- The Food and Drug Administration has rejected Reata Pharmaceuticals' experimental pill for a rare genetic kidney disease. The company announced Friday that regulators want to see data from a new trial to evaluate whether the drug, called bardoxolone methyl, keeps patients from getting sicker as well as its effects on the heart.
- The FDA's so-called complete response letter comes 11 weeks after expert advisers convened by the FDA voted unanimously against approving the drug to treat Alport syndrome. Bardoxlone is being developed for five other types of kidney conditions, but the agency's stance has raised concerns among analysts about the drug's prospects elsewhere.
- Reata has begun submitting a second experimental drug to the FDA for a rare movement disorder that currently has no treatment options. That drug has been given special regulatory status that involves closer collaboration with the agency and an approval decision could be possible by the end of 2022.
With the Dec. 9 negative vote from FDA advisers, Reata's announcement of the agency's rejection with a request for a new round of clinical trials comes as little surprise.
Wall Street analysts had already dismissed the possibility of an FDA approval. Because Alport syndrome has no available treatments, Stifel analyst Annabel Samimy wrote in a Dec. 9 note to clients that it was bardoxolone's best chance for approval.
Although expected, the rejection could add to investors' increasingly negative views on smaller biotech companies, in part driven by questions of whether the FDA has, in some cases, gotten tougher on drug reviews. The agency doesn't appear to be issuing more rejections than in the past, however.
Shares in Reata rose by more than 20% in Monday morning trading. The surge followed the company's release of fourth quarter earnings, in which executives announced that they plan to complete submission of the movement disorder drug by the end of the first quarter.
Company executives also disclosed that they have asked the FDA for permission to change a Phase 3 study of bardoxolone in a different type of inherited kidney disease. The requested changes would increase the number of patients enrolled in the trial by more than 50% and double the length of treatment time before making a final measurement of its effectiveness.