Home » Duchenne: the despondency of patients, don’t leave us without the only drug

Duchenne: the despondency of patients, don’t leave us without the only drug

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Duchenne: the despondency of patients, don’t leave us without the only drug

With a letter to the European Commission and the European medicines agency EMA, the associations Uildm Italian Union Fight against Muscular Dystrophy, Parent Project aps and the Italian Federation of Rare Diseases Let’s unite jointly ask to review the decision relating to the drug “Translarna” (Ataluren) for Duchenne Muscular Dystrophy. The Committee for Medicinal Products for Human Use gave a negative opinion on the renewal of the conditional authorization due to the lack of efficacy of the drug (here is the news in Italian on the Aifa website). In light of all the available data, we read on the EMA website, the cost/benefit balance is negative and there is no evidence in its favor which, ten years after the first conditional approval, justifies its authorization.

However, this decision has thrown hundreds of patients with this pathology and their families across Europe into despair who will no longer be able to benefit from a drug that for years has been the only treatment option for patients with nonsense mutation. The associations report that «according to the scientific community, after years of use, Translarna is a drug with a high safety profile, easy to use in children due to the method of administration, which allows a slowdown in the progression of the pathology as widely documented in the register Stride.” According to the associations’ joint statement, the Stride registry includes data collected over the last 5 years and offers a unique opportunity to directly observe the long-term benefits of Translarna in delaying the loss of the ability to walk. The data shows a lag of 3.5 years compared to the population included in the Cinrr natural history register.

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The associations say they are aware that the primary endpoints of the study were not achieved from a statistical point of view, but they highlight how treatment with Traslarna allows a improvement of the quality of vita as it slows down the effects of the pathology. Confirmation also comes from the families of the patients involved in the trial who note clear improvements and positive repercussions on the quality of life in general. This result is very important for all the patients who have been included in the trial to date and for their families.

Parent Project aps in December it promoted the Buy Some Time campaign, collecting testimonies from families across Europe on their experience with the drug in question, as well as launching a petition on Change.org, which collected more than 14,000 signatures in just a few months. «Translarna is not “the cure”, we know this well» declare the signatories of the letter «But it allows us to save time. And time is of the essence for patients with nmDMD, as the disease progresses in the absence of specific treatments. The time gained could allow access to new upcoming therapies. With the shutdown of Translarna some children and adults will undoubtedly lose some skills within a year or two. Maintaining the ability to walk is not the only goal, our children want to maintain the freedom to carry out daily functions, such as using a laptop, driving a wheelchair, feeding themselves or going to the bathroom.”

«We Parent Project families are saddened by the risk we are running of once again losing a therapeutic opportunity that has contributed for almost 10 years to counteract the damage caused to our children by Duchenne Muscular Dystrophy» declares Ezio Magnano, President of Parent Project aps. «Translarna is not the cure, but it is an important part of a combined therapy that has contributed to giving more life and quality of life to our children. We will do everything possible to ensure that our message is heard by the European Commission.”

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The comment of president of Uniamo Annalisa Scopinaro: «Transalarna has proven to be a drug with a high safety profile, easy to administer and, from Stride data, there are slowdowns in the progression of the pathology. For people who have no therapeutic alternatives, these are more than sufficient reasons for its marketing. In general, we hope for greater participation of patient representatives in the construction of research designs and in the entire subsequent trial process, in order to identify the correct endpoints and evaluate, through PROMs and PREMs, not only the clinical implications but also the quality improvement of life”

«For years we have been working on the quality of people’s lives, both from a medical-health point of view and at a social inclusion level. They are two sides of the same coin, which allow us to give hope to our families. We are aware that Translarna is not the definitive solution, but in recent years it has offered many more opportunities – to do, to plan the future – to those living with Duchenne Dystrophy”, he declares Marco Rasconi, National President of Uildm.

Photo at Hans Moerman on Unsplash

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