“Today is a good day for the history of this disease because the approval by the FDA, which we hope will be followed by that of the European Medicines Agency EMA in the short term, is not only a bureaucratic act but represents a fundamental step to allow the access to the drug to a greater number of patients”. So Mario Sabatelli, president of the medical-scientific commission of theItalian Multiple Sclerosis Association (Aisla) and clinical director of the adult area of the NeMO Center at the Gemelli Polyclinic in Rome, comments on the approval by the US Food and Drug Administration (FDA) of tofersen, the first drug for the treatment of adults affected by ALS who have the gene mutation of superoxide dismutase 1 (Sod1). The hope of the patient community – reads a note – is that the work of the EMA will be accelerated which, in December 2022 accepted the application from Biogen, the company that manufactures the drug, and is awaiting an answer.
On advanced therapies, unfortunately – underlines Aisla – Europe has been losing ground in recent years. It is clear that the possibility is given by need to streamline the regulatory process and, precisely for this reason, the development of advanced therapies should increasingly involve companies, institutions, researchers and, last but not least, patient associations. The great global debate should focus on the ability to bring ever more immediate responses to people’s needs.
Many do not know they have Sod1 mutation, immediate genetic testing is now imperative
For people with ALS in Italy – recalls the note – today the method envisaged since last October 2021 of administering tofersen through the early access program, compassionate use continues to be active: every neurologist can request for their ALS patients with mutation in the Sod1 gene, regardless of disease progression, access to the investigational drug. In Italy there are an estimated 120 people with ALS-SOD1 mutation. Since early access to the drug was opened, just under half have been treated, of which 30 at the NeMO Clinical Centres.
“In the light of this magnificent news – declares Fulvia Massimelli, national president of Aisla – we will continue ours commitment to guaranteeing the possibility for all people with ALS to access the genetic test and, fundamental aspect, to have results in a short time. The data that the scientific community has made available tell us, in fact, that the essential condition for the drug to be effective is to intervene as early as possible”.
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