- Provention Bio disclosed Thursday that the Food and Drug Administration found "deficiencies" in the approval application for the company's Type 1 diabetes therapy. While not yet a rejection, the agency's feedback suggests an approval decision won't be reached by the original deadline of early July.
- According to Provention, the FDA has yet to provide many details about what's wrong with the application. But the company did note how, during a recent discussion, the agency raised issues with a study evaluating the drug product used to make Provention's therapy, called teplizumab. As such, regulators told the company additional data would be needed to resolve its concerns.
- While Provention executives believe they can clear up these problems, investors appear skeptical. The company's share price opened down 34% on Friday before rising to trade down 20% at mid-morning.
Provention is the latest drugmaker to be surprised by apparent shortfalls in its approval submission. Last month, Acadia Pharmaceuticals said that after being told twice there were no potential issues with its application, the FDA suddenly changed course and found "deficiencies" just weeks before the approval decision deadline.
"We were extremely surprised and disappointed to receive such a communication from the FDA, and to receive it so late in the review cycle," Steve Davis, Acadia's CEO, said during a subsequent call with investors.
Acadia's application, meant to get a drug called pimavanserin cleared to treat dementia-related psychosis, has since been rejected. Other companies, including TherapeuticsMD and Vanda Pharmaceuticals, have in past years seen their applications knocked back after receiving similar FDA feedback about "deficiencies" that prevented the discussion of marketing requirements.
In Provention's case, executives said they're hoping to soon meet with the FDA to discuss whatever issues there are in the teplizumab application. "As additional facts and timelines evolve, we will be in a much better position to update you," Ashleigh Palmer, Provention's CEO, said on a Thursday investor call.
One thing Provention does know is the FDA has concerns about a so-called bridging study, which looked at how teplizumab gets absorbed and takes effect in the body when made with drug product from two different sources.
Historically, this drug product was manufactured by Eli Lilly, which tried its hand at developing teplizumab over a decade ago. It was Lilly's drug product that was used in the pivotal trial now supporting teplizumab's approval.
But Provention is hoping to have AGC Biologics, a Seattle-based contract manufacturer, make the drug product should teplizumab hit the market.
On April 2, Provention executives spoke to the FDA about an upcoming meeting in which outside experts would offer the agency advice regarding the teplizumab filing. Provention claims that during this discussion, the FDA said it did not believe the profiles of the two drug products were comparable based on data from the bridging study. Additional data would therefore be needed, according to the company.
On the April 8 investor call, Palmer noted how the FDA followed up with Provention earlier in the day, writing that the agency is willing to go over comparability data at the same time it's reviewing the larger approval application. "Specifically, the agency stated the timely discussions may help to avoid delays in addressing the agency's concerns," he said.
Even with that assurance, Provention is bracing for a longer review. "We recognize and want to make it clear that these circumstances increase the likelihood that there will be a delay in the timelines within which teplizumab has the potential to be approved by the FDA," Palmer said.
Analysts foresee delays as well. Alethia Young of Cantor Fitzgerald, for instance, thinks it's now more likely the July 2 approval deadline won't be met, and that teplizumab's launch will be pushed back to next year.
"We think it is interesting that the FDA still plans to move forward with the May 27 advisory committee, though we think this may be a sign that the agency is committed to working with the company through the concerns," Young wrote in a note to clients.
In the meantime, Provention shares are sitting at their lowest value since October 2019. They were going for just under $7 Friday morning, a fraction of the $17 price they hit in December.
The New Jersey-based biotech has garnered attention for its work with teplizumab, as clinical testing showed the drug appears to substantially delay the development of Type 1 diabetes — a disease that affects more than 1 million people in the U.S. alone, according to the American Diabetes Association.