- Dutch biotech UniQure (QURE +22.6%) catapults into uncharted territory with the pricing of its gene therapy drug Glybera (alipogene tiparvovec). It is set to debut in Germany at €1.1M ($1.4M).
- Glybera treats an ultra-rare disease called lipoprotein lipase deficiency (LPLD), a condition that clogs the blood with fat. The European Commission approved it in November 2012 but the launch was delayed because the company needed to collect six-year follow up data on its benefits.
- UniQure's commercial partner, Italian firm Chiesi, has filed a pricing dossier with German regulators, who will issue an assessment of the drug's benefits by April 2015. Chiesi seeks a retail price of €53,000 per vial. Under German rules, the launch price will be valid for 12 months.
- At this price, the full regimen cost will be €1.1M for a typical LPLD patient weighing 62.5 kg, who will require 42 injections from 21 vials. The number of eligible patients is tiny, only 150 - 200 across Europe.
- Former #1 Soliris (eculizumab), made by Alexion Pharmaceuticals (ALXN +0.7%) is now a distant second at ~$540,000.
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