Amryt Pharma PLC (LON:AMYT, NASDAQ:AMYT) said it received positive feedback from the American drug regulator as it seeks sign-off for its Myalept drug for use in patients with partial lipodystrophy (abnormal distribution of fat).
In the US, it is currently used to treat complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.
In Europe, the label is broader and includes familial or acquired partial lipodystrophy (PL) in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control.
In its bid to grow the reach of Myalept in the States it is in dialogue with the Food & Drug Administration on the proposed development plan and study design to support an indication for patients with PL.
Amryt has received what is called a Type C written response from the regulator saying it is willing to consider an efficacy supplement based on six-months efficacy and safety data from its trial in PL patients.
Agreement was also received on Amryt’s responses addressing PL study design comments from previous FDA discussions, which have been incorporated into its study protocol.
The company is carrying out a 12-month phase III trial to evaluate the safety and efficacy of a daily subcutaneous Myalept treatment (also known as metreleptin) in patients with PL.
“Today’s news represents further progress as we grow the reach of our commercial products across both territories and indications,” said chief executive, Dr Joe Wiley.
“We now have a very clear path forward for our global clinical study of metreleptin in PL which, if successful, could offer the potential to address a broader population of patients in need.”