Five months ago, when the FDA raised multiple concerns over ChemoCentryx’s filing for a drug to treat vasculitis, the company’s stock plummeted.
But ChemoCentryx answered those questions and on Friday scored an approval for Tavneos, an oral treatment for vasculitis, which leads to inflammation and destruction of small blood vessels. It's the first approval for ChemoCentryx in its 24-year history and, as a result, shares surged by 56% in premarket trading.
“With this approval, we achieve our vision of becoming an integrated biopharmaceutical company, with discovery, development and U.S. commercial capabilities,” ChemoCentryx CEO Thomas Schall told investors in a conference call on Friday morning. “And we are ready for a fast launch.”
Tavneos is an oral and adjunctive treatment to be used in combination with standard therapy. The FDA approval is for adult patients with severe anti-neutrophil cytoplasmic antibody-associated (ANCA) vasculitis.
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The autoimmune disorder can lead to organ failure, particularly in the kidneys, and is often fatal if not treated. With a high variability in symptoms, identifying the disease is difficult. More than 1 in 10 patients die within a year of diagnosis.
The condition is rare. Of the 50,000 vasculitis patients in the United States, ChemoCentryx estimates that 9,500 fit Tavneos’ label.
Schall said that the company will charge between $150,000 and $200,000 for an annual regimen, which fits in the “orphan corridor,” he said. Tavneos received orphan drug designation from the FDA in 2014.
“We firmly believe that Tavneos has the potential eventually to be a billion-dollar medication in this indication alone,” Schall said. “But we also know that we have a long distance to ride in our aspirations to make Tavneos a blockbuster.”
Tavneos is the first new therapy sanctioned for ANCA vasculitis in more than a decade. It's also the first FDA therapy that selectively inhibits the C5a receptor, which is central to the pathogenesis of ANCA vasculitis.
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Tavenos survived close scrutiny in May when a panel of experts convened by the FDA voted 10-8 that the benefit-risk profile of the drug supported its approval. Reviewers at the FDA had set the stage for a tough advisory committee by publishing a briefing document that raised concerns about the med's data.
ChemoCentryx will now focus on developing Tavneos for other indications including lupus nephritis and hidradenitis suppurativa, Schall said.