Velmanase alfa for treating alpha-mannosidosis
Closed for comments This consultation ended on at Request commenting lead permission
3 The technology
3.1 Velmanase alfa (Lamzede, Chiesi) is an enzyme replacement therapy produced using recombinant DNA technology. It is intended to replace natural alpha-mannosidase enzyme outside the central nervous system to help with the degradation of mannose-rich oligosaccharides. Velmanase alfa is administered once a week by intravenous infusion at a dose of 1 mg/kg. It has a marketing authorisation in the UK for treating 'non-neurological manifestations in patients with mild to moderate alpha-mannosidosis'.
3.2 The most common adverse reactions listed in the summary of product characteristics for velmanase alfa include weight gain, immune-related responses, diarrhoea, headache, arthralgia (joint pain), increased appetite and pain in the extremities. For full details of adverse reactions and contraindications, see the summary of product characteristics.
3.3 The list price of velmanase alfa is £886.61 per 10 mg vial (excluding VAT; company's evidence submission). The company has a commercial arrangement, which would have applied if the technology had been recommended.
How are you taking part in this consultation?
You will not be able to change how you comment later.
You must be signed in to answer questions