FDA Grants RMAT Designation to AT132

Audentes announced the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to AT132 for the treatment of X-linked Myotubular Myopathy (XLMTM).

"We are pleased that the FDA has granted RMAT designation to AT132, an important regulatory milestone that highlights the transformative potential of AT132 as a therapy to treat XLMTM, a rare, congenital disease characterized by extreme muscle weakness, respiratory failure and early death," said Mary S. Newman, Senior Vice President, Regulatory Affairs at Audentes. "This news follows our recent announcement of the Priority Medicines, or PRIME, designation received from the European Medicines Agency, and we look forward to collaborating closely with the FDA, EMA, our XLMTM clinical experts and the patient community as we seek to rapidly advance the AT132 development program toward global regulatory approvals."

Established under the 21st Century Cures Act, RMAT designation is a program designed to expedite the development and approval of regenerative medicine products, including gene therapy products. An investigational therapy is eligible for the RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates a potential to address unmet medical needs for that disease or condition. The designation includes all the benefits of the FDA's Fast Track and Breakthrough Therapy designations, and enables the ability to work more closely and frequently with the FDA to discuss surrogate or intermediate endpoints to support the potential acceleration of approval and satisfy post-approval requirements.

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 timepoint 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.

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