Despite Failed Phase 3 Study, FDA Staff Says Biogen's ALS Drug May Have Clinical Benefit

 

• FDA's Peripheral and Central Nervous System Drugs Advisory Committee will virtually meet on Wednesday, March 22, to discuss Biogen Inc's 
  BIIB-0.69%+ Free Alerts
   ALS drug tofersen.
• FDA staff, citing a briefing document, said Biogen's investigational ALS drug might have a "clinical benefit" on a rare and aggressive form of the disease, despite failing a broader late-stage clinical trial last year. 
• "Despite the notable limitations of a failed study and the many post hoc exploratory analyses conducted after [the Phase III trial], the Division considers that the data may suggest a treatment effect of tofersen in SOD1-ALS," the FDA wrote. "This is a very rare and devastating disease; therefore, it is of utmost importance that we give full consideration to all available data."
• Tofersen did not achieve the primary goal of its Phase 3 VALOR trial, failing to prove it could slow SOD1-ALS patients' functional decline better than a placebo. Biogen said the p-value for the primary endpoint was p=0.97.
• "This is a situation where there is a negative clinical study that failed to show a statistically significant treatment effect in the prespecified primary analysis population," the report says. 
• It notes, however, that the study was limited in its ability to determine whether the drug affected the overall population because of how it was designed. 
• At the same time, the data indicate that targeted drug use was shown to reduce "a biomarker that is correlated with disease progression and prognosis in patients with ALS."
• The FDA accepted Biogen's application for full approval of tofersen in July. Last October, the agency extended its application review by three months, with a PDUFA date of April 25. 

https://www.benzinga.com/general/biotech/23/03/31433167/despite-failed-phase-3-study-fda-staff-says-biogens-als-drug-may-have-clinical-benefit