Velmanase alfa for treating alpha-mannosidosis

Velmanase alfa (Lamzede, Chiesi) is an enzyme replacement therapy produced using recombinant DNA technology. It is intended to replace the natural alpha‑mannosidase enzyme outside the central nervous system to help with the degradation of mannose-rich oligosaccharides. Velmanase alfa is given once a week by intravenous infusion at a dose of 1 mg/kg body weight. It has a marketing authorisation in the UK for treating 'non-neurological manifestations in patients with mild to moderate alpha‑mannosidosis', but the company focused its decision problem on people under 18 years and people who turn 18 while on treatment.

The most common adverse reactions 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 for velmanase alfa.

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. This makes velmanase alfa available to the NHS with a discount. The size of the discount is commercial in confidence. It is the company's responsibility to let relevant NHS organisations know details of the discount.