Products / Pipeline

Erythropoietic Protoporphyria (EPP)

EPP is CLINUVEL's lead clinical indication for SCENESSE® (afamelanotide 16mg)

Clinical and regulatory progress:

In December 2014, the European Commission approved SCENESSE® to prevent phototoxicity in adult patients diagnosed with EPP, following a recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP). You can read the announcement here.

CLINUVEL is currently working to make SCENESSE® available across Europe. If you have EPP and would like to receive updates on our program, please contact us.

About Erythropoietic Protoporphyria (EPP)

erythropoietic-protoporphyria-reaction-thumb
EPP symptoms on the lips

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.

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 16mg) 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). You can view the results here.

In November 2013 CLINUVEL announced results from its US Phase III study of SCENESSE® (CUV039). You can read the results here.

 Regulatory status

SCENESSE® (afamelanotide 16mg) 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 16mg) under Law 648/96 for Italian patients diagnosed with EPP. For more information, see this page.

In December 2014, the European Commission approved SCENESSE® to prevent phototoxicity in adult patients diagnosed with EPP, following a recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP). You can read the announcement here.

CLINUVEL is currently working to make SCENESSE® available across Europe. If you have EPP and would like to receive updates on our program, please contact us.

 

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.

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