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Firdapse® (amifampridine) for LEMS

Firdapse®, currently approved in the European Union, is the first and only approved drug for the symptomatic treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) in adults, a rare autoimmune disease with the primary symptoms of muscle weakness. In LEMS, the body’s own immume system attacks connections between nerves and muscles and disrupts the ability for nerve cells to send signals to muscle cells.

Firdapse at a Glance

  • First specific approved therapy in the European Union for the treatment of LEMS
  • Manufactured and commercialized by BioMarin in the European Union
  • Designated as an orphan drug internationally

Regulatory Status

In December 2009, Firdapse received marketing authorization from the European Commission making it the first approved therapy for the symptomatic treatment of LEMS. Firdapse has been designated an orphan drug in the European Union, which grants market exclusivity for 10 years.

Commercialization Plan

Firdapse is commercialized by BioMarin in the European Union.

In October 2012, BioMarin licensed North American rights to Firdapse to Catalyst Pharmaceutical Partners. For more information on Firdapse development plans in the United States, please refer to the Catalyst website.

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BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS

Products

Brineura
Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

Global Medical Information

Contact Global Medical Information

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