International research-focussed healthcare group Chiesi, has announced that the European Commission has granted the marketing authorisation for Lamzede® (velmanase alfa).
This is the first enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha-mannosidosis (AM) – an ultra-rare, progressive, and debilitating disease. Patients present with a broad range of symptoms and their long-term prognosis is generally poor, with reduced life expectancy.1
The authorisation means velmanase alfa is now approved for use in the 31 European countries covered by the European Medicines Agency (EMA).
The Marketing Authorisation has been granted under “exceptional circumstances” according to the EU legislation, which aims to enable treatment of extremely rare disorders for which traditional large-scale clinical studies are not feasible.
Exposure to velmanase alfa has been investigated in 34 patients ranging in age from 6 to 35 years, in five clinical studies. Improvements were detected in disease-specific biomarkers, motor and lung function, and quality of life measures for up to four years of observation.
Alessandro Chiesi, region Europe head of the Chiesi Group said: “Lamzede is the first approved treatment with disease modifying potential for alpha-mannosidosis and therefore, our next goal is to make this treatment available to patients in Europe as soon as possible.”
He added the marketing authorisation of Lamzede in the EU was “a significant stepping stone for patients suffering from this devastating disease and reinforces the Chiesi Group’s role in the complex and challenging world of rare diseases”.
The active substance of Lamzede is velmanase alfa, which is a recombinant form of human alpha-mannosidase (ATC code: A16AB15). Lamzede is an enzyme replacement therapy intended to provide or supplement natural alpha-mannosidase – an enzyme that helps with the degradation of mannose–rich oligosaccharides and so prevents their accumulation in various tissues in the body.
Velmanase alfa was designated as an orphan medicinal product on 26 January 2005.
For the EU Summary of product characteristics for Lamzede, visit: http://ec.europa.eu/health/documents/community-register/2018/20180323140282/anx_140282_en.pdf
- Malm D, Nilssen Ø. Alpha-mannosidosis Orphanet J Rare Dis. 2008;3:21