We wanted to take a moment today and let you know where things stand from PPMD’s perspective since the drisapersen Advisory Committee Meeting on November 24 (click here for a full summary or that meeting or download presentations made throughout the day). As you are aware, no decision has been made to date. Based on the requirements by the Prescription Drug User Fee Act (PDUFA), the FDA is required to tell BioMarin something on or prior to December 27. Of course, we will let you know the minute we hear from either the company or the agency and we will do our best to help interpret the potential implications of whatever decision is made.
Community-wide efforts supporting drisapersen have not slowed down since the Ad Comm last month. PPMD has been in regular contact with leadership at BioMarin, providing benefit/risk data as requested to help strengthen their ongoing discussions with the agency. We have also spoken to leadership at the FDA, sharing our experience about the Advisory Committee Meeting process and helping analyze what worked and what needs improvement before future Duchenne-related meetings anticipated in the New Year (including eteplirsen potentially taking place on January 22, 2016).
Perhaps most importantly, we have worked to connect key opinion leaders (KOLs) in the Duchenne community with both BioMarin and the FDA. PPMD felt that their perspective – members of the Duchenne clinical community who had worked directly with families throughout the drisapersen trial – was underrepresented at the Ad Comm. KOLs arguably have the most experience with therapies like drisapersen on a day-to-day basis. They, along with families and patients, make the decision to prescribe these therapies. Therefore, it is critical that clinicians also weigh–in on the discussion. Several KOLs (including a few clinicians) have stepped forward and – like so many of you who participated in the Open Public Hearing portion of the Ad Comm including PPMD’s Pat Furlong – will speak to the importance of options.
Other foundations are working with BioMarin as well, taking a variety of approaches to help ensure that the decision the FDA ultimately makes is one that is educated and reflects this community’s experience with drisapersen. We appreciate the efforts by families and patients who are raising awareness around their experience with drisapersen. Regardless of the various strategies we may take, everyone in this community wants options. Everyone wants to end Duchenne.
We’ve waited for years, decades for an approval in Duchenne. And though two weeks should just be a drop in the bucket for us, it somehow seems longer. Next week we will have information that will alter the course of Duchenne drug development and the clinical trial process in rare disease. PPMD will continue to keep you updated and informed, and once a decision is made we promise to share with you next steps.
For more information about the FDA Ad Comms, please visit the links and resources that we at PPMD have compiled over the past few months with the assistance of our community members, regulatory advisors, and federal agency partners: