On June 20, 2019, Jenny Mosier, Executive Director of Michael Mosier Defeat DIPG Foundation, gave a presentation to the Food and Drug Administration (FDA) regarding development of ONC201 as a potential therapy for DIPG patients. In her remarks, Mosier urged the FDA (1) to include DIPG patients in the development of ONC201, (2) to utilize all tools available to expedite development and approval of ONC201 for DIPG, and (3) while ONC201 continues to be studied, to take steps within the trial or through expanded access to enable as many patients as possible to gain access to the drug.
The public hearing was held by the FDA’s Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee, to gauge investigator interest in exploring potential pediatric development plans for ONC201, including discussion of diseases to be studied, patient populations to be included, and possible study designs in the development of this product for pediatric use.
Oncoceutics, Inc., the company developing ONC201 presented its early findings and plans for development of ONC201. Two current DIPG families gave passionate and moving presentations about their experience taking ONC201. Al Musella of The Musella Foundation also advocated for prompt action to move forward ONC201 development and approval.
Michael Mosier Defeat DIPG Foundation has made grants of over $166,000 in support of development of ONC201 and to help increase availablilty of ONC201 for DIPG patients, including through Oncoceutics’ expanded access program.
You can view written materials available from the meeting on the FDA website. FDA provided a live webcast of the meeting, and they plan to post the archived webcast after the meeting conclusion. It should appear here once available.