FDA appoints Public Private Partnership Liaison to ARC

To focus on the challenges faced by AL amyloidosis, a particularly complex and multi-systemic disease, and bridge the gaps in drug discovery and development .

We are excited to announce that the FDA has approved the appointment of Preston Dunnmon, Medical Officer, Office of New Drugs (OND), Office of Drug Evaluation I (ODEI), Division of Cardiovascular and Renal Products (DCRP) within the Center for Drug Evaluation and Research (CDER), to serve as the CDER Public Private Partnership (PPP) Liaison to ARC.

Under FDA’s Critical Path Initiative, Advancing Regulatory Science Initiative and similar programs, the FDA engages with other government, academic, scientific, patient and industry organizations to foster scientific collaborations in the format of a Public Private Partnerships (PPP). By appointing PPP Liaisons to existing consortia like ARC, these individuals are able to facilitate, inform, and help the external groups understand CDER’s current thinking in a role separate from any regulatory review process. The overarching goal of these partnerships is to leverage expertise and resources from all stakeholders to advance rigorous science to bridge gaps in drug discovery and development, and project results generated by these PPPs are made broadly available to the public to benefit public health.

Dr. Dunnmon’s appointment will allow ARC to bring together the entire AL amyloidosis community – patients, academia, industry, and regulatory – in a series of meetings and discussions at the FDA that will focus on defining AL amyloidosis, its pathophysiology, its incidence and prevalence, and potential treatment pathway(s) for it in the setting of the multiple/simultaneous competing risks of plasma cell reduction therapies. Understanding these basic aspects of the disease will then allow consideration of clinical trial designs and endpoints that may succeed in assessing the potential for new treatments to decrease the mortality, improve the function, and/or improve the quality of life of patients with AL. These meetings are expected to kick off in 2019 and continue into 2020.

“One of the highest priorities that arose out of ARC’s 2018 Research Strategy Roundtable and subsequent white paper, ‘Advancing Amyloidosis: A Research Roadmap,’ was the need to address the challenges faced by AL amyloidosis, a particularly complex and multi-systemic disease, in treatment and clinical trial settings,” says Isabelle Lousada, Chief Executive Officer of ARC. “In the past year we have seen several treatments gain approval for ATTR amyloidosis. However, there still remains a significant unmet need for patients with AL amyloidosis, for whom there are no approved therapies.”

“The efforts of ARC support our shared goal to identify a clear path(s) forward to bringing new, safe, and efficacious therapies to patients with AL amyloidosis,” says Dr. Dunnmon. “I believe that bringing the stakeholders in academia, industry, and regulatory together to tackle the challenges faced by patients with AL amyloidosis will be a good thing.”