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UK NICE rejects the use of Kanuma (sebelipase alfa) to treat lysosomal acid lipase deficiency- Alexion

Read time: 1 mins
Last updated:27th Jun 2017
Published:16th Feb 2017
Source: Pharmawand

The UK's National Institute for Health and Care Excellence has published its a final evaluation which rejects the use of Kanuma (sebelipase alfa), from Alexion, to treat infants, children and adults with the rare inherited genetic disorder lysosomal acid lipase deficiency (LAL-D).

In clinical trials with Kanuma, five out of nine infants survived beyond three years of age, achieving normal developmental milestones, the firm noted. But the high cost of the drug - nearly £500,000 per patient - could not be justified by its long-term treatment benefits.

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