Three parents representing 800 families and their children affected by Duchenne Muscular Dystrophy and a nonsense mutation are calling on the European Medicines Agency (EMA).
The Committee for Medicinal Products for Human Use (CHMP) decided to withdraw Ataluren (Translarna) from the European market in September 2023 and then again in January, May and October 2024, despite all the field evidence supported by experts in paediatric diseases.
What is Ataluren (Translarna)?
Ataluren is the only approved drug for people with Duchenne muscular dystrophy (DMD), which results from a nonsense mutation in the dystrophin gene. [Read More: What is Ataluren (Translarna)?]
Letter from Families to the European Medicines Agency (EMA) for Ataluren (Translarna)
Dear European Commissioners,
We are 3 parents representing 800 families and their children with Duchenne Muscular Dystrophy with a nonsense mutation.
We are contacting you to ask for an emergency health action.
Our children, all Europeans, have been suffering for a year and a half at risk of being left without the only drug available to them, Translarna, as they have no therapeutic alternative. Translarna was authorised 10 years ago by EMA.
The CHMP in September ’23 and again in January, May and October ’24, decided, against all the evidence in the field supported by experts in our children’s disease, to withdraw Translarna from the European MKT.
On 12 December 2024, after a preparatory webinar with the families who clearly highlighted the public health risk of this case, your SC decided to find a viable solution to guarantee at least the therapy to the patients currently being treated, some of whom have been being treated for 10 years.
A decision aimed at avoiding a health emergency.
From the studies carried out and from RWE in fact, 10 years of observations, children undergoing therapy risk losing their ability to walk 3.5 years earlier than expected by the natural history of the disease, an absolute tragedy, considering not only the social impacts, but also the fact that transformative gene therapies are now close to MKT.
In short, a condemnation without appeal for our children (we attach the report of the Italian experts, including Prof. Mercuri and Prof. Luca Bello, 2 of the 4 most important KOLs in the world, both in Italian and in the English translation).
We are therefore very happy with this solution, of which however to date we have not yet had the details and for which we ask you for a guarantee intervention.
We are Europeans, we read in the newspapers that we are undergoing strong pressure to separate, we are convinced that this decision can, given the criticality that it brings with it, give a first sign of strength and compactness of our Europe.
We are ready to appeal to the EU court if necessary, but we are convinced that your reading of the context will help to make the right choice for our/your children.
Sincerely.
Alice Cadalora (Italy)
Elena Semenzato (Italy)
Emil Wirsz (Germany)
