05.11.2012 - The European Commission declined a market authorisation for the orphan drug Elelyso in spite of a positive risk-benefit assessment.
The drug couldn’t be approved due to specific requirements of the European Union (EU) Orphan Drug Regulation. Pfizer Inc. and Israel’s Protalix BioTherapeutics, Inc. developed taliglucerase alfa, as an enzyme replacement therapy (ERT) for the treatment of Gaucher disease. They filed in November 2010 for marketing approval in the EU. In June 2012 the CHMP issued its opinion on the plant cell-expressed recombinant form of human glucocerebrosidase, stating that it could not recommend the drug for approval, because Irish drugmaker Shire’s velaglucerase alfa had received prior Marketing Authorisation with orphan drug designation for the same condition. Therefore, Shire's treatment has orphan market exclusivity in the EU for a ten-year period commencing on its authorisation in August 2010. Pfizer pursued a request for derogation from Shire's orphan market exclusivity based on a number of factors. In detail, Pfizer had been seeking an exemption to orphan exclusivity on the basis of an "insufficient supply" of enzyme replacement therapy for Gaucher's disease and the drug’s expected "major contribution to patient care." But the reasoning of the pharma giant was not enough to convince the CHMP. In USA, Australia, and Japan, Genzyme's orphan drug Cerezyme dominates the Morbus Gaucher market.
"We are disappointed by the EC's decision on taliglucerase alfa and believe it is important, given the history of past shortages, for the Gaucher disease community in the EU to have a third treatment option available," said Diem Nguyen, General Manager, Pfizer Biosimilars. "We will continue to work closely with our partner, Protalix, to make taliglucerase alfa available to the Gaucher disease community in other countries." Taliglucerase alfa was approved by the FDA in May 2012 for the long-term ERT of adults with a confirmed diagnosis of Type 1 Gaucher disease and was approved by Israel's Ministry of Health in September 2012.
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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.