Parma, IT, April 4, 2018 - Chiesi, an international research-focussed healthcare group, today announced that the European Commission has granted the marketing authorisation for Lamzede® (velmanase alfa), 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 disease1. As a result of this marketing authorisation, 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 aim to enable treatment of extremely rare disorders for which traditional large-scale clinical studies are not feasible. Velmanase alfa has been investigated in 34 patients, both children and adults.
“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,” comments Alessandro Chiesi, Region Europe Head, Chiesi Group. “The marketing authorisation of Lamzede in the EU is 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.”
For the EU Summary of Product Characteristics for Lamzede, please visit http://ec.europa.eu/health/documents/community-register/2018/20180323140282/anx_140282_en.pdf
Notes to Editors
About Lamzede®▼(Velmanase alfa)
The active substance of Lamzede is velmanase alfa, 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 thus prevents their accumulation in various tissues in the body. Velmanase alfa was designated as an orphan medicinal product on 26 January 2005.
The treatment should be supervised by a physician experienced in the management of patients with alpha mannosidosis or in the administration of other enzyme replacement therapies (ERT) for lysosomal storage disorders.
A total of 34 patients (ranging in age from 6 to 35 years) were exposed to velmanase alfa 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.
Velmanase alfa was initially developed by Zymenex A/S, which was acquired by Chiesi in 2013. With this acquisition, Chiesi has established an integrated biotech research group based in Hillerod (Denmark) and Lidingö (Sweden) focussed on the development of protein-based therapeutics.
AM is an ultra-rare disorder caused by the genetic absence or malfunction of alpha-mannosidase, an enzyme involved in the cellular breakdown of complex sugar molecules, oligosaccharides. The deficiency in alpha-mannosidase activity leads to the progressive toxic accumulation of oligosaccharides in the cells of many tissues and organs. The most frequent symptoms include facial coarsening, intellectual disability, progressive motor function disturbances and physical disability, hearing impairment, impaired speech, immunodeficiency and recurrent infections, psychiatric symptoms, and skeletal abnormalities. The long-term prognosis is generally poor, with reduced life expectancy1. Velmanase alfa is administered via weekly intravenous infusions to replace the missing or malfunctioning enzyme causing the disease.
About Chiesi Group
Based in Parma, Italy, Chiesi Farmaceutici is an international research-focused Healthcare Group, with over 80 years of experience in the pharmaceutical industry, present in 26 countries. Chiesi researches, develops and markets innovative drugs in the respiratory therapeutics, specialist medicine and rare disease areas. Its R&D organisation is headquartered in Parma (Italy), and integrated with 6 other key R&D groups in France, the USA, the UK, Sweden, and Denmark to advance Chiesi's pre-clinical, clinical and registration programmes. Chiesi employs more than 5,000 people.