Clinuvel Pharmaceuticals Ltd has received approval from The Australian Therapeutic Goods Administration (TGA) for registration of its drug SCENESSE.
SCENESSE has been developed for the prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP) and is the first treatment approved for EPP patients in Australia.
EPP is a poorly characterised rare metabolic disorder causing lifelong absolute light intolerance. Due to a genetic defect, EPP patients suffer debilitating acute phototoxic reactions (anaphylactoid reactions and second-degree burns) after just a few minutes of exposure to visible light (including sun and artificial light).
Burns and reactions may last days to weeks. Without treatment patients must withdraw from light exposure to prevent phototoxicity, leading to lifelong social isolation.
SCENESSEhas been shown to reduce the incidence and severity of phototoxic reactions and increase the amount of time patients can expose to light without incurring phototoxicity.
The drug is administered as a controlled-release subcutaneous injectable implant in an outpatient setting. Each SCENESSEimplant contains 16mg of the active ingredient afamelanotide which is released into the body over a period of approximately 10 days.
CLINUVEL’s Chief Scientific Officer, Dr Dennis Wright said the notification completes a nine-month review by the TGA following validation of the SCENESSEdossier.
SCENESSEwill be registered in Australia for the indication “the prevention of phototoxicity in adult patients with EPP”.
The drug will be available as a prescription medication in Australia, to be administered by trained and accredited healthcare professionals every two months.
CLINUVEL will implement a comprehensive training and accreditation program, ensuring that healthcare professionals are provided with information in line with the approval.
As an innovative chemical entity, SCENESSEwill be subject to additional safety monitoring, as well as a formal risk management plan and regular pharmacovigilance (drug safety) reporting to the TGA.
This is in line with the drug’s European and US marketing authorisations, received in 2014 and 2019 respectively, and allows for long-term follow up of patient safety.
Following inclusion of SCENESSEon the ARTG, CLINUVEL is legally allowed to supply the product within Australia. CLINUVEL is engaging with Pharmaceutical Benefits Advisory Committee to make the drug available on the Pharmaceutical Benefits Scheme in Australia.
SCENESSEwas granted an orphan drug designation in 2010, recognising the potential of the drug to treat a rare (affecting fewer than 2,000 individuals in Australia) metabolic disease. This designation – a regulatory mechanism to encourage development of medicinal products for patients with severe and neglected diseases – entitled CLINUVEL to a waiver of registration fees as well as review under the TGA’s priority registration pathway.
It is deeply satisfying to share with all involved that SCENESSE– an innovative, Australian-developed drug – has been approved to treat Australian EPP patients,” Dr Wright said.
“Our team is particularly grateful to individuals and families within the Australian EPP patient community who have supported our development program over the past 15 years as we work towards enabling treatment access.
“The TGA is the third global regulatory agency to evaluate and approve SCENESSEfor EPP. Our stepwise approach to regulatory engagement continues to be validated by today’s news while we progress discussions in other regions where EPP patients lack access to treatment,” Dr Wright said.