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

M&AUKSweden

24.08.2016 Pharma major Pfizer is putting the cherry on top of its multi-billion-dollar takeover of Medivation with the acquisition of AstraZeneca’s antibiotics for a cool US$1.5bn. With the divestment, AZ aims to further sharpen its focus.

FinancingUK

23.08.2016 Cambridge University-allied investor Cambridge Innovation Capital has raised £75m (€87m) to reinvest in technology and healthcare companies. Among the backers were Woodford Investment and Winton Ventures.

R&DDenmark

19.08.2016 Together with partner Johnson & Johnson, Danish biotech Genmab is now one step closer to FDA approval with its combination treatment for multiple myeloma after its submission of a supplemental Biologics License Application. In July, Genmab was granted a Breakthrough Therapy Designation (BTD) for its blood cancer therapy.

ResearchUKEU

17.08.2016 Philip Hammond, the UK’S chancellor has announced the government will guarantee funding for EU-backed "structural and investment fund" projects whose timelines extend beyond the UK’s departure from the EU, provided the projects are signed prior to this year's Autumn Statement on the UK budget. The decision ensures continued funding for farmers, companies and institutions involved in the EU’s Horizon 2020 project. The move could cost the UK government up to £6bn (€6.9bn).

Stock marketsGermany

15.08.2016 Back in April, the American FDA gave the green light for the colorectal cancer-screening test Epi procolon. Now Berlin-based diagnostics specialist Epigenomics is considering a secondary listing in the US. A draft of the required registration document was confidentially submitted to the US Securities and Exchange Commission (SEC).

LicensingDenmark

11.08.2016 Danish Genmab AS has granted Gilead Sciences license to use its DuoBody technology platform to develop bispecific antibodies for the treatment of HIV. The deal is worth up to €252m.

Clinical developmentHungaryIreland

09.08.2016 Hungarian Gedeon Richter and Irish Allergan are staying on course in the face of dissappointing Phase III results for depression drug cariprazine. The partners will now begin another Phase III study needed for approval.

ImmunotherapySpain

08.08.2016 Araclon Biotech’s Alzheimer’s vaccine ABvac40 is going forward into Phase II after showing a good safety profile in Phase I. The promising trial results were presented at the Alzheimer’s Association International Conference.

ApprovalEU

04.08.2016 After a two-year pilot period, the EMA has published its report on its adaptive pathways approach. The agency plans to “explore the concept further” and will continue to accept proposals.

BusinessPolandUK

01.08.2016 Life science venture capital group Epidarex has launched Nodthera Ltd, a biotech based on research by Poland-based drug discovery company Selvita. The start-up aims to develop novel inhibitors of the NLRP3 inflammasome.

Quick news provided by GlobeNewswire

View more stock news

Events

All Events

Partner-Events

Current issue

All issues

Product of the week

Products

Stock list

All quotes

TOP

  • NEWRON (CH)22.65 CHF9.69%
  • VERNALIS (UK)45.00 GBP4.65%
  • KARO BIO (S)40.00 SEK4.44%

FLOP

  • PROTHENA PLC (IE)50.02 USD-7.70%
  • FLAMEL TECHNOLOGIES (F)13.85 USD-6.98%
  • BIONOR PHARMA (N)0.44 NOK-6.38%

TOP

  • DIAMYD MEDICAL -B- (S)7.25 SEK68.6%
  • KARO BIO (S)40.00 SEK28.6%
  • MOLMED (I)0.42 EUR27.3%

FLOP

  • BIONOR PHARMA (N)0.44 NOK-27.9%
  • NOVOZYMES (DK)284.40 DKK-14.3%
  • RENEURON (UK)2.75 GBP-12.4%

TOP

  • NICOX (F)10.26 EUR442.9%
  • SAREUM HOLDINGS (UK)0.74 GBP252.4%
  • GENMAB (DK)1126.00 DKK100.9%

FLOP

  • BB BIOTECH (D)46.74 EUR-81.5%
  • EVOCUTIS (UK)0.04 GBP-80.0%
  • CIRCASSIA LIMITED (L)94.70 GBP-70.9%

No liability assumed, Date: 25.08.2016