UAE-approved drug offers lifeline to older children with spinal muscular atrophy

A new spinal muscular atrophy (SMA) drug approved in the UAE has been hailed as “a game-changer” in treatment of the condition.

As reported in The National, regulators in the Emirates have given the go-ahead for the use of Itvisma, a drug that will enable gene therapy treatment to be given to older SMA sufferers.

For several years, doctors in the UAE have successfully been administering another gene-therapy drug, Zolgensma, to children with SMA.

However, this medicine is suitable only for infants, while Itvisma, now approved by the Emirates Drug Establishment after the thumbs up in November from the US Food and Drug Administration (FDA), can be given to children older than two, as well as teenagers and adults.

Treatment is likely to come at a high cost, with Zolgensma, which works in a similar way, having been described as the most expensive drug in the world, with a price tag of $2 million per treatment.

The drug, which received FDA approval in 2019, needs to be administered only once, as does Itvisma.

Offering fresh hope

Giles Lomax, chief executive of SMA UK, a charity that supports people with the condition and works with health authorities to advance treatment, said Itvisma was “a really great step in the right direction”.

“It’s a huge game-changer for us,” he said. “Although the drug is expensive – gene therapies are expensive – over time they average themselves out.

If children are diagnosed really early within that first 12 months, if they’re eligible they can go straight on to Zolgensma. [Itvisma] is really for those who missed that window. If you don’t quality [for Zolgensma], there’s the option for you to go on to it.”

Unlike many other neurodegenerative conditions, such as Alzheimer’s, which have proved difficult to develop drugs for, huge strides have been made in SMA treatment in recent years.

This is because most cases are caused by a mutation in a s