BioMarin Update for the Duchenne Community: 01 June 2016

We are sharing the following update from BioMarin.

We would like to update the patient community in light of the recent announcement regarding the EMA marketing application.

BioMarin is discontinuing clinical and regulatory development of drisapersen as well as the other three follow-on products: BMN 044, BMN 045 and BMN 053

Why have we taken this decision?

• EMA is the regulator which decides whether a drug may be prescribed in Europe, similar to the FDA in the United States.
• The role of the Committee for Medicinal Products for Human Use (CHMP) as part of the EMA is to assess the benefits of new therapies relative to the risks associated with taking them.
• Our discussion with the CHMP clearly indicated that they intended to issue a negative opinion, meaning that we would not receive a Marketing Authorisation or licence.
• We are extremely disappointed that drisapersen did not meet the regulatory standards in the US or Europe.
• We believe that we would encounter the same issues with BMN 044, 045 and 053 and for this reason we are also discontinuing these programs.

What will be the Impact on Patients Receiving Drug in Clinical Trials?

As we wind down the current Duchenne clinical development programs, BioMarin plans to work with doctors, patient groups, and regulatory authorities to develop a path forward for those patients currently being treated with drisapersen, BMN 044, BMN 045 and BMN 053.

While we develop the transition plan, clinical trial sites may continue to access drug from BioMarin. The decision on whether to continue to treat will be made by the investigator along with the patient/family, and also requires agreement by local ethics committees/IRBs and national competent authorities. When the transition plan is completed, we will be able to provide more information. Our intent is to make drug available while a plan is finalized.

What About Second-Generation BioMarin Products in Development for Duchenne?

Second generation anti-sense oligonucleotides are in the early research stage, a process which can take years to yield potential candidates to be considered for clinical trials.

Next Steps

Your physician/investigator and study team remain the best source of support and information for you and your family. You should contact them if you have any questions or concerns. 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, while deeply disappointed in the outcome, BioMarin extends its sincere appreciation for the patients, families, investigators, and advocacy groups who contributed so much to the advancement of DMD research in efforts toward developing treatments for Duchenne.