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The complete response letter from the FDA cited a lack of effectiveness in slowing the loss of kidney function in patients with Alport syndrome.
The US Food and Drug Administration has issued a Complete Response Letter (CRL) to Reata Pharmaceuticals, Inc. concerning the New Drug Application (NDA) for bardoxolone methyl in the treatment of chronic kidney disease (CKD) caused by Alport syndrome.
Announced on February 25, the letter indicated that the FDA cannot currently approve the NDA, as it does not believe the submitted data demonstrated bardoxolone’s effectiveness in slowing the loss of kidney function in patients with Alport syndrome and reducing the risk of progression to kidney failure. They requested further data to support the efficacy and safety of bardoxolone.
“This outcome is a significant disappointment for our company, as well as the many patients, families, and investigators who have participated in our development program for bardoxolone in Alport syndrome patients,” said Warren Huff, Reata’s Chief Executive Officer in a statement. “We will continue to work with the FDA to confirm our next steps on our Alport syndrome program.”
Concerns about bardoxolone were initially discussed during the Cardiovascular and Renal Drugs Advisory Committee held in December 2021. The committee members voted unanimously against its approval, citing questionable efficacy and concerns with the safety profile in the CARDINAL or BEACON trials.
The FDA stated in the letter that the issues could be resolved by providing evidence of effectiveness from an adequate and well-controlled study showing a clinically relevant effect on the rate of loss of kidney function in patients with Alport syndrome.
The company will additionally need to address if bardoxolone has a clinically relevant effect on the QT interval and show that the clinical benefit of bardoxolone will outweigh the risk. In their press release, Reata noted they plan to work closely with the FDA “to bring this important medicine to patients in the US.”
Currently, bardoxolone is being studied in the phase 3 FALCON study for the treatment of CKD caused by autosomal dominant polycystic kidney disease, the open-label EAGLE study for patients with CKD caused by Alport who participated in the CARDINAL trial, and the phase 3 AYAME study for the treatment of diabetic kidney disease being conducted in Japan.