Most Expensivest: NOVARTIS Wins Backing in Europe for £1.79 Million Gene Therapy



Cost watchdogs at England’s National Institute for Health and Care Excellence (NICE) have repeatedly been against drugmakers pricing over new drugs. However, in a review of Novartis’ pricey gene therapy, Zolgensma points out that the agency is considering more than just cost.


NICE endorsed Zolgensma, which is listed at £1.79 million per dose, for babies aged up to 12 months with type 1 muscular atrophy (SMA). If the draft guidance is concluded, the drug will become the most expensive med ever approved by NICE.


Because Zolgensma is a “potentially curative one-off gene therapy” that can offer “exceptional benefit” to patients, reviewers at the cost-effectiveness agency agreed that it is worth the high cost in certain patients.


NICE recommends that a “national multidisciplinary clinical team” review drug use for babies aged seven to 12 months. Type 1 SMA patients who fall outside NICE’s negotiated deal but within the medicine’s approved label will also be eligible for treatment consideration from the team. The deal advises treatment with Zolgensma before SMA symptoms developing.

Currently, NICE recommends Biogen’s Spinraza for presymptomatic SMA patients or patients with type 1, 2, or 3 diseases under a managed access deal. That med costs £450,000 for the first year and £225,000 for subsequent years. However, NICE and Biogen penned a deal for an undisclosed discount.


In 2019, when NICE expanded its Spinraza access agreement, the agency said between 600 and 1,200 children and adults in the U.K. are living with SMA. The eligible number of Zolgensma patients is much less: NICE remarks that around 65 babies born in England are diagnosed with SMA annually and that about 60% of that number have type 1 SMA.

The Zolgensma deal increases the growth odds for Novartis in England – and likely beyond because many other countries look to NICE when coming up with their own reimbursement decisions.


It is also another threat to a major Biogen med. Biogen is awaiting a highly-anticipated FDA decision on Alzheimer’s drug candidate Aducanumab this year, but, in the meantime, its top drugs have been facing increasing competition.


Novartis’ Zolgensma raked in $920 million in 2020, a 151% increase at constant currencies. Biogen’s Spinraza recorded $2.05 billion, a slight reduction from its 2019 performance.