The Italian Government recently approved the protein restoration therapy Translarna (ataluren) for the treatment of Duchenne muscular dystrophy (DMD), and included it in its medication list for reimbursement by the Italian National Health System. The oral drug treatment, developed by biopharmaceutical company PTC Therapeutics, will now be available in Italy after being advocated by both specialists and DMD families’ associations.
Following the decision, under Law 648/96, the formalization of the approval will happen through its publication at the official Italian state journal, the Gazzetta Ufficiale, which is expected to happen within the next weeks. The list of medications that is up for reimbursement by the healthcare system can be found in the website of the Italian Medicines Agency’s (AIFA).
The AIFA’s Technical and Scientific Committee was part of the decision, as it had granted a positive opinion on the inclusion of the therapy to become available and be reimbursed in a meeting last October. “We are very pleased with the positive opinion from AIFA,” stated the Chief Commercial Officer of PTC Therapeutics, Inc., Mark Rothera.
In addition, a group of specialist physicians and the Parent Project Onlus were also crucial in the process of giving Italian patients access to Translarna. “The story of Translarna, which began in 1998, has taught us a lot about Duchenne muscular dystrophy and the potential for new agents to treat the underlying cause of the disease,” said the President of Parent Project Onlus, Filippo Buccella. “We are delighted that AIFA’s decision means we can now bring the first therapy approved for nonsense mutation DMD to patients and families in Italy.”
Law 648/96 enables a new cohort of DMD patients to have access to Translarna, which has been demonstrated to be beneficial in the formation of functioning proteins, since DMD causes alterations in the genetic codes that prematurely halts the synthesis of an essential protein. PTC Therapeutics recently released the results of its phase 2b clinical trial, revealing the effectiveness of Translarna on the treatment of male patients five years or older suffering from nonsense mutation Duchenne muscular dystrophy (nmDMD). Translarna had already received marketing authorization in the European Economic Area, but was not available in Italy until now.
“DMD is a devastating and a rapidly progressing muscle wasting disorder and every day counts. We are committed to working with regulators, payers and the DMD community to enable Translarna to reach all patients who may benefit as soon as possible wherever reimbursed EAP mechanisms exist,” added Rothera.
Affecting primarily men, DMD is a progressive disorder caused by the shortage of functional dystrophin protein, which is determinant for the skeletal stability, diaphragm and heart muscles. In severe cases, it may lead patients to lose the capacity to walk, as well as induce life-threatening lung and heart complications.