Company seeks accelerated approval based on changes in neurofilament as a surrogate for efficacy; with controversy over FDA’s Aduhelm approval in Alzheimer’s still raging, agency is again faced with deciding whether biomarker data in a neurodegenerative disease with high unmet need are sufficient to justify approval in the face of a failed clinical endpoint study.
With Biogen, Inc.’s tofersen now under review for a subgroup of ALS patients, the US Food and Drug Administration is once again faced with deciding whether biomarker data in a neurodegenerative disease with high unmet need justify accelerated approval in the face of a failed clinical endpoint study.
On 26 July, Biogen announced the FDA had accepted its application for tofersen, an antisense oligonucleotide, for treatment of superoxide...
Exploring How Everyday Industrial Compounds Enter Our Systems, Their Health Implications, and the Ongoing Quest for Safer Alternatives .
With the deadline for comments on the US FDA’s draft guidance less than a month away, the Pink Sheet offers an infographic outlining what the agency is expecting in diversity action plans and when that might be implemented.
The agency also should explain the potential consequences for sponsors if enrollment goals are not met, stakeholders say in comments on the DAP draft guidance. Some commenters urged the FDA to apply waiver criteria flexibly, especially for rare diseases.
The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.
Brazil has also published its recent regulatory reliance regulations in English, a move designed to increase confidence in its decisions as it presses on with aligning its national practices with global best practices.
The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.
The FDA and sponsors of two new accelerated approval drugs for PBC have taken steps in study design, initiation and reporting transparency to ensure timely completion; Intercept’s COBALT trial of obeticholic acid was negatively impacted by product safety labeling changes and a high crossover rate to commercial drug.
