FibroGen’s new PhIII data set it up for quick OK, analysts say. But can it avoid a black box warning?
Less than two months out from its PDUFA date, FibroGen has rolled out a slate of Phase III data on roxadustat that analysts say all but seals the deal on a broad OK for anemia associated with CKD. A black box warning, though, may still dim its prospects.
FibroGen presented data from six different Phase III studies at the American Society of Nephrology Kidney Week, covering a broad spectrum of CKD patients both dialysis-dependent and non-dialysis-dependent. Notably, the AstraZeneca-partnered drug scored some positive numbers on MACE and MACE+ rates in a pooled safety analysis, suggesting non-inferiority to placebo on cardio outcome.
“Based on the data, we think roxadustat sets a high bar for both efficacy and safety as the potentially first-in-class HIF-PHI drug,” Cowen analyst Yaron Werber wrote in a note. “We have high conviction that roxadustat will receive broad approval for both NDD- and DD-CKD by its 12/20/20 PDUFA.”
Just weeks ago, FibroGen’s rivals at Akebia were slammed despite hitting the primary and key secondary endpoints, precisely because of failing the all-important MACE non-inferiority standard. As patients’ anemia worsens over time, decreased oxygenation often results in an increased risk of cardiovascular complications and even death, FibroGen CMO Peony Yu said in a statement.
While the erythropoietin stimulating agents currently taken by anemic CKD patients are tied to increased CV risk at higher hemoglobin levels, roxa-treated patients experienced lower CV risks with higher Hb levels.
“This is important to roxa and its commercial outlook, since the increased CV risk associated with higher Hb cost ESAs a blackbox in 2011 and reduced revenue by more than a half,” Geoffrey Porges of SVB Leerink wrote. “This post-hoc analysis won’t necessarily guarantee a blackbox-free label for roxa, but it demonstrated very encouraging signals that roxa is a safer oral agent for the treatment of chronic kidney disease (CKD) anemia.”
Then there’s the reduction in total cholesterol, LDL cholesterol and triglycerides, all hopeful signs for a group at high risk of cardiovascular complications and even death.
Some analysts had already projected roxa’s peak sales at $2 billion or more, with Jefferies’ Michael Yee predicting that FibroGen “could have the whole $2-3B+ NDD market to itself” given Akebia’s flop.
Mizuho essentially echoed that sentiment this morning, casting approval in DD highly likely for Akebia but a more difficult path forward for the NDD indication. But the dialysis setting still represents a $2 billion US market.
“New data from Akebia suggests lower MACE in US-treated patients not on dialysis (vs. Ex-US) driven by regional protocol differences, and we expect this to be a key discussion point with FDA,” a note from the firm read.
Back in 2018, FibroGen and AstraZeneca marked the first time a top global pharma secured approval for a potential blockbuster drug in China before even filing in the United States or Europe — although the Chinese OK only covered dialysis-dependent patients.