BioMarin's amifampridine phosphate gets EU marketing approval for LEMS

The European Commission has granted marketing approval for US drugmaker BioMarin Pharmaceutical's 3,4-diaminopyridine (amifampridine phosphate), for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS).

Amifampridine phosphate, developed by the pharmaceutical unit of France's Paris Public Hospital Authority (AP-HP) and licensed from EUSA Pharma SAS, is the first approved treatment for LEMS, thereby conferring orphan drug protection and providing 10 years of market exclusivity in Europe. It has also received orphan drug designation in the USA, the company noted.

BioMarin acquired the rights to the drug along with its $58.5 million acquisition last year of Huxley Pharmaceuticals, to which EUSA had on-licensed the compound, and the US firm is obliged to pay sales milestones of up to $36 million.