There is little time left for the definitive answer on the regulatory situation of Translarna™ (ataluren), the only drug intended for Duchenne muscular dystrophy (DMD) due to nonsense mutations. After the communication from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency on 24 January, in which the second negative opinion was issued for the renewal of the conditional marketing authorization following the review procedure, the European Community has communicated that the decision relating to Translarna™ (ataluren) will be communicated during the week of April 22nd, we read on Osservatorioomalattierere.it.
“After the second negative opinion from the CHMP we based ourselves on the average completion times of the procedures – explains the Parent Project Aps association in a press note – which usually involves 60 days, but without precise indications we could only make assumptions about possible dates. Now, finally, an official communication has been received indicating the week of April 22nd as the period in which a decision will be made.”
“It is important to underline that this communication does not specify a precise date but provides a time frame within which a definitive response is expected from the European Commission. While waiting for the week of April 22 – concludes the note – Parent Project Aps remains vigilant and hopes that the decision takes into account the experiences of patients.”
In fact, it was the Italian scientific community that appealed, together with patient associations, to the European Commission and the EMA, with a letter signed by 28 experts, including Prof. Eugenio Mercuri, who clearly confirm that a sudden interruption of accessibility to this drug – the clinicians explain in the letter – while other potential therapeutic avenues are still under clinical study, could affect their quality of life, as well as that of their parents, relatives and healthcare workers.
“Consistently postponing the critical phase of loss of walking is in fact an important result – the clinicians explained in the letter – which is reflected in a better quality of life and higher social and educational outcomes, even if other therapeutic approaches will also be necessary in a coordinated manner to treat this disorder. For these reasons we wish to express our support for the maintenance of Translarna (ataluren) as a medical option in the European Union and respectfully request that you grant a positive opinion on the review of the renewal of the conditional authorisation.”
It is therefore difficult for families to wait without being able to do anything: a group of parents, especially mothers, have addressed a heartfelt letter to all the main government institutions, including President Sergio Mattarella.
There were various demonstrations of attention and support. Action MEP, Fabio Massimo Castaldo, sent an official letter to the Health and Food Safety Directorate of the EU Commission, through which he expressed “concern” and asked to “intervene urgently” so that the drug translarna, used for the treatment of Duchenne muscular dystrophy can continue to be used. “It is a drug that represents the only therapeutic option for Duchenne Muscular Dystrophy (DMD) – explained Castaldo – a rare genetic disease that affects approximately one male out of every five thousand, which is characterized by progressive muscular degeneration and which ultimately leads to the loss of all muscle mass and premature death. Translarna has been a beacon of hope for these patients by bringing a tangible delay in the loss of the ability to walk and other functions crucial to their quality of life.”
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