Despite having a favorable vote in a recent FDA AdComm meeting, Provention Bio announced the FDA has issued a CRL for teplizumab for the delay of type 1 diabetes in at-risk individuals.
In a move that will come as a disappointment to many, Provention Bio Inc. has announced the receipt of a Complete Response Letter (CRL) detailing the US Food and Drug Administration's (FDA) reasons for opting against approval of teplizumab for the delay of type 1 diabetes.
Announced in a statement on July 6, Provention Bio reports receipt of the CRL on the evening of July 2 and pointed out the FDA felt a single, low-dose pharmacokinetic bridging study did not demonstrate pharmacokinetic comparability, stating “As PK remains the primary endpoint for demonstration of comparability between the two products, you will need to establish PK comparability appropriately between the intended commercial product and the clinical trial product or provide other data that adequately justify why PK comparability is not necessary”.
"We want to recognize the patients, their families, study investigators, clinicians and T1D champions that have played such a crucial role in the development of teplizumab and thank our partners and our team of dedicated employees and consultants for their outstanding contributions. We also want to acknowledge the efforts of Drs. Yanoff and Unger and the review team at the FDA, who have worked so closely and transparently with us throughout the priority review of our BLA for this Breakthrough Therapy drug," said Ashleigh Palmer, co-founder and CEO of Provention Bio.
While the move may come as a surprise to some after the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted 10-7 to support the benefit-risks of teplizumab for delaying clinical type 1 diabetes in May, Provention Bio remains optimistic as they hope to contribute additional pharmacokinetic data from a substudy in patients receiving 12 days of therapy in the ongoing phase 3 PROTECT trial later in Q3 2021. Provention Bio also pointed out the FDA’s CRL cited several additional considerations related to product quality and deficient during a recent inspection at the manufacturing facility used by the company that will need to be resolved before approval.
However, the CRL from the FDA did not cite any clinical deficiencies related to the efficacy and safety data packages submitted in the BLA and also confirmed the acceptability of the proposed proprietary name for teplizumab.
“We know the T1D community is urgently awaiting therapeutic advancements to address their medical needs and believe our collective passion and commitment will continue to drive us forward to meet this goal. We will continue to work collaboratively with the FDA to hopefully secure approval of teplizumab and bring the first disease-modifying therapy for T1D to at-risk patients as soon as possible,” added Palmer.
This is a breaking news story and will be updated when more information becomes available.