MA granted for drug tested in just 34 patients

An absence of comprehensive data did not prevent Italian family-owned drugmaker Chiesi Farmaceutici from receiving Marketing Authorization (MA) for Lamzede (velmanase alfa).

The MA was granted under European Union (EU) ‘exceptional circumstances’ legislation whereby the applicant is unable to provide comprehensive data on efficacy and safety in normal conditions of use, in this case because the condition concerned is an ultra-rare disorder.

So, after being investigated in just 34 patients, Lamzede has become the first enzyme replacement therapy for the treatment of non-neurological manifestations in patients with mild to moderate alpha mannosidosis (AM), a condition presenting a broad range of symptoms with generally poor long-term prognosis and reduced life expectancy.