Melbourne-based Clinuvel Pharmaceuticals (ASX:CUV) has announced its registration dossier for SCENESSE (afamelanotide 16mg) has been accepted by the Australian Therapeutic Goods Administration (TGA).
The TGA is evaluating SCENESSE under its Priority Review pathway as the first proposed therapy for adult patients with the rare metabolic disorder erythropoietic protoporphyria (EPP).
The pathway provides a formal mechanism for accelerated assessment of new therapies in Australia, reducing the TGA’s target review timeframe to 150 working days.
The company said the scientific dossier supporting SCENESSE includes data from the clinical development program that facilitated approval of the product in the US and European Union. This includes data on safety and effectiveness obtained from long-term use of the drug prescribed by EPP experts internationally in compassionate use, special access and post-authorisation programs.
SCENESSE is the world's first photoprotective drug. Its active component, afamelanotide, is an analogue of the naturally occurring alpha-melanocyte-stimulating hormone.
Afamelanotide has been designed to provide photoprotection. It acts as an antioxidant to prevent phototoxic reactions experienced by EPP patients when they expose to light sources.
SCENESSE is approved for adult EPP patients in the US and Europe.
“We rejoice each step towards our objectives,” said CLINUVEL’s regulatory affairs manager, Dr Monique Baldwin. "We are all committed to file SCENESSE in territories where there is a clinical need. The prospect of working with the TGA to implement a similar distribution program as in Europe and the USA is exciting.”