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

BusinessGermanyFrance

15.04.2014 German flavour and scent specialist Symrise has announced its plans to acquire French pet-food ingredients maker Diana Group for €1.3bn.

BioeconomySpain

14.04.2014 Spanish Neol Biosolutions and the Center for Second Generation Biofuels are starting a partnership to scale-up Neol's waste-to-oil technology.

EvidenceUKSwitzerland

11.04.2014 There is no evidence that neuraminidase inhibitors Tamiflu and Relenza prevent complications of influenza or reduce hospital admissions. In addition, it is unclear whether they can stop the spread of influenza in case of outbreaks, says a report by the Cochrane Collaboration.

BusinessSwitzerlandSpain

09.04.2014 Roche is extending its involvement in the area of point-of-care diagnostics by taking over US company Iquum. In the field of epigenetics, the Swiss pharma company has started a cooperation with the Spanish company Oryzon.

Status ReportSwitzerland

08.04.2014 The Swiss Biotech Report 2014 presented in Zurich sees an encouraging upturn in the country's biotech scene. However, the acceptance of the mass immigration initiative poses a significant threat.

NanobiotechnologyItaly

07.04.2014 Scientists from the University of Udine have designed, produced and tested a prototype for a "DNA origami" nanorobot that can potentially deliver biological activators in-vivo.

EpidemicEUFranceGermanyItaly

02.04.2014 As the Ebola epidemic in Guinea worsens, Europe is deploying the specialists of EMLab to provide diagnostics at the focus of the outbreak.

BioeconomyFranceEU

01.04.2014 The European Commission has approved a French state package aid worth €43m for green chemistry project IEED.

MonitoringEU

27.03.2014 In Europe, bacterial resistance to prevalent antimicrobials is common, while multi-resistance remains low, EFSA and ECDC now report.

FinancingFrance

25.03.2014 Immunotherapy developer Transgene SA has successfully raised a total of €65.5m via a two-step capital increase – including a rights issue and a private placement.

Events

All Events

Partner-Events

Frankfurt am Main

7th DVFA Life Science Conference

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • THROMBOGENICS (B)22.00 EUR16.5%
  • BAVARIAN NORDIC (D)14.56 EUR10.3%
  • CLAVIS PHARMA (N)2.18 NOK6.9%

FLOP

  • CYTOS (CH)0.15 CHF-94.8%
  • DIAGENIC (N)0.79 NOK-38.3%
  • PAION (D)3.08 EUR-18.5%

TOP

  • WILEX (D)1.04 EUR79.3%
  • CO.DON (D)3.10 EUR74.2%
  • BIOTECH PHARMACON (N)18.20 NOK56.9%

FLOP

  • CYTOS (CH)0.15 CHF-94.8%
  • BIONOR PHARMA (N)2.08 NOK-48.6%
  • DIAGENIC (N)0.79 NOK-45.9%

TOP

  • SILENCE THERAPEUTICS (UK)261.00 GBP6041.2%
  • IXICO (UK)69.50 GBP776.4%
  • PLETHORA (UK)10.75 GBP593.5%

FLOP

  • CYTOS (CH)0.15 CHF-96.5%
  • EVOCUTIS (UK)0.25 GBP-91.9%
  • AGENNIX (D)0.04 EUR-78.9%

No liability assumed, Date: 15.04.2014