- The US just approved a new treatment for a devastating rare genetic disease.
- Called Zolgensma, it’s a cutting-edge, one-time treatment called a “gene therapy” to treat spinal muscular atrophy at its genetic root.
- That means it should work longer than the typical drug – but that also translates to a much higher price tag.
- Zolgensma, made by the drugmaker Novartis’ AveXis unit, will cost $US2.125 million, the company said on Friday.
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The US Food and Drug Administration on Friday approved a cutting-edge new treatment for a devastating rare genetic disease called spinal muscular atrophy.
The treatment, Zolgensma, made by Novartis’ AveXis unit, is a one-time therapy that works to treat the disease at the genetic level. That means the drug’s effects should last a long time, though it’s still not known whether the treatment is permanent.
Novartis priced the product at $US2.125 million, or $US425,000 a year if paid over a five-year instalment plan. The $US2.125 million price tag makes Zolgensma the most expensive drug in the world.
On a conference call with reporters on Friday, Novartis executives called the price tag fair and reasonable, citing the value the treatment will deliver for patients.
‘A lifetime of possibilities’
“Zolgensma could create a lifetime of possibilities for the children and families impacted by this devastating condition,” Novartis CEO Vas Narasimhan said in a statement.
Spinal muscular atrophy affects an estimated 10,000 to 25,000 people in the US, according to the SMA Foundation.
The Swiss drug giant previously suggested that a price tag of up to $US5 million could be appropriate for the therapy because treating spinal muscular atrophy is already very expensive and Zolgensma is expected to have long-term benefits for those who get it.
Spinraza, the first therapy for spinal muscular atrophy, for instance, costs up to $US750,000 for the first year of treatment and $US375,000 in subsequent years.
Spinal muscular atrophy is the top genetic cause of death for infants
The condition, which affects muscle movement, is the leading genetic cause of death in infants. Babies born with spinal muscular atrophy have impaired reflexes and lessened muscular and head control, lacking the ability to sit without help.
Novartis expects that babies born with spinal muscular atrophy under age 2 will be its target population for Zolgensma, executives said on the call.
The company said that it would launch the drug immediately and that it expected about 1,100 patients to be eligible for it at launch.
Zolgensma’s price tag “falls within the upper bound” of a cost-effective price, Dr. Steve Pearson, the president of the Institute for Clinical and Economic Review, a group that measures the cost-effectiveness of new drugs, said on Friday. The group found that fair price ranges would be about $US1 million to $US2 million, depending on how the figures are calculated.
“Zolgensma is dramatically transforming the lives of families affected by this devastating disease,” Pearson said in a statement, adding: “Insurers were going to cover Zolgensma no matter the price, and Novartis has spoken publicly about considering prices that approached $US5 million. It is a positive outcome for patients and the entire health system that Novartis instead chose to price Zolgensma at a level that more fairly aligns with the benefits for these children and their families.”
Payers like health insurers will have the option to pay for Zolgensma over up to five years, Novartis said, adding that more than 15 payers were in “advanced discussions” around contracts with its AveXis unit.
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