The license to Biogen included licenses to intellectual property that Ionis had acquired from Cold Spring Harbor Laboratory and University of Massachusetts.In November 2016, the new drug application was accepted under the FDAs priority review process on the strength of the Phase III trial and the unmet need, and was also accepted for review at the European Medicines Agency (EMA) at that time. It was approved by the FDA in December 2016 and by EMA in May 2017 as the first drug to treat SMA. Subsequently, nusinersen was approved to treat SMA in Canada (July 2017), Japan (July 2017), Brasil (August 2017), Switzerland (September 2017), and China (February 2019). Society and culture
Economics
Nusinersen list price in the USA is US$125,000 per injection which puts the treatment cost at US$750,000 in the first year and US$375,000 annually after that. According to The New York Times, this places nusinersen "among the most expensive drugs in the world".In October 2017, the authorities in Denmark recommended nusinersen for use only in a small subset of people with SMA type 1 (young babies) and refused to offer it as a standard treatment for all other people with SMA quoting an "unreasonably high price" compared to the benefit.Norwegian authorities rejected the funding in October 2017 because the price of the medicine was "unethically high".
However, at the higher dose levels (eight and 16 times the human dose equivalent), liver carcinomas and adenomas, lung adenomas, and adenomas of the Harderian gland occurred. No mutagenic effects were seen in assays. Testicular atrophy was noted in dogs and rats at four and eight times the human exposure, respectively. Limitations of use
It should not be used in patients with type 1 diabetes, diabetic ketoacidosis, pancreatitis, Fredrickson types I and V dyslipidemias, and severe renal impairment. Drug interactions
Juvisync should not be used with: strong CYP3A4 inhibitors, cyclosporine, danazol, gemfibrozil, and other fibrates. Caution should be used and the patient should be monitored if they are taking the following: amiodarone, dronedarone, ranolazine, calcium channel blockers, niacin, digoxin, coumarin anticoagulants, and colchicine. References
External links
"Juvisync". Drug Information Portal. U.S. National Library of Medicine.