2018 ceremony sees highest number of finalists in category for rare disease and the first orphan drug to win the prestigious Innovative Product Award.
London, 15th November, 2018. Innovations from Biogen and Amicus have become the latest medicines to win prestigious UK Prix Galien medals following a glittering ceremony at London’s House of Commons.
Amicus’ Galafold (Innovative Product) and Biogen’s Spinraza (Orphan Product) won the two main prizes on an evening dominated by treatments for rare disease. All bar one of the medicines shortlisted for the final were for orphan conditions, with Galafold becoming the first orphan product in the history of UK Prix Galien to win the Innovative Product Award.
The 2018 UK Prix Galien also saw the introduction of a new category for Medical Technology, in which Medtronic was awarded a commendation for its vascular device, Solitaire.
Announcing the results, Sir Michael Rawlins – who has been Chair of the UK Prix Galien judges for 22 years – said: “The quality and depth of innovation that emerges from the UK life sciences industry continues to change lives and make a real difference to people all over the world. That innovation is the reason why UK Prix Galien exists – to honour excellence in medical R&D and celebrate the achievements of all those on whom we rely for the discovery and development of new innovation.
“The 2018 awards produced a fantastic range of entries, sparking fierce competition and robust debate. Congratulations must go not only to the winners, but to all entrants for putting up such a strong showing.”
Innovative Product Award
The Innovative Product Award was won by Amicus’ Galafold, an oral medication for the treatment of Fabry disease. Fabry disease is a rare lysosomal metabolic storage condition that results in progressive, irreversible nervous-system disease, cardiac problems, renal disease and stroke.
“For the 855 patients in the UK, Fabry disease will cut their life expectancy by 15-20 years,” said Sir Michael. “Galafold is a highly innovative, precision-targeting pharmacological chaperone that utilises the body’s own enzyme to enable the breakdown of accumulated glycosphingolipid compounds.
Identification of the sub-group of patients most likely to respond has been achieved by creating a precision screening technique during the clinical trial programme. Ultimately, Galafold will reduce costs to the NHS and was approved under the NICE highly specialised programme in 2017.”
Dupixent, Sanofi’s treatment for moderate-to-severe atopic dermatitis, was highly commended.
Orphan Product Award
The Orphan Product Award, first introduced as a UK Prix Galien category in 2006, recognises innovation in conditions that affect very small numbers of patients. “Many of these conditions are either untreatable or treated inadequately,” said Sir Michael. “Pharmaceutical companies face a number of hurdles in developing drugs for these conditions, particularly the likely size of the market and the small number of patients available for clinical trials.”
The 2018 Orphan Product Award was won by Spinraza, Biogen’s treatment for spinal muscular atrophy (SMA). SMA, is a devastating neurodegenerative disease marked by debilitating muscle weakness; each year approximately 1 in every 10,000 babies receives an SMA diagnosis and it is the leading cause of infant mortality, with only supportive therapies available until now.
“Spinraza is an antisense oligonucleotide and is the first disease-modifying therapy for SMA,” said Sir Michael. “Clinical studies show powerful evidence of improved survival alongside patients achieving and/or maintaining developmental motor milestones closer to those expected for their age and inconsistent with the progressive decline associated with the natural history of the disease. Spinraza’s Prix Galien victory is well deserved and was the unanimous decision of the judging panel.”
In the same category, Qarziba, Eusa’s treatment for Neuroblastoma, was highly commended.
Medical Technology Award
In the newly introduced Medical Technology category, Medtronic’s Solitaire was awarded a commendation. “The Solitaire Platinum revascularisation device reduces stroke-related disability by showing nearly 70% relative improvement in functional outcomes at 90 days in patients suffering large vessel occlusions,” said Sir Michael. “This is life changing for the patient, their families, the NHS and society. The development of Solitaire has resulted in a cost-effective method of treating large vessel occlusion stroke that could improve or save the lives of over 4000 patients a year.”
Karen Westaway, Chief Executive of ValueBase, owners of Prix Galien’s UK franchise, said: “The 2018 Awards yet again confirmed the depth and diversity of innovation in UK life sciences. The success of products for rare disease – which, for the first time, dominated the shortlist of finalists – shows that innovation is not confined to Big Pharma or treatments for major headline conditions. As our Parliamentary Sponsor, Kevin Barron MP, put it, UK Prix Galien has underlined the value of treatments that focus on the ‘few, not the many’. It’s great to see these innovations coming through and the work of the scientists that develop them being rewarded and applauded. That’s the essence of UK Prix Galien. And it once again confirms that UK life sciences are as strong as ever.”