CLINUVEL has announced that the European Medicines Agency (EMA) has requested further data from using SCENESSE in adolescent patients to allow a label expansion.
The company is seeking expansion to include the treatment of adolescent (12-17-year-old) patients with erythropoietic protoporphyria (EPP) patients.
It made a formal submission to the EMA in September 2022. The company said it will now conduct a post-authorisation study (CUV052) involving 12 adolescent patients who will be evaluated following one dose of SCENESSE. Results from the study are expected to be reported to the EMA in 2024.
The company said that to date, over 13,500 doses of SCENESSE have been administered to adult EPP patients.
It said clinical experts increasingly request SCENESSE for adolescent EPP patients. However, the cost of treatment for all paediatric patients requires prior authorisation from national health insurers to be fully reimbursed.
“We see a growing pool of adolescent patients treated under expert supervision, thereby assisting us to expand the database of safety and effectiveness data,” said CLINUVEL’s chief scientific officer, Dr Dennis Wright.
“Although, there is a good scientific reason to allow adolescent patients access to SCENESSE now, the EMA maintains its risk threshold for the drug product notwithstanding its safe use the past two decades.
“Once we have obtained additional clinical data, anticipated in 2024, the drug is expected to be available to all adolescent patients, giving them an approved treatment option for the first time.
“The convenience of being treated once every two months is reported to be an advantage for adolescent patients. It is estimated that 10 per cent of the total EPP European population are of 12 to 18 years of age,” added Dr Wright.