In February 2012 Clinuvel submitted a marketing authorisation application for SCENESSE® (afamelanotide) for erythropoietic protoporphyria (EPP) with the European Medicines Agency. For more information, see the company's announcement.
Phase III results – Europe and Australia
Clinuvel has completed two Phase III studies of SCENESSE® (afamelanotide) in patients with EPP.
In 2010 Clinuvel completed its first Phase III study of SCENESSE® (afamelanotide) in patients with EPP (CUV017). A total of 91 patients completed the 12-month study, in which an 11-point Likert scale and physician assessments through case report forms (CRF) were used to evaluate pain as a principal symptom of phototoxicity. The duration of daily (sun)light exposure was used to assess the willingness of patients to expose themselves during all seasons. Melanin density (reflecting changes in skin pigmentation, measured by spectrophotometry) and quality of life (Short Form 36 surveys) were also evaluated.
In an analysis of the total number of days (frequency distribution) on which patients experienced pain in the specific pain severity categories (severe, moderate, mild and none), a significant reduction of frequency was observed in patients on active drug [p=0.0023]. Characteristic to EPP, the majority of phototoxic reactions occurred during spring and summer.
In analysing the average pain severity experienced by the total number of patients, the assessment of all individual daily pain scores was significantly lower in patients receiving SCENESSE® compared to those receiving placebo [p=0.0017].
An additional evaluation of the pain scores in patients willing to modify behaviour by continuous exposure to daily (sun)light showed a positive trend toward a reduction in average pain score following active drug treatment [p=0.1654]. You can view the full Phase III results from our CUV017 EPP study here.
In 2011, Clinuvel completed a second Phase III study of SCENESSE® in patients with EPP (CUV029). Sixty-eight patients completed the nine month study across eight academic centres in Europe. Patients were evenly distributed between two treatment groups across all study sites, receiving either afamelanotide or placebo treatment every 60 days.
Results of the study showed that SCENESSE® was well tolerated, allowed EPP patients to expose their skin to sunlight during the middle of the day without or with reduced pain and improved their Quality of Life (QoL). Overall the study demonstrated a strong clinical benefit to patients, despite their deeply learned behaviour to avoid reactions caused by sun exposure.
View the full Phase III results from our CUV029 study here.
Read more about our erythropoietic protoporphyria program for SCENESSE®.
Phase II results - US
In 2011 Clinuvel completed a Phase II study (CUV030) SCENESSE® in adult US EPP patients. This six-month, randomised, multicentre, double-blind, placebo-controlled US study (CUV030) was primarily designed to confirm the efficacy and safety of subcutaneous bioresorbable afamelanotide implants (SCENESSE®) in reducing the severity of phototoxic skin reactions in patients with the rare light intolerance disorder erythropoietic protoporphyria (EPP), allowing them to lead ‘more normal’ lives.
Results of the study showed that SCENESSE® was well tolerated, allowed EPP patients to expose their skin to sunlight during the middle of the day and improved their Quality of Life (QoL). Read the company's Phase II US EPP results announcement.
Phase II results – Europe
In 2007 Clinuvel completed a Pilot Phase II study (CUV010) of SCENESSE® assessing the drug’s ability to reduce the number and severity of phototoxic reactions. In all five patients, the time to provoke pain (photoprovocation) was markedly prolonged (range: 384% - 1266%). Read the company’s Phase II EPP results announcement.