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On the Horizon

New Medications

ApoC-111 inhibitors

Volanesorsen
Currently in clinical trials due to be reported in the spring of 2017,  Volanesorsen is an innovative antisense drug,  An antisense drug essentially works with the proteins which activate genes, either enhancing or reducing their production.

Volanesorsen is designed to reduce the production of ApoC-III, a protein produced in the liver that plays a central role in the regulation of plasma triglycerides.

Volanesorsen has now reported on its Phase 3 study in patients with FCS (APPROACH).  Read the press release here

You can read more about the European Medicines Agency authorisation process here.

The company developing the drug are called Akcea Therapeutics.  The link to their site is here.

 

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 of this year. 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 will be 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. There will also be in place a restricted access programme to ensure that Glybera is used appropriately.’

For further information on Glybera please see the following:

Summary from the European Medicines Agency
European Medicines Agency

Summary from Uniqure,  the company who have developed the gene therapy
uniQure

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