Today, 5th June, Audentes Therapeutics announced that PRIME status has been granted by the European Medicines Agency for their AT132 product (the gene therapy being developed to treat x-linked myotubular myopathy). The PRIME scheme was developed by the EMA to enhance support for the development of medicines that target an unmet medical need. It means that the Agency can offer ‘proactive support’ to those companies developing innovative products, enabling accelerated assessment of these desperately awaited treatments.
This PRIME designation provides the company with a ‘rapporteur’ to help with building knowledge, ahead of any potential authorisation application to bring a product to market, and scientific advice at key development milestones to facilitate quicker access of new patients to a new medicine.
“This PRIME designation has been enabled by the positive interim data reported from the first dose cohort of ASPIRO, the Phase 1 / 2 clinical trial of AT132, and we look forward to collaborating with the EMA to accelerate the clinical development of this potentially transformative therapy for XLMTM patients.” said Mary S. Newman, Senior Vice President, Regulatory Affairs.
AT132 has now been granted PRIME and Orphan Drug designations by the EMA, and Rare Pediatric Disease, Fast Track and Orphan Drug designations by the FDA.
5th June 2018