Zolgesma: Does the 2.1 million drug from Novartis not deliver what it says on the tin?

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In 2018, Novartis launched a gene therapy for spinal muscular atrophy. It is now becoming apparent that the drug, which is expensive to treat, will bring less than expected.

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Novartis’ spinal muscular atrophy drug is not believed to be as effective as Novartis originally announced.

KEYSTONE

Spinal muscular atrophy is a genetic condition that leads to ongoing muscle wasting.  In the picture: Kim Knebel from Vaud, who was named Miss Handicap in 2009.

Spinal muscular atrophy is a genetic condition that leads to ongoing muscle wasting. In the picture: Kim Knebel from Vaud, who was named Miss Handicap in 2009.

KEYSTONE

Novartis promised to stop the disease with just a single infusion.  Cost: $ 2.1 million.

Novartis promised to stop the disease with just a single infusion. Cost: $ 2.1 million.

KEYSTONE

  • In 2018 Novartis launched the drug Zolgensma. It should be able to cure the hereditary disease spinal muscular atrophy with a single treatment.
  • But this one treatment has its price: the infusion with Zolgensma costs 2.1 million francs.
  • Now competitors from Novartis say that it has not been conclusively clarified whether Zolgensma really works reliably. They advocate combination studies with different drugs – which would make the treatment even more expensive.

There was great excitement when Novartis launched a drug for spinal muscular atrophy in 2018. The genetic disease, which leads to continued muscle wasting, could be combated for the first time with just a single treatment, the pharmaceutical company promised.

There was great excitement when it became known how expensive the drug called Zolgensma is: the target price for the treatment is 2.1 million US dollars. And finally, there was great excitement when Novartis announced in February 2020 that it would offer treatments Zolgensma to be raffled: Treatment should be free for a hundred children.

“Still need for action”

Now it turns out: Zogensma may not be the miracle cure for which it was advertised. That reports the «NZZ on Sunday»(Article behind payment barrier). Because: The competition from Novartis, specifically the pharmaceutical giants Roche and Biogen, are starting to test the Novartis drug in combination with their own drugs.

According to the two pharmaceutical companies, there is “still a need for medical action” in the treatment with Zolgensma. Novartis, however, says treatment with Zolgensma alone is enough to contain spinal muscular atrophy for a lifetime. “We stand firmly behind the established efficacy and safety data for Zolgensma as a one-off treatment option,” said Novartis.

Combined treatments

The background to this: Roche and Biogen also offer their own drugs for spinal muscular atrophy. At Roche, the corresponding drug must be taken daily as a syrup – at Biogen, a syringe is administered directly into the spinal fluid three times a year.

When Novartis’ Zolgensma came out, which promised to treat spinal muscular atrophy with just a single treatment, there was also skepticism. Parents of children with the genetic disease had ensured that the children would be treated with therapies from Roche or Biogen in addition to Zolgesma.

“Not all patients respond to Zolgensma”

Roche and Biogen are now taking advantage of this and want to implement the combination therapy from Zolgensma and their own substances. Experts agree: “There is evidence that not all patients respond to Zolgensma,” says pharmaceutical analyst Philippa Salter of “NZZ am Sonntag”.

And Stefan Schneider, pharmaceutical analyst at Bank Vontobel, also says: “We just don’t know at the moment how good the results will be and how long the gene therapy will last.” One thing is clear: the treatment against spinal muscular atrophy, which is already hardly affordable, becomes even more expensive with the combination therapies.

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