Amryt receives positive feedback from the FDA on the path forward for Myalept indication for partial lipodystrophy.

Amryt provides an update on its engagement with the FDA on Myalept and its proposed development plan and study design to support an indication for patients with partial lipodystrophy (PL). Myalept / Myalepta (metreleptin) is approved in the US (under the trade name Myalept) as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (GL) and in the EU (under the trade name Myalepta) as an adjunct to diet for the treatment of leptin deficiency in patients with congenital or acquired GL in adults and children two years of age and above and familial or acquired PL in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control. The prevalence of PL patients severely affected by their disease is thought to be similar to GL in the US. Therefore, if approved for treatment of this patient sub-group, this represents approximately double the current US market opportunity. Building on previous FDA interactions, Amryt has recently received additional feedback via a Type C written response on the proposed development plan and study design to support an indication for patients with PL. The FDA confirmed that they are willing to consider an efficacy supplement based on 6-months efficacy and safety data from a randomized, placebo-controlled trial in PL patients. Safety data will continue to be collected up to the completion of the 12-month treatment period and the overall assessment of the benefit-risk ratio for PL patients will take into consideration anti-leptin neutralizing activity. Agreement was also received on Amryt’s responses addressing PL study design comments from previous FDA discussions, which have been incorporated into the study protocol (APG-20). The 12-month randomized, placebo-controlled Phase III trial to evaluate the safety and efficacy of daily subcutaneous metreleptin treatment in patients with PL will enroll approximately 80 patients globally. The study will enroll patients with severe metabolic consequences of their disease as reflected by blood glucose control and/or triglyceride levels on optimal background treatment.