The Orphan Drug Award 2010 was won by Firazyr.

Orphan Drug Award:

The Orphan Drug Award was introduced as a dedicated category at the previous UK Prix Galien in 2008, following a special award for orphan products in 2006. The term ‘orphan condition’ is used to describe conditions that affect a very small number of patients in a given population - many of which are either untreatable or treated very inadequately. It is estimated that there are 6000 orphan diseases – which, in total, affect about 30 million EU citizens. 

Parliamentary Sponsor, Lord Walton of Detchant – a Crossbench Life Peer and  former Prix Galien judge – said the decision to give a new award for orphan drug discovery was “far-sighted” and commended the industry on its developments in this important area of medicine. “In many branches of medicine, there are many diseases formerly unresponsive to any pharmaceutical intervention which are now known to be due to single gene disorders. Many of these are rare, and as drugs begin to emerge capable of circumventing the genetic defect, the costs of development may be huge, but because of the rarity of the relevant conditions, the income derived from marketing such remedies may be relatively limited,” he said. “This is a huge dilemma for the industry to face. It is good to know that there have been several outstanding submissions in the orphan drug category this year.”

Professor Sir Michael Rawlins concurred. “For orphan diseases that are potentially treatable with medicines, pharmaceutical manufacturers face a number of hurdles, including concerns about the size of the market and difficulties – because of the small numbers of patients – in their development. The judging panel was delighted by how many companies are now working in these areas – and with the extremely high calibre of the entries to this category.”

Winner: Firazyr^® (Shire Pharmaceuticals)

The Orphan Drug Award 2010 was won by Shire Pharmaceuticals for its hereditary angioedema (HAE) treatment, Firazyr. HAE is a rare condition characterised by recurrent episodes of oedema formation in the soft tissues of the extremities, face, genitals as well as in the mucous membranes of the gastrointestinal tract and larynx.  These attacks are often painful, disfiguring and debilitating.   And involvement of the larynx can be lethal.

The exact prevalence of hereditary angioedema is uncertain – but estimates suggest that 1:10,000 to 1:50,000 persons are affected.  Hereditary angioedema is an autosomal dominant disease caused by the absence or dysfunction of C1-esterase inhibitor.  While the exact trigger for attacks of hereditary angioedema is unknown, it follows activation of the complement cascade with increased local production of bradykinin.   Firazyr selectively – and competitively – inhibits the action of bradykinin – thus halting oedema formation and rapidly alleviating symptoms. In clinical trials, Firazyr significantly reduced the duration of HAE attacks.   In 90% of patients only one injection was required to achieve effective control of the HAE attack.

“Apart from its novel mechanism of action and clear evidence of its clinical effectiveness, the jury were also extremely impressed by the clinical development programme of Firazyr,” said Professor Sir Michael. “Attacks of hereditary angioedema occur spontaneously and without warning.  Patients tend to seek help from their nearest Accident and Emergency Department or Emergency Room.  Organising clinical trials under such circumstances is what might be called ‘a challenge’! That Shire managed to undertake the clinical development of this product in such an unpromising environment – is hugely to their credit.”

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