EPP results and clinical relevance

Friday, November 4th, 2011

A protoporphyrin IX molecule

Over recent months I have written several times of the need for Clinuvel to prove clinical relevance in our trials with the use of SCENESSE® (afamelanotide) in erythropoietic protoporphyria (EPP). In orphan populations the need to demonstrate how a novel drug assists in their daily activities and improve their lives is at the forefront of the regulators’ minds. And so it should be, after all the objective of the pharmaceutical industry is to develop drugs which address either disease or symptoms adequately and safely. The results the company released yesterday from our Phase II US study of the drug in EPP (CUV030) have given us important data towards demonstrating clinically relevant improvement of patients’ lives. (more…)

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Clinical relevance – the value of patient experiences

Friday, September 30th, 2011

Since 2006 Clinuvel has trialed SCENESSE® in a truly unique group of individuals: patients living with erythropoietic protoporphyria (EPP), a rare genetic blood disorder which causes an absolute intolerance to light.

EPP prevents patients from leading ‘normal’ lives, especially outdoors. It is one of the few diseases that manifest clinically with initially invisible symptoms which cause severe dermal pain for several days. This not only presents a challenge for diagnosis and treatment, but also for generating meaningful clinical trial results – those which are measurable numerically and are used by regulatory authorities to evaluate the efficacy of a drug in a patient population. Here, real life patient experiences during a trial can play an important role in providing clinical relevance and analysing hard data. (more…)

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Porphyrias: a disease grouping by cause, not symptoms

Monday, April 18th, 2011

Held biennially, the Porphyrins & Porphyrias conference (P&P) is the world’s largest gathering on the porphyrias – a group of metabolic disorders causing biochemical disruptions in the pathway of the body which synthesizes haem (heme).

As a result of each of these disruptions, the body presents with unique symptoms ranging from skin symptoms and phototoxicity – as those seen in erythropoietic protoporphyria and congenital erythropoietic porphyria – through to acute attacks of abdominal pain, seen most commonly in acute intermittent porphyria. In short, no two porphyrias are clinically identical yet they are discussed as a single group of disorders with a similar cause. As a matter of fact, there are eight variations of porphyrias, each with a specific clinical manifestation of disease. (more…)

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Mikey’s story part three – EPP

Tuesday, April 12th, 2011

As part of an ongoing series, Clinuvel has released its third video featuring Mikey, an Australian man with erythropoietic protoporphyria (EPP). EPP is a rare disorder in which a chemical, called protoporphyrin IX, accumulates in the skin. When exposed to UV and visible light, the protoporphyrin IX molecules in the skin undergo a chemical reaction which results in swelling scarring and can leave those with EPP in considerable pain. (more…)

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Allergic to the sun?

Monday, February 21st, 2011

As the Northern Hemisphere approaches the end of winter, we are beginning to see more mentions of ‘sun allergies’ in popular press, online and in social media.

Most people are not actually ‘allergic’ to the sun, but rather are suffering the first symptoms of seasonal or light-induced skin disorders, known medically as photodermatoses. Photodermatoses cause a person’s skin to react to even the briefest sunlight exposure by burning, swelling or developing a rash. Most distressingly, these diseases can have sudden onset where people, who may have never experienced them before, see symptoms following their first day in the spring sun. (more…)

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How I explain EPP to the world

Friday, February 18th, 2011

As part of our ongoing focus on erythropoietic protoporphyria (EPP), we’ve invited Victoria to share her experiences with EPP as a guest blogger. You can read Victoria’s first post here.

When I was first diagnosed with EPP there was a huge sense of relief: someone had actually listened to me and believed that something wasn’t quite right.

That said, I am not overjoyed at having the condition as it restricts my life so much. When Spring starts to approach I get anxious as I dread having a reaction; my freedom to go out feels more restricted. (more…)

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A clinical success: study retention rates

Thursday, February 10th, 2011

We were delighted this week to be able to announce the successful completion of our first Phase II study conducted in the US, a placebo controlled, randomised trial of SCENESSE® (afamelanotide) for patients diagnosed with erythropoietic protoporphyria (EPP) (CUV030). (more…)

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Childhood reactions and two decades to diagnose

Friday, February 4th, 2011

As part of our ongoing focus on erythropoietic protoporphyria (EPP), we’ve invited Victoria to share her experiences with EPP on the blog.

My name is Victoria; I am 26 years old and live in England. I was diagnosed with EPP at the age of 24. I fought for many years to get people to listen to me and understand EPP. I want to help raise awareness for this condition and also encourage those who suffer in silence to fight to get someone to listen to them. This is part of my story. (more…)

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Mikey’s story part two – EPP

Monday, January 17th, 2011

Today Clinuvel released the second instalment of our video series featuring Mikey, a 44 year old Australian man who has the severe light intolerance disorder, Erythropoietic Protoporphyria (EPP). You can view the new video on our website or on YouTube.

(more…)

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Compassionate use – navigating the regulatory landscape to ‘do good’

Friday, December 3rd, 2010

As snow begins to fall around Clinuvel’s European office, the team in Australia is preparing for a long hot summer. The seasons are at the forefront of our minds at Clinuvel, since our lead drug SCENESSE® appears to have a dramatic impact on the ability of patients to expose themselves to sun. We try to test the drug under the most extreme conditions, meaning trials must be conducted in spring and summer. As the seasons change, we begin to see more requests and enquiries from the southern hemisphere, in particular from patients with erythropoietic protoporphyria (EPP), seeking access to the drug outside of formal trial programs. (more…)

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