Dive Brief:
- The Food and Drug Administration has rejected a potentially new kind of treatment for HIV developed by Gilead, a decision that, according to the company, was based on issues related to manufacturing and quality controls.
- Gilead said the agency has concerns about the interaction between an injectable solution of the drug, called lenacapavir, and the vials in which it's contained. Specifically, the FDA fears that tiny glass particles could form in the solution because the drug isn't compatible with vials made of borosilicate glass. Recently, the FDA halted at least 10 studies testing injectable lenacapavir due to this possible packaging problem.
- In a statement, Merdad Parsey, Gilead's chief medical officer, said the company intends to produce a "comprehensive plan and corresponding data to use a different vial type," and that it will work with the FDA over the coming months to ensure lenacapavir becomes available to patients.
Dive Insight:
Gilead, the world's largest developer of HIV medications, has for some time highlighted lenacapavir as one of its most important experimental programs. The drug has a unique way of preventing HIV from replicating. And, if approved, it would be the only treatment given every six months, which is much less often than currently available options.
As such, Wall Street came to have high expectations for lenacapavir. According to the investment bank Mizuho Securities, the consensus among analysts has been that sales from the drug could exceed $1.1 billion by 2030.
Those estimates may be a bit more ambitious, now that the FDA has issued a rejection. In a note to clients, Baird & Co. analysts wrote that the lenacapavir launch will likely be delayed by a year because of the agency's decision.
"The formal response is not entirely surprising, given the earlier concerns by the FDA about glass particle formation with injectable lenacapavir," Baird analyst Brian Skorney wrote in the March 1 note. "But given Gilead has increasingly worked to emphasize the upside potential of this program ... we think this update will make it difficult for management to drum up excitement around the program for the time being."
Still, Skorney and his team argue the negative outcome is only an "incremental setback" for lenacapavir, and that the FDA will ultimately approve the drug once Gilead addresses the manufacturing and quality control concerns.
The company's share price remained relatively unchanged Tuesday morning, perhaps reflecting that investors were prepared for a rejection.
Gilead formally asked the FDA for approval last summer, after generating positive results from a late-stage clinical trial that tested its drug in a small crop of HIV patients who had tried other therapies but no longer responded to them. The trial found lenacapavir was able to suppress the virus, with around 80% of patients who were given the drug and a "optimized background regimen" showing viral loads so low they were undetectable after six months of treatment.
The trial tested both an injectable and an oral form of lenacapavir, and the package submitted to the FDA included an approval application for each. While the vial issues are specific to the injectable solution, Gilead spokesperson Brian Plummer notes that the oral tablet is meant to be used as part of treatment initiation.
"Therefore, for this [application] submission, the approval of the lenacapavir tablet is contingent on the approval of lenacapavir injection," Plummer wrote in an email to BioPharma Dive.