In late February, the US Food and Drug Administration (FDA) declined to grant marketing approval to the novel agent bardoxolone methyl as a treatment for Alport syndrome, which means this rare genetic disease that causes early onset progressive kidney dysfunction remains without a specific treatment.
Despite bardoxolone's rejection by the FDA because of what the agency said was inadequate evidence of efficacy for the first agent filed as a treatment for Alport syndrome, researchers and leaders from the Alport syndrome patient community see the experience as a small step forward in the effort to find effective interventions.
They believe the drug's development, testing, and regulatory submission blazed a path that previously did not exist for investigational Alport syndrome treatments and should aid development of future candidate treatments.
Bardoxolone's pivotal trial "demonstrated that it is possible to conduct an international clinical trial in Alport syndrome, and this could help with planning future studies," said Rachel Lennon, BMBS, PhD, professor of nephrology at the University of Manchester, UK.
"I am very optimistic about the prospects for new treatments for patients with Alport syndrome in the next 5 to 10 years," Lennon said in an interview.
"Having completed the first clinical trial...which was fully enrolled, and with other, available studies [now in progress] being filled with target numbers of patients with Alport syndrome, I believe our community has clearly demonstrated its motivation to change our outcomes and our families' stories by participating in research and in clinical trials," said Lisa Bonebrake, executive director of the Alport Syndrome Foundation, who also lives with the condition.
