An Important Milestone
Currently, amifampridine is the only evidence-based, FDA-approved treatment for adult patients with Lambert-Eaton Myasthenic Syndrome (LEMS). The European Medicines Agency (EMA) has also approved amifampridine in the treatment of LEMS, and the European Federation of Neurological Societies recommends amifampridine as a first-line treatment for patients with LEMS. Amifampridine has been granted “orphan drug designation” by the FDA for the treatment of patients with congenital myasthenic syndromes (CMS) and MuSK-positive myasthenia gravis (MuSK-MG). There is an ongoing investigational program for amifampridine in the treatment of patients with spinal muscular atrophy (SMA).
Catalyst has undertaken the clinical development of a stable salt form of the amifampridine base compound, in order to provide patients access to a well-studied and consistent product. This novel formulation allows for storage and transportation at room temperature and will be produced using Good Manufacturing Practices (GMP).
Now there’s a prescription-ready treatment for your adult patients with LEMS.
In October 2012, Catalyst announced a strategic collaboration with BioMarin Pharmaceuticals, Inc., which included the in-licensing of the North American rights to amifampridine and responsibility for its clinical development program. Amifampridine had previously received orphan medicinal product designation by the European Union (EU) and has been marketed in the European Commission (EC) for the treatment of LEMS since 2010.
Support for the clinical efficacy of amifampridine for treatment of patients with LEMS has been documented in several published randomized, double-blind, placebo-controlled studies and two double-blind studies with an active comparator in patients with LEMS. Data from the randomized controlled studies demonstrate statistically significant improvements across a number of independent measures of neurological function, including Quantitative Myasthenia Gravis (QMG) score and compound muscle action potential (CMAP), which have been demonstrated to be clinically relevant in patients with LEMS.
To date, amifampridine is the only evidence-based, FDA-approved treatment for adult patients with Lambert-Eaton Myasthenic Syndrome (LEMS).
For more information about amifampridine, please choose from the list of clinical publications and reviews below.
Oh SJ and Sieb JP. Update on Amifampridine as a Drug of Choice in Lambert-Eaton Myasthenic Syndrome. US Neurology. 2014; 10(2): i-vii Go to publication
Oh SJ, Shcherbakova N, Kostera-Pruszczyk A, et al. Amifampridine phosphate (Firdapse®) is effective and safe in a phase 3 clinical trial in LEMS. Muscle Nerve. 2016;53(5):717-725. Go to publication
Lindquist S, Stangel M. 3,4-Diaminopyridine (amifampridine) for the treatment of Lambert–Eaton myasthenic syndrome. Expert Opin Orph Drugs. 2014; 2(3):293-300. Go to publication
Sedehizadeh S, Keogh M, Maddison P. The use of aminopyridines in neurological disorders. Clin Neuropharmacol. 2012; 35(4):191-200. Go to publication
Harms L, Sieb JP, Williams AE, et al. Long-term disease history, clinical symptoms, health status, and healthcare utilization in patients suffering from Lambert Eaton myasthenic syndrome: Results of a patient interview survey in Germany. J Med Econ. 2012; 15(3):1-10. Go to publication
Green DM, Jones AC, Brain KR. Content variability of active drug substance in compounded oral 3,4-diaminopyridine products. J Clin Pharm Ther. 2012; 37:53–7. Go to publication
Lindquist S and Stangel M. Update on treatment options for Lambert–Eaton myasthenic syndrome: focus on use of amifampridine Neuropsychiatr Dis Treat. 2011;7(1):341–349. Go to publication
Quartel A, Turbeville S, Lounsbury D. Current therapy for Lambert-Eaton myasthenic syndrome: development of 3,4-diaminopyridine phosphate salt as first-line symptomatic treatment. Curr Med Res Opin. 2010; 26(6):1363-75. Go to publication
Raust JA, Goulay-Dufaÿ S, Le Hoang MD, et al. Stability studies of ionised and non-ionised 3,4-diaminopyridine: hypothesis of degradation pathways and chemical structure of degradation products. J Pharm Biomed Anal. 2007; 43(1):83-8. Go to publication