NICE recommends Chiesi’s alpha-mannosidosis enzyme replacement therapy

10 Nov 2023
www.pmlive.com
Drug ApprovalGene Therapy
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 National Institute for Health and Care Excellence (NICE)s and can be continued in Lamzedehovelmanase alfa on treatment in line with a commercial agreement, Chiesi said.alpha-mannosidosis
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.
Alpha-mannosidosisssocialysosomal storage disordersigns and symptoms, including difficulty coMAN2B1ing movement, breathing problems, deafness, speech difficulties and bonealpha-mannosidasend 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 fdeafness ospeech difficultiesent tbone abnormalities by NICE for the condition.
Lamzedean Iqbal, head of medical affairs, global rare dialpha-mannosidase 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 montMucopolysaccharide Diseasesnded 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.
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