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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.

In June 2018 CLINUVEL submitted a new drug application with the US FDA seeking marketing authorisation for SCENESSE® . You can read the announcement here.

In January 2019 the FDA set a PDUFA date of 8 July 2019 and granted SCENESSE® a Priority Review. You can read the announcement here.

In June 2019 the FDA extended the PDUFA date of SCENESSE®  to 6 October 2019. You can read the announcement here.

About erythropoietic protoporphyria (EPP)

EPP symptoms on the lips

EPP is a rare metabolic disorder that causes severe anaphylactoid reactions to light (phototoxicity). Patients incur physical burns and ulcers, and are in a state of crisis following light exposure, summarised as phototoxicity. This usually occurs within minutes of exposure to bright lights, especially sunlight.

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.

EPP symptoms can be acute, or delayed (subacute), most often expressed as generalised oedema, effusion in tissue and distortion of the skin. As little as a few minutes of light outdoors (even when it is overcast or transmitted through a window) or artificial light exposure may be sufficient to evoke EPP symptoms.

Phototoxicity is unresponsive to traditional pain and burn management techniques and patients can be incapacitated for days before reactions subside. Most patients withdraw from light exposure in order to manage their phototoxic symptoms.

Approximately 10,000 people globally are affected by EPP, an estimated 4,000 in the US.

Read more about Erythropoietic Protoporphyria

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 EMAFDATGA and Swissmedic for EPP.

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.