|Melbourne, Australia, 05 September 2022||ASX:
ADR Level 1:
- submission to European Medicines Agency to expand label
- 90-day review time
- SCENESSE® to treat adolescent EPP patients, 12-17 years of age
- four adolescent EPP patients treated with SCENESSE®, treatment well tolerated
- safety record of the drug after 16 years of continuous use
- approximately 21% of EPP patients are younger than 18 years of age
CLINUVEL today announced that it has submitted a formal application to the European Medicines Agency (EMA) to expand the approved indication for SCENESSE® (afamelanotide 16mg) to include the treatment of adolescent erythropoietic protoporphyria (EPP) patients. The submission follows the treatment of the first adolescent patients in the European Union, with SCENESSE® well tolerated by these patients to date.
“Having gained experience with the treatment of EPP patients over the past sixteen years, including those under real-world conditions, we have confidence in the safety profile of SCENESSE®,” CLINUVEL’s Chief Scientific Officer, Dr Dennis Wright said. “With experience gained from the recent treatment of adolescent patients, we believe the clinical benefit-risk assessment for use in this patient population supports the widening of the approved indication.”
EMA AUTHORISED INDICATION, “LABEL”, EXPANSION SUBMISSION
CLINUVEL has submitted a variation to its marketing authorisation dossier for SCENESSE® to the EMA, providing scientific data supporting the expansion of the authorised indication to include adolescent patients aged 12 years and older. In parallel, the Company has updated its paediatric investigation plan for SCENESSE® to reflect the proposed label expansion.
The EMA has a review timeline of 90 days, which may be extended if there are Agency requests for further information from the Company, including possible “clock stops”. The earliest possible approval of the variation is in December 2022.
“Having worked closely with the EPP community – patients, their families, and expert physicians – for nearly two decades, we are excited at the prospect of facilitating treatment for a larger group of patients, particularly knowing the severe impact EPP has during the development stages of adolescents, who are forced to live in isolation. We look forward to the EMA’s decision later this year,” Dr Wright said.
ADOLESCENT EPP PATIENT TREATMENT
SCENESSE® is the only approved therapy for EPP, a rare metabolic disorder which causes severe light intolerance (phototoxicity). To date, authorities in Europe, the USA and Australia have approved the drug for adult EPP patients.
To date, four adolescent EPP patients and one adolescent patient with xeroderma pigmentosum (XP) have received SCENESSE® treatment. As part of global pharmacovigilance, CLINUVEL is closely monitoring the effects of the drug in the younger patient population. Based on the data received, the safety profile and clinical benefit of SCENESSE® in these patients has been consistent with that seen in adults. Estimates from expert centres and population data suggest 21% of EPP patients are under 18 years of age.