On the Horizon

New Medications

Glybera (Alipogene Tiparvovec)
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

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

Valorensorsen 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.

Valorensorsen 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.