CDKL5 Patient-Focused Drug Development Meeting (Full Program)

IFCR has partnered with LouLou Foundation to sponsor a CDKL5 Deficiency Disorder Externally-Led Patient-Focused Drug Development Meeting (PFDD) with the FDA.

The purpose of this meeting is for individuals affected by CDKL5 Deficiency and their caregivers to communicate their perspectives on living with CDKL5 Deficiency to drug development stakeholders and the FDA. This feedback will help them with drug development an appropriate clinical trial design for future CDKL5 trials.

Meeting Agenda

1:30 - 1:40pm – Welcome and overview
Karen Utley, President IFCR

1:40 - 1:50pm – Opening remarks
Dr Michelle Campbell, Division of Neurology Products, Office of New Drugs, CDER, FDA

1:50 - 2:05pm – Background on CDKL5 deficiency Disorder
Dr Tim Benke, Children’s Hospital Colorado
Dr Orrin Devinsky, NYU Langone Comprehensive Epilepsy Center

2:05 - 2:15pm – Goals and objectives for the meeting, overview of discussion format
James Valentine, MHS, Moderator

2:15 - 3:25pm – Session 1: CDD patient voice: Symptoms and daily impacts
• Panel: Kathy DeSimone, Marissa Bishop, Jenny Feinman, Diana Dinescu, Rick Upp
• Live Polling
• Moderated discussion among all meeting participants

3:25 - 3:35pm Break

3:35 - 4:45pm – Session 2: CDD patient voice: Current and future approaches to treatments
• Panel: Amanda Jaksha, Ed Fennell, Rita Fredericks, Kristin Kozera, Martha Rodgers Boyles
• Live Polling
• Moderated discussion among all meeting participants

4:45 - 4:55 pm – Closing remarks
Larry Bauer, Hyman, Phelps & McNamara

4:55 - 5:00 pm – Next steps
Majid Jafar, Founder Loulou Foundation