RE: The poster22 Nov 2020 19:15
Post 4
You say the p3 trial failed. It didn't meet the endpoints but the company has worked with the EMA and I recall it being said that the EMA wanted the company to make the submission!
"Chronocort® Europe: preparing for commercialisation
Diurnal’s second product candidate, Chronocort®, provides
a drug release profile that the Group believes better mimics the body’s natural cortisol circadian rhythm, which current therapies are unable to replicate, and is designed to improve disease treatment for adults with CAH, as measured by androgen (male sex hormone) control.
During the year, Diurnal submitted an MAA to the EMA for
Chronocort® as a treatment for adult and adolescent patients with CAH. The MAA subsequently passed validation with the EMA in March 2020, confirming that the submission is sufficiently complete to begin the formal review process. The pMAA submission followed a positive meeting with the EMA and written formal Scientific Advice confirming the clinical and regulatory pathway for Chronocort®, based on detailed analysis of data from the Group’s Phase 3 study conducted in a total of 122 patients enrolled across 11 clinical sites, the largest ever interventional clinical trial in CAH, and an open-label
safety extension study for patients completing treatment in
the Phase 3 study. This extension study is assessing the impact of treatment with Chronocort® over an extended period, regardless of whether the patients were initially treated with Chronocort® or standard of care.
A significant proportion of patients eligible to enter the
follow-on study did so, and patient retention rates in this
study have been high, with a number of patients on this trial having been treated for over 42 months at the latest data-cut in April 2020. Patients on this trial have, to date, shown sustained benefit from extended Chronocort® treatment.
Shortly after the financial year end, Diurnal received the first formal set of questions from the EMA (“Day 120 questions”) and is working to provide responses to these, including data from the latest data-cut of the Chronocort® extension study taken during the financial year, in line with the EMA’s timetable.
Assuming responses to these (and any subsequent) questions are acceptable to the EMA, Diurnal anticipates receiving recommendation for approval of Chronocort® in Europe in Q1 2021, with formal approval to follow in Q2 2021. In parallel with the MAA submission, Diurnal will apply for confirmation of Orphan Drug Status for Chronocort® in CAH.
Assuming the EMA approves Chronocort® for the treatment of CAH, Diurnal subsequently intends to submit a line extension in Europe for the treatment of AI, a much larger market opportunity, once an existing Orphan Drug Designation for the product Plenadren® in the treatment of adult AI has expired. This planned submission will use existing clinical data, along with data from a planned study comparing Chronocort® with Plenadren®.