Minoryx Therapeutics and Neuraxpharm Group announced that the Marketing Authorization Application (MAA) for Minoryx’s lead candidate leriglitazone (NEZGLYAL®) has been submitted to the European Medicines Agency (EMA) for the treatment of pediatric and adult male patients with cerebral adrenoleukodystrophy (cALD). EMA has now validated the MAA file and it is under review by the Committee for Medicinal Products for Human Use (CHMP).
This new MAA is based on recent data from the successfully concluded NEXUS study, a 96-week, pivotal, open-label study designed to evaluate the efficacy and safety of once-daily oral dosing of leriglitazone in pediatric patients with cALD. The study met the primary endpoint and all 20 evaluable patients remained clinically stable whilst on treatment and 7 out of 20 evaluable patients (35%) met the arrested disease criteria, which is significantly greater than the 10% self-arrest rate that would be expected from natural history (p<0.05). Leriglitazone was well tolerated with no discontinuations due to adverse events.
The submission is further supported by data from the previously concluded ADVANCE study, a 116 patient, double-blind, placebo-controlled study conducted in Europe and the United States in which leriglitazone reduced the incidence and progression of cerebral lesions in adults. Furthermore, data from the ongoing extensive compassionate use program in adult and pediatric patients with cALD have also been submitted. Leriglitazone was generally well tolerated in the ADVANCE study and compassionate use programme.
“We believe the new and positive data from Nexus provide compelling evidence for the efficacy of leriglitazone giving hope to patients and their families that a new treatment will be available in the near future,” said Marc Martinell, CEO, Minoryx. “Leriglitazone, if approved, would be the only pharmacological treatment for patients suffering from this devastating orphan disease with a major unmet medical need.”
“We are very pleased that the clinical development program and compassionate use data have shown the efficacy of leriglitazone across all age ranges,” said Arun Mistry, Chief Medical Officer, Minoryx. “We will continue to work closely with the EMA through the evaluation process to maximize the chance for this therapy to reach all patients in desperate need.”
Dr. Jörg-Thomas Dierks, CEO, Neuraxpharm said;“The EMA’s validation of the leriglitazone MAA submission is a key step forward as we look to bring this much needed treatment to patients. Given the terrible impact of this devastating disease and the lack of effective options, we remain hopeful of a positive outcome from the CHMP.”
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