The most expensive drug in the world costs $ 2 million



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The gene therapy drug Zolgensma has become the most expensive drug in the world, with a unique treatment sold at a price of 2.1255 million US dollars (A3 million dollars).

Swiss drug manufacturer Novartis has received US approval for its Zolgensma gene therapy for the treatment of spinal muscular atrophy (SMA), the leading genetic cause of death in infants.

Novartis executives defended the award, saying that a single treatment was more valuable than an expensive long-term treatment costing several hundred thousand dollars a year.

The US Food and Drug Administration has approved Zolgensma in children under two years of age with ADS, including those with no symptoms yet.

Approval concerns babies with the most lethal form of the hereditary disease as well as those with types where debilitating symptoms may occur later.

Novartis is awaiting European and Japanese approval later this year. Zolgensma will compete with Biogen Spinraza, the first approved treatment for ADS.

The disease often causes paralysis, breathing difficulties and death within a few months for infants born with the most severe type I form.

ADM affects about one in 10,000 live births, with 50% to 70% of them having Type I disease.

Some neurologists consider that gene therapy is becoming the treatment of choice for newborns with severe AS, while recognizing that families may choose to wait for the data relating to the condition. safety and long-term effectiveness of Zolgensma.

Novartis is investigating whether the death of a critically ill baby being treated with Zolgensma was treatment-related.

The FDA has approved Zolgensma on the basis of clinical trials of 36 patients aged 2 weeks to 8 months.

According to the agency, patients treated with Zolgensma showed a significant improvement in developmental milestones such as head control and the ability to sit down.

The most common side effects of Zolgensma are elevated liver enzymes and vomiting. The FDA is asking Zolgensma's label to include a warning stating that a serious and serious liver injury may occur.

With additional studies underway, Novartis said it has so far treated more than 150 patients with Zolgensma, acquired when AveXis was purchased for $ 8.7 billion last year.

Wall Street badysts expect a turnover of $ 2 billion by 2022, according to a survey of Refinitiv. Spinraza sales reached $ 1.7 billion last year and are expected to reach $ 2.2 billion in 2022.

Roche is currently developing risdiplam, an oral medication, and plans to apply for approval later this year.

Australian Associated Press

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