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Novartis' $2.15m SMA drug Zolgensma becomes most expensive FDA-approved therapy

Published on 28/05/19 at 09:52am

The FDA has approved Novartis subsidiary AveXis’ $2.15 million gene therapy Zolgensma for children with spinal muscular atrophy (SMA).

The treatment has become the first FDA-approved gene therapy for children with SMA.

The drug has been approved for patients living with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene, under the age of two.

Zolgensma is designed to address the genetic root cause of SMA in providing a functional copy of the SMN gene. The treatment is delivered intravenously with a one-time infusion.

The therapy will cost $2.15 million per patient. However insurers will be able to pay $425,000 a year over five years. The hefty price tag makes Zolgensma the most expensive drug ever approved by the FDA.

“This is potentially a new standard of care for babies with the most serious form of SMA,” said pediatric neurologist at Children’s Hospital Los Angeles, Dr Emmanuelle Tiongson, who has provided Zolgensma to patients under an expanded access programme. “The job now is trying to negotiate with insurers that this would be a long-term savings.”

Novartis is also offering refunds if the treatment does not work and upfront discounts to payers who commit to standardised coverage turns.

Wall Street analysts have forecast sales of $2 billion by 2022. For comparison Biogen’s SMA treatment Spinraza generated sales worth $1.7 billion last year. The drug is forecast to reach $2.2 billion by 2022.

SMA is the leading genetic cause of infant death. Around 450-500 children are born with SMA each year.

Louis Goss

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