Novato, Calif.-based BioMarin Pharmaceutical has just launched its fourth orphan drug product, Firdapse (amifampridine phosphate), in Germany and the UK, nearly four months after the European Commission approved it.

BioMarin expects to launch Firdapse in all major EU countries by the end of the year. Firdapse is a new molecular entity indicated for the symptomatic treatment of a rare autoimmune disorder, Lambert Eaton myasthenic syndrome (LEMS). BioMarin says it will discuss the regulatory pathway in the U.S. for the product with the FDA in the second quarter of this year.

The success of BioMarin and other orphan drug marketers has placed the sector on the radar of big pharma - GlaxoSmithKline set up a specific unit in February to develop products for rare diseases, while Pfizer and Novartis also have expressed interest in this area (Also see "Orphaned No Longer: Big Pharma Embraces Drugs for Niche Markets" - In Vivo, 1 February, 2010.). There even has been speculation that BioMarin is a takeover target Also see "Dispute With Departing Board Member Creates Speculation BioMarin Is For Sale" - Pink Sheet, 29 March, 2010.).

Around 2,000 to 4,000 patients in the EU have LEMS at any one time. Most of these have small cell lung cancer. It is associated with the development of auto-antibodies to neuromuscular junctions, specifically voltage-gated calcium channels, thereby reducing the release of the neurotransmitter acetylcholine, and is characterized by muscle weakness and fatigue. The product is not inexpensive - in the UK, 100 x 10mg tablets cost £2,017 ($ 3,104), around a month's supply.

Amifampridine (3,4-diaminopyridine) base has been used as an ad hoc hospital preparation for more than 20 years for LEMS, but has never been properly developed or marketed. The phosphate salt is a new proprietary formulation, and the CHMP granted it a positive opinion under its "exceptional circumstances" provisions last October.

The European Commission said that LEMS was encountered so rarely that the company could not be expected to provide comprehensive data on the use of amifampridine; however, BioMarin is obliged to collect further evidence of its safety and efficacy.

BioMarin acquired rights to Firdapse by a circuitous route from its originators, the pharmaceutical unit (AGEPS) of the Paris Public Hospital Authority. This unit licensed rights to the product to EUSA Pharma (which developed it as Zenas ), which in turn passed them to a private company, Huxley Pharmaceuticals.

Huxley was acquired by BioMarin last October for $15 million up front, plus potential additional payments of over $40 million for achieving LEMS-related milestones, and with the possibility of further milestone payments if the product is developed successfully for multiple sclerosis.

BioMarin set up commercial sales and marketing operations in Europe in 2006, and has offices in Dublin, Ireland, and the UK.

- John Davis (j.davis@elsevier.com)

BioMarin Launches Firdapse In Germany And The UK

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