biotech

At €1 million a pop, expectations are high. 
But European regulators have given the initial thumbs up to Glybera (alipogene tiparvovec), the western world’s first gene therapy, paving the way for approval by the European Commission.

The treatment is for a rare type of pancreatic disorder in which patients have damaged copies of a gene which is essential for breaking down the fat content in foods.  Affecting one person in a million, the disease causes a build up of lipids in the blood stream, leading to abdominal pain and potentially fatal inflammation of the pancreas.

The decision marks an important advance for gene therapy, a field which has been hotly debated for many years due to shortcomings of previous investigational therapies.  Approval for Glybera, which was previously rejected on three occasions, will make it the first gene therapy to be available outside a clinical trial in Europe or the US.  China approved a gene therapy for head and neck cancer in 2004. Read full post »

With the 24-hour news cycle, new and social
media at our fingertips, and even a presidential initiative on “transparency and open government” — federal agencies must adapt to this change in paradigm. The Food and Drug Administration (FDA) along with the Centers for Medicare & Medicaid Services (CMS) recently held a summit in Washington, DC, where its top policy makers engaged biotech and pharmaceutical executives in an open dialogue on driving pre- and post-regulatory approval and commercialization strategies for products coming to market. Read full post »