28.11.2014 - The first gene therapy for a rare genetic condition — UniQure's Glybera — is on its way to the European market. But there is a hefty price tag: Chiesi, responsible for the marketing of the orphan drug, is asking for a record price of €1.1m per patient.
Only a few hundred patients suffer from lipoprotein lipase (LPL) deficiency in Europe. The metabolic disorder leads to the patient's inability to process fatty acids in the blood, which can lead to pancreatitis. There was no effective treatment for the rare disorder, until Glybera was granted marketing authorisation in the EU two years ago— the first gene therapy to be approved in Europe. The drug, developed by Dutch biotech firm UniQure with the help of their insect cell-based manufacturing process, aims to restore the LPL enzyme activity. However, the market launch of the drug was delayed until longer-term data was available. Italian commercialisation partner Chiesi has now priced the drug at €53.000 per vial, bringing the total cost of one patient's treatment to over €1m.
Currently, the German Federal Joint Committee (G-BA), the highest decision-making body of the joint self-government of physicians, hospitals and health insurance funds in Germany, is assessing the benefits of Glybera. A decision is expected by April 2015 at the latest. The high price asked for the orphan drug will likely be subject to some debate, especially as it will serve as a benchmark for future gene therapies. In a next step, UniQure plans to seek approval for Glybera in the US.
25.11.2014 The European biotech IPO slump has finally come to an end. A brand new industry report shows that compared to previous years, the number of biotech IPOs more than doubled in 2014, with London and Paris exchanges leading the pack.
19.11.2014 It’s not every day that a company is pushed into the top ten of its sector, but that day has come for Actavis. Christmas came early for the new pharma giant after completing a US$66bn (€53bn) takeover battle for the Botox manufacturer Allergan.
18.11.2014 Merck KGaA and US rival Pfizer have signed a immuno-oncology alliance worth billions: Merck will share the development and commercialisation of its cancer antibody with its US partner – for €2.3bn.
14.11.2014 Cosily coinciding with World Diabetes Day on 14th November, French biopharma Les Laboratories Servier has struck a US$1bn deal with US pharma Intarcia Therapeutics for the exclusive rights to Intarcia’s unprecedented therapy for type 2 diabetes.
12.11.2014 Glitz and glamour are not words often used to describe science and mathematics. But once a year, celebrities from the science and showbiz world come together at the Breakthough Prizes, a glitzy television bash that honours scientists for their achievements. French life scientist Emmanuelle Charpentier was among this year’s winners.
04.11.2014 Galecto Biotech has entered an agreement with Bristol-Myers Squibb for the acquisition of the company and its lead drug, providing the Swedish company with the potential of reaching over US$400m.