Rare Breakthrough Therapy Designation for Amifampridine Phosphate


Amifampridine PhosphatePatients suffering from the rare neuromuscular disorder Lambert-Eaton myasthenic syndrome have recently received life changing news. On August 27, 2013 the FDA decided to grant Amifampridine Phosphate a Breakthrough Therapy Designation, also known as BTD.  Marketed by BioMarin as the brand name Firdapse, Amifampridine Phosphate offers a crucial new hope for patients enduring this fatal disease, which is also called LEMS. Amifampridine Phosphate is already licensed in the EU to treat patients with Lambert-Eaton myasthenic syndrome, having been approved in January 2010.

Lambert–Eaton Myasthenic SyndromeLambert-Eaton myasthenic syndrome is a severely debilitating and deadly condition which affects neuromuscular transmission. The insufficient release of the acetylcholine synaptic vesicles creates a devastating impairment in the patient’s nerve signal transmissions. While researchers are not one hundred percent certain of the cause of this rare disease, most patients with LEMS have been determined to have an underlying autoimmune condition of a specific origin, thus predisposing them to this uncommon illness. Patients with LEMS will exhibit symptoms such as extreme muscle weakness and fatigue, numbness, tingling, drooping eyelids and difficulty with speech. Lambert-Eaton myasthenic syndrome becomes dire for patients when they experience respiratory muscle failure, as well as subsequent diagnoses of cancer, which is unfortunately common for this patient population. Almost sixty percent of patients with LEMS also develop small-cell lung cancer. Research scientists and physicians are still trying to determine that LEMS leads to small-cell lung cancer, and not the other way around. However, the comorbid conditions of both LEMS and small-cell lung cancer often lead to unresectable outcomes.

Current Phase III clinical trials are underway to continue to evaluate both the safety and efficacy of Amifampridine Phosphate. The current trial is double-blind, multicenter, placebo-controlled and randomized, and available to all patients with diagnosed cases of LEMS.  Roughly 3,000 patients in the United States and Canada are currently diagnosed with LEMS, making this presynaptic neuromuscular transmission condition one that indelibly impairs lives.

LGM Pharma provides API Amifampridine Phosphate, CAS number 446254-47-3, for research and development purposes. Clients can be assured of continuous support throughout the R&D process, as well as quality API products.

 

Products currently covered by valid US Patents are offered for R&D use in accordance with 35 USC 271(e)+A13(1). Any patent infringement and resulting liability is solely at buyer risk.

  • Share: