On August 21, 2018, Audentes Therapeutics, Inc. (Nasdaq: BOLD ) announced that the U.S. FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to AT132, its treatment for X-linked Myotubular Myopathy (XLMTM).
Audentes is a biotechnology company that develops gene therapy products for patients for rare diseases.
Audentes has pursued a number of advanced regulatory pathways for AT132, including:
- Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, Fast Track and Orphan Drug designations by the FDA
- Priority Medicines (PRIME) and Orphan Drug designations by the European Medicines Agency (EMA)
AT132 for X-linked Myotubular Myopathy
As stated in the company announcement:
“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.”
What is an RMAT Designation?
Congress included several provisions related to cell and gene therapies in the 21st Century Cures Act, the most important of which is the RMAT designation. RMAT stands for “Regenerative Medicine Advanced Therapy” designation.
Sponsors of cell and gene therapies are now eligible to obtain an RMAT designation if their product is intended to treat serious or life-threatening diseases, and there is preliminary clinical evidence that it can address unmet medical needs.
To learn more, see Audentes’ RMAT announcement here.
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