BrainStorm discovered a statistical error while preparing the stem cell therapy's BLA, and corrected data show a statistically significant treatment difference for a key secondary endpoint in less advanced ALS. However, the Phase III trial still failed its primary clinical efficacy endpoint.
BrainStorm Cell Therapeutics Inc. is citing the regulatory environment, the US Food and Drug Administration’s “urgency” on drugs for amyotrophic lateral sclerosis, as well as a newly corrected subgroup analysis for a secondary endpoint in its Phase III trial, in explaining why it plans to seek approval of the stem cell therapy NurOwn.
The success of a biologics license application submission would seem to face long odds given the Phase III trial failed...
The Pink Sheet explores how regulatory approvals for Leqembi differ around the world, and looks at what is coming next.
Upcoming FDA user fee goal dates include novel products from a busy lung cancer pipeline, a single-dose passive immunization against RSV for infants, what could be the first oral HAE acute treatment, and a first-in-class HAE preventive antibody.
The agency said it will consider knee extensor muscle strength as a potential intermediate clinical endpoint to support accelerated approval, but Stealth CEO Reenie McCarthy said announcing it via complete response letter is confusing and inefficient.
The European Medicines Agency says that a regulatory reliance pilot that it is supporting to speed up assessments of post-approval changes appears to be accelerating evaluation timelines in non-EU countries.
The EU product information for Novo Nordisk’s semaglutide medicines is to be updated to include non-arteritic anterior ischemic optic neuropathy as a side effect with a frequency of “very rare.”
The Pink Sheet explores how regulatory approvals for Leqembi differ around the world, and looks at what is coming next.
Experts from EUCOPE explain why the Council of the EU’s position on the proposed overhaul of the general pharmaceutical legislation could offer more predictability for companies than the commission’s initial offering.
