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.

Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease

In a press release, Sanofi Genzyme announced today "that the first patient has been enrolled and received an infusion in a pivotal Phase 3 clinical trial named COMET for the investigational therapy neoGAA. NeoGAA is a second-generation enzyme replacement therapy being studied for the treatment of Pompe disease... COMET is a Phase 3 randomized, multi-center, multi-national, double-blinded study to compare the efficacy and safety of repeated bi-weekly infusions of neoGAA and alglucosidase alfa in treatment-naïve patients with late-onset Pompe disease... Approximately 96 patients, ages 3 and up, are expected to be enrolled in the study, which will last up to 3 years, including a 49-week blinded treatment period and a 96-week open-label treatment period."

A detailed description of the COMET clinical trial can be found on ClinicalTrials.gov (Identifier: NCT02782741): Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease (COMET).

Read more

• Press release (external link)
• Press release (PDF, 135 kB)

Update

• The first patient in the UK has been enrolled and dosed at the University of Newcastle upon Tyne:
  November 17, 2016 - press release (external link)
  November 17, 2016 - press release (PDF, 455 kB)

Disclaimer: The IPA does not endorse any of the products, medications, treatments or information reported herein. Articles on the IPA web pages are intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.

For Immediate Release

July 4, 2016

As the International Pompe Association (IPA) has previously announced, on Thursday, June 9^th, Biomarin Pharmaceuticals stated it was planning to out‐license the further development of BMN‐701 during Goldman Sachs 37^th Annual Global Healthcare Conference. BMN‐701 (also known as Reveglucosidase alfa) is an investigational enzyme replacement therapy for Pompe disease that is currently in Phase II/III trials. The IPA was disappointed to learn of this development, but remained hopeful for the continuation of the program.

Unfortunately, this announcement was followed by a Community Update from Biomarin on Wednesday, June 22^nd that announced that Biomarin was discontinuing “the clinical development of the BMN 701 (reveglucosidase alfa) Pompe program.” (http://worldpompe.org/index.php/news/591‐biomarin‐update‐for‐the‐pompe‐community). Biomarin has stated: “This decision is not based on concerns for patients’ safety or efficacy.”

The IPA Board appreciates that many factors must have gone into Biomarin’s decision to discontinue its Pompe program. Clinical development of a treatment is difficult in the best of circumstances; it is even more difficult in the rare disease field. The IPA believes that the Pompe community is fortunate to have as much interest in developing new therapies and treatments as we do. Many rare diseases do not have any treatments on the horizon.

However, the IPA also believes that the development of a treatment is, and should be, a community effort. It takes close collaboration between the scientific/medical community and the patient community to understand the disease in question, and how to treat it. Then, when the time is right, it takes industry to move potential treatments from the scientist’s laboratory to the patient in the form of clinical trials and market approvals. However, successful clinical development of a therapy is impossible without the contribution and collaboration of all three parties in our experience.

The Board of the IPA is disappointed in Biomarin’s decision to discontinue its Pompe program. We are disappointed that a potential new therapeutic option will not be pursued, but more than that we are disappointed with the way the global patient community and the patients in the trial were informed about this development.