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