Welcome to the International Pompe Association (IPA)
For Immediate Release
July 4, 2016
As the International Pompe Association (IPA) has previously announced, on Thursday, June 9th, Biomarin Pharmaceuticals stated it was planning to out‐license the further development of BMN‐701 during Goldman Sachs 37th 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 22nd 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.
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 email@example.com. 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.
- 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.
Since it was founded in 1999, the International Pompe Association (IPA) has worked directly with the different industry partners who have been active in the field of Pompe disease. Through its Board, the IPA has sought to be a conduit of information between the patient community and industry.
On Thursday, June 9th, one of our industry partners, Biomarin Pharmaceuticals, made the announcement during Goldman Sachs 37th Annual Global Healthcare Conference that it was planning to out-license the further development of BMN-701. BMN-701 (also known as Reveglucosidase alfa) is an investigational enzyme replacement therapy for Pompe disease that is currently in Phase II/III trials.
A recording of this announcement may be found at: http://investors.bmrn.com/events.cfm
The Board of the IPA understands that this news is unexpected to the Pompe community.
If current trial participants have questions regarding this announcement, they should contact their trial center. A list of trial centers can be found at: https://clinicaltrials.gov/ct2/show/NCT01924845?term=bmn+701&rank=3
In addition, the IPA has been in contact with Biomarin and has been communicating the need for an update to the Pompe patient community as soon as possible to explain the short-term and long-term effect of this announcement on Biomarin’s Pompe program, and more importantly the effect on the Pompe patient community. As soon as more information is available, the IPA will post it on the IPA website (http://www.worldpompe.org) and send it to our Members and Affiliates.
The IPA Board