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Rare Disease: Catalyst Pharmaceutical Receives 20th FDA Breakthrough Therapy Designation

August 27, 2013

Catalyst Pharmaceutical Partners, a pharmaceutical company focusing on developing and commercializing novel drugs to treat rare neuromuscular and neurological diseases, announces on August 27, 2013, that orphan drug Firdapse (Amifampridine Phosphate) receives FDA Breakthrough Therapy Designation (BTD) #20. Firdapse, which receives FDA Orphan Drug Designation in November 2009, receives FDA BTD for the symptomatic treatment of patients with Lambert-Eaton Myasthenic Syndrome (LEMS).

Firdapse is marketed by BioMarin in Europe. The drug is now in a Phase III study that is designed to help receive FDA approval.   

LEMS is a rare autoimmune disease with primary symptoms of muscle weakness of the limbs. It is a result of an autoimmune reaction in which antibodies are formed against presynaptic voltage-gated calcium channels in the neuromuscular junction. The prevalence of LEMS is 3.4/1,000,000. Approximately 60% of patients with LEMS have an underlying malignancy, most common of which is small cell lung cancer. Patients who develop LEMS are usually over 40, though it can occur at any age.

The Blog Post, “FDA Breakthrough Therapy Designation Chart”, will always have the latest FDA Breakthrough Therapy (BT) designations, so please bookmark using URL

The latest “FDA Breakthrough Therapy Designation Statistics Chart” Blog Post is located at URL, so please bookmark.

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