Zolgensma Gene Therapy: Babies Gain Approval To Use World's Most Expensive Drug

Zolgensma Gene Therapy spinal muscular atrophy SMA most expensive drug novartis Zolgensma U.S. Food and Drug Administration National Health Services genetic disorder Type 1 SMA Zolgensma cost

By Lilian Scott , Parent Herald Jul 13, 02:00 am

Photo: (Photo : BSIP/Universal Images Group via Getty Images)

A handful of babies across the world who have Spinal Muscular Atrophy (SMA) had been cleared to receive the Zolgensma gene therapy, known as the world's most expensive drug at $2.1 million for one-off treatment.

Developed by the pharmaceutical company Novartis, the Zolgensma gene therapy passed the U.S. Food and Drug Administration (FDA) standards in 2019 and was conditionally approved in the U.K. and other countries around the same time. Because of its hefty price tag, availing of the drug hasn't been easy for many parents with children suffering from this genetic illness.

In early 2021, the National Health Services (NHS) in the U.K. arranged a deal with the manufacturers to secure the world's most expensive drug following additional studies that proved a single dose of Zolgensma could dramatically help the babies with muscular conditions crawl, sit and walk. The therapy could also do away with a ventilator for young patients who usually exhibit difficulty in breathing.

Hope for Babies with SMA

Ten-month-old Baby Edward will receive the drug by August 2021 after his mother, Megan Willis, campaigned for the treatment. Willis, 29, said that when her son was diagnosed with SMA in November 2020, she did not think he would have a hopeful future. However, after learning of Zolgensma and discussing the options with the NHS, the grateful mom said that they now see a glimmer of hope for Edward.

Two-year-old Riley Cadle-Birch just had his intravenous therapy at the Bristol Royal Hospital for Children in the U.K. following the NHS clearance. Like Willis, Jade Cadle-Billingham, the boy's mother, also found renewed hope that Riley will have a better life ahead of him because of this life-changing drug.

In New Delhi, Ayaansh Gupta, 3, has also received Zolgensma thanks to more than 65,000 donors. Another baby in the same region has gotten his treatments as well with the help of a charitable organization's draw.

In Egypt, two babies have had the therapy following a directive from their president, while one baby in Denmark got the green light to receive the drug through the help of the Boston Children's Hospital in the U.S.

Why SMA Treatment is Expensive

SMA is a genetic disorder that affects the nervous system, where the brain starts to malfunction and impedes muscle control or movement. Babies who develop this rare genetic condition passed down to families show physical signs when they are about three months old. The babies usually fail to reach certain developmental milestones, such as lifting their heads or rolling over. They also do not learn the ability to walk or experience frequent breathing difficulties.

According to the experts, SMA develops because of a deficiency in the child's chromosomes. If they don't develop SMA, they can still pass the broken genes down to their children or grandchildren as they are carriers.

The treatment for SMA is expensive because the disorder is so rare and requires extensive studies by highly knowledgeable and trained experts. It took years for Novartis to develop the Zolgensma gene therapy, which has now proved effective among children with Type 1 SMA.

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