Erythropoietic Protoporphyria | Clinuvel Pharmaceuticals news and discussion blog

MAA: a moment in Clinuvel’s EPP story

Thursday, February 9th, 2012

Those who have taken an interest in Clinuvel will have learned with joy that, on Monday February 6^th, the company announced its first official filing for SCENESSE® (afamelanotide) with the European Medicines Agency. It has taken our teams around six years to arrive at this point. Benchmarked against peer companies, it is a relatively swift development path for a first-in-class drug; we first publicly announced our erythropoietic protoporphyria (EPP) program in September 2006. It is an opportune moment to reflect briefly on how we reached this milestone and then discuss the steps that must be taken from here. (more…)

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…)

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…)

The importance of clinical relevance

Tuesday, August 2nd, 2011

Development of novel drugs is truly like no other business: one attempts to address questions that may have never been previously posed – let alone answered – in the pursuit of improving the lives and quality of life of patients. As I eluded to in my recent letter to shareholders, the team is now well into the analysis of results from our erythropoietic protoporphyria (EPP) program; two studies from the US and Europe. This is a complex and time consuming task that requires one to collate and make sense of thousands of data points to answer a seemingly straight forward question: does this trial show that the drug is safe and effective?

Obtaining an answer needs to be understood from the concept of clinical relevance. Put simply, results don’t just need to show that a treatment or intervention has an effect on a disease. Rather, they need to indicate that that effect is relevant to the current clinical understanding, treatment and care for the disease or indication. They need to show that the drug’s effect is having a positive, meaningful impact upon a patient’s prognosis and care. This is a crucial point to consider in the development of protocols and in the careful analysis of results, as it is how regulators will review the results. (more…)

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…)

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…)

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…)

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…)

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.

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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…)

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…)

The importance of clinical relevance

Allergic to the sun?

Mikey’s story part two – EPP

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