Baby Ben Kutschke was diagnosed at three months with spinal muscular atrophy, a rare inherited disorder which is the leading genetic cause of death in infancy globally. It leaves children too weak to walk, talk, swallow or even breathe.
So when in 2021 his parents heard about Zolgensma – a one-time therapy costing millions of dollars that promises to replace genes needed for the body to control muscles – they had high hopes.After treatment with the $2.25 million therapy at almost eight months old, Ben was able to hold his head up for a few seconds – a significant milestone, his mother Elizabeth Kutschke told Reuters.
Zolgensma, launched in 2019 by Swiss-based healthcare group Novartis as a "potential cure" for SMA, was the most expensive drug in the world at the time. It has worked well for many. Novartis' data presented in March shows that depending on the timing of treatment, most patients have gone on to swallow, breathe, or even walk independently, said Sitra Tauscher-Wisniewski, vice president at Novartis Gene Therapies. Some are able to run and climb.
"The perception that Zolgensma is going to be a complete cure ... is not coming to fruition from the data we have seen over the last four years," said Dr. Roger Hajjar, director of the Mass General Brigham Gene & Cell Therapy Institute. The multimillion-dollar pricing strategy is not unique. More recently, the first hemophilia gene therapy approved by the U.S. Food and Drug Administration was priced by CSL Behring at $3.5 million; 26 more gene therapies are in late-stage development, according to IQVIA.
"One of the arguments is you're saving all of these millions of dollars down the line," said Stacie Dusetzina, professor of health policy at Nashville's Vanderbilt University School of Medicine. The fact that some children need treatment with other expensive drugs after Zolgensma shows that the gene therapy represents "poor value," said Steven Pearson, ICER president.
The treatment works best when infused as soon as possible after birth – some patients can begin to suffer from degeneration before birth – and Ben Kutschke was a relative latecomer. Zolgensma is becoming available to younger babies across the U.S.: Most states screen newborns for it, and health insurers typically cover Zolgensma for children with the most severe form.Zolgensma delivers the gene through a modified virus. Ben had already been exposed to the virus in nature and had developed antibodies, which would neutralize the replacement genes in his body.
The body develops antibodies to the virus used, so each therapy using it can only be administered once. Doctors recommended a third treatment, Biogen's Spinraza.The most recent Novartis data, updated in March, shows this was not unusual. Its study found that 24 of 81 children given Zolgensma as of May 2022 had been subsequently treated with other SMA drugs. Novartis declined to provide details on them.
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