Posted by Michael Wonder on 15 Jan 2020
Reform at the FDA—in need of reform
14 January 2020 - On 23 December 2016, the US FDA announced the approval of nusinersen, the first drug for the devastating disease spinal muscular atrophy.
The approval was based on an interim analysis of 82 patients in a single randomised trial; 40% of treated children improved, compared with none in the control group.
The FDA had requested that the interim analysis be conducted. A press release emphasised the speed of agency action, with the review division director saying, “We worked hard to review this application quickly.”
