In infants with spinal muscular atrophy (SMA), treatment with Spinraza (nusinersen), the first FDA-approved drug for SMA, reduces the risk of death or permanent ventilation, according to new results from the Phase 3 ENDEAR study (NCT02193074).
Biogen presented the data at the British Paediatric Neurology Association (BPNA) annual conference Jan. 11-13 in Cambridge, England.
Read more at SMA News
Currently, there are two approved treatments for SMA: risdiplam and nusinersen. These treatments were initially approved temporarily under Managed Access Agreements while the NHS and NICE gathered data to assess their cost-effectiveness. Now, as these agreements come to an end, both treatments face a crucial review.
SMA UK are proud to join together with Treat SMA and MDUK to bring you this webinar about NICE’s upcoming Multi Technology Assessment for Spinraza and Risdiplam. 2024 sees the end of the managed access agreements (MAAs) for the two treatments. An expert committee will gather to assess the new clinical and real-world evidence, along…
This year the Managed Access Agreement for Spinraza and Risdiplam expires. The MAA was put in place five years ago so the pharmaceutical companies could gather more evidence to resubmit to NICE for approval on the NHS with Biogen extending their MAA by a year.
