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Orphan drugs from Biogen and Amicus win UK Prix Galien

New medical technology category added for the first time


Amicus’ Galafold and Biogen’s Spinraza have won the two top prizes at the UK Prix Galien, the biennial awards which recognise the most innovative and high impact new treatments for patients.

The awards were once again held at the House of Commons in London – the scene of high drama earlier in the day when Prime Minister Theresa May received a decidedly cool reception from MPs for her Brexit deal.

There was a much warmer welcome at the awards, hosted by ValueBase, for the pharma and biotech companies with products on the shortlist, which was dominated by orphan medicines.

The Parliamentary Sponsor was Sir Kevin Barron, MP, a former a shadow health minister, while current health minister Lord O’Shaughnessy was also on hand to praise the industry’s work and hand out the awards.

All bar one of the finalists were medicines for orphan conditions, and Galafold became the first orphan product in the history of UK Prix Galien to win the Innovative Product Award.

Galafold (migalastat) is an oral medication for the treatment of Fabry disease, a rare lysosomal metabolic storage condition. The drug makes for a more convenient formulation than existing treatments, which are intravenous Enzyme Replacement Therapies.


Amicus receive their award from health minister Lord O'Shaughnessy

Sir Michael Rawlins, chairman of the MHRA and former NICE chairman was once again chairman of the Prix Galien awards, and having served for an exceptional 22 years, steps down from the role this year.

He commented on Galafold: “For the 855 patients in the UK, Fabry disease will cut their life expectancy by 15-20 years. Galafold is a highly innovative, precision-targeting pharmacological chaperone that utilises the body’s own enzyme to enable the breakdown of accumulated glycosphingolipid compounds.”

He added that Galafold would also reduce costs to the NHS and was approved under the NICE highly specialised programme in 2017.

Sanofi’s treatment for moderate-to-severe atopic dermatitis Dupixent was highly commended in the same category.

Biogen pg

Biogen receive their award for Spinraza

In the dedicated orphan drug category, the winner was Biogen’s antisense oligonucleotide Spinraza (nusinersen), the first disease-modifying therapy for spinal muscular atrophy (SMA), a cruel muscle-wasting rare disease in children.

Sir Michael Rawlins commented: “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.”

However, as is frequently the case with products recognised at the UK Prix Galien awards, Spinraza is currently not widely available on the NHS in England (or Northern Ireland and Wales), NICE having rejected its use in draft guidance in August.

In the same category, Qarziba, Eusa’s treatment for neuroblastoma, was highly commended.

A new Medical Technology category was introduced to the awards this year, reflecting the growing important innovation in the field.

Medtronic’s Solitaire, a revascularisation device that reduces stroke-related disability won a commendation, and was called “life changing for the patient, their families, the NHS and society” by Sir Mike Rawlins.

“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," said Karen Westaway, Chief Executive of ValueBase, owners of Prix Galien’s UK franchise.

“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.”

Sir Michael Rawlins

Finally Sir Mike received a special award himself from Karen Westaway, who thanked him for his 22 years of outstanding service as chairman of the Prix Galien awards.

Article by
Andrew McConaghie

16th November 2018



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