News

Gene therapy back on track
Enlarge image

BusinessNetherlands

Gene therapy back on track

23.07.2012 - A ray of hope for Dutch gene therapy specialist uniQure B.V.: EMA's CHMP
recommended approval of its product Glybera to treat lipoprotein lipase (LPL) deficiency in patients with severe or multiple pancreatitis attacks.

EMA said Glybera (alipogene tiparvovec) is the first gene therapy drug to receive a positive CHMP recommendation. The news is a relief for Amsterdam-based uniQure, after having received three negative opinions before. CHMP said it reversed its prior negative opinions following a draft positive opinion in June from the Committee for Advanced Therapies (CAT),which conducted additional analyses of the data for the adeno-associated virus (AAV) vector encoding LPL gene. CHMP said Glybera should be approved under EMA's exceptional circumstances clause, and that the company be required to establish a patient registry to monitor outcomes. uniQure expects a final decision from the European Commission within three months.

UniQure's predecessor, Amsterdam Molecular Therapeutics Holding N.V., first applied for European approval of the gene therapy in December 2009 for all patients with LPL deficiency. CHMP subsequently issued three negative opinions for Glybera, the most recent of which came in April following arequest by the EC to consider the product in a restricted patient population with severe or multiple pancreatitis attacks. Amsterdam Molecular dissolved into uniQure this February.

LPL disease is a very rare, inherited disease which affects 1-2 people per million. Patients with the disease are unable to handle fat particles in their blood plasma, which leads to recurring severe abdominal pain andpancreatitis. "After today's positive recommendation, Glybera is poised tobecome the first in a class of gene therapy products approved in Europe to treat orphan diseases, rare conditions with a very high unmet medical need", Jörn Aldag, CEO of uniQure, said.

http://www.european-biotechnology-news.com/news/news/2012-03/gene-therapy-back-on-track.html

FinancingDenmark

18.10.2014 After raising US$220.5m (€172m) with its IPO, fast Forward Pharma’s lucky streak hits an obstacle on the road to success with shares falling 17% on its first day of trading.

M&AUKItaly

17.10.2014 One of the biggest pharma mergers of the year has been called off. Abbvie has dropped plans to acquire UK’s Shire following new US tax rules.

FinancingUKBelgium

15.10.2014 Bicycle Therapeutics wins €25m backing by major pharma venture capitalists for the development of a new class of oncology drugs.

FinancingGermanySwitzerlandNetherlands

13.10.2014 Biotechnology IPOs are back in fashion – German Probiodrug AG wants to raise €32m with its IPO, whilst Swiss Molecular Partners AG hopes the launch of its IPO will gain a revenue of €145m.

BusinessIreland

10.10.2014 Actavis has announced plans to merge with American pharma company Durata - a move that will strengthen their infectious diseases portfolio.

RegulationEU

07.10.2014 In an unprecedented move, the European Medicines Agency has approved a system allowing the future analysis of clinical trial data.

FinanceNetherlandsEU

06.10.2014 Another fund from the Netherlands has raised €92m in its first close and will invest the money in companies developing innovative therapeutics, medical devices and diagnostics.

FinanceNetherlandsUKSwitzerland

03.10.2014 Dutch venture capitalist LSP has achieved its monetary milestone by raising €80m for its fifth fund - with a goal of €150m.

PoliticsEULithuaniaPoland

02.10.2014 New EU commission chief Jean-Claude Juncker is on the defensive after members of Parliament and NGOs criticised his reorganisation of the health technology units.

EbolaEUFrance

30.09.2014 In the face of the Ebola outbreak sweeping the West African countries, the EMA has started to review investigational treatments for the deadly virus disease.

Events

All Events

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • MOLOGEN7.65 EUR18.42%
  • BB BIOTECH150.50 EUR5.10%
  • CO.DON2.69 EUR4.26%

FLOP

  • 4SC0.80 EUR-8.05%
  • CYTOS0.16 CHF-5.88%
  • WILEX2.15 EUR-4.44%

TOP

  • BIOFRONTERA2.85 EUR29.5%
  • ADDEX3.30 CHF6.5%
  • BB BIOTECH150.50 EUR2.6%

FLOP

  • CYTOS0.16 CHF-33.3%
  • 4SC0.80 EUR-31.6%
  • PAION2.20 EUR-27.9%

TOP

  • SANTHERA81.90 CHF2072.4%
  • CO.DON2.69 EUR174.5%
  • PAION2.20 EUR144.4%

FLOP

  • CYTOS0.16 CHF-95.9%
  • 4SC0.80 EUR-54.8%
  • THERAMETRICS0.07 CHF-50.0%

No liability assumed, Date: 20.10.2014