Share Price, page-17699

Is it possible that the following statement caused problems for the CHMP and the EU acceptance of Daybue:
"While the exact method by which trofinetide (Daybue) treats Rett syndrome is not yet fully understood it is believed to target core pathophysiology through several proposed pathways. Research suggests it works by mimicking the effects of a naturally occurring neuroprotective peptide in the brain etc"

Kens

While regulatory agencies prefer that the exact mechanism of action of a drug can be described, there are multiple drugs approved by both the FDA and the EC for which the mechanism of action is unknown. The key issues for both agencies are efficacy and safety; whether the benefits of the drug outweigh the risks.

I previously provided the example of Sanofi’s Rezurock for GVHD which received a negative opinion from the CHMP last October. Sanofi sought a re-examination and Rezurock was approved in January.

The CHMP documents from October show that the negative CHMP opinion was based on perceived unclear/unproven efficacy, especially as there were a number of side effects.
At the time of the CHMP negative opinion, Sanofi comments emphasized the company’s belief that the drug is proven to be efficacious and safe.

Below, in addition to Sanofi's comments, I’ve provided Acadia’s recent comments on the negative trend vote for trofinetide. Comments from the companies are fairly similar in emphasis, making me think that proven benefits v. risk is the main sticking point for the trofinetide application.

I say “proven” benefits, because in Sanofi’s case, the CHMP wasn’t convinced that the pivotal trial data used to support Rezurock’s marketing application truly proved its benefit.

Sanofi

“Sanofi is confident in the body of clinical and real-world evidence that underscores Rezurock’s consistent efficacy and well-established safety profile for treating third-line chronic graft-versus-host disease. We will continue to work closely with the European Medicines Agency with the aim of bringing this treatment to patients in the EU who are waiting.”

Rezurock is supported by safety and efficacy results from several clinical studies and real-world evidence….

Rezurock is currently approved in 20 countries, including the US, UK and Canada for the treatment of patients 12 years and older….

More than 17,000 patients living with cGVHD worldwide have been prescribed Rezurock since its first approval in the US in July 2021.

Acadia

“While the negative trend vote is disappointing and not what we hoped for, we believe the strong data that supported the approval of trofinetide for the treatment of Rett syndrome in the United States, Canada, and Israel speak to the meaningful benefits that trofinetide can deliver,” said Catherine Owen Adams, Chief Executive Officer. “We now have more than 1,000 patients on active treatment globally, from newly diagnosed 2-year-olds to adults who have lived with their disease for decades. Our ongoing real-world experience study in the U.S. continues to show outcomes that closely mirror the impact observed in rigorous randomized clinical trials conducted across a broad age range. We look forward to working with the EMA and other stakeholders to advance trofinetide as an important potential treatment option in the EU. Our commitment to the Rett syndrome community in the EU remains steadfast, and we are fully dedicated to making trofinetide available to individuals and families who urgently need a new therapeutic option.”