NICE okays enzyme replacement therapy for alpha-mannosidosis


Chiesi’s Lamzede (velmanase alfa) will now be funded on the NHS in England and Wales as an option for treating the non-neurological signs and symptoms of mild to moderate alpha-mannosidosis following a recommendation by the National Institute for Health and Care Excellence (NICE).

The treatment must be started in people under 18 years of age, and can be continued in people who turn 18 while on treatment, in line with a commercial agreement.

Velmanase alfa is the first enzyme replacement therapy recommended by NICE for alpha-mannosidosis and is given to patients once-a-week by intravenous infusion.

“We are pleased that eligible patients in England and Wales will now be able to access velmanase alfa on the NHS for the first time,” said Dr Kamran Iqbal, Head of Medical Affairs, Global Rare Diseases, Chiesi UK&I.

“The discovery and availability of new treatment options is crucial for those affected by this progressive disease, and we have worked tirelessly over several years with key stakeholders including NICE, patient groups and clinicians to address uncertainties that are common in rare diseases and ensure access for those in need. We are immensely grateful for the hard work and commitment of all involved.”

Alpha-mannosidosis is an extremely rare, genetic condition, thought to affect around 25 people in England.

“On behalf of our alpha-mannosidosis community, I am pleased at the news that NICE has made the decision to make velmanase alfa available to suitable patients in our community in England and Wales,” said Bob Stevens, Group Chief Executive, MPS Society. “This marks a real change for this community because now they have the possibility of treatment and this is another example of innovative science benefitting our rare patients.”

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