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.
04.05.2016 Barcelona-based epigenetics expert Oryzon Genomics has closed a debt funding round of €10.5m, bringing the total money raised since last year to €27m. The funds will serve to advance the company’s two LSD1 inhibitors in cancer and neurodegeneration.
03.05.2016 Ipsen is strengthening its ties to long-time development partner Oncodesign, a Dijon-based cancer treatment biotech. The French pharma is handing over the pre-clinical pharmacology for its oncology research programmes to Oncodesign.
29.04.2016 Swiss bank Group UBS has raised US$471m (€412m) for the UBS Oncology Impact Fund, which was set up to invest in early-stage cancer treatments. The money raised by the fund, nearly half of which comes from investors in Asia, is the largest amount ever raised for such a cancer investment fund.
27.04.2016 More revenue, more jobs, more financing, more R&D expenditure – all signs point towards sustainable growth in the German biotech sector. These are the results of the most recent company survey 2016, published by biotechnologie.de. The report was once again conducted alongside the biotech standards defined by the organisation for economic cooperation and development (OECD).
21.04.2016 An expert panel’s final report on the fatal drug trial earlier this year in France states that the death of one of the participants was most likely caused by the drug’s toxicity and not by any violation of the rules.
21.04.2016 For up to US$685m (€606m), Dutch ArgenX has outlicensed its human antibody programme ARGX-115 to AbbVie. The pre-clinical immuno-oncology candidate targets a protein believed to contribute to immunosuppressive effects of T-cells.
19.04.2016 British drug developer Heptares Therapeutics and mAb maker Kymab Ltd have partnered up in yet another immuno-oncology collaboration. The companies plan to discover antibody meds targeting a superfamily of receptors.
15.04.2016 Not every company is put off by the current stock market climate. Swiss biopharma company GeNeuro has gone public at Euronext Paris, raising €33m in the process. Others, meanwhile, have to find different ways to raise money.
13.04.2016 Despite political disturbances, 2015 was a good year for the biotech scene in Switzerland. Net sales increased by 5.1% to CHF5,133m and 400 additional people found jobs in the sector. As usual, the new numbers were presented at the annual Swiss Biotech Day – which set new records as well.