Fast Track designation for aTyr’s Resolaris to treat limb girdle muscular dystrophy 2B and removes partial clinical hold for Resolaris.

aTyr Pharma, Inc., a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe, rare diseases, announced that its product candidate Resolaris was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of limb girdle muscular dystrophy 2B (LGMD2B), making it the first known therapeutic candidate for the treatment of LGMD2B to receive the designation. In addition, the FDA removed its partial clinical hold on a dosing ceiling for Resolaris in clinical trials.

“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 first product candidate based on the Physiocrine pathway,” said John Mendlein, Ph.D., CEO of aTyr Pharma. “Combined with our Phase1b/2 data in LGMD2B, adult facioscapulohumeral muscular dystrophy (FSHD) and early onset FSHD patients, we believe we are building a clinical and regulatory foundation for future development of Resolaris to treat patients across multiple rare genetic myopathies with an immune component.”

aTyr previously announced results from a completed Phase 1b/2 open-label, intra-patient dose escalation trial testing doses of Resolaris up to 3.0 mg/kg biweekly in patients with LGMD2B. Based on the clinical trials completed to date, Resolaris has demonstrated a favorable safety profile without signs of immuno-suppression of circulating immune cells. 78% of the LGMD2B patients in the trial (7 of 9) recorded increases in their muscle function at 14 weeks as measured by manual muscle test (MMT), a validated assessment tool. Overall, the LGMD2B patients had a mean increase of MMT scores from baseline of 6.2%.

“We appreciate the FDA’s responsiveness to our request to remove the partial clinical hold that provides dosing flexibility based on our data for Resolaris,” commented Sanjay Shukla, M.D., MS, chief medical officer of aTyr Pharma. “We also believe that during our safety and dose ranging Phase 1b/2 clinical trials we have potentially identified a dose for the next phase of clinical development with a favorable safety profile and potential clinical activity across different rare muscle indications.”

(Source: PR Newswire)