Novartis gets okay for SMA gene therapy, with $2m price tag

After all the anticipation, Novartis has the FDA approval it has been seeking for Zolgensma, its gene therapy for spinal muscular atrophy, and has priced it at the low end of expectations.

The FDA green light came ahead of the weekend and means that Novartis will move straight ahead with a launch of Zolgensma (onasemnogene abeparvovec) for children with all types (1-3) of SMA aged less than two years, with 60 centres across the US already geared up to deliver the therapy.

Older patients with the disease will have to wait for the outcome of clinical trials and a possible label extension. The FDA’s approved label covers around 80% of all infants with SMA.

Zolgensma will be launched at a price of $2.125 million, which Novartis says works out at $425,000 per year over five years, around half the cost of 10 year’s treatment with Biogen’s Spinraza (nusinersen), which until now was the only FDA-approved drug to treat SMA.

Novartis maintains that the current 10-year cost of chronic SMA therapy, which is given over the patient's lifetime, can often exceed $4 million in just the first 10 years of life.

Nevertheless, Zolgensma’s price has already created a Twitter debate on the subject of pharmaceutical pricing policies, but is around half the level Novartis had previously suggested was cost-effective as a one-shot treatment – and crucially has the backing of the authoritative Institute for Clinical and Economic Review (ICER).

“Zolgensma is dramatically transforming the lives of families affected by this devastating disease, and given…new efficacy data for the pre-symptomatic population, the price announced today falls within the upper bound of ICER’s value-based price benchmark range,” said Steven Pearson, ICER’s president.

The organisation’s assessment makes the assumption that Zolgensma’s clinical benefits for children with the devastating rare disease will be long-lasting, and that the US “widely and rapidly adopts the recommendation to add screening for SMA to routine newborn screening.”

ICER recommended last month that the price of Spinraza be reduced from $750,000 for the first year of treatment with, and $375,000 annually thereafter, by a substantial margin. The drug made $1.7 billion for Biogen last year and is its most important growth product after setbacks in its Alzheimer’s disease pipeline. Analysts at Clarivate have suggested Zolgensma could be the first gene therapy blockbuster with sales of $2.1 billion by 2023.

Novartis’ AveXis unit – which developed the gene therapy and was bought by the Swiss big pharma for $8.7 billion last year – says that it is “working closely with payers to create five-year outcomes-based agreements and novel pay-over-time options” to help with access to the gene therapy.

Acting FDA Commissioner Ned Sharpless said the approval “marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases.” Zolgensma is only the second gene therapy to be approved in the US after Spark’s Luxturna for a rare eye disease.

“With each new approval, we see this exciting area of science continue to move beyond the concept phase into reality,” added Sharpless.