Acclaimed as the “most expensive drug in the world” the U.K.’s National Health Service approved a novel gene therapy drug called Zolgensma. This drug could possibly be a life-altering innovation for babies and children suffering from a rare genetic disorder. The drug will be utilized to treat babies and young children suffering from Spinal Muscular Atrophy, which a rare and often fatal genetic disease that leads to paralysis, muscle weakness, and progressive loss of movement.
NHS England in a statement said, “Zolgensma, which has a reported list price of £1.79 million (Rs. 18 crores) per dose and is labeled the most expensive drug in the world, will be available to patients at a price that is fair to taxpayers after a landmark confidential deal struck by NHS England.”
Hence, Thus, these children who have been diagnosed with severe type 1 SMA have a life expectancy of just two years.
How does drug Zolgensma help?
Manufactured by Novartis Gene Therapies (which is part of U.S. pharmaceutical Novartis), Zolgensma has been proven to help babies with SMA to breathe without a ventilator, sit up on their own, and crawl and walk after a single infusion treatment.
It is predicted that Zolgensma can prolong the life of young children by facilitating rapid and sustained improvement in their motor functions. This is possible as the chief constituent in the drug, i.e. onasemnogene abeparvovec goes into the nerves and restores the gene, which then produces proteins essential for nerve function and controlling muscle movement.
The NHS stated that as many as 80 babies and young children annually can gain benefits from gene therapy.
NHS England Chief Executive Sir Simon Stevens said the deal was a “life-changer for youngsters with this cruel disease and for their families.”
He further added, “Spinal Muscular Atrophy is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers”
Commenting on the impact of COVID-19 on the health care systems, Stevens stated, “Although the health service is still under real pressure from covid, and NHS England is also focused on leading the national covid vaccination rollout, today’s agreement is an important reminder that the NHS is looking after millions of other patients too, for whom real medical advances are now possible.”