EMA hands Italy's Chiesi a win in its long campaign to advance new drugs for rare diseases
Five years after Italy’s Chiesi built up its rare disease efforts through acquiring Zymenex, the EMA has come through with a marketing OK for a drug obtained in that deal.
Lamzede is an enzyme replacement therapy for the ultra-rare alpha-mannosidosis, a multi-faceted debilitating disease that is estimated to affect 470 people in the EU. The treatment is approved specifically for non-neurological manifestations in patients with mild to moderate forms of the disorder, a first for this patient population.
The disease is caused by a gene mutation that leads to severe deficiency in alpha-mannosidase, an enzyme that breaks down glycoproteins. As a result, a toxic build-up of sugars takes place in the body, causing a variety of symptoms from skeletal abnormalities and muscle problems to hearing loss and motor skill impairments. Current treatments include bone marrow transplant and peripheral blood stem cell transplantation, in addition to other therapies targeting individual symptoms.
Designated an orphan drug back in 2005 when it was still in Zymenex’s hands, Chiesi’s Lamzede works by delivering, intravenously, a recombinant form of alpha-mannosidase dubbed velmanase alfa to patients. Falling under “exceptional circumstances,” the treatment’s approval came after several clinical studies involving a total of 34 patients and a positive CHMP opinion in January.
The largest of those trials, a Phase III, enrolled 25 patients, and found reduction in serum oligosaccharides for both the adult and pediatric sub-groups. Those under 18 showed positive improvement in functional tests like the three-minute stair climbing test and six-minute walking test, while the adult group achieved stabilization compared to the placebo arm (who experienced more decline than the drug arm).
“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,” said Alessandro Chiesi, head of Europe at the 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.”
Chiesi building in Germany. CHIESI DEUTSCHLAND