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The trial of the immunotherapy combination, although approved in other renal cell carcinoma indications, did not meet the endpoint of disease-free survival in patients with localized disease.

If approved, N-803 — with the brand name Anktiva — plus the Bacillus Calmette-Guérin vaccine would be the first immunotherapy combination for this indication in 23 years. The Prescription Drug User Fee Act target action date is May 23, 2023.

If approved, tofersen will be the first treatment that targets a genetic cause of ALS. The FDA assigned a Prescription Drug User Fee Act action date of Jan. 25, 2023, but said it will hold an advisory committee meeting for this application.

If approved, this would be the first therapy to treat patients with Rett syndrome, a rare genetic neurological disorder mostly in girls.