Genetic Therapy and the Industrial Uncertainties
Last week, EMA approved Strimvelis for the treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID). It is the second genetic therapy approved by EMA after Glybera was cleared in 2012. The usual discussion goes around rarity, investment and pricing, which have been set at very high levels with a few unknowns on efficacy and long term safety. But it has to raise a parallel question, from the industrial and economic perspective.