EPP is Clinuvel's lead clinical indication for SCENESSE® (afamelanotide)
- European marketing authorisation application submitted
- US Phase II EPP trial completed, US Phase III trial commenced 2012
- Orphan designated indication
About Erythropoietic Protoporphyria (EPP)
EPP is a rare life-long genetic disease found mainly in fair-skinned people. It is characterised by severe phototoxicity (intolerance of light) of the skin resulting in intolerable pain, swelling and scarring, usually of exposed areas such as the face, hands and feet. Reactions can vary from mild to extreme with hospitalisation and powerful pain killers required in the worst cases.
Children and adults living with EPP must avoid sunlight and even reflected light for life, often staying indoors or wearing protective clothing. Conventional sunscreens have little to no effect.
Since sun avoidance is recommended, patients lead lives where they are in the sun for very limited time. This can prevent normal social activities and the intense pain that is experience interferes with normal daily activities and can prevent adequate sleep.
Approximately 10,000 people globally are affected by EPP, an estimated 4,000 in the US.
It is hoped that with regular use, SCENESSE® will be able to provide medicinal photoprotection to EPP patients, allowing them to lead more normal lives. Read more about EPP...
EPP patient Mikey explains what effect the sun has on his skin.
Read more about Erythropoietic Protoporphyria
Keep up to date with our clinical trials, EPP blogs and videos.
Clinical results – EPP
In January 2009 Clinuvel announced interim results from its lead Phase III study of SCENESSE® (afamelanotide) in patients diagnosed with EPP (CUV017). The data from the first 14 Swiss patients to complete the 12 month study period were analysed, showing SCENESSE® was of clinical benefit in EPP. For more information, see the company's announcement.
In December 2009 Clinuvel announced preliminary results from its lead Phase III study of SCENESSE® in 100 patients diagnosed with EPP (CUV017). For more information, see the company's announcement.
Clinuvel released full results from the CUV017 study in July 2010. For more information, see the company's announcement.
In November 2011 Clinuvel announced results from it first US Phase II study of SCENESSE® (CUV030). You can read the results here.
In December 2011 Clinuvel announced results from its second Phase III study of SCENESSE® (CUV029), completing its pre MAA European program for the drug. You can view the results here.
Regulatory status
SCENESSE® (afamelanotide) has been granted Orphan Drug Designation by the EMA, FDA, TGA and Swissmedic for EPP.
In May 2010, the Italian Medicines Agency allowed for the prescription and reimbursement of SCENESSE® (afamelanotide) under Law 648/96 for Italian patients diagnosed with EPP. For more information, see the company's announcement.
In February 2012, Clinuvel submitted a marketing authorisation application for SCENESSE® (afamelanotide) with the European Medicines Agency. For more information, see the company's announcement.
A Phase III study of SCENESSE® (afamelanotide) in EPP commenced in the USA in early 2012, results from which are expected in early 2013.
References
- Harms JH, et al. ‘Mitigating photosensitivity of erythropoietic protoporphyria patients by an agonistic analog of alpha-melanocyte stimulating hormone.’ Photochem Photobiol. 2009 Nov-Dec;85(6):1434-9.
- Murphy GM. ‘Diagnosis and Management of the Erythropoietic Porphyrias’, Dermatologic Therapy 2003;16:57-64.
- Thunell S, Harper P, Brun A. ‘Porphyrins, Porphyrin Metabolism and Porphyrias. IV. Pathophysiology of Erythropoietic Protoporphyria - Diagnosis, Care and Monitoring of the Patient’. Scand J Clin Lab Invest 2000;60:581-604.
- Todd DJ. ‘Clinical Implications of the Molecular Biology of Erythropoietic Protoporphyria’, J Eur Acad Dermatol Venerol 1998;11:207-13.
