Ionis Pharmaceuticals has received $40 million in milestone earnings from Biogen following Japanese regulatory approval of the price of Spinraza (nusinersen), Biogen’s treatment for spinal muscular atrophy (SMA).
“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 CEO of Ionis.
Brooke added that Spinraza’s approval by U.S, European, Canadian, Brazilian and now Japanese regulatory agencies, Ionis is now “closer to achieving our shared goal with Biogen that all patients with SMA will have access to this life-changing medicine.”
Biogen is working to bring the treatment to other countries, including Switzerland, Israel, South Korea and Australia, where it is now under review by regulatory agencies, said B. Lynne Parshall, Ionis’ chief operating officer.
Biogen licensed global development, manufacturing and commercialization rights to Spinraza from Ionis in August 2016, paying Ionis a $75 million license fee. In so doing, it assumed responsibility for all activities and costs associated with the drug. Ionis is eligible to receive tiered royalties on Spinraza sales up to a percentage in the mid-teens, in addition to up to $150 million in milestone payments based on regulatory approvals.
Spinraza became the first approved SMA treatment in December 2016, when the U.S. Food and Drug Administration (FDA) supported its use in children and adults. The FDA made that decision within three months of Biogen’s regulatory filing.
SMA, a motor neuron or neuromuscular disease, is the most common genetic cause of death in infants under 2 years of age, according to the National Institutes of Health. It can affect any race and gender and is caused by a mutation in the survival motor neuron gene 1 (SMN1) . In healthy people, the SMN1 gene produces a protein critical to the functioning of nerves that control muscles. Without the SMN protein, those nerve cells work poorly and eventually die, leading to debilitating muscle weakness.
Spinraza is an antisense oligonucleotide (ASO) that Ionis co-developed — along with Biogen — based on its proprietary antisense technology. ASOs are short synthetic strings of nucleotides designed to selectively regulate gene expression by binding to target RNA. Spinraza is thought to work by increasing the amount of full-length SMN protein in people with SMA.
Biogen sponsors one of the largest Expanded Access Programs worldwide, offering Spinraza free of charge to patients requesting it in countries that have not yet approved its use. About 600 children with infantile-onset SMA in 24 countries began treatment under such programs, the release said.