News

First gene therapy in Europe
Enlarge image

MedicineNetherlandsEU

First gene therapy in Europe

02.11.2012 - Amsterdam-based uniQure biotherapies B.V. has received the first market authorisation for a gene therapy in the western world.

Today, the European Commission confirmed the EMA’s recommendation for market authorisation for the gene therapy Glybera (alipogene tiparvovec), a treatment for patients with lipoprotein lipase deficiency (LPLD) suffering from recurring acute pancreatitis. The rare, inherited disease affects about 350-700 patients in Europe. Patients are unable to metabolise fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis), a potentially lethal condition. Up to now, no gene therapy has been approved in the EU or the US. Glybera consists of an Adeno-associated Virus (AAV) vector expressing the faulty LPL enzyme.

“This therapy will have a dramatic impact on the lives of these patients. Currently their only recourse is to severely restrict the amount of fat they consume”, commented Professor John Kastelein from Academic Medical Center of the University of Amsterdam. “By helping to normalise the metabolism of fat, Glybera prevents inflammation of the pancreas, thereby averting the associated pain and suffering and, if administered early enough, the associated co-morbidities.”

Uniqure’s CEO Jörn Aldag stressed that Glybera is a proof-of-concept for the company’s gene therapy platform: “The final approval of Glybera from the EC marks a major step forward in making gene therapies available not only for LPLD but also for a large number of rare diseases with a very high unmet medical need.” Experts estimate the uniQure’s virus-based gene substitution therapy Glybera could spend €1m annually per patient. “Prices for orphan drugs like Provenge, Cerezyme or Naglazyme range from a 100,000-450,000 annually,” Aldag told EuroBiotechNews. “Since our therapy has already proven efficacy over several years after just one treatment, I think it's reasonable to start at the high end of this range.

In a next step, uniQure will go to special hospitals in Europe where patients with acute pancreatitis are treated and provide the doctors with its companion diagnostics chip. The firm, that took over the IP from Amsterdam Molecular Therapeutics (AMT), aims at identifying patients in about 20 hospitals throughout the EU.

© eurobiotechnews.eu/tg

http://www.european-biotechnology-news.com/news/news/2012-04/first-gene-therapy-in-europe-approved.html

BusinessUK

03.03.2015 After weeks of speculation, AstraZeneca has announced its plans to spin out its early-stage antibiotic R&D. The decision marks the company’s aims to “sharpen its focus on three main therapy areas”.

GenericsEUUKHungaryGermanyItalyGreeceAustriaLuxembourgFranceSpain

26.02.2015 It is the first biosimilar monoclonal antibody in many European markets: Mundipharma and Hospira are launching Celltrion’s Remicade generic.

M&AUK

23.02.2015 British drug developer Heptares has been acquired by Japanese biopharma play Sosei for US$400m (€352m).

bioethanolGermanyUKBelgiumFrance

19.02.2015 The price of oil has been sliding for months, and the European bioethanol market is taking it hard. CropEnergies AG has now stopped production at its British unit Ensus UK.

BusinessFranceGermany

17.02.2015 After Christopher Viehbacher’s unceremonious ousting last year, Sanofi has been on the look out for a new CEO. Now, sources say Bayer’s Olivier Brandicourt is set to fill the position.

BusinessUKSwitzerland

12.02.2015 Glaxosmithkline is intent on bolstering its vaccine portfolio further and has bought all shares of Swiss development partner Glycovaxyn.

InvestmentsUKEU

10.02.2015 Interest in biotechnology is on the rise among British investors. Not just one, but two major financiers are planning investment funds in the field.

IVFUKEU

04.02.2015 In a historic move, the UK is set to become the first country to allow the creation of babies with the genetic material of three people – a mother, a father and a female mitochondrial donor.

Events

All Events

Partner-Events

Planegg/Martinsried

1st Munich Biotech Pitch

Berlin (DE)

Proteomic Forum 2015

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • MEDIGENE4.64 EUR7.16%
  • FORMYCON16.89 EUR4.07%
  • BIOFRONTERA1.99 EUR3.65%

FLOP

  • HBM102.00 CHF-2.86%
  • SYNGENTA327.00 CHF-2.15%
  • STRATEC BIOMEDICAL45.12 EUR-2.10%

TOP

  • WILEX3.17 EUR47.4%
  • EVOLVA1.70 CHF18.9%
  • MEDIGENE4.64 EUR16.0%

FLOP

  • MOLOGEN5.25 EUR-12.5%
  • SANTHERA98.45 CHF-10.2%
  • 4SC0.77 EUR-9.4%

TOP

  • SANTHERA98.45 CHF2417.9%
  • WILEX3.17 EUR395.3%
  • FORMYCON16.89 EUR119.4%

FLOP

  • CYTOS0.37 CHF-86.9%
  • MOLOGEN5.25 EUR-56.0%
  • PAION2.10 EUR-52.5%

No liability assumed, Date: 03.03.2015