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UniQure BV’s gene therapy Glybera won the backing of European drug authorities on its fourth attempt, making it the first approved such treatment in a western nation.
The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended that Glybera be approved as a treatment for lipoprotein lipase deficiency, or LPLD, the London-based regulator said today in a statement. The drug is intended for people with a rare fat-processing disorder who have severe or multiple pancreatitis attacks despite restricting their fat intake, the agency said.
“It’s phenomenal because it’s the first gene therapy the EMA has approved,” Joern Aldag, UniQure’s chief executive officer, said in an interview. The treatment will only need to be administered once to be effective and may have peak annual sales between 50 million euros ($61.1 million) and 300 million euros, depending on how it’s priced, he said.
Glybera was developed by Amsterdam Molecular Therapeutics. (AMT) The company sold itself to Amsterdam-based UniQure this year after European authorities initially rejected the drug. <snip>
