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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The effects of photosensitivity disorders on patient quality of life

Friday, October 22nd, 2010

A new study from the University of Manchester has uncovered some of the impacts that photosensitivity disorders have upon individual and family quality of life. While several research papers have illustrated the trend towards reduced psychological health in this group, this is the first to compare specific aspects of lifestyle in photosensitive and healthy individuals. (more…)

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Mikey’s story – EPP

Tuesday, September 7th, 2010

Today Clinuvel presents the first in a series of webcast videos featuring 44 year-old Mikey, an Australian man with the severe light intolerance disorder Erythropoietic Protoporphyria (EPP).

EPP is a rare, inherited, metabolic disorder characterised by extreme phototoxicity; toxic reactions to UV and visible light, particularly blue light. The symptoms of this photosensitivity may include those which are not visible to the naked eye, including itching, burning, prickling and most notably, intense pain. (more…)

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Summary of today’s announcements

Wednesday, July 14th, 2010

Today’s announcement of the results of CUV017 has brought with it a stream of updates from Clinuvel, on the website, YouTube and beyond.

In this post we summarise the days releases and developments.

(more…)

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Orphan drug development legislation and regulation landscape

Monday, May 17th, 2010

Previously we have discussed the shifts which take place in the regulatory centers of the world specifically London and Maryland, which present an opportunity to the industry to respond to expressed need.  Perhaps the greatest concern of every individual is access to health and quality of life.

This implies a strong directive for drug developers to respond to the identified needs of the patients and patient communities. But amongst those with therapeutic needs are some with more unaddressed medical requirements: patients with ‘orphan’ diseases.

Orphan diseases are rare and frequently untreated diseases. The previously referred shift towards patient driven drug development made a few voices heard and legislators in the European Community and United States started to act on behalf of the population. Here the legislators and regulators provided incentives to drug developers by reducing fees, providing exemptions and protection from competitors.

(more…)

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Changing the rules in pharmaceutical development: wider significance of Clinuvel’s progressive communication channels

Monday, May 17th, 2010

Communication remains key, whereby content of news and material facts dominate over frequency of news flow. Here our adagium no news signifies focus and good news. A total devotion to this one project, and execution on clinical and regulatory development while finding a balance in available funds, are the core activities en route to commercialisation of afamelanotide.

The significance of communication is equally seen in the way regulators, patients and the medical community gain access to information, data and obtain feedback. Clinuvel is well aware of this and consistency in our operations helps to communicate its objectives.

(more…)

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Patient driven drug development

Monday, May 17th, 2010

Pharmaceutical development is undergoing a defining shift following the ever-increasing regulatory rigor in the process of evaluating a new product. Our recent announcement reflects the conservative view we hold on 21st century drug development.

During development the innovator often seeks to create a market for the new product, a common concept applicable to various industries. The same holds true for afamelanotide: we identified and developed our lead product in an under-served market of porphyria. Adaptation to chemistry and technology, novel communication platforms, deep knowledge and relationships with academia facilitated today’s entry to market.

(more…)

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Brutal: EPP and School sports

Friday, May 7th, 2010

During my high school years, age 12 – 18, I attended an all boys boarding school in Sydney.  The school was/is well known, among other things, for its sporting prowess.  Senior teams are almost unbeatable. This could be put down to the camaraderie built by living in close proximity to your team mates for the whole of the sporting season;  it could also be put down to the fact that every student was expected to play sport. In my time at the school, during the summer you could choose cricket, tennis or basketball and in winter you could choose any sport you wanted, as long as the only sport you wanted to play was rugby union!  Obviously, with a diagnosis of EPP (unheard of previously in the school’s experience) my parents met with the school’s head teachers and arranged for me to be excused from outdoor activity.

(more…)

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Shadow-jumping: EPP and work

Wednesday, April 21st, 2010


This week is Porphyria Awareness Week in the US, and guest blogger Mikey is discussing his experience of the impact that his EPP has had on his professional life.

Direct and indirect sunlight, from reflected light, is a real “killer” for me. If I can find a shadow, I can put exposed skin into that shadow to prevent that area getting burnt.

Unless you’ve got EPP you wouldn’t understand.

(more…)

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Boiler suit, gloves, balaclava and a hat: EPP and the outdoors

Friday, April 9th, 2010

Clinuvel has invited guest blogger Mikey to discuss EPP on our blog; This is the second post in the series discussing the impact of EPP on life outdoors. We are grateful to Mikey for being able to share his experiences.

I was diagnosed with EPP when I was about 7.  Australia is a great country.  It is vast in its beauty and diverse in the hazards it provides!  One of those, for someone with EPP is the “great outdoors”.  I reckon this is the best country in the world.  I’ve lived in New York, and travelled extensively around the world – I’m always pretty pleased when I return home.

(more…)

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