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

BusinessUKSwitzerland

29.07.2015 Newly founded Mereo BioPharma has acquired three mid-stage clinical assets from Novartis. In turn, the Swiss pharma concern will hold a stake in the UK company. Other investors are also on board.

M&AIreland

28.07.2015 Irish Allergan is bolstering its product pipeline with a takeover of CNS specialist Naurex. At the same time, the company is divesting its global generics business to Teva.

Public marketGermanyFranceBelgiumFinlandDenmarkUK

24.07.2015 Biotech companies across Europe continue to raise cash on the public markets, generating €3bn in total financing proceedings in the first half of 2015. Compared to 2014, this is almost twice as much.

FinanceFrance

23.07.2015 US money keeps flowing into Europe's biotechs. As the race for the peanut allergy market heats up, NASDAQ listed French biotech DBV has announced the successful closing of US$281m (€256m) share offering for its allergy products.

BioplasticsFrance

21.07.2015 French biotech Deinove has launched a R&D programme to produce the versatile chemical intermediate muconic acid by using a designer variant of the Deinococcus bacteria.

BusinessFrance

16.07.2015 Sanofi has announced plans to restructure its business in five standalone global business units starting in January 2016. The goal is to promote growth and help the company launch a catalogue of new drugs over the next five years.

R&DUK

15.07.2015 GlaxoSmithKline (GSK) has become the first drugmaker to join the Francis Crick Institute. It is a consortium made up of six scientific and academic institutes to conduct research and drug development in the UK. The new institute is set to become a world-leading centre of biomedical research and innovation.

FundingUK

13.07.2015 UK drug developer ReNeuron has pulled in £68.4m (€96m) for its cell therapy pipeline for stroke and blindness treatments. Biotech guru Neil Woodford increases his fund’s stake in ReNeuron.

BusinessSweden

10.07.2015 Karolinska Development has slashed the reported value of its portfolio by almost half by revising its valuation model for early stage companies and divesting its shares in pain med maker Pharmanest.

M&AIreland

08.07.2015 Horizon Pharma plc. has set its sights on US-American speciality pharma company Depomed Inc. After getting nowhere with the rival’s board, Horizon has now launched a US$3bn hostile bid for the pain and CNS specialist.

Events

All Events

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • SERODUS (N)2.60 NOK4.00%
  • SUMMIT (UK)4.03 GBP3.87%
  • FLAMEL TECHNOLOGIES (F)23.88 USD3.83%

FLOP

  • BASILEA (CH)103.50 CHF-13.10%
  • ZELTIA (E)3.69 EUR-8.44%
  • DIAMYD MEDICAL -B- (S)9.20 SEK-6.60%

TOP

  • IXICO (UK)34.00 GBP51.1%
  • DBV TECHNOLOGIES (F)78.26 EUR50.9%
  • ZEALAND PHARMA (DK)149.00 DKK34.8%

FLOP

  • BIOTEST (D)25.40 EUR-63.7%
  • CHRONTECH PHARMA (S)0.01 SEK-50.0%
  • BIOTECH PHARMACON (N)9.74 NOK-43.4%

TOP

  • ADOCIA (F)88.47 EUR565.7%
  • FORMYCON (D)28.89 EUR326.7%
  • DBV TECHNOLOGIES (F)78.26 EUR301.5%

FLOP

  • CHRONTECH PHARMA (S)0.01 SEK-88.9%
  • NEUROVIVE PHARMACEUTICAL AB (S)22.00 SEK-70.3%
  • NEOVACS (F)1.02 EUR-69.1%

No liability assumed, Date: 30.07.2015