BioMarin Update for the Duchenne Community: 7th July, 2016

BioMarin is providing a further update to the Duchenne Community following our announcement of the withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on compounds BMN 044, BMN 045 and BMN 053.

Update for Patients Receiving Drisapersen, BMN 044, 045 or 053 in Clinical Trials

As clinical development has been halted, BioMarin is discontinuing dosing in all clinical trials for drisapersen, BMN 044, BMN 045 and BMN 053. The intended target date for receipt of final doses is on or before Friday, 05 August, 2016. Clinical Investigators have been informed and will be contacting patients to schedule final dosing and safety follow-up visits over the coming weeks. Patients currently receiving home dosing must return to the clinic for any/all doses received after July 10, 2016. We are currently working with advocacy organizations to determine if they may be able to facilitate access through a separate compassionate use program.

Have the Trials been Stopped by Health Authorities or Local Ethics Committees?

Ethics committees at some sites have withdrawn their approval for drisapersen studies. Based on the regulatory outcomes for drisapersen, some ethics committees have concluded that the benefits to drisapersen treatment are unclear and do not justify continued approval of the protocol(s) under which patients currently have access to treatment. Drisapersen, BMN 044, BMN 045 and BMN 053 are investigational drugs, and access to them is provided in clinical studies. When ethics committees make such decisions, the investigator must discontinue treatment.

The Trials Are Stopping, What Are the Options? Can We Go to a Different Site?

All options regarding present and future treatment must first be discussed with your physician. As our clinical development efforts have halted, BioMarin is halting dosing in all trials at all sites, so transfer to another site is not possible. When the BioMarin trial is stopped at your site, your doctor must comply with the protocol instructions for continued safety monitoring and the health authority and/or ethics committee requirements. Currently there is no mechanism for continued access outside a clinical study; however, BioMarin is working hard with a patient advocacy organization to see if another mechanism can be identified to continue access. If such a mechanism is possible, it would be under the supervision of a different entity than BioMarin. BioMarin will keep the Duchenne community informed of any important developments regarding continued access.

What about Compassionate Use?

Questions regarding compassionate use and expanded access have been asked by members of the community. Compassionate use programs vary according to country. Generally, compassionate use programs are for drugs that are being actively developed/studied. For experimental drugs that are not being actively developed or studied, we are determining what, if any, options for access exist. We are working with a patient advocacy organization and request your continued patience while BioMarin determines whether other options exist.

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 options. BioMarin remains in close contact with all investigators and will provide further updates to the Duchenne community through patient associations when additional information is available.

Again, BioMarin extends its sincere appreciation to patients, caregivers, families, and advocacy groups, and the enormous contribution you have made to these clinical trials.