The National Institute for Health and Care Excellence (NICE) has recommended Chiesi’s Lamzede (velmanase alfa) to treat the non-neurological signs and symptoms of mild-to-moderate alpha-mannosidosis, an extremely rare inherited condition affecting around 25 people in England.
The treatment must be started in patients aged under 18 years and can be continued in those who turn 18 while on treatment in line with a commercial agreement, Chiesi said.
Alpha-mannosidosis is a lysosomal storage disorder caused by inheriting a faulty copy of the MAN2B1 gene from both parents, resulting in impaired production of the enzyme alpha-mannosidase, which is needed to break down certain sugars in the body.
The condition is associated with a broad range of signs and symptoms, including difficulty controlling movement, breathing problems, deafness, speech difficulties and bone abnormalities, and the most severe forms are associated with rapid progression and early death.
Lamzede, which is designed to supplement or replace the alpha-mannosidase enzyme via a once-weekly intravenous infusion, is now the first and only enzyme replacement therapy recommended by NICE for the condition.
Dr Kamran Iqbal, head of medical affairs, global rare diseases, Chiesi UK and Ireland, said: “We are pleased that eligible patients in England and Wales will now be able to access [Lamzede] on the NHS for the first time.
“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.”
Bob Stevens, group chief executive of the Society for Mucopolysaccharide Diseases, added: “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.”
NICE’s final evaluation of Lamzede comes just two months after the agency recommended another of Chiesi’s enzyme replacement therapies, Elfabrio (pegunigalsidase alfa), to treat adults with Fabry disease, a rare genetic disorder that can lead to progressive damage to vital organs, including the heart, kidney and brain.