Posted by Michael Wonder on 21 Aug 2018
Audentes announces regenerative medicine advanced therapy designation granted by the FDA to AT132 for the treatment of X-linked myotubular myopathy
21 August 2018 - Designation granted based on positive interim data from ASPIRO clinical trial.
Audentes Therapeutics today announced that the U.S. FDA granted Regenerative Medicine Advanced Therapy designation to AT132 for the treatment of X-linked Myotubular Myopathy (XLMTM).
RMAT designation for AT132 was granted based on positive interim clinical data from ASPIRO, the ongoing Phase 1 / 2 clinical study of AT132 for XLMTM. These data show encouraging initial evidence of safety and efficacy in the first dose cohort, including significant improvements in neuromuscular function as assessed by the CHOP-INTEND scale, increased respiratory function as demonstrated by reductions in ventilator dependence and gains in maximal inspiratory pressure (MIP), a measure of respiratory muscle strength. In addition to these functional outcome measures, muscle biopsy results from the first three patients treated in the study at the 24-week time-point demonstrate highly efficient tissue transduction as indicated by vector copy number, robust myotubularin protein expression as assessed by western blot, and significant improvement in histology.
