Regulatory evaluation of Glybera in Europe — two committees, one mission

Regulation: Sell help not hope

“…In the United States and Europe, regulatory structures actively support therapeutic development, even for rare or orphan diseases. The gene-therapy product Glybera (alipogene tiparvovec), approved by the European Medicines Agency in 2012 for a condition that can cause life-threatening pancreatitis, is an example of establishing efficacy for a complex, innovative product with a small, but rigorous trial 9 . However, many companies are not equipped, scientifically or technologically, to develop therapies from promising biological advances in the five or so years that their investors demand.…”

Innovative medicines: new regulatory procedures for the third millennium

“…The drug was approved for the treatment of a clinically heterogeneous condition with a risk of life-threatening pancreatitis on the basis of data from 27 patients only with results referring to a primary end point different from that originally set, and following some divergent opinions of different EMA bodies [5].…”

Clinical Development of Gene Therapy Needs a Tailored Approach: A Regulatory Perspective from the European Union

“…In a complex multisystem disorder, which is a rare disease, an alternative approach that is different from a traditional approach based a single or composite clinical end point(s) could be one based on the totality of evidence (see also Melchiorri et al, 2013). This could include expression of the therapeutic gene (the transgene) and the levels of resulting protein, in addition to applicable traditional clinically relevant end points for the target indication.…”

“…The regulatory process for this product has been commented on extensively, highlighting the challenges posed by such a novel technology (English, 2011;Flemming, 2012;Miller, 2012;Melchiorri et al, 2013).…”