Keywords: BioMarin, Amifampridine, LEMS, EU approval,

BioMarin’s amifampridine phosphate gets EU marketing approval for LEMS

Article | 6 January 2010

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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.

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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.

"We are excited to bring the first approved therapeutic option to the European Union to treat LEMS, a rare, serious and debilitating disease as we expand our commercial portfolio with our fourth approved product," said Jean-Jacques Bienaime, chief executive of BioMarin. "We are on track to launch amifampridine in the EU in mid-March and look forward to meeting with the [Food and Drug Administration] in early 2010 to determine the necessary regulatory path for this product in the USA. We will also evaluate the amifampridine phosphate opportunity in a number of countries outside the USA and Europe, as well as the best development strategy for amifampridine in other indications," he added.

The prevalence of LEMS is estimated at four to 10 per million, or approximately 2,000 to 5,000 patients in the EU and 1,200 to 3,100 patients in the USA, noted BioMarin, adding that approximately 50% of LEMS patients diagnosed have small cell lung cancer.