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Novartis’ £1.79M gene therapy Zolgensma scores cost watchdog’s backing, threatening Biogen’s Spinraza

March 8, 2021 Eric Sagonowsky


Cost watchdogs at England’s National Institute for Health and Care Excellence (NICE) have clashed repeatedly with pharma companies over the price of new medicines. But a review of Novartis’ expensive gene therapy Zolgensma shows the agency is looking at more than just cost.

NICE endorsed Zolgensma, which costs £1.79 million per dose, for babies aged up to 12 months with type 1 spinal muscular atrophy (SMA). If the draft guidance is finalized, the med would become the most expensive drug ever to be approved by the cost-effectiveness agency.

Because Zolgensma is a “potentially curative one-off gene therapy” that can provide “exceptional benefit” to patients, NICE reviewers concluded it’s worth the high cost in certain patients.

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For babies aged seven to 12 months, a “national multidisciplinary clinical team” should discuss using the medicine, NICE said. Type 1 SMA patients who fall outside of NICE’s negotiated deal but within the drug’s approved label will also be eligible for treatment consideration from the team. The deal recommends treatment with Zolgensma before SMA symptoms develop.

RELATED: Novartis cites ‘transformative’ data on Zolgensma as it rolls out SMA gene therapy in Europe

Currently, NICE recommends Biogen’s Spinraza for presymptomatic SMA patients or patients with type 1, 2 or 3 disease under a managed access deal. That drug costs £450,000 for the first year and £225,000 for subsequent years, but NICE and Biogen inked a deal for a confidential discount.

In 2019, when NICE expanded its Spinraza access deal, the agency said between 600 and 1,200 children and adults in the U.K. are diagnosed with SMA. The eligible pool of Zolgensma patients is much smaller: NICE says around 65 babies are born in England each year with SMA, and about 60% have type 1 SMA.

RELATED: Biogen’s sales slide on Spinraza, Tecfidera as pivotal Alzheimer’s decision nears

The Zolegnsma deal adds opportunity for growth for Novartis in England—and potentially beyond, because many other countries look to NICE when making their own reimbursement decisions.

It’s also another threat to a key Biogen drug. Biogen is awaiting a much-anticipated FDA decision on Alzheimer’s drug candidate aducanumab this year, but, in the meantime, its stalwart drugs have been facing increasing competition.

Novartis’ Zolgensma generated $920 million last year, a 151% increase at constant currencies. Biogen’s Spinraza pulled in $2.05 billion, a slight decrease from its 2019 performance.



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