Aifa approves drug against SMA, families: “A revolution, finally”

Sma, Aifa approved the Novartis drug

Aifa, the Italian drug agency, has approved Zolgensma, the drug against SMA, spinal muscular atrophy, as well as the most expensive in history. The price of the treatment in the US is 2.1 million dollars, while in Italy the agreement reached with Novartis would be approximately 1.5 million euros per treatment, but with deferred payment over time and based on the results obtained.

“Today is a very important day for Novartis Italy” reads the company’s press release “but above all for patients and their families. The Italian Medicines Agency AIFA has approved the first gene therapy against SMA, and our country is among the forerunners in Europe to guarantee the reimbursement of this treatment. Our mission is to reimagine medicine. And today everyone can touch it, recognizing the value of pharmaceutical research. Once again it is important to emphasize how these advances can be built through the efforts of those who work in the scientific community, listening to patients, and collaborating with health authorities, so that innovation is an early and accessible right to all “.

“The first gene therapy for spinal muscular atrophy is now a reality also in Italy and represents a real revolution for our entire community”. So Families SMA, the association that represents people with SMA and their families, welcomes the news. The drug will be provided free of charge by the National Health Service for type 1 SMA patients up to 13.5 kg and pre-symptomatic patients with 2 copies of the SMN2 gene.

What is Sma

In Italy about 40-50 children are born each year with spinal muscular atrophy, a rare neuromuscular disease – the leading cause of infant genetic death – characterized by the progressive loss of motor skills. It mainly affects children, making everyday gestures such as sitting and standing difficult, and in severe cases, swallowing and breathing. Type 1 SMA – the most severe form of the disease for which access to gene therapy is expected – affects 60% of new born with the disease (about 20-25 children each year) and until a few years ago it was difficult to survive over two years.

Zolgensma is the trade name of a gene therapy, onasemnogene abeparvovec, capable of correcting the genetic defect underlying the disease, which affects about one new born every 10 thousand, that is, more or less 40 children a year in our country.

Although not a definitive cure, the innovative aspect of Zolgensma is that it intervenes directly on the genetic defect with a single administration, so it is carried out only once in a lifetime.

An incredible opportunity for a disease that up to 4 years ago did not foresee any possibility of fighting it and that today – with genes – can count on three effective therapies capable of arresting the course of the disease.


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