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On the Horizon

New Medications

Volanesorsen / Waylivra

In February 2019 the European Medicines Agency licenced Waylivra / Volanesorsen in Europe as “an adjunct to diet in adult patients with genetically confirmed familial chylomicronaemia syndrome (FCS) and at high risk for pancreatitis in whom response to diet and triglyceride lowering therapy has been inadequate”.

You can read more about the EMA’s decision here.

To decide whether the medicine will be made available to patients the National Institute of Health and Care Excellence (NICE) will be meeting later this year (2019).


ApoC-11 mimetic peptide*

This is a drug being developed for people with ApoC-11 deficiency, but as a cofactor for LPL there is a question whether it would potentiate LPL in other populations.  Infusion of ApoC-11 potentiates has been given either via fresh frozen plasma or by therapuetic plasma exchange.  It can substantially lower triglycerides to prevent pancreatitis.


DGAT inhibitors*
Diacylglycerol O-acyltranferase (DGAT) mediates triglyceride synthesis.  DGAT is expressed in the gut, liver and adipose and there are two forms:  DGAT1 and DGAT2.


Pradisgastat (DGAT1 inhibitor)*
There has been an open-label, crossover study of six patients with FCS who were given 3 fixed-dose sequences over 21 days.  At the higher doses trigylcerides were lowered by up to 70%.  The medicine is given orally.  Pradisgastat is made by Novartis.  You can read an abstract about it here.


MTP inhibitors*
Microsomal triglyceride transfer protein (MTP) is a protein that assembles the various lipoprotein components, such as lomitapide (Juxtapid, Aegerion), currently approved for patients with homozygous familial hypercholesterolaemia.  Small case reports have shown that it may reduce triglcerides in patients with FCS.  However, side effects include nausea, vomitting and diarrhea.


Gene Therapy

Glybera (Alipogene Tiparvovec)
STOP PRESS:  uniQure, the company that developed Glybera has made the decision not to pursue the renewal of its marketing authorization in Europe when it is scheduled to expire in October 25th 2017. That effectively means that the product will no longer be available commercially after that time. Please see the press release for further details.

Glybera was the first gene therapy to be authorised for use in Europe. (there is a gene therapy used in throat cancer in China). As an innovative medicine for an ultra-rare population the therapy had a difficult pathway to authorisation. You can read about it here.

Glybera was authorised under ‘exceptional circumstances’.  the European Medicines Agency decided ‘that the benefits of the medicine outweigh its risks in patients with severe or multiple pancreatitis attacks, a subgroup of severely affected patients with a high unmet medical need. The data examined indicated a reduction in the number of pancreatitis attacks which could be of benefit to these patients.’

Under the terms of the authorisation, the company was required to provide further study data as well as data from a registry to be set up to monitor outcomes in patients treated with Glybera.



* information taken from a National Lipid Association scientific Session, Philadelphia May 18-21 2017.  You can read the report here.