Provention Bio, Inc. announced that the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the U.S. Food and Drug Administration (FDA) voted 10 yes and 7 no on the question, “Does the information provided in the background documents and presentations by the Applicant and FDA show that the benefits of teplizumab outweigh the risks in support of approval to delay clinical type 1 diabetes mellitus?”.
The EMDAC based its recommendation on safety and efficacy data from the pivotal TN-10 Study in which a single 14-day course of teplizumab delayed insulin-dependent, clinical-stage disease by a median of at least two years in presymptomatic patients with Stage 2 type 1 diabetes (T1D) compared to placebo. Acknowledging the significant unmet medical need facing early-stage T1D patients, the Committee Members discussed the strengths and limitations of the clinical data and provided opinions on the proposed indication statement and potential post-marketing studies.
“We want to first and foremost thank everyone throughout the T1D community, the patients, the caregivers and the clinicians for their support today,” said Ashleigh Palmer, co-founder and CEO, Provention Bio. “We especially want to thank T1D clinicians, patients and families for having the courage and conviction to so articulately describe during today’s open public hearing, as well as in the over 180 letters submitted to the EMDAC docket, what it means to live with T1D and how meaningful a delay in onset of two or more years would be. We also want to thank TrialNet, the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), the investigators and clinical teams, and all the patients and their families who participated in the TN-10 study and contributed to teplizumab-related foundational scientific and clinical research over the past two decades. Finally we would like to thank the EMDAC for their thorough discussion and deliberations resulting in today’s favorable vote. We know the T1D community and at-risk patients and their families in particular are waiting urgently for access to clinical advancements to address their significant medical needs. We remain committed to working closely with the FDA to hopefully secure approval of teplizumab and potentially bring the first disease-modifying therapy in T1D to at-risk patients as soon as possible.”
The FDA granted Breakthrough Therapy designation to teplizumab and priority review designation for the Biologics License Application (BLA). The Prescription Drug User Fee Act (PDUFA) action date is July 2, 2021. The FDA will consider today’s vote as it reviews the BLA, although it is not obligated to follow the Committee’s recommendation. The previously disclosed matter surrounding Pharmacokinetic (PK) comparability was not a topic for discussion during the Advisory Committee meeting. The Company reiterates previous guidance that the FDA’s PK comparability considerations are likely to result in a delay in potential BLA approval timelines.