In 2010, Clinuvel announced that it would commence a clinical trial of a new drug for vitiligo. In late 2012 the first treatment results from this program were announced with follow up results release in September 2013. The company has received a large number of questions related to its next trial and has released this Q&A to help people understand the steps to be taken to develop SCENESSE® in vitiligo. We’ve included a number of links throughout this document, as well as on Clinuvel’s website, to help people understand the technical content in a user-friendly manner.


The biennial Porphyrins & Porphyrias conference is the only international meeting of global key opinion leaders on the group of diseases known as porphyrias. Convening last week (15-19 May) in Lucerne, Switzerland, P&P has become an important fixture on Clinuvel’s calendar, providing an opportunity to review updates from across the globe and discuss our program with international experts. Our specific interest in the porphyrias lies with erythropoietic protoporphyria (EPP), one of the rarer forms of the disease grouping and the lead indication for our drug SCENESSE® (afamelanotide 16mg implant). Indeed there were a number of presentations reviewing updates in thinking on EPP, including presentation of results from Clinuvel’s program. I’ll return to those shortly, however, as there have been significant advances in another form of porphyria and it would be remiss not to at least mention these here. (more…)

Today, February 28, marks Rare Disease Day: an annual event to help highlight the effects of rare diseases on individuals, families and the community, and to raise awareness in the hope of finding treatments, or even cures. As a snapshot, a rare disease is defined in Europe as one which affects less than 1 in 200,000 people. Some 6,000-8,000 rare diseases have been identified to date, and it’s estimated that up to 30 million people across the European Union and another 30 million across the US are affected by a rare disease. Clinuvel takes an active stance on patients’ rights – we’re working hard to get SCENESSE® approved for the rare light intolerance erythropoietic protoporphyria (EPP). This year’s theme Rare Diseases Without Borders resonates with us particularly, as it recognises the global challenges that rare disease patients, and those working with them, face. Cultural and linguistic challenges aside, to get a new drug to rare disease patients one must find those patients then tackle myriad national, regional and local laws and regulations every step of the way. In short, a more cohesive, multinational approach is needed to better address the needs of rare disease patients. (more…)

I find vitiligo to be a fascinating, yet devastating disorder: almost overnight, patients see their skin colour erode and their identity change. Vitiligo has a serious impact on individuals and their family and professional relationships, something I’ve discussed before when looking at treatment challenges. Because of this impact, we’ve been very careful of the way in which we discuss vitiligo publicly to ensure that, rather than adding to the distress of those affected, we can have a positive impact on disease awareness while we execute our clinical program. I’ve had the privilege to speak with individuals from a broad range of backgrounds living with, and treating, this disorder, with conversations ranging from disease impact, to treatments, to prevalence. During these discussions I also try to seek feedback on how we present the program: what we are doing and how we can do it better. This feedback has, in turn, led to changes, large and small, in our communications (and hopefully will continue to do so). Yet, one story is often at the forefront when discussing vitiligo with investors, journalists and the broader public which we haven’t discussed until today: the case of Michael Jackson’s very public battle with vitiligo and pigment change. Often these conversations boil down quickly to the “Michael Jackson skin disease”. (more…)

Clinuvel Non-Executive Director Brenda Shanahan has been honoured at The Australian Financial Review and Westpac Group Woman of Influence 2012 Awards, being recognised as the second most influential woman in the category of Boards and Management from a field of over 350 nominees.

Mrs Shanahan has been a Director of Clinuvel since 2007, serving as Non-Executive Chair from late 2007 until July 2010. In addition to her role at Clinuvel Mrs. Shanahan is the Chair of the St Vincent’s Institute, an independent medical research institute in Melbourne, as well as a Non-Executive Director of Challenger Limited (ASX: CGF), Bell Financial Group (ASX: BFG) and DMP Asset Management. You can read Mrs. Shanahan’s full biography on Clinuvel’s website and more about the Women of Influence 2012 Awards at The Financial Review.

Last week I gave a snapshot of the formal process undertaken by the European Medicines Agency (EMA) to review a Marketing Authorisation Application (MAA) dossier under the Centralised Procedure. While this provides some context, looking at historical review timelines provides some sense of how long a future application may take. This is far from an exact science, but given the queries we have received of late on approvals timelines, I felt it appropriate to provide some internal perspective. The EMA’s timelines are confidential, with only set time points at which companies are allowed to publish information (CHMP meetings in particular). Once a drug has been approved by the EMA, however, the Agency issues a summary of the entire review process, known as an EPAR, or European Public Assessment Report. This document outlines the timeframes required by the EMA to perform the review, along with the names of the co-rapporteurs and a summary of the product in some depth, much of which likely reflects the dossier submitted, albeit in an abbreviated version. (The EPARs are published online by the EMA and are free to download.) (more…)

For many years we’ve taken an active interest in the time required by regulatory authorities globally to approve orphan drugs. Clearly this kind of activity is required for forecasting and planning, but it also helps in communications to give our broader audience a realistic idea of the timelines ahead and some of the hurdles we may face. Predicting timelines in drug development is an imprecise business fraught – each case or drug needs to be indivudally assessed, there are no comparables – but it is nonetheless one method by which we can anticipate the period of time required before any decision from the European Medicines Agency (EMA) is published. (more…)

Following much hype and fanfare at the Olympics, news reports have begun to trickle out on the costs of the Games and the longer term impact they might have on the British economy. While not immune to Europe’s economic spluttering across the Channel, many of the issues facing Britain’s bottom line are unique, and they may have broader implications for drug development, more specifically for drug pricing and reimbursement. (more…)

Since we publicly announced our vitiligo program in 2010, the entire Clinuvel team has aimed to gain a better understand this disease, its possible causes and how SCENESSE® (afamelanotide) may become a vital tool in a dermatologist’s arsenal for vitiligo. We’ve also worked closely with the broader vitiligo community to better communicate the program’s goals and limitations (if you missed them, you can see the first two videos in a series of interviews between Mr Lee Thomas and our CEO here) as well as reaching out to individuals with vitiligo to better understand how this disease impacts upon lives. For me, one of the most prominent recurring topics in discussions, emails, phone calls and online is the lack of treatment for vitiligo and the frustrations of physicians and patients alike at treatment inconsistency. (more…)

A new article was published last week looking at a small pilot study of SCENESSE® (afamelanotide) in acne vulgaris. Before discussing this piece specifically, I think it is relevant to briefly review melanocortins and their receptors. The active ingredient in SCENESSE®, afamelanotide, has been the focus of over 80 peer reviewed journals (as well as being mentioned in at least 100 more) since the initial formulatory work with the drug began in the 1980s. In the last two years alone the drug and its actual or theoretical application in the clinic have been presented at least 20 times at various global scientific forums. This growing library of resources reflects the enthusiasm of the medical community to put a new compound through its paces, both in vitro and in vivo. (more…)