Firdapse amifampridine: QT/QTc study data

A double-blind QT study in 59 healthy volunteers showed that Firdapse given at therapeutic and supratherapeutic doses met the primary endpoint by demonstrating no effect compared to placebo on heart rate or cardiac depolarization. Additionally, no subjects developed new, clinically relevant electrocardiographic or morphological changes following treatment with Firdapse. Furthermore, there was no significant effect of Firdapse on cardiac repolarization. Catalyst said that during a pre-IND meeting in 2010, FDA requested thorough QT study results for Firdapse given at exposures higher than typical therapeutic levels be included in an NDA for the product.

Last month, FDA requested a "slight modification" in the analysis of the endpoints of an ongoing Phase III trial of Firdapse in about 36 patients with LEMS. Catalyst said it does not believe the modification will require changes in data collection or the number of patients needed for enrollment. The company said it anticipates meeting previously disclosed timelines for submission of an NDA to FDA for Firdapse to treat LEMS. Catalyst previously said it plans to submit an NDA in 1H15 (see BioCentury, Jan. 13). ...