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.
20.08.2014 Biopharmaceutical contract manufacturer Fujifilm Diosynth Biotechnologies is expanding its cell culture manufacturing capacity further with a new 2,000l single-use bioreactor at the company’s site in Billingham, UK.
08.08.2014 Boehringer Ingelheim is walking away from Swedish Orexo's prostaglandin inhibition project OX-MPI. Germany's largest researching pharmaceutical company had been responsible for the project's research and development since 2005.