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17.05.2010
Clinuvel Pharmaceuticals Ltd.: Italian patients request UV-drug: afamelanotide available for rare sun disorder
Clinuvel Pharmaceuticals Ltd.: Italian patients request UV-drug: afamelanotide available for rare sun disorder
Italian National Health System to reimburse afamelanotide for patients with porphyria (EPP)
Italian drug regulators have, for the first time, approved the use and reimbursement of a drug under clinical investigation while it is in advanced regulatory review process worldwide. Patients diagnosed with the rare light and UV intolerance erythropoietic protoporphyria (EPP) will now be able to gain access to the photoprotective drug – known under the generic name afamelanotide – on prescription. The decision by the Italian Medicines Agency (AIFA) precedes approval of formal marketing authorisation by European or US drug regulatory agencies.
Afamelanotide is developed by the Australian-based company Clinuvel and has been recognised for its innovative medicinal value by Italian regulators in the treatment of EPP, a severe disease for which no other therapy exists. Patients diagnosed with EPP can now be prescribed the drug by a physician and the full cost will be reimbursed by the Italian National Health System (Sistema Sanitario Nazionale, SSN).
Clinuvel’s Chief Scientific Officer, Dr Hank Agersborg commented: “AIFA has taken a leadership role in the treatment of absolute light and UV intolerance as seen in this kind of porphyria. We have seen a world-first in UV treatment: patients and physicians requesting regulators to allow access to the novel drug. Perhaps it is significant in today’s world that patients become more and more empowered in influencing drug development.”
“Five years ago, Clinuvel made a decision to develop afamelanotide specifically for patient populations who were most severely and acutely affected by UV and light,” Clinuvel’s CEO Dr Philippe Wolgen said. “Today’s news comes as a welcome surprise but supports our choice to develop afamelanotide in these categories of patients.”
“Following the completion of the complex study of afamelanotide in Italian EPP patients, we continued to support their treatment through a compassionate use program. Unfortunately, the indefinite free supply of afamelanotide was not sustainable for Clinuvel as a small enterprise, and this approval by the Italian regulators not only makes the drug available to these trial patients, but also provides further incentive to the company to focus on orphan drug development,” Dr Wolgen said.
EPP is characterised by “intolerable pain”: patients’ skin burns, blisters and scars when exposed to normal levels of light and sun. The disease is incurable and affects patients for life. As a result, EPP patients are forced to spend most of their lives indoors. Sunscreens are of no use as they don’t block out visible light (the blue spectrum), which is the cause and trigger of toxic reactions in EPP.
Afamelanotide works by activating pigment, melanin, in the skin providing a barrier between light and skin cells. To date, the photoprotective drug has been shown to be clinically safe and able to reduce the incidence and severity of reactions in EPP patients. Full results from a European Phase III study are expected shortly. Initial clinical observations from an Italian cohort of patients from this study will be presented at the Sidemast Italian Dermatological Congress (Congresso Sidemast Dermatologia) in Rimini, Italy, on Friday May 21.
Under the Italian Law 648/96 AIFA may approve access in exceptional circumstances to, and reimbursement for, treatments which are unapproved in Italy, or treatments in clinical development for patients with disorders where no alternative therapy exists.
Previously, only 36 drugs had been approved under Law 648/96 since its introduction in 1996. Afamelanotide is the first drug “under clinical investigation” and pharmaceutical therapy in the symptomatic treatment of porphyria to be approved under this Law. Although the exact prevalence of EPP in Italy is unknown, it is estimated that 60 patients will initially benefit from afamelanotide treatment.
“The 648/96 Italian listing is a confirmation of our global regulatory strategy and will see the company generate revenue from afamelanotide for the first time. The revenues will enable us to accelerate the development of a paediatric dose for juvenile EPP patients,” Dr Wolgen said.
For more information contact:
Australia
Lachlan Hay
Head of Global Network and Communications
Clinuvel Pharmaceuticals Limited
T: +61 3 9660 4900
E: investorrelations@clinuvel.com
Europe
Daniela Schaefer
Head of Business Operations Europe
Clinuvel Pharmaceuticals Limited
T: +41 44 253 75 00
About afamelanotide
Afamelanotide is a first-in-line therapeutic being developed by Clinuvel. An analogue of α-MSH, afamelanotide is a linear peptide which activates the skin to produce eumelanin, the dark pigment which is known to have photoprotective properties (providing skin protection against light and UV radiation). Increased pigmentation of the skin appears a few days after administration of afamelanotide and lasts up to 60 days. Afamelanotide is administered underneath the skin as a dissolvable implant approximately the size of a grain of rice.
About Clinuvel Pharmaceuticals Limited
Clinuvel Pharmaceuticals Ltd is a leading and innovative Australian company focused on the development of afamelanotide, its proprietary first-in-class photoprotective drug. Clinuvel has identified five groups of patients with a clinical need for photoprotection. Currently, Clinuvel is in its final stages to complete testing of afamelanotide in Phase II and III trials in Australia and Europe. Clinuvel’s ongoing focus is to demonstrate the safety and efficacy of afamelanotide. Pending positive clinical results, Clinuvel aims to file afamelanotide for its first market approval for the orphan indication porphyria (EPP).
Clinuvel is currently testing afamelanotide in five clinical indications: http://www.irw-press.com/dokumente/Clinuvel_170510_EN.pdf
Phase I and II human clinical trials using afamelanotide have demonstrated that the drug is well tolerated and no significant safety concerns have been identified to date. Following successful conclusion of the development program, Clinuvel will work closely with global regulators to facilitate marketing approval of afamelanotide.
About Erythropoietic Protoporphyria (EPP)
Porphyrias are a group of inherited disorders with enzymatic deficiency in the blood synthesis pathway (also called porphyrin pathway). They are broadly classified as erythropoietic porphyrias based on the site of the overproduction and main accumulation of porphyrin. They manifest with either skin problems, neurological complications or gastro-intestinal problems (occasionally all).
EPP is a rare genetic disease found mainly in people with fair skin. It is characterised by severe phototoxicity (or intolerance to light) of the skin resulting in intolerable pain, swelling, and scarring, usually of the exposed areas such as the face, hands and feet. The pain experienced and expressed by EPP patients when their skin is exposed to light is reported as intolerable. EPP patients are often forced to remain indoors, severely affecting their quality of life.
Clinuvel is an Australian biopharmaceutical company focussed on developing its photoprotective drug, afamelanotide for a range of UV-related skin disorders resulting from exposure of the skin to harmful UV radiation. Pharmaceutical research and development involves long lead times and significant risks. Therefore, while all reasonable efforts have been made by Clinuvel to ensure that there is a reasonable basis for all statements made in this document that relate to prospective events or developments (forward-looking statements), investors should note the following:
• actual results may and often will differ materially from these forward-looking statements;
• no assurances can be given by Clinuvel that any stated objectives, outcomes or timeframes in respect of its development programme for afamelanotide can or will be achieved;
• no assurances can be given by Clinuvel that, even if its development programme for afamelanotide is successful, it will obtain regulatory approval for its pharmaceutical products or that such products, if approved for use, will be successful in the market place
Level 11 / 330 Collins Street
Melbourne, Victoria 3000
Australien
T +61 3 9660 4900
F +61 3 9660 4999
www.clinuvel.com
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Italian National Health System to reimburse afamelanotide for patients with porphyria (EPP)
Italian drug regulators have, for the first time, approved the use and reimbursement of a drug under clinical investigation while it is in advanced regulatory review process worldwide. Patients diagnosed with the rare light and UV intolerance erythropoietic protoporphyria (EPP) will now be able to gain access to the photoprotective drug – known under the generic name afamelanotide – on prescription. The decision by the Italian Medicines Agency (AIFA) precedes approval of formal marketing authorisation by European or US drug regulatory agencies.
Afamelanotide is developed by the Australian-based company Clinuvel and has been recognised for its innovative medicinal value by Italian regulators in the treatment of EPP, a severe disease for which no other therapy exists. Patients diagnosed with EPP can now be prescribed the drug by a physician and the full cost will be reimbursed by the Italian National Health System (Sistema Sanitario Nazionale, SSN).
Clinuvel’s Chief Scientific Officer, Dr Hank Agersborg commented: “AIFA has taken a leadership role in the treatment of absolute light and UV intolerance as seen in this kind of porphyria. We have seen a world-first in UV treatment: patients and physicians requesting regulators to allow access to the novel drug. Perhaps it is significant in today’s world that patients become more and more empowered in influencing drug development.”
“Five years ago, Clinuvel made a decision to develop afamelanotide specifically for patient populations who were most severely and acutely affected by UV and light,” Clinuvel’s CEO Dr Philippe Wolgen said. “Today’s news comes as a welcome surprise but supports our choice to develop afamelanotide in these categories of patients.”
“Following the completion of the complex study of afamelanotide in Italian EPP patients, we continued to support their treatment through a compassionate use program. Unfortunately, the indefinite free supply of afamelanotide was not sustainable for Clinuvel as a small enterprise, and this approval by the Italian regulators not only makes the drug available to these trial patients, but also provides further incentive to the company to focus on orphan drug development,” Dr Wolgen said.
EPP is characterised by “intolerable pain”: patients’ skin burns, blisters and scars when exposed to normal levels of light and sun. The disease is incurable and affects patients for life. As a result, EPP patients are forced to spend most of their lives indoors. Sunscreens are of no use as they don’t block out visible light (the blue spectrum), which is the cause and trigger of toxic reactions in EPP.
Afamelanotide works by activating pigment, melanin, in the skin providing a barrier between light and skin cells. To date, the photoprotective drug has been shown to be clinically safe and able to reduce the incidence and severity of reactions in EPP patients. Full results from a European Phase III study are expected shortly. Initial clinical observations from an Italian cohort of patients from this study will be presented at the Sidemast Italian Dermatological Congress (Congresso Sidemast Dermatologia) in Rimini, Italy, on Friday May 21.
Under the Italian Law 648/96 AIFA may approve access in exceptional circumstances to, and reimbursement for, treatments which are unapproved in Italy, or treatments in clinical development for patients with disorders where no alternative therapy exists.
Previously, only 36 drugs had been approved under Law 648/96 since its introduction in 1996. Afamelanotide is the first drug “under clinical investigation” and pharmaceutical therapy in the symptomatic treatment of porphyria to be approved under this Law. Although the exact prevalence of EPP in Italy is unknown, it is estimated that 60 patients will initially benefit from afamelanotide treatment.
“The 648/96 Italian listing is a confirmation of our global regulatory strategy and will see the company generate revenue from afamelanotide for the first time. The revenues will enable us to accelerate the development of a paediatric dose for juvenile EPP patients,” Dr Wolgen said.
For more information contact:
Australia
Lachlan Hay
Head of Global Network and Communications
Clinuvel Pharmaceuticals Limited
T: +61 3 9660 4900
E: investorrelations@clinuvel.com
Europe
Daniela Schaefer
Head of Business Operations Europe
Clinuvel Pharmaceuticals Limited
T: +41 44 253 75 00
About afamelanotide
Afamelanotide is a first-in-line therapeutic being developed by Clinuvel. An analogue of α-MSH, afamelanotide is a linear peptide which activates the skin to produce eumelanin, the dark pigment which is known to have photoprotective properties (providing skin protection against light and UV radiation). Increased pigmentation of the skin appears a few days after administration of afamelanotide and lasts up to 60 days. Afamelanotide is administered underneath the skin as a dissolvable implant approximately the size of a grain of rice.
About Clinuvel Pharmaceuticals Limited
Clinuvel Pharmaceuticals Ltd is a leading and innovative Australian company focused on the development of afamelanotide, its proprietary first-in-class photoprotective drug. Clinuvel has identified five groups of patients with a clinical need for photoprotection. Currently, Clinuvel is in its final stages to complete testing of afamelanotide in Phase II and III trials in Australia and Europe. Clinuvel’s ongoing focus is to demonstrate the safety and efficacy of afamelanotide. Pending positive clinical results, Clinuvel aims to file afamelanotide for its first market approval for the orphan indication porphyria (EPP).
Clinuvel is currently testing afamelanotide in five clinical indications: http://www.irw-press.com/dokumente/Clinuvel_170510_EN.pdf
Phase I and II human clinical trials using afamelanotide have demonstrated that the drug is well tolerated and no significant safety concerns have been identified to date. Following successful conclusion of the development program, Clinuvel will work closely with global regulators to facilitate marketing approval of afamelanotide.
About Erythropoietic Protoporphyria (EPP)
Porphyrias are a group of inherited disorders with enzymatic deficiency in the blood synthesis pathway (also called porphyrin pathway). They are broadly classified as erythropoietic porphyrias based on the site of the overproduction and main accumulation of porphyrin. They manifest with either skin problems, neurological complications or gastro-intestinal problems (occasionally all).
EPP is a rare genetic disease found mainly in people with fair skin. It is characterised by severe phototoxicity (or intolerance to light) of the skin resulting in intolerable pain, swelling, and scarring, usually of the exposed areas such as the face, hands and feet. The pain experienced and expressed by EPP patients when their skin is exposed to light is reported as intolerable. EPP patients are often forced to remain indoors, severely affecting their quality of life.
Clinuvel is an Australian biopharmaceutical company focussed on developing its photoprotective drug, afamelanotide for a range of UV-related skin disorders resulting from exposure of the skin to harmful UV radiation. Pharmaceutical research and development involves long lead times and significant risks. Therefore, while all reasonable efforts have been made by Clinuvel to ensure that there is a reasonable basis for all statements made in this document that relate to prospective events or developments (forward-looking statements), investors should note the following:
• actual results may and often will differ materially from these forward-looking statements;
• no assurances can be given by Clinuvel that any stated objectives, outcomes or timeframes in respect of its development programme for afamelanotide can or will be achieved;
• no assurances can be given by Clinuvel that, even if its development programme for afamelanotide is successful, it will obtain regulatory approval for its pharmaceutical products or that such products, if approved for use, will be successful in the market place
Level 11 / 330 Collins Street
Melbourne, Victoria 3000
Australien
T +61 3 9660 4900
F +61 3 9660 4999
www.clinuvel.com
NEWSLETTER REGISTRIERUNG:
Aktuelle Pressemeldungen dieses Unternehmens direkt in Ihr Postfach:
<a href="http://www.irw-press.com/alert_subscription.php?lang=en&isin=AU000000CUV3">http://www.irw-press.com/alert_subscription.php?lang=en&isin=AU000000CUV3</a>
Mitteilung übermittelt durch IRW-Press.com. Für den Inhalt ist der Aussender
verantwortlich.
Kostenloser Abdruck mit Quellenangabe erlaubt.