Biogen’s SPINRAZA® (nusinersen) Receives Positive Recommendation from NICE for Funding in the United Kingdom for the Treatment of Infants, Children and Adults with Spinal Muscular Atrophy

By Cure SMA | Published On May 15, 2019

Biogen’s SPINRAZA® (nusinersen) Receives Positive Recommendation from NICE for Funding in the United Kingdom for the Treatment of Infants, Children and Adults with Spinal Muscular Atrophy

Biogen Inc. today announced that The National Institute for Health and Care Excellence (NICE) in the United Kingdom has recommended funding for SPINRAZA (nusinersen) on the National Health Service (NHS). The positive recommendation is for the treatment of infants, children and adults with 5q spinal muscular atrophy (SMA), including pre-symptomatic and symptomatic SMA Types 1, 2 and 3.

Established in 1999, NICE provides advice and standards on value in healthcare to the NHS. The NICE recommendation was based on the comprehensive set of data for nusinersen highlighting its clinically meaningful benefits for individuals in all age groups with SMA. New data on the efficacy and safety of nusinersen was presented at the 71st Annual Meeting of the American Academy of Neurology in Philadelphia (May 4-10). As of March 31, 2019, more than 7,500 individuals with SMA are being treated with SPINRAZA worldwide, based on patients across the post-marketing setting, Expanded Access Program (EAP) and clinical trial participants.

Cure SMA Provides Seed Funding for Spinraza

From 2003 to 2006, Cure SMA provided the very first research funding needed to begin investigation into the therapeutic approach behind SPINRAZA. We would like to thank and acknowledge Cold Spring Harbor Laboratory (CSHL) and the University of Massachusetts Medical School for generating critical intellectual property for the program that was licensed to Ionis Pharmaceuticals. We specifically thank Drs. Adrian Krainer, Yimin Hua and colleagues at CSHL for years of dedication to and hard work on the preclinical development of Spinraza for SMA, and Drs. Ravindra Singh and Elliot Androphy for their work funded by Cure SMA in originally identifying the ISSN1 gene sequence, which is the sequence targeted in Spinraza.

For More Information

To learn more about Spinraza please visit www.curesma.org/spinraza.

Topics: Research, Front Page News

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