Zolgensma is a novel drug that was approved by the US Food and Drug Administration (FDA) in May 2019 as an one-time gene therapy for children with spinal muscular atrophy (SMA), said the National University Hospital in an e-mail reply to The New Paper yesterday.

Valued at US$2.125 million (S$2.9 million)for each patient, it is said to be the most expensive drug in the world.

SMA is diagnosed through genetic testing. Besides treatment, patients are usually supported symptomatically, with physiotherapy to manage muscle weakness and prevent complications like contractures.

They are also supported by medical means such as appropriate seating and mobility devices, respiratory support, as well as regular therapy.

"Aside from Zolgensma, other options of treatment for SMA include the use of other drugs and supportive treatment.

"These alternative options for SMA include Spinraza, a recurring therapy given intrathecally every four months, and Risdiplam, an oral medication administered daily," said NUH.

Zolgensma is administered intravenously as a single dose over 60 minutes.

While it has a hefty price tag, the other treatments are also expensive. The Spinraza costs more than $1 million for the five or six injections required in the first year of treatment, and an annual amount of a little over $500,000 for three injections each year subsequently. The Risdiplam oral treatment costs around $460,000 a year.

Zolgensma manufacturer, US-based biopharmaceutical company Novartis Gene Therapies, has stated that the drug is designed to target the genetic root cause of SMA by replacing the function of the missing or non-working survival of motor neuron 1 (SMN1) gene with a new, working copy of a human SMN gene.

Child neurologist Wendy Liew of Dr Wendy Liew Paediatric and Child Neurology Centre said while none of the three FDA-approved treatments for SMA are available in Singapore, they can be imported under the Health Sciences Authority's (HSA) Special Access Route.

On HSA's website, it is stated that the named-patient application allows a licensed hospital or clinic to import and supply an unregistered therapeutic product that presents a life-saving treatment option to the patient whose condition would be clinically compromised without the requested therapy, and that there is no effective alternative therapy registered in Singapore.

TNP understands the approval of such applications usually takes 14 working days and needs to be done before buying the treatment for import.

When asked if any treatment is superior to the others, Dr Liew said: "There are many considerations (when selecting a treatment), such as the age of the patient, the route of administration, the expected benefit, the anticipated adverse effects and the cost of each treatment.

"At the moment, all three treatments have been proven to work, so it is hard to say that one is definitely better than the others."

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Hefty price tag for treatment of spinal muscular atrophy - The New Paper

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