Myalepta 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 .
In 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 .
Has been available in Germany since Oct 18 .
Aegerion 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 The Final Evaluation Document for this evaluation has been withdrawn.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. .
NICE 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 .
Aegerion is being acquired by Amryt Pharma. Presume this will further delay plans for UK launch .
NICE TA production delayed to enable the company to prepare and submit additional information for consideration. Presume this is also delaying plans for UK launch .
Approved in EU .
EU 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 .
Novelion expects CHMP opinion in Q2 18 .
Company (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 .
Licensing application submitted in EU for metreleptin as a treatment for generalised lipodystrophy and subset of patients with partial lipodystrophy .
Aegerion 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 .
No longer listed in AZ pipeline. Plans for EU filing potentially changed following sale to Aegerion .
EU filing planned for Q4 2015 .
Aegerion Pharmaceuticals announced that it has entered into a definitive agreement with AstraZeneca to acquire Myalept™ .
Q2 Launched in the US .
Approved 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 .
AstraZeneca acquires global rights for development, manufacture and commercialisation of metreleptin .
An 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 .
EU filing planned for 2015 .
EU filing will be 1H 14 .
FDA has granted priority review status for the treatment of metabolic disorders associated with inherited or acquired lipodystrophy .
Orphan 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) .
US 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 .
Initial 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 .
Granted orphan drug and fast track status in US in patients with rare inherited and acquired forms of lipodystrophy .