An interview held with Catherine Owen Adams at the T D Cowen...

An interview held with Catherine Owen Adams at the T D Cowen Conference overnight provides some valuable insight into the recent European CHMP Negative Opinion. (The interview is available on the Acadia website).

Around the 24 minute mark, Owen Adams is asked about the Negative Opinion for DayBue.

As she tells it, the key issue that the CHMP reviewers had was with the two scales that were used for the Lavender trial primary outcome measures– the RSBQ and the CGI-I. They did not consider these primary outcome measures to be “clinically meaningful”.

When asked by the interviewer for further clarification, she uses examples. If a girl went from not being able to use her hands to using her hands, that would be considered by the reviewers to be clinically meaningful. If a girl progressed from being able to verbalize just one word to four words, that would be considered clinically meaningful.

Thus, the plan of attack for the re-examination by a new panel of reviewers is to use the same data but bring it to life by providing detail on the skills gained.

As Owen Adams describes it, Acadia will repackage the trial data, adding patient voices.

A second way in which the voice of the patient will be incorporated is by including input on the European experience of DayBue. This was not yet available for the original submission, but as the named patient program has been operating for a while now, it is now possible to add European voices to the submission for re-examination.

“European voices” will include European DayBue patients/carers, European advocacy communities and KOLs in the form of the European physicians who have been treating Rett patients with DayBue.
Owen Adams says it is possible to bring all of these voices into the process as part of the Scientific Advisory Committee.

I am heartened somewhat after listening to this interview. The “not addressing the core symptoms” reason given in the CHMP opinion now makes more sense to me. What the reviewers want is specific, concrete evidence of skills gained after being treated with DayBue.

I am confident that Catherine Owen Adams and her team will be giving this their very best shot.