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