At the “Steps Forward in Pompe Disease” 8th European Symposium on November 11-12, 2016 in Amsterdam (NL), organised by Sanofi Genzyme, one session was dedicated to a review of 10 years of enzyme replacement therapy. A company perspective was presented by Henk Schuring, Vice President and Head of Rare Neurological Diseases (Sanofi Genzyme), a doctor’s perspective was given by Ans van der Ploeg (Erasmus MC Rotterdam, The Netherlands), and insights into the patient community perspectives were given in a speech delivered by Tiffany House, President of the IPA. We want to share this speech with the Pompe Community:
Past, Present and Future
2016 has been an interesting year for the Pompe Community. It is the ten-year anniversary of the approval of a treatment for Pompe by the EMA and FDA: a milestone that should be celebrated. The majority of patients around the world now have access to treatment. The IPA recognizes that this treatment is not a cure, and its effectiveness varies for patients. But what we know without a doubt is that it is an important first step in the treatment of Pompe Disease. The natural history of Pompe disease is clear: patients will exhibit progressive muscle weakness. What is also clear after ten years of commercial treatment, and seven years of clinical trials before that, is that enzyme replacement therapy (ERT) slows or halts the disease process for most patients.
The ten-year anniversary of the approval of ERT is a chance to look back at how far we have come, and also to look towards the future and where we want to go. I think to truly understand where we are today, we need to go back even further than the last 10 years. We need to take a moment to remember the days and months and years before treatment was a reality. Before there were promising trials in the works.