FDA Rare Disease Listening Session

  • Friday, August 16, 2019

The APF will be participating in a FDA Rare Disease Listening Session on August 26th at 2pm. These sessions are intended to be a resource for the FDA to engage with patients or their advocates. It is an excellent opportunity to share our patient perspective with the FDA to help inform regulatory decision-making and educate review staff about rare diseases.

Our FDA counterpart shared that our meeting request received the most interest of any other. Our objective for this one-hour session is to help the FDA understand the impact of living with porphyria, to differentiate the porphryias and the burden of living with each disease and focus on our need for treatment for each type of porphyria! We are excited for this opportunity and the impact it will have on our community.