- This past May, Portola Pharmaceuticals won a long-awaited approval for its anticoagulant reversal agent Andexxa. So far, however, roll-out of the medicine has been limited to only a few dozen treatment centers as the biotech works to win regulatory approval of a new, large-scale production process.
- Portola inched a step closer to that goal last week, submitting what's known as a "prior approval supplement" to the Food and Drug Administration for clearance of its second-generation manufacturing, now contracted out to Swiss giant Lonza.
- If the FDA signs off, Portola says it could quickly launch Andexxa more broadly. Already, three production runs using the new process have produced enough drug supply to stock 1,000 hospitals, the company said in an Aug. 31 statement.
Despite finally winning approval for Andexxa (coagulation Faxtor Xa (recombinant), inactivated-zhzo) in May, shares in Portola have lost nearly a third of their value since then. Part of that slide might be due to the minimal commercial returns the company has earned from Andexxa while it waits to launch the drug more broadly.
In the run-up to Andexxa's approval, investors were frustrated by a series of setbacks that kept the blood thinning antidote from market for years.
Pushing the drug across the finish line earned Portola a temporary boost to its stock, but shares have since drifted back down. Under an early-supply program, only about 40 hospitals and treatment centers have been offered access to Andexxa.
The drug is the first and only antidote approved for patients treated with Xarelto (rivaroxaban) and Eliquis (apixaban) who need reversal of anticoagulation due to uncontrolled bleeding.
Now, clearance of the new manufacturing process is one last hurdle before Portola can market the drug as widely as it intends.
For Andexxa's clinical studies, the company relied on AGC Biologics for drug product supply. But, following a Complete Response Letter from the FDA in 2016, Portola decided to switch to a larger, 10,000 liter-scale process from Lonza.
The only snag? The Biologics License Application for Andexxa referenced the manufacturing process at AGC Biologics. So, while Andexxa has won FDA approval, Portola still needs to get sign-off on Lonza's manufacturing.
The company expects a standard turnaround from the FDA, with a decision sometime in the first quarter of 2019.
In the meantime, preparations are underway for a broader launch.
"We ... are helping those hospitals waiting for access to Gen 2 get Andexxa added to formulary in advance of potential approval," Tao Fu, Portola's chief commercial and business officer, said on an earnings call earlier this month. "And we will be increasing our sales force in anticipation of the Gen 2 launch early next year."
Currently, Portola employs about 70 sales staff but will increase that number to about 130 so it can adequately cover the expanded roll-out.
Until then, investors will likely be keeping any excitement over Andexxa's prospects in check.