29.05.2012 - A positive recommendation by EMA's CHMP committee raises hope for patients with cystic fibrosis in Europe.
London – The EU agency recommended Kalydeco (ivacaftor) from the US-biotech Vertex for approval. The treatment targets patients from the age of six years on who have a G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene.
Kalydeco was already approved by the FDA at the end of January this year. According to the European agency, Kalydeco offers an innovative therapeutic approach for patients with cystic fibrosis: it is the first treatment that targets the underlying mechanism of the disease, by restoring the function of the mutated CFTR protein.
Currently available therapies for patients with cystic fibrosis only address the consequences of the disease, not the underlying defect. Clinical studies showed that Kalydeco improved pulmonary function in cystic fibrosis patients with the specific G551D-CFTR mutation. About half of the world's CF patients reside in Europe, where about 35,000 people have the disease. The European Commission will consider the CHMP's recommendation in its final decision on Kalydeco. According to PM live, the European recommendation comes almost four months after Kalydeco was cleared by the US Food and Drug Administration (FDA), with the US agency taking just three months to review Vertex' marketing application. This backs outcomes from a recent report that described how the FDA had a faster approval process than the EMA.
Update: After releasing this news, Vertex corrected trial data of Kalydeco.
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.