The IPA Board was provided with the following statement regarding Biomarin's BMN-701 study. If you have any questions or concerns please contact the IPA at info@worldpompe.org. If and when more information is available the IPA will inform you as soon as possible.
BioMarin greatly appreciates the many patients, families, and physicians who have participated in our Pompe program, as well as your ongoing support of the entire patient community. After much deliberation, BioMarin has decided to discontinue the clinical development of the BMN 701 (reveglucosidase alfa) Pompe program. However, BioMarin remains open to external opportunities for the development of this compound. This decision is not based on concerns for patients’ safety or efficacy.
Does my doctor know about this decision?
Yes, your study doctor has been made aware of this decision.
What will happen to patients currently receiving drug in the clinical trials?
BioMarin plans to stop providing investigational drug to study sites within the month of July.
What are patients’ options when the trial is stopped at their study site?
All options regarding present and future treatment should first be discussed with your physician. In contrast to many other rare diseases, there is an approved treatment option for Pompe disease. As with any treatment option, this should be discussed with your physician.
What about compassionate use?
BioMarin’s policy for treatment under compassionate use requires that the drug be under development. Since BioMarin’s plans are to discontinue development of BMN 701, we are not planning a compassionate use program at this time.
Who is the best source for additional information?
Your physician remains the best source of support and information for you and your family. You should contact them with any questions you have regarding treatment options. BioMarin remains in close contact with all investigators and study teams and will provide updates when new information becomes available.
Read more
- Biomarin Pompe Community Update (PDF, 70 kB)
- BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) – ClinicalTrials.gov Identifier: NCT01924845
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.