Author Archives: Charles Moore

Ionis Earns $40 Million Spinraza SMA Regulatory Milestone Payment From Biogen

Ionis Pharmaceuticals has received $40 million in milestone earnings from Biogen following Japanese regulatory approval of the price of  Spinraza, Biogen's treatment for spinal muscular atrophy. So far, Ionis has earned more than $435 million from Biogen in Spinraza-related regulatory approvals and sales royalties, it said in a press release. The market performance speaks to the overwhelming success that the drug is having in treating the SMA patient population. Spinraza has been approved by U.S, European, Canadian, Brazilian and now Japanese regulatory agencies. Ionis is now working to bring the treatment to other countries including Switzerland, Israel, South Korea and Australia, where it is now under review by regulatory agencies Biogen licensed global development, manufacturing and commercialization rights to Spinraza from Ionis in August 2016. 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 supported its use in children and adults. The FDA made that decision within three months of Biogen's regulatory filing. 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.

Cure SMA Launches National Newborn Screening Campaign

The national nonprofit spinal muscular atrophy (SMA) support and advocacy organization Cure SMA has launched a grassroots campaign to have required newborn-screening for SMA in every state. The campaign was announced at the 2017 Annual SMA Conference held June 29-July 2 in Orlando, Florida. It was attended…

Researchers Design Exoskeleton to Help SMA Kids Walk, Some for First Time

The Spanish National Research Council (CSIC) recently unveiled a prototype of what it believes is the world’s first assistive exoskeleton designed to enhance mobility for children with the degenerative illness spinal muscular atrophy (SMA), a rare disease that affects 1 in 10,000 babies born in Spain. The 26-pound device, made primarily of…

SMA Survey