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13-08-2023, 02:33 PM
By Deena Beasley
August 12, 20235:05 PM GMT+8Updated 11 hours ago
LOS ANGELES, Aug 12 (Reuters) - 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.
They were disappointed.
......
Ben is one of a growing number of patients with spinal muscular atrophy (SMA) whose doctors are turning to additional drugs on top of the gene therapy, six top U.S. neurologists told Reuters.
Their experience raises broader questions around other high-cost gene therapies coming to market, sometimes after accelerated regulatory approvals, drug pricing experts said.
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.
......
Zolgensma has been given to more than 3,000 children globally, with 2022 sales of US$1.4 billion representing 91% of gene therapy sales worldwide
......
In the U.S., where costs are borne by government health programs such as Medicaid as well as private insurance, IQVIA estimated Zolgensma sales totaled US$434 million last year.
......
It has worked well for many.
......
But ...... almost one-third of children in an ongoing study went on to be given other drugs.
......
"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.
"Following the injection of Zolgensma you are basically going from a deadly disease to a more chronic disease state," he said, adding that many patients would not survive without this treatment.
......
Novartis said Zolgensma has been "transformative,"
......
Notably, it has dropped the term "potentially curative" – common in its analyst calls in 2018 and 2019 – from descriptions of Zolgensma, instead calling it a "one-time treatment."
......
The Institute for Clinical and Economic Review (ICER), a drug pricing research group, has said Zolgensma's maximum price should be US$900,000 – less than half its current cost.
The two other available SMA treatments are also not cheap.
Biogen's Spinraza, injected into the spine, has a U.S. list price of US$800,000 in the first year followed by maintenance doses at US$400,000 a year. Roche's Evrysdi, an oral solution, costs US$100,000 to US$340,000 per year depending on how much the patient weighs.
Both Biogen and Roche said studies of their drugs as treatments for patients who do not respond sufficiently to Zolgensma have been encouraging so far.
.....
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 ...... and Ben Kutschke was a relative latecomer.
......
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.
......
Babies can also be ineligible due to issues such as liver problems – Zolgensma's label warns it can cause these. Last year, two children in Russia and Kazakhstan died of acute liver failure several weeks after receiving it.
......
Its study found that 24 of 81 children given Zolgensma as of May 2022 had been subsequently treated with other SMA drugs.
......
it can be difficult to get insurance coverage after Zolgensma. Some health plans explicitly exclude add-on therapies if a child received it
......
It took nearly six months of paperwork and appeals – supported by Ben's medical team – before payment for Spinraza was approved.
Since then Ben, who now uses a wheelchair at age 2, began talking a lot more. He was suddenly able to move his lower body
https://www.reuters.com/business/healthc...023-08-12/
August 12, 20235:05 PM GMT+8Updated 11 hours ago
LOS ANGELES, Aug 12 (Reuters) - 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.
They were disappointed.
......
Ben is one of a growing number of patients with spinal muscular atrophy (SMA) whose doctors are turning to additional drugs on top of the gene therapy, six top U.S. neurologists told Reuters.
Their experience raises broader questions around other high-cost gene therapies coming to market, sometimes after accelerated regulatory approvals, drug pricing experts said.
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.
......
Zolgensma has been given to more than 3,000 children globally, with 2022 sales of US$1.4 billion representing 91% of gene therapy sales worldwide
......
In the U.S., where costs are borne by government health programs such as Medicaid as well as private insurance, IQVIA estimated Zolgensma sales totaled US$434 million last year.
......
It has worked well for many.
......
But ...... almost one-third of children in an ongoing study went on to be given other drugs.
......
"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.
"Following the injection of Zolgensma you are basically going from a deadly disease to a more chronic disease state," he said, adding that many patients would not survive without this treatment.
......
Novartis said Zolgensma has been "transformative,"
......
Notably, it has dropped the term "potentially curative" – common in its analyst calls in 2018 and 2019 – from descriptions of Zolgensma, instead calling it a "one-time treatment."
......
The Institute for Clinical and Economic Review (ICER), a drug pricing research group, has said Zolgensma's maximum price should be US$900,000 – less than half its current cost.
The two other available SMA treatments are also not cheap.
Biogen's Spinraza, injected into the spine, has a U.S. list price of US$800,000 in the first year followed by maintenance doses at US$400,000 a year. Roche's Evrysdi, an oral solution, costs US$100,000 to US$340,000 per year depending on how much the patient weighs.
Both Biogen and Roche said studies of their drugs as treatments for patients who do not respond sufficiently to Zolgensma have been encouraging so far.
.....
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 ...... and Ben Kutschke was a relative latecomer.
......
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.
......
Babies can also be ineligible due to issues such as liver problems – Zolgensma's label warns it can cause these. Last year, two children in Russia and Kazakhstan died of acute liver failure several weeks after receiving it.
......
Its study found that 24 of 81 children given Zolgensma as of May 2022 had been subsequently treated with other SMA drugs.
......
it can be difficult to get insurance coverage after Zolgensma. Some health plans explicitly exclude add-on therapies if a child received it
......
It took nearly six months of paperwork and appeals – supported by Ben's medical team – before payment for Spinraza was approved.
Since then Ben, who now uses a wheelchair at age 2, began talking a lot more. He was suddenly able to move his lower body
https://www.reuters.com/business/healthc...023-08-12/
