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Orphan drugs dominate UK Prix Galien shortlist, medtech debuts

Seven treatments for rare conditions have been selected for the final


The shortlist for one of the UK’s most prestigious pharma industry prizes has been announced, and illustrates the growing number of orphan drugs reaching the market.

Seven treatments for rare conditions have been selected for the final of UK Prix Galien 2018, with one – Galafold from Amicus – becoming the first orphan drug to be shortlisted in both the Orphan Product and Innovative Product categories.

Held every other year at the House of Commons in London, the awards focus solely on how much the medicines use innovation to advance treatment for patients, and this year sees the addition of a medical technology category.

Previous years had seen the introduction of a Real World Evidence (RWE) award, however the UK Prix Galien judges failed to shortlist any entrants in this category, suggesting the field is still maturing.

The final includes representation from eight biopharmaceutical companies and one global medical technology company. The shortlist was drawn up by the UK Prix Galien’s independent panel of distinguished physicians, chaired by former NICE chairman Sir Michael Rawlins.

This year, the judges concluded that just two entrants were worthy of being shortlisted for the Innovative Product Award; Sanofi (Dupixent) and Amicus (Galafold)

The most recent winner of the Innovative Product Award, at the 2016 UK Prix Galien, was Novartis’ Entresto – the first new treatment for heart failure in 15 years.

Seven companies have been shortlisted for the Orphan Product Award; Amicus (Galafold), Biogen (Spinraza), Biomarin (Brineura), Eusa (Qarziba), Intercept (Ocaliva), Kyowa Kirin (Crysvita) and Shire (Revestive).

The 2016 Orphan Drug Award was won by Holoclar, which in 2015 became the first stem cell therapy to receive European marketing approval. The therapy is used to treat moderate-to-severe forms of limbal stem cell deficiency (LSCD), a rare eye condition that can lead to blindness.

Only one company was shortlisted for the inaugural Medical Technology Award; Medtronic (Solitaire Platinum). The Solitaire Platinum revascularisation device used for clot retrieval from occluded blood vessels in the brain due to ischemic stroke.

Karen Westaway, Chief Executive of ValueBase – owners of Prix Galien’s UK franchise – said: “UK Prix Galien is always evolving and 2018 is no different. The introduction of a prize for Medical Technology is in recognition of the sector’s rich history in developing innovative devices, diagnostics and medical equipment."

Welcoming the increased investment in orphan conditions, she noted that 41% of drugs approved by the FDA in 2016 were for orphan conditions, with the picture similar in Europe. Looking ahead to the awards night itself, she concluded:  It’s going to be a fiercely contested final. We look forward to welcoming UK pharma to the House of Commons in the autumn to find out who wins these prestigious prizes.”

Visit Prix Galien UK for more details

Article by
Andrew McConaghie

21st June 2018

From: Research



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