Posted by Michael Wonder on 23 May 2018
NICE says no to Chiesi’s rare disease drug
23 May 2018 - England’s cost effectiveness watchdog NICE has published draft guidance not recommending Chiesi’s enzyme replacement therapy Lamzede (velmanase alfa).
The drug treats alfa-mannosidosis, a very rare and debilitating lysosomal storage disorder (LSD) that affects around 25 people in England.
The enzyme replacement therapy is given lifelong once a week by intravenous infusion at a dose of 1 mg/kg. The list price is £886.61 per 10 mg vial – producing a cost of hundreds of thousands of pounds a year for each patient.
