Britain Approves 'World's Most Expensive Drug'. Cost: Rs 18
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The 'most expensive drug in the world' has been approved by the United Kingdom's National Health Service (NHS) to cure a rare genetic disorder. The one-off gene therapy Zolgensma, manufactured by Novartis Gene Therapies, has a reported list price of ?18 crore (£1.79 million) per dose, NHS England said.

The drug treats Spinal Muscular Atrophy (SMA), a rare and often fatal genetic disease that causes paralysis, muscle weakness, and progressive loss of movement. It will be used for babies and young children suffering from SMA. Zolgensma, has helped babies to reach milestones, showed studies.

The treatment is given as a single intravenous infusion and contains a replica of the missing gene SMN1, NHS said. The active ingredient onasemnogene abeparvovec passes into the nerves and restores the gene, which then produces proteins essential for nerve function and controlling muscle movement, it explained.

The latest data suggested that Zolgensma can provide rapid and sustained improvement in motor function for young children with type 1 SMA and prolong their lives. “This deal is a life-changer for youngsters with this cruel disease and for their families," NHS England chief executive Sir Simon Stevens said.

The approval of the drug marked the second medical treatment now available for youngsters with SMA, after Spinraza became available on the NHS to eligible patients in May 2019. The NHS is ready to fast-track the introduction of the highly complex and innovative gene therapy, it said.

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