SCENESSE(afamelanotide 16mg) 新型复色疗法治疗皮肤惧光症,白癜风
Clinuvel has developed the concept of medicinal photoprotection and has refined the technology over the last five years to optimise the photoprotective effect of SCENESSE® (afamelanotide 16mg). The company has formulated a 16mg controlled-release subcutaneous implant for the delivery of SCENESSE®.
Roughly the size of a grain of rice, the implant is injected underneath the skin. Skin pigmentation is activated immediately and the photoprotective effect lasts up to two months.
SCENESSE® has a relatively short half life (less than 50 minutes) and trials to date have raised no serious safety concerns; thus far the drug is well tolerated by patients. The implant drug product has been chosen for its ease of use in dermatology.
SCENESSE® is the first melanocortin to be developed into a pharmaceutical product. To date, Clinuvel has invested over A$100 million developing the SCENESSE® implant drug product as a therapeutic photoprotective drug for patients who are most at risk from UV and sun exposure and as a novel repigmentation therapy in vitiligo.

New Drugs Online Report for afamelanotide
Information
Generic Name: afamelanotide  
Trade Name: Scenesse 
Entry Type: New molecular entity  
Development and Regulatory status
UK: Approved (Licensed) 
EU: Approved (Licensed) 
US: Phase II Clinical Trials 
UK launch Plans: Available only to registered users
Actual UK launch date:  
Comments
Feb 15: Approved in the EU [15].
23/02/2015 16:24:23 
Oct 14: EU positive opinion for afamelanotide to be used for prevention of phototoxicity in adults with EPP [14].
24/10/2014 15:21:05 
March 14: EMA review period extended to June 2014. [13]
25/03/2014 11:37:52 
Feb 12: Filed in the EU for prophylactic treatment in adult patients with EPP [10].
07/02/2012 22:04:55 
Clinuvel plans to file afamelanotide in the EU as a prophylaxis of the phototoxic symptoms of EPP before the end of 2011 (8). 
14/12/2011 10:33:22 
March 11: Filing anticipated H2 2011 [6]
17/03/2011 10:49:40 
Filing expected in 2010 (4).
28/05/2010 13:45:45 
Orphan status in Eu and US [1].
02/09/2009 08:47:36 
Trial or other data
Clinuvel plan to submit a Paediatric Investigational Plan (PIP) in the EU [12].
12/03/2013 10:43:02
May 12: NCT01605136 is a PIII placebo-controlled 6 month RCT enrolling between 75 and 100 eligible patients who will receive afamelanotide (16mg implants) or placebo on days 0, 60 and 120. The number and severity of phototoxic reactions, the type and duration of sun exposure, treatment-emergent adverse events and the use of concomitant medication will be recorded by patients in study diaries between Days 0 and 180. Quality of life will be measured using the DLQI and EPP-QoL. A subset of patients will be photoprovoked on the lower back and dorsal surface of the hand and the minimal symptom dose (MSD) will be determined on Days 0, 30, 60, 90 and 120. The study is planned to start Jun 12 and to complete Apr 13 [11]. 
29/05/2012 15:53:12
Dec 11: Results reported from the PIII CUV029 European study in 74 patients. 68 patients (91.9%) completed the study and 17,377 days were evaluated. Patients on afamelanotide (given as an implant every 60 days) had half as many phototoxic reactions as those on placebo (p=0.044), had a lower total median pain score (6.0 v 17.5; p=0.035) and total lower maximum pain score per phototoxic episode (4.0 v 6.0; p=0.018). Patient kept diaries showed that the patients in the afamelanotide group were able to spend more time in direct sunlight during periods of highest light and UV intensity (p=0.005). For the majority of study days, patients in the afamelanotide group were able to spend up to seven times longer in direct sunlight without experiencing pain. Afamelanotide patients could spent more time in direct sunlight on days when they reported no pain (10 AM-3 PM, p=0.005; 10 AM-8 PM, p=0.003) and on days when they reported no pain or mild pain (10 AM-3 PM, p=0.032; 10 AM-8 PM, p=0.012). Using a a specialised EPP-specific QoL questionnaire (EPP-QoL), patients on afamelanotide reported greater improvement in their QoL and the difference was significant on Days 120 (p=0.005) and 270 (p=0.011). Most common AEs were associated with implant administration (pain or bruising following injection), transient nausea, headache and the common cold [9]. 
21/12/2011 16:09:32
Nov 11: Results of the 6-month US double blind, PII RCT (CUV030) reported. 77 patients were randomized to afamelanotide implant or placebo at study start, after 60 days and 120 days, and were followed up to 180 days. Based on diary records, afamelanotide increased patients´ ability to expose their skin to direct sunlight, based on time spent outside. Patients on afamelanotide spent significantly more time in direct sunlight between the most intense hours of 10 AM and 3 PM (p=0.036) and between 10 AM and 8 PM (p=0.025). They reported a 3-fold increase in the median amount of time in direct sunlight vs placebo and many reported no pain or only mild pain compared to their previous life of experiencing severe phototoxic reactions. Data from an EPP-specific QoL assessment tool showed an improvement from baseline for patients on afamelanotide vs placebo at 60 days (p=0.001), 120 days (p=0.003) and 180 days (p<0.001). The drug appeared to be safe and well-tolerated [7]. 
09/11/2011 10:55:24
Jul 10: A 12 month European and Australian PIII study has reported that afamelanotide significantly reduced average daily pain severity scores vs placebo and allowed patients to expose their skin to sunlight and spend more time outdoors. It was well tolerated with no serious safety issues identified. Complete results will be presented at the 19th Congress of the European Association for Dermatology and Venereology in October [5].
15/07/2010 11:19:50
Afamelanotide could become the first-line treatment for EPP (4).
28/05/2010 13:46:04
Dec 09: Phase III, multicentre, randomised, double-blind, placebo-controlled trial (CUV 017) results announced for afamelanotide in erythropoietic protoporphyria (EPP) - interim analysis of data from the first 4 out of 12 months of treatment. The study used a crossover comparison of afamelanotide (3 doses) and placebo (3 doses) in 100 patients. At dose intervals of 2 months, each patient received a single dose of either treatment. Analysis of 2 treatment arms demonstrated an overall reduction in the average number of phototoxic reactions. 35 patients with severe and/or moderate pain reported the greatest reduction in mean number of reactions (95% CI). Analysis of pain severity was positively correlated with treatment, indicating that patient pain scores differed significantly between treatment groups (95% CI). Interim quality of life data reported a dramatic improvement in the pts ability to engage in outdoors activities. This study will be concluded by the end of December 2009. (3)
22/12/2009 08:56:19
Aug 09: Clinuvel expects to complete its first EU and Australian PIII trial (CUV017) in erythropoietic protoporphyria (EPP) by Q4 2009. Pending results, the company will file in Europe shortly after. The study started in Jun 07 and was expected to enrol 50-70 patients. Positive interim results - the reduction of the number and severity of phototoxic skin reaction - were reported in Jan 09. The company has now received European regulatory approval to begin a confirmatory multicentre 6 month PIII study (CUV029). The trial will be conducted in 5 centres in EU and will involve around 40 patients. Afamelanotide is delivered through a biodegradeable, subcutaneous, controlled-release implant [1,2].
02/09/2009 09:26:04
Evidence Based Evaluations
EPAR  http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/002548/WC500182309.pdf 
NHSC 

References  
Available only to registered users
 Category
BNF Category: Management of skin conditions (13.01)
Pharmacology: Melanocyte stimulating hormone stimulant  
Epidemiology: EPP is a porphyria that affects at least 1 in 140,000 people in the UK. Protoporphyrin accumulates in the skin and causes severe photosensitivity: burning, excruciating pain and erythema, lasting 2-3 days and unresponsive to non-opiates.  
Indication: Erythropoietic protoporphyria  
Method(s) of Administration  
Implantation 
Subcutaneous 
Company Information
Name: Clinuvel 
US Name: Clinuvel 
Further Information
Anticipated commissioning route (England) - 
High cost drug list? Yes
Implications Available only to registered users
Scenesse获批准成为治疗遗传性疾病的新方法
不能耐受阳光的一种罕见的遗传性疾病很快就会有一种新的治疗选择。
澳大利亚Clinuvel制药公司的一款用于该种疾病的药物已获得欧洲药品管理批准推荐。该生物技术公司寄希望于Scenesse，与红细胞生成protoporPHyria防止光毒性首先在成人医学（EPP）。
在暴露于阳光之后，EPP患者会感觉到暴露于阳光的皮肤有一种刺痛感，延长暴露可导致一种令人伤残的疼痛，随后出现红肿。
Scenesse计数器疾病的影响，从而影响欧盟人数少于10000人，通过刺激色素称为黑色素保护皮肤的生产。
欧洲药品管理局表示，其新药专家委员会根据特殊情况及实施安慰剂对照试验反映的困难，已决定推荐批准Scenesse。欧洲药品管理局人用医药产品委员会(CHMP)的上市许可推荐通常会在几个月内得到欧盟的支持。
这种不能耐受阳光的一种罕见的遗传性疾病将得到新的治疗方法！