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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‘Measuring’ vitiligo: the challenges of clinical and treatment evaluation

Monday, April 4th, 2011

Since our announcement last year that Clinuvel would commence a new program for SCENESSE® (afamelanotide) in nonsegmental vitiligo, the company has received vast interest in the application of the drug in this disease. Of the enquiries that best captured the essence of this program, one stood out: a US based analyst asked how the company intended to objectively measure the response to treatment, the repigmentation of vitiliginous lesions, in its trial. (more…)

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FDA Basics website reworks new drug approvals data

Friday, March 18th, 2011

We’ve spoken often about drug approvals by regulatory agencies, particularly approvals for new drugs, or new molecular entities (NMEs). A quick – and useful – data update on this front has just been posted by the FDA to their FDA Basics website (a project we blogged about in 2010).

The FDA has posed itself two questions which it seeks to answer, head over the their website to see the results:

Is it true FDA is approving fewer new drugs lately?

Is the U.S. really slower than Europe in approving new drugs?

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New drug approval rates

Friday, February 25th, 2011

Novel drug development is a business which is not well understood; perhaps one which is not well explained. To be able to launch a novel drug proposition, one needs tenacity, expertise and a talented team to succeed. Unlike a ‘follow-on’ product where an abundance of safety data on the molecule in one or other formulation or therapeutic application exists, those few companies working with new drugs must clear all the necessary stringent barriers from the outset.

This makes novel drug development a risky endeavour, amplified by a level of uncertainty even when it is believed that the necessary regulatory hurdles have been cleared and the drug development process seems complete. By looking at several new molecules being developed for obesity (Lorcaserin, Contrave and Qnexa particularly), one can see how uncertain the outcome remains, even when so much work and so many years have been invested; others have written extensively about these cases and I encourage avid readers to review the publications. (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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FDA online regulation

Monday, January 17th, 2011

Effective communication is demanded more than ever in present-day drug development. I’ve blogged before about the role of patients in this process and the importance of acknowledging patient driven drug development. In a time of advanced technologies and communications it is my intention that Clinuvel be at the forefront of any developments in this area and that the company seeks new ways to discuss our program, online and off.

We also work in a highly regulated environment: very little of what the company does is without scrutiny from this perspective, either from our own regulatory team or from external organizations established for this purpose. Despite the hightened reviews, there remains a discord in the communications realm, with a lack of regulation surrounding tools referred to broadly as ‘social media’. (more…)

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Time to look forward

Monday, December 20th, 2010

Many of you who follow the company closely will notice the increase in communications in recent weeks. As our clinical and regulatory workload increases behind the scenes, it is our intention to keep our numerous stakeholders abreast of relevant updates in the field and with Clinuvel’s work.

While it is commonplace to review the year past during the festive season, I take this time to indulge in the progress and challenges that lie ahead for Clinuvel. (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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Pain measurement in clinical studies: The Likert Scale

Friday, August 13th, 2010

surveyWhen assessing new therapeutic goods through clinical trials, researchers must obtain information on the degree of a patient’s physical response to therapy. This data then undergoes detailed statistical analyses in order to determine the safety and efficacy (effectiveness) of the drug or treatment.

Central to Clinuvel’s clinical trial design, and the value of these studies, is measurement of the severity of phototoxic reactions (adverse reactions to light or UV radiation) in trial patients. In order to do this accurately, a symptom severity scale has been developed based on the method of ‘Likert scaling’.

(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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