Ionis Pharmaceuticals announced that it has earned a $40 million milestone payment from Biogen associated with the pricing approval of Spinraza (nusinersen) in Japan.

Spinraza was approved for individuals with infantile-onset spinal muscular atrophy (SMA) by the Pharmaceuticals and Medical Devices Agency in Japan in June 2017. To date, Ionis has earned more than $435 million from Biogen related to Spinraza.

"We are grateful to the Japanese SMA community, including the patients who participated in the clinical studies, along with their families and the investigators, for their dedication, perseverance and support. Each of them played a pivotal role in bringing Spinraza to the Japanese market and we are pleased that Spinraza is now available to Japanese infantile-onset individuals with SMA," said Stanley T. Crooke, chairman and chief executive officer of Ionis Pharmaceuticals. "Spinraza is now approved in the US, EU, Japan, Canada and Brazil, which brings us closer to achieving our shared goal with Biogen that all patients with SMA will have access to this life-changing medicine."

"We expect to see continued strong patient demand for Spinraza as the launch continues in the U.S. and with the commercial rollouts in Europe, Japan and around the globe," said B. Lynne Parshall, chief operating officer at Ionis Pharmaceuticals. "Biogen is working to expand access to Spinraza, with marketing authorization applications currently under review in Switzerland, Israel, South Korea and Australia, and additional filings planned. We believe the speed and breadth at which Spinraza has achieved marketing approval across geographies so far is a recognition of the immense value Spinraza may bring to patients."

Biogen licensed the global rights to develop, manufacture and commercialize Spinraza from Ionis Pharmaceuticals in August 2016 and is now responsible for all development, regulatory and commercialization activities and costs for Spinraza. Spinraza was first approved by the U.S. Food and Drug Administration (FDA) on December 23, 2016, within three months of regulatory filing.

Spinraza is an antisense oligonucleotide (ASO), using Ionis Pharmaceuticals' proprietary antisense technology, that is designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to SMN protein deficiency. Spinraza alters the splicing of SMN2 pre-mRNA in order to increase production of full-length SMN protein. It was discovered and co-developed by Ionis Pharmaceuticals, a leader in antisense therapeutics, and Biogen. ASOs are short synthetic strings of nucleotides designed to selectively bind to target RNA and regulate gene expression. Through use of this technology, Spinraza has the potential to increase the amount of full-length SMN protein in individuals with SMA.

Spinraza must be administered via intrathecal injection, which delivers therapies directly to the cerebrospinal fluid (CSF) around the spinal cord, where motor neurons degenerate in patients with SMA due to insufficient levels of SMN protein.

In 2016, in response to the urgent need for treatment for the most severely affected individuals living with SMA, Biogen sponsored one of the largest, pre-approval Expanded Access Programs (EAP) in rare disease free of charge. The EAP has successfully led to the initiation and ongoing treatment of almost 600 eligible individuals with infantile-onset SMA (most likely to develop Type 1) across 24 countries.