New Clinical Data Further Support Efficacy of Nusinersen in Spinal Muscular Atrophy

Nusinersen (Spinraza, Biogen), the first therapy approved for the treatment of spinal muscular atrophy (SMA), demonstrated long-term safety and efficacy in pre-symptomatic infants with the disease, according to data from a phase 2 clinical study.

SMA, a rare, genetic neuromuscular disease, causes severe, progressive muscle atrophy and weakness. The leading genetic cause of infant mortality, the disease affects approximately 1 in 10,000 live births.

Nusinersen, which was approved in December 2016, is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide that is administered intrathecally. Nusinersen achieved the first drug approval for the treatment of SMA.

The NURTURE study included 25 pre-symptomatic patients with SMA who received their first dose of nusinersen before 6 weeks of age.

According to the data, as of March 2019:

• One hundred percent of patients were alive without a need for permanent ventilation.
• The median age of patients in the study was approximately 3 years old.
• One hundred percent of the infants were sitting independently, compared with the natural history of the disease for which no patients with SMA type 1 would be able to do so and patients with SMA type 2 would need assistance.
• Patients were approaching the maximum mean score of 64 on the CHOP INTEND measure of motor function—63.4 for patients with 3 SMN 2 copies and 62.1 for those with 2 SMN 2 copies, demonstrating the powerful impact of early nusinersen treatment.
• Nusinersen demonstrated longer term efficacy up to approximately 4 years, with patients continuing to make progress and showing no signs of loss of motor function.
• Nusinersen was well-tolerated, with no new safety concerns identified after up to approximately 4 years of treatment.

The results are being presented at the CURE SMA Annual SMA Conference in Anaheim, California and the 5^th Congress of the European Academy of Neurology in Oslo, Norway.

“These study results demonstrate the durable impact of pre-symptomatic, proactive treatment on transforming the natural course of this disease,” Darryl De Vivo, MD, Sidney Carter Professor of Neurology and Pediatrics, Columbia University Irving Medical Center, said in a statement. “We are seeing an extensive number of patients continually meeting child motor development milestone and making unprecedented gains in a previously hopeless and often fatal condition. Spinraza is setting patients on a path toward survival, greater mobility and independence, which is helping improve outcomes for patients of all ages.”

Nusinersen costs $750,000 for year 1 of treatment, and approximately $375,000 per year thereafter.

Reference

Biogen Announces New Data Further Establishing Spinraza (nusinersen) as a Foundation of Care in Spinal Muscular Atrophy [news release]. Biogen. https://www.multivu.com/players/English/8560251-biogen-nurture-study-spinraza-spinal-muscular-atrophy-treatment-data/. Accessed July 1, 2019.