BioMarin Update for the Duchenne Community: 17 June 2016

BioMarin is providing a further update to the Duchenne Community in light of recent announcements of withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on products BMN 044, BMN 045 and BMN 053.

Is there any Update for Patients Receiving Drug in Clinical Trials?

BioMarin is exploring options for those patients currently on treatment who are interested in continuing access to the limited remaining supplies of drisapersen, BMN 044, BMN 045 and BMN 053. This is, however, a complicated process because drisapersen did not meet the standards for approval by the U.S. and European regulatory authorities. As investigational therapies without a regulatory path forward, this creates a unique situation. BioMarin must follow the rules, regulations and policies of individual countries, health authorities and individual sites. We will keep the community updated as soon as additional details are available.

Can the Trials be Stopped by Health Authorities or Local Ethics Committees?

Yes. Drisapersen, BMN 044, 045 and 053 remain investigational drugs and all access to them is provided exclusively in clinical studies. All clinical studies require approval by national health authorities and by local ethics committees. Both have mandates for the protection of patient welfare. Health authority and ethics committee reactions to drisapersen’s failure to meet the regulatory standard(s) for approval can and do differ. Patients may only continue to receive drug if these approvals are in place. If either health authority or ethics committee revokes their approval of the current trial(s), then the investigator must discontinue treatment.

If the Trial is Stopped at My Site, What Are the Options?

All options regarding present and future treatment should first be discussed with your physician. If a trial is stopped at your site, your doctor must comply with the health authority and/or ethics committee. Continued access outside a clinical study is currently not possible, however, BioMarin is working hard to see what can be done.

What About Compassionate Use?

Questions regarding compassionate use and expanded access have been asked by members of the community. Most compassionate use programs require that the drugs are being actively developed/studied. Therefore, this is a somewhat unique situation since the decision has been made to discontinue development. We request your continued patience while potential options are explored.

Continued Communication with your Physician

Your physician/investigator and study team remain the best source of support and information for you and your family. You should continue to contact them with any questions regarding treatment and treatment options. As before, BioMarin remains in close contact with all investigators and study teams and will provide updates when new information becomes available. We will also provide further updates to the patient community through patient associations and advocacy groups as soon as additional specific information is available.

Again, BioMarin extends its sincere appreciation to patients, families, investigators, and advocacy groups who have been part of this clinical trial process.