Out-of-pocket payment for the treatment would vary as per insurance coverage, but the Swiss drugmaker will let insurers make payments over a five-year period - at $425,000 per year and will give partial rebates if it doesn't work.
The USFDA on May 24 approved ‘Zolgensma’, a $2.125 million medicine sold by Novartis, which treats spinal muscular atrophy, as per a Mint report. This is the most expensive medicine ever to get a USFDA approval.
Spinal muscular atrophy is an inherited disorder due to a faulty gene that weakens a child’s muscles and affects their ability to swallow or breathe, killing most afflicted by it within a couple of years.
The treatment is a one-time infusion gene therapy that takes about an hour and the USFDA has approved it for children under two years of age who are confirmed to have any of the three types of the disease by a genetic test, the paper stated.
Out-of-pocket payment for the treatment would vary as per insurance coverage, but the Swiss drugmaker will let insurers make payments over a five-year period - at $425,000 per year and will give partial rebates if it doesn't work, it added.
Patient testing found that children with the most severe form of the disorder who got Zolgensma within six months had limited muscle problems. Those who received the treatment earlier fared best, as the medicine cannot reverse muscle damage already suffered, thus making an early diagnosis of the utmost importance, the paper said.
Novartis is working with states to make by next year genetic testing mandatory at birth so as to detect the disorder early.
Doctors said it is too early to judge how long the benefit lasts but expressed hope it would be for a lifetime as a few treated under patient testing have grown to four and five with no recurrence or symptoms.
The USFDA listed vomiting and possible liver damage as side effects of Zolgensma, which means that patients must be monitored for the first few months after treatment, it added.
NGO Institute for Clinical and Economic Review (ICER), which rates the value of expensive new medicines has justified the cost of Zolgensma as it ‘dramatically transforms the lives of families affected by this devastating disease’. ICER President Dr Steven D Pearson termed the treatment’s price a positive outcome for patients and the health system, the report stated.
Another approved treatment for the disorder that affects around 400 babies per year in the US is Biogen’s ‘Spiranza’, which is administered every four months, and costs $750,000 for the first year and $350,000 per year after that, the paper noted.
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