Biogen’s Key Drug for the Treatment of Spinal Muscular Atrophy




Biogen (BIIB) is a global biopharmaceutical company with a focus on core areas of multiple sclerosis (or MS), neuroimmunology, Alzheimer’s disease, dementia, spinal muscular atrophy (or SMA), and amyotrophic lateral sclerosis (or ALS).

Approved by the FDA for the treatment of SMA in pediatric and adult patients in December 2016, Spinraza is one of Biogen’s key products. Biogen presented Phase 3 end-of-study data in April 2017 that showed a significant improvement in motor function in children with later onset SMA compared to untreated children. These findings continued to support the efficacy and favorable safety profile of the drug for a broad range of individuals with SMA.

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Spinraza is the first approved treatment for SMA in the European Union (or EU). Reviewed under the accelerated assessment program, the drug received marketing authorization in the EU in June 2017. The drug also received approval in Japan in July 2017 for treating infantile SMA and in September 2017 for treating pediatric and adult patients with SMA.

Disease profile

SMA is characterized by the loss of motor neurons in the spinal cord and lower brain stem, which leads to severe and progressive muscular atrophy and weakness. Patients suffering from a severe type of SMA can become paralyzed.

Sales growth

Sales of Spinraza grew from $4.6 million in 2016 to $883.7 million in 2017. Of its total revenue in 2017, $657 million came from US sales, and $226.7 million came from sales in the rest of the world. Biogen expects the growth rate of the drug to moderate over time as patients transition to dosing every four months.

In the next part of the series, we’ll take a look at Amgen’s Repatha.


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