Audentes Therapeutics Inc. said the European Medicines Agency granted priority medicines, or Prime, designation to its drug candidate AT132.
AT132 is being developed to treat X-linked myotubular myopathy, a rare inherited disease characterized by extreme muscle weakness, respiratory failure and early death. XLMTM occurs almost exclusively in newborn males, with an estimated 50% mortality rate by 18 months of age.
The EMA's Prime designation helps optimize development plans and expedite the evaluation of certain drugs, so they reach patients as soon as possible.
San Francisco-based Audentes said AT132 previously received orphan drug status from the EMA. The U.S. Food and Drug Administration already granted AT132 rare pediatric disease, fast track and orphan drug designations.
