AMYT.L Share News (AMYT)

(Sharecast News) - Commercial-stage biopharmaceuticals company Amryt Pharma has received further feedback from the United States Food and Drug Administration (FDA) on Myalept, it said on Tuesday, and its proposed development plan and study design to support an indication for patients with partial lipodystrophy (PL).
The AIM-traded firm said 'Myalept', or metreleptin, is approved in the US as an adjunct to diet, as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (GL).

It is also approved in the European Union 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 had failed to achieve adequate metabolic control.

The prevalence of PL patients severely affected by their disease was thought to be similar to GL in the US, and thus, if approved for treatment of that patient sub-group, it would represent a doubling of Amryt's current US market opportunity.

It said on Tuesday that it recently received additional written feedback on its proposed development plan and study design to support an indication for patients with PL.

The FDA confirmed that it was willing to consider an efficacy supplement based on six-months efficacy and safety data from a randomised, placebo-controlled trial in PL patients.

Safety data would 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 would take into consideration anti-leptin neutralising activity.

Agreement was also received on Amryt's responses addressing PL study design comments from previous FDA discussions, which were incorporated into the study protocol.

The board said the 12-month randomised, placebo-controlled phase 3 trial to evaluate the safety and efficacy of daily subcutaneous metreleptin treatment in patients with PL would enrol around 80 patients globally.

It said the study would enrol patients with severe metabolic consequences of their disease as reflected by blood glucose control and triglyceride levels, on optimal background treatment.

Amryt said it had completed feasibility assessments to identify investigators and sites with potentially eligible PL patients, adding that the study would be initiated by the end of 2021.

"Today's news represents further progress as we grow the reach of our commercial products across both territories and indications," said chief executive officer 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."

At 0900 GMT, shares in Amryt Pharma were down 3.26% at 208p.