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    Clinuvel's Program - Erythropoietic Protoporphyria (EPP) PDF Print E-mail

    EPP is Clinuvel's lead clinical indication for SCENESSE (afamelanotide)

    Clinical and regulatory progress:
    • European Phase III EPP trials completed see here
    • Confirmatory European Phase III EPP trials commenced
    • US EPP trials due to commenced; see FDA approval announcement here
    • Orphan designation indication - read more

    About Erythropoietic Protoporphyria (EPP)

     

    EPP scarring on lips
    EPP symptoms on the lips
    Also known as EPP, erythropoietic protoporphyria is a rare inherited porphyrin metabolism disorder that affects between one in 200,000 and one in 750,000 people. This disorder causes a chemical known as protoporphyrin IX to accumulate in the skin. When the skin is exposed to the sun, these molecules undergo a chemical reaction that results in swelling, excruciating pain and scarring.

    ...sometimes described as like having hot needles stuck into the skin

    The pain is sometimes described as like having hot needles stuck into the skin. The lifelong pain experienced by these patients can be so severe that they require continuous treatment with analgesics to cope with the incessant pain. Typically, these patients become socially isolated because of the lack of an efficacious treatment and their need to continuously avoid sunlight.

    Sun avoidance by remaining indoors or wearing sun protective clothing including cotton gloves and a wide brimmed hat is the first line in EPP management. Drugs such as B-carotene, cysteine and cimitedine have been used and because the disease is inherited, genetic counselling is recommended.

    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.

    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.

    Clinical results – EPP

    Clinuvel released full results from the CUV017 study in July 2010

    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.

    Regulatory status

    SCENESSE (afamelanotide) has been granted Orphan Drug Designation by the EMA, FDA and Swissmedic for EPP.

    In May 2010, the Italian Medicines Agency allowed for the prescription and reimbursement of SCENESSE under Law 648/96 for Italian patients diagnosed with EPP. For more information, see the company's announcement.

    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