The European Medicines Agency (EMA) has granted Orphan Drug Designation to Resolaris (ATYR1940) for the treatment of all types of limb girdle muscular dystrophy (LGMD). This will speed up its development and help get it to patients quicker.
aTyr Pharma recently reported the results of its phase I/II trial testing Resolaris in people with LGMD 2B. The drug was found to be safe and also improved the muscle function of seven out of nine participants after 14 weeks of treatment.
This is promising news, though it’s worth keeping in mind that this was a small-scale trial and the average improvement in muscle strength was small (+6.2%). The trial has been extended to see the effect of Resolaris in the longer term.
John Mendlein, Chief Executive Officer of aTyr Pharma, said:
The Resolaris program has now been granted Orphan Drug Designation for both LGMD and facioscapulohumeral muscular dystrophy (FSHD) from the FDA and the EMA, as well as Fast Track designation for both rare myopathies with an immune component from the FDA. We are pleased with the development of Resolaris from a clinical and regulatory standpoint. We look forward to continuing to develop this potential therapy to help treat patients with these rare myopathies, for which there are limited or no treatment options, across the globe.
More information on the Orphan Drug Designation
More information on the LGMD 2B trial results
Read the latest LGMD research news