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Myalepta Amryt Pharmaceuticals

Amryt Pharmaceuticals
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12 November 2021
London MI Service

New Medicines

MyaleptaGeneralised lipodystrophy and partial lipodystrophy


New molecular entity
Amryt Pharma
Novelion Therapeutics

Development and Regulatory status

March 2021
Mar 21Myalepta is available in the UK. NHS list price for 30 vials of 3mg is 17,512.50; 5.8mg is £35,025.00; 11.3mg is £70,050 [30].
Apr 20In Jan 20, NICE granted the request to enable the company to prepare and provide further evidence for consideration. NICE planned to formally invite participating stakeholders to provide evidence submission and for the HST committee to consider the evidence submitted in October 2020. This has now been delayed due to the COVID-19 pandemic [28].
Dec 19Has been available in Germany since Oct 18 [27].
Aug 19Aegerion Pharmaceutical has requested reconsideration of this topic with NICE in line with the HST Interim Methods and Process guide, to undertake further work to address the concerns outlined by the committee. The Final Evaluation Document for this evaluation has been withdrawn. An appeal hearing will not be held. [26].
Jul 19NICE received two appeals against the Final Evaluation Determination of the HST evaluation from both Aegerion Pharmaceuticals and Lipodystrophy UK. The appeal panel will convene on Friday 30 August 2019 [26].
Jun 19Aegerion is being acquired by Amryt Pharma. Presume this will further delay plans for UK launch [25].
Aug 18NICE TA production delayed to enable the company to prepare and submit additional information for consideration. Presume this is also delaying plans for UK launch [24].
Jul 18Approved in EU [23].
Jun 18EU positive opinion for use as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients with confirmed congenital generalised LD (Berardinelli-Seip syndrome) or acquired generalised LD (Lawrence syndrome) in adults and children 2 years of age and above. Also with confirmed familial partial LD or acquired partial LD (Barraquer-Simons syndrome) in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control [22]. 03/06/2018 09:05:15
May 18Novelion expects CHMP opinion in Q2 18 [21].
Jan 17Company (now Novelion following acquisition of Aegerion by QLT and re-naming) announces that the EMA has accepted the MAA for review: an opinion from the CHMP is expected in December 2017 with final EC decision Q1 2018 [20].
Dec 16Licensing application submitted in EU for metreleptin as a treatment for generalised lipodystrophy and subset of patients with partial lipodystrophy [19].
May 15Aegerion is preparing to file a MAA in the EU for metreleptin to treat certain complications of leptin deficiency in patients with generalised lipodystrophy (GL) in 2016 [18].
Mar 15No longer listed in AZ pipeline. Plans for EU filing potentially changed following sale to Aegerion [17].
Dec 14EU filing planned for Q4 2015 [15].
Nov 14Aegerion Pharmaceuticals announced that it has entered into a definitive agreement with AstraZeneca to acquire Myalept™ [16].
Mar 14Q2 Launched in the US [14].
Feb 14Approved in the US as replacement therapy to treat the complications of leptin deficiency, in addition to diet, in patients with congenital generalized or acquired generalized lipodystrophy [10].
Feb 14AstraZeneca acquires global rights for development, manufacture and commercialisation of metreleptin [12].
Dec 13An FDA panel voted 10-2 that metreleptin´s benefits didn´t outweigh its risks for patients with partial lipodystrophy (LD) and hypertriglyceridaemia and diabetes. Instead, the panel voted 11-1 in favour of the drug in patients with generalized LD, a complete loss of leptin, which is a very small patient population. An FDA decision on licensing is expected by 24 Feb 14 [9].
Dec 13EU filing planned for 2015 [11].
Jun 13EU filing will be 1H 14 [7].
Jun 13FDA has granted priority review status for the treatment of metabolic disorders associated with inherited or acquired lipodystrophy [7].
Jul 12Orphan designation granted by the European Commission to Aptiv Solutions (UK) Limited for metreleptin for the treatment of a number of lipodystrophies: familial partial lipodystrophy (EU/3/12/1022), Lawrence syndrome (EU/3/12/1024), Berardinelli-Seip syndrome (EU/3/12/1025) and Barraquer-Simons syndrome (EU/3/12/1023) [5].
Apr 12US filing has been completed for the use of metreleptin to treat diabetes and/or hypertriglyceridaemia in paediatric and adult patients with rare forms of lipodystrophy. Amylin has requested priority review [4].
Dec 10Initial sections (clinical and non-clinical) of a rolling submission to the FDA for a Biologics License Application (BLA) have been submitted. The chemistry, manufacturing and controls (CMC) section of the BLA will be submitted by the end of 2011 [1].
Dec 10Granted orphan drug and fast track status in US in patients with rare inherited and acquired forms of lipodystrophy [1].


An analogue of the human hormone, leptin
Familial partial lipodystrophy affects approximately 0.2 in 10,000 people in the European Union, Barraquer-Simons syndrome & Lawrence syndrome each affect 0.1 in 10,000 people & Berardinelli-Seip syndrome affects 0.05 in 10,000 people [13].
Generalised lipodystrophy and partial lipodystrophy

Further information


Trial or other data

Feb 21NICE recommends metreleptin for use in England and Wales as an option for treating the complications of leptin deficiency in lipodystrophy for people who are 2 years and over and have generalised lipodystrophy. Also as an option for treating the complications of leptin deficiency in lipodystrophy for people who are 12 years and over, have partial lipodystrophy, and do not have adequate metabolic control despite having standard treatments (it is only recommended if they have an HbA1c level above 7.5%, or fasting triglycerides above 5.0 mmol/litre, or both) [29].
Dec 13The FDA briefing for its advisory panel states that metreleptin appears to be effective in a subgroup of patients with generalized lipodystrophy (congenital or acquired) with severe insulin resistance resulting in diabetes mellitus and/or severe hypertriglyceridaemia not adequately controlled with other therapies. However, as the pivotal study did not have a control group, it is unclear whether the benefit was due to the drug or by a change of diet or compliance with other medicines. The FDA reviewers raise concerns about the potential of metreleptin to cause T-cell lymphoma and affect the immune system [8].
May 13Bristol-Myers Squibb and AstraZeneca announced the results from a sub-group analysis in patients 12 to <18), and data were analyzed at month 12 of metreleptin treatment, where available (n=27). In adolescents, metreleptin reduced mean HbA1c from 9.8±1.8% at baseline to 7.7±1.7% at month 12 (mean decrease 2.3±0.4%) and triglycerides from 1378±2024 mg/dL at baseline to 385±446 mg/dL at month 12 (mean decrease of 44±14%). Mean ALT decreased from 105±97 U/L to 59±108 U/L ( mean decrease of 46±40%), and mean AST decreased from 87±89 U/L to 57±118 U/L (mean decrease of 31±38%). In younger children, confirmed diabetes and hypertriglyceridaemia were uncommon; however, mean LFTs were markedly elevated at baseline and decreased with metreleptin treatment (mean ALT decreased from 193±202 U/L to 155±274 U/L (mean decrease 38±47%, and AST decreased from 119±112 U/L to 90±144 U/L (mean decrease 30±29%). Reductions in ALT and AST did not reach statistical significance due to the small sample size but were clinically meaningful. The most common AE related to treatment with metreleptin were decreased weight (n=3, 7.7%), hypoglycaemia (n=3, 7.7%), fatigue (n=2, 5.1%), and nausea (n=2, 5.1%) [6].
Aug 12BMS buy out Amylin
Nov 08Amylin is investigating metreleptin in an open label clinical trial (NCT00677313) in 30 patients (aged ≥5) for the treatment of diabetes mellitus and/or hypertriglyceridaemia associated with lipodystrophy. The trial started Nov 2008 and is due to complete Nov 2011 [2]
Apr 06Placebo controlled PIII (NCT00896298) trial of metreleptin in pts with lipodystrophy started. 24 pts aged btn 6 yrs & 70 yrs with partial or generalised lipodystrophy will be recruited in the US. Metreleptin will be injected subcutaneously twice a day, given at 100% of the estimated replacement dose during the first month, which is a dosage of 0.03mg/kg for female children, 0.04mg/kg for adult females, & 0.02 mg/kg for all males, & then at 200% subsequently. The study will complete in Jul 12 [3].
Jan 06Lipodystrophy syndromes are characterized by abnormalities in adipose tissue distribution with loss of subcutaneous fat, and often manifest in childhood or adolescence. It is associated with multiple severe metabolic abnormalities including extreme insulin resistance, very high triglyceride levels, difficult-to-control diabetes and hepatic steatosis resulting in a high risk of acute pancreatitis, accelerated atherosclerosis, diabetes complications and liver cirrhosis. Patients with lipodystrophy typically also have a deficiency of leptin, a hormone secreted by fat cells that plays a key role in regulating metabolism [1].

Evidence based evaluations