April 25, 2023 - FDA Approves Tofersen

Tofersen, a drug pioneered by Dr. Smith alongside Ionis Pharmaceuticals (Frank Bennett), and UCSD (Don Cleveland), was approved by the FDA for the treatment of one form of familial ALS. The approval of Tofersen is not only a milestone in the history of ALS, but also a milestone for the Center which has now been involved in the development of 2 out of the 5 drugs available for ALS.

Pictured above is the UCSD and CNS SOD1 Antisense team. Not pictured: Dr. Bennett (Ionis Pharmaceuticals). From left to right: Tim Miller, Dr. Smith, Don Cleveland, Melissa McAlonis-Downes.

Above is an early photo of the antisense team that was taken outside of Ionis's original laboratory in Carlsbad. Pictured from left to right: Tim Miller, Don Cleveland, Brett Monia, Tom Condon, Dr. Smith, Frank Bennett.

https://alsagoldenwest.org/fda-approves-treatment-of-als-associated-with-a-mutation-in-the-sod1-gene/

https://investors.biogen.com/news-releases/news-release-details/fda-grants-accelerated-approval-qalsodytm-tofersen-sod1-als#:~:text=for%20Alerts%20Search-,FDA%20Grants%20Accelerated%20Approval%20for%20QALSODY%E2%84%A2%20(tofersen)%20for%20SOD1,a%20Genetic%20Cause%20of%20ALS&text=CAMBRIDGE%2C%20Mass.%2C%20April%2025,GLOBE%20NEWSWIRE)%20%2D%2D%20Biogen%20Inc.