HP&M’s Frank Sasinowski and Kurt Karst Highlight Regulatory Innovations in Neurotherapeutics at the ASENT 19th Annual Meeting

On March 15-17, 2017, the American Society for Experimental Neurotherapeutics (ASENT) held its 19th Annual Meeting in Rockville, MD. The meeting brought together clinical investigators, pharmaceutical industry sponsors, officials from drug regulatory agencies, and patient advocacy organizations to address and advance the science of neurotherapeutics.

On Wednesday, March 15th, Hyman Phelps, & McNamara’s (HP&M’s) Frank J. Sasinowski presented on regulatory innovations in neurological disorder therapies, including discussing the recent approvals of Spinraza for SMA and Exondys 51 for DMD. His presentation highlighted issues and opportunities related to:

• Role of patient advocates in the drug approval process;
• Reliance on historically-controlled trials in rare diseases;
• Cumulative distribution as a means to establish clinical meaningfulness; and
• Use of Accelerated Approval in neurological diseases.

Slides from Frank Sasinowski’s presentation are available here.

On Friday, March 17th, HP&M’s Kurt R. Karst presented on repurposing drugs, including providing an overview of the regulatory pathways for repurposed drugs, legislative attempts o address repurposing (e.g., the OPEN Act), and the current environment surrounding orphan drugs and drug repurposing. Late breaking topics discussed included the proposed OPEN Act and the recent Congressional inquiry into potential abuses of the Orphan Drug Act (see our previous post here).  Slides from Kurt Karst’s presentation are available here.