PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the initiation of a reimbursed expanded access program (EAP). PTC's EAP program is intended to make Translarna™ (ataluren) available to patients before commercial availability in certain countries. Where mechanisms exist and in accordance with local regulations, PTC will make Translarna available to nonsense mutation Duchenne muscular dystrophy (nmDMD) patients through funded EAP programs.
Funded Named Patient Programs have already been authorized in Turkey and Spain. Today, the French National Agency for Medicines and Health Products Safety (ANSM), has granted a Temporary Authorization for Use (Autorisation Temporaire d'Utilisation de cohorte). Government allocations to hospitals will allow payment for Translarna for patients included in the ATU cohort program.
"For children and young men living with DMD, a rapidly progressing muscle wasting disorder, every day counts. We are committed to working with regulators, payors and the DMD community to enable Translarna to reach patients as soon as possible wherever reimbursed EAP mechanisms exist," stated Mark Rothera, Chief Commercial Officer of PTC Therapeutics, Inc.
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