World’s Most Expensive SMA Drug Shows Effectiveness in Infants

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Tests of gene therapy with the drug “Zolgensma” in children under six months give a positive result, reports Reuters, referring to the manufacturer’s report.

The interim results of the third phase of trials of the drug “Zolgensma” manufactured by the Swiss pharmaceutical company Novartis, which were treated in infants of the first 6 months of life with spinal muscular atrophy (SMA), have been published. It is a rare genetic neuromuscular disorder characterized by loss of motor neurons, resulting in impaired swallowing, respiratory and basic motor functions.

Reportedthat two-thirds of participants in the STR1VE-EU study who underwent gene therapy before reaching 6 months of age had an acquisition of age-related motor skills uncharacteristic of the natural course of SMA 1. Also, according to report, the majority (about 92%) of children did not require constant respiratory support, and two-thirds were able to eat on their own.

The widespread use of the drug, which costs US $ 2.1 million per patient, was temporarily suspended in the United States a month ago as the Food and Drug Administration (FDA) requested his additional study in older children.

Registration of the drug “Zolgensma” (onasemnogen abeparvovec) in Russia started in July 2020. Until then, its import into the territory of the Russian Federation could only be carried out for health reasons for specific patients in accordance with the requirements determined by the federal law No. 61-F3 (on the circulation of medicines). This required decisions of medical commissions of medical institutions, as well as approval for import from the Ministry of Health of the Russian Federation. At the same time, payment for treatment was carried out both at the expense of charitable fees and within the framework of state guarantees, reports portal “Family of SMA”.

In addition, last year in Russia for the treatment of this disease, the drug “Spinraza” (nusinersen, manufactured by “Biogen”) was approved, received orphan status in 2020.

Also Roshe stated on the intention to register in Russia its drug for the treatment of SMA Risdiplam in the first quarter of 2020.



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