2020 Announcements News Regulatory

Australian TGA approves SCENESSE®

Executive summary

  • SCENESSE® approved by Australian TGA under Priority Review
  • First approved therapy for erythropoietic protoporphyria (EPP) in Australia
  • SCENESSE® indicated for the prevention of phototoxicity in adult EPP patients, to be administered every two months
  • Follow up of patients as part of CLINUVEL’s commitment

Melbourne, Australia, 27 October 2020
CLINUVEL PHARMACEUTICALS LTD today announced that the Australian Therapeutic Goods Administration (TGA) has approved the registration of its drug SCENESSE® (afamelanotide) for the prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP).1 SCENESSE® is the first treatment approved for EPP patients in Australia.


TGA approval for registration
The TGA evaluated the SCENESSE® dossier under Section 25 of the Therapeutic Goods Act (1989) and notified CLINUVEL in line with Section 25(3) of its decision to approve the drug’s registration on the Australian Register of Therapeutic Goods (ARTG). The notification completes a nine-month review by the TGA following validation of the
SCENESSE® dossier.

SCENESSE® will 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, SCENESSE® will 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 SCENESSE® on 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.

SCENESSE® was 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.

First systemic photoprotective drug for erythropoietic protoporphyria (EPP)
SCENESSE® is the first approved treatment for EPP patients in Australia and the only drug to have been evaluated for safety and effectiveness in randomised placebo-controlled clinical trials in this disease anywhere in the world.

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.

SCENESSE® has 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 SCENESSE® implant contains 16mg of the active ingredient afamelanotide which is released into the body over a period of approximately 10 days. Afamelanotide – an analogue of the naturally occurring hormone alpha-melanocyte stimulating hormone – binds to melanocortin-1 receptors on cells and provides total body protection from light and UV (systemic photoprotection) to EPP patients. Over 10,000 doses of afamelanotide have been administered to over 1,400 individuals worldwide, with a positive safety profile maintained to date.

“It is deeply satisfying to share with all involved that SCENESSE® – an innovative, Australian-developed drug – has been approved to treat Australian EPP patients,” CLINUVEL’s Chief Scientific Officer, Dr Dennis 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 SCENESSE® for 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.

“Our consistency, persistence in the dialogue with the TGA over more than 10 years has led to this landmark decision,” CLINUVEL’s Regulatory Affairs Manager, Dr Monique Baldwin said. “Our full attention now turns to actively progressing discussions with the Pharmaceutical Benefits Advisory Committee to have SCENESSE® included in the Pharmaceutical Benefits List and enabling access for Australian EPP patients.”

1 SCENESSE® (afamelanotide 16mg) is approved in the European Union as an orphan medicinal product for the prevention of
phototoxicity in adult patients with erythropoietic protoporphyria (EPP). SCENESSE® is approved in the USA to increase “painfree” light exposure in adult EPP patients with a history of phototoxicity. Information on the product can be found on CLINUVEL’s website at www.clinuvel.com.

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