23 Mar 2021 07:00
Amryt Receives Positive Feedback from the FDA on the Path Forward for Myalept® (metreleptin) Indication in Partial LipodystrophyAmryt Receives Positive Feedback from the FDA on the Path Forward for Myalept® (metreleptin) Indication in Partial Lipodystrophy
DUBLIN, Ireland, and Boston MA, March 23, 2021, Amryt (Nasdaq: AMYT, AIM: AMYT), a global, commercial-stage biopharmaceutical company focussed on acquiring, developing and commercializing novel treatments for rare diseases, today 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 3 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.
Amryt has completed feasibility assessments to identify investigators and sites with potentially eligible PL patients. It is anticipated that the study will be initiated by the end of 2021.
Dr Joe Wiley, CEO of Amryt Pharma, commented today: “Today’s news represents further progress as we grow the reach of our commercial products across both territories and indications. 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.”
About AmrytAmryt is a global commercial-stage biopharmaceutical company focused on acquiring, developing and commercializing innovative treatments to help improve the lives of patients with rare and orphan diseases. Amryt comprises a strong and growing portfolio of commercial and development assets.
Amryt’s commercial business comprises two orphan disease products - metreleptin (Myalept®/ Myalepta®) and lomitapide (Juxtapid®/ Lojuxta®).
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 partial lipodystrophy (PL) in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control. For additional information, please follow this link.
Juxtapid®/ Lojuxta® (lomitapide) is approved as an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the rare cholesterol disorder, Homozygous Familial Hypercholesterolaemia ("HoFH") in the US, Canada, Columbia, Argentina and Japan (under the trade name Juxtapid®) and in the EU, Israel and Brazil (under the trade name Lojuxta®). For additional information, please follow this link.
Amryt's lead development candidate, Filsuvez® (Oleogel-S10) is a potential treatment for the cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa (“EB”), a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment. Filsuvez® has been selected as the brand name for Oleogel-S10. The product does not currently have regulatory approval to treat EB.
Amryt’s pre-clinical gene therapy platform, AP103, offers a potential treatment for patients with Dystrophic EB, and is also potentially relevant to other genetic disorders. For more information on Amryt, including products, please visit www.amrytpharma.com.
The person making this notification on behalf of Amryt is Rory Nealon, CFO/COO and Company Secretary.
Financial Advisors
Shore Capital (Edward Mansfield, Daniel Bush, John More) are NOMAD and Joint Broker to Amryt in the UK. Stifel (Ben Maddison) are Joint Broker to the company in the UK.
Forward-Looking Statements
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Contacts
Joe Wiley, CEO / Rory Nealon, CFO/COO, +353 (1) 518 0200, ir@amrytpharma.com
Edward Mansfield, Shore Capital, NOMAD, +44 (0) 207 468 7906, edward.mansfield@shorecap.co.uk
Tim McCarthy, LifeSci Advisors, LLC, +1 (212) 915 2564, tim@lifesciadvisors.com
Amber Fennell, Consilium Strategic Communications, +44 (0) 203 709 5700, fennell@consilium-comms.com