Biogen’s big breakthrough drug Spinraza runs into a barrier as NICE turns thumbs down on reimbursement
Once again, UK regulators have proven that they are unwilling to pay a heavy price for the breakthrough drugs that the FDA enjoys hustling through to quick approvals.
In this case, the UK’s NICE is at least temporarily closing the door on Spinraza, Biogen’s landmark new therapy for rare cases of SMA, despite discounting the $750,000 US list price for the first year of therapy.
NICE acknowledged that it would only have to foot the bill for a relatively small group of patients and that the drug, nusinersen, had passed muster as a new therapy for a severe unmet medical need. But without a better idea of the longterm impact of the drug on patients, some of whom face a death sentence at the age of 2, it wasn’t possible to conclude that the price was justified.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.