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.

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 9^th, one of our industry partners, Biomarin Pharmaceuticals, made the announcement during Goldman Sachs 37^th 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

My sister, Helen Walker was diagnosed in 1990 with Pompe’s Disease. Little was known about this rare disease in Australia at that time. One morning Helen serendipitously happened to watch a TV interview with Professor John Hopwood on the research his group in Adelaide was doing on Glycogen Storage Diseases. She rang him the next day and had a long conversation. His interest in her situation resulted in a firm professional friendship between them. He informed her of his connections with the scientific work developing therapies at Erasmus University and at Sophia Children’s Hospital in Rotterdam.

John also arranged contact with three other known Pompe patients in Australia: Together they formed the Australian Pompes Association with Helen as President.

Through the Australian Diagnostic Service for Pompes Disease in Adelaide, Helen was able to inform managing physicians of the existence of the Association as new diagnoses were confirmed. Doctors were requested to inform their patients of the existence of the APA. And so the organisation grew in membership.

Helen’s gratitude and respect for John Hopwood and the research team in Adelaide was never forgotten. She owed so much for the success of her endeavours to this dedicated group and as a consequence she left a substantial amount from her estate to the South Australian Health and Medical Research Institute for future research.

I first met Professor Hopwood at Helen’s funeral in November 2012. John flew to Melbourne to speak at her funeral. Earlier this year he rang me to announce his imminent retirement and his wish to launch a memorial benevolent fund in honour of Helen at The Institute. He invited me to attend the launch and to speak. On May 10 this year I attended SAHMRI’s launch of the beneficiary fund called ‘The Walker Society’.

The establishment of the South Australian Health and Medical Research Institute (SAHMRI) began in 2007 when the South Australian Government commissioned the Review of Health and Medical Research in South Australia which recommended the establishment of a dedicated research institute for South Australia.

In response to this recommendation, the South Australian Government committed to establish SAHMRI in 2008, and the Federal Government allocated $200 million to build the research facility. In December 2009, SAHMRI was incorporated as the state’s first independent health and medical research institute.