PET PET
  • My Account
  • Subscribe
Become a Friend Donate
  • About Us
    • People
    • Press Office
    • Our History
  • Get Involved
    • Become a Friend of PET
    • Volunteer
    • Campaigns
    • Writing Scheme
    • Partnership and Sponsorship
    • Advertise with Us
  • Donate
    • Become a Friend of PET
  • BioNews
    • News
    • Comment
    • Reviews
    • Elsewhere
    • Topics
    • Glossary
    • Newsletters
  • Events
    • Upcoming Events
    • Previous Events
  • Engagement
    • Policy and Projects
      • Resources
    • Education
  • Jobs & Opportunities
  • Contact Us
  • About Us
    • People
    • Press Office
    • Our History
  • Get Involved
    • Become a Friend of PET
    • Volunteer
    • Campaigns
    • Writing Scheme
    • Partnership and Sponsorship
    • Advertise with Us
  • Donate
    • Become a Friend of PET
  • BioNews
    • News
    • Comment
    • Reviews
    • Elsewhere
    • Topics
    • Glossary
    • Newsletters
  • Events
    • Upcoming Events
    • Previous Events
  • Engagement
    • Policy and Projects
      • Resources
    • Education
  • Jobs & Opportunities
  • Contact Us
  • My Account
  • Subscribe
  • Privacy Statement
  • Advertising Policy
  • Thanks and Acknowledgements
PETBioNewsNewsGene therapy nears approval as European regulator makes recommendation

BioNews

Gene therapy nears approval as European regulator makes recommendation

Published 19 March 2013 posted in News and appears in BioNews 667

Author

Dr Rosie Morley

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.

The European Medicines Agency has recommended European Union market approval for a gene therapy, Glybera, to treat patients with severe cases of a rare genetic condition called lipoprotein lipase deficiency...

The
European Medicines Agency (EMA) has recommended European Union market approval for a gene therapy, Glybera, to treat patients with severe cases of a rare genetic condition called
lipoprotein lipase deficiency (LPLD). If the
treatment is approved by the European Commission, which usually follows the
recommendations of the EMA, Glybera will be the first commercially available
gene therapy in Europe and the USA.

'This
is a watershed moment. Gene therapy holds incredible promise for people,
especially those with rare disease. It paves the way for the approval of other
treatments of this kind', said Dr Tim Coté, former director of the US Food and
Drug Administration's office of orphan products development.

Due
to mutations in the LPL gene, patients with LPLD lack the necessary enzyme to break down fat from digested food,
which can cause fat globules to build up in the blood resulting in severe
abdominal pain and pancreatitis. The only way to manage the disease at present
is for patients to control the fat content in their diet. Glybera works by
using a virus vector to introduce a working copy of the gene.

Jörn
Aldag, chief executive at UniQure, the company that is developing the drug,
said: 'Now, for the first time, a treatment exists for these patients that
not only reduces this risk, but also has a multi-year beneficial effect after
just one treatment'.

The
approval of Glybera has not been without problems. LPLD is very rare affecting one or two people per million, so it is difficult to assess the risks and
safety of the drug in the usual way. Glybera originally received a negative
response from the EMA, and the recent recommendation is only for use in
patients with a severe form of the disease.

Gene
therapy is aimed at patients with genetic diseases and essentially replaces
their faulty genes with working ones. The only other commercially available gene
therapy is Gendicine, which was approved for the Chinese market in 2004 to treat
cancer.

Gene
therapy has received bad press in the past after the trial of a drug to treat
children with severe combined immunodeficiency was suspended due to some
participants developing leukaemia. Another setback to the development of gene
therapies was the death of a participant after he reacted adversely to the
injected virus in a gene therapy trial.

Related Articles

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
14 September 2016 • 3 minutes read

Second gene therapy wins approval in Europe

by Rachel Reeves

The European Commission has granted marketing authorisation for a gene therapy to treat children with an extremely rare, life-threatening genetic disorder...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
28 November 2014 • 2 minutes read

World's first commercialised gene therapy set to be 'world's most expensive drug'

by Arit Udoh

Glybera, the first gene therapy to go on sale in Europe, is set to cost €1.1m (£870,000) per patient, making it the world's most expensive drug...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
Comment
30 June 2014 • 3 minutes read

Patient organisations challenge process for accessing orphan and ultra orphan medicines in Wales

by Emma Hughes

In Wales, the subject of access to medicines has recently been in the spotlight. We are calling on the National Assembly to review the use of the 'exceptionality' rule in determining whether a patient can access a treatment through the IPFR process...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
Reviews
3 April 2013 • 4 minutes read

Film Review: The Bourne Legacy

by Dr Greg Ball

The Bourne Legacy sees a return of the Bourne franchise, only this time without its eponymous hero, but rather a whole new breed of biologically enhanced undercover CIA agents...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
5 March 2013 • 2 minutes read

Pair of gene-targeted melanoma drugs show promise in late-stage clinical trials

by Dr Tamara Hirsch

Two drugs targeting advanced melanoma linked to a mutation in the BRAF gene are more effective than current chemotherapy at slowing the progress of the skin cancer, clinical trial results indicate...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
23 November 2012 • 2 minutes read

Gene therapy in mice restores sense of smell

by Holly Rogers

Scientists have restored the sense of smell in mice using a gene therapy designed to repair the cilia - hair-like formations on nasal cells that are important in olfaction...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
26 July 2009 • 2 minutes read

Gene therapy and drug studies show promise for treatment of cystic fibrosis

by Dr Rebecca Robey

Scientists have used two new techniques to fix defects in lung cells from people with cystic fibrosis, raising hope for new treatments for the disease in the future. The first study, published in the journal Plos Biology, used a gene therapy technique to treat the cells, whilst the second study, published in the American Journal of Respiratory Cell and Molecular Biology, used a drug called miglustat....

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
9 June 2009 • 2 minutes read

Cancer patients travel to China for gene therapy

by BioNews

Terminally ill cancer patients from Europe and America are travelling to China to receive a new gene-based drug, the world's first commercially available gene therapy treatment. Chinese biotech firm SiBiono Genetech launched the drug in January, initially for the treatment of head and neck squamous cancers (a type of skin...

Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the output from a DNA sequencing machine.
CC BY 4.0
Image by Peter Artymiuk via the Wellcome Collection. Depicts the shadow of a DNA double helix, on a background that shows the fluorescent banding of the sequencing output from an automated DNA sequencing machine.
News
9 June 2009 • 1 minute read

Gene therapy treatment launched in China

by BioNews

A Chinese biotech firm has been granted permission to market the world's first commercial gene therapy medicine, New Scientist magazine reports. The country's medicines authority has approved Gendicine, a new cancer treatment, after promising results in a clinical trial. It will be launched in January by the firm SiBiono GeneTech...

Leave a Reply Cancel reply

You must be logged in to post a comment.

« US court ruling frees regulator to police stem cell therapies

Data-Label The UK's Leading Supplier Of Medical Labels & Asset Labels

RetiringDentist.co.uk The UK's Leading M&A Company.

Find out how you can advertise here
easyfundraising
amazon

This month in BioNews

  • Popular
  • Recent
13 June 2022 • 2 minutes read

Drop in diversity of blood stem cells leads to old-age health issues

8 August 2022 • 2 minutes read

Placenta and organ formation observed in mouse embryo models

8 August 2022 • 2 minutes read

Complex structures of the human heart bioengineered

8 August 2022 • 1 minute read

Brain tumour gene also linked to childhood cancers

8 August 2022 • 2 minutes read

Lower hormone doses may improve IVF egg quality

8 August 2022 • 2 minutes read

Boosting muscle cell production of gene therapy proteins

Subscribe to BioNews and other PET updates for free.

Subscribe
  • Twitter
  • Facebook
  • Instagram
  • LinkedIn
  • YouTube
  • RSS
Wellcome
Website redevelopment supported by Wellcome.

Website by Impact Media Impact Media

  • Privacy Statement
  • Advertising Policy
  • Thanks and Acknowledgements

© 1992 - 2022 Progress Educational Trust. All rights reserved.

Limited company registered in England and Wales no 07405980 • Registered charity no 1139856

Subscribe to BioNews and other PET updates for free.

Subscribe
PET PET

PET is an independent charity that improves choices for people affected by infertility and genetic conditions.

  • Twitter
  • Facebook
  • Instagram
  • LinkedIn
  • YouTube
  • RSS
Wellcome
Website redevelopment supported by Wellcome.

Navigation

  • About Us
  • Get Involved
  • Donate
  • BioNews
  • Events
  • Engagement
  • Jobs & Opportunities
  • Contact Us

BioNews

  • News
  • Comment
  • Reviews
  • Elsewhere
  • Topics
  • Glossary
  • Newsletters

Other

  • My Account
  • Subscribe

Website by Impact Media Impact Media

  • Privacy Statement
  • Advertising Policy
  • Thanks and Acknowledgements

© 1992 - 2022 Progress Educational Trust. All rights reserved.

Limited company registered in England and Wales no 07405980 • Registered charity no 1139856