FDA Panel Rejects Lexicon Pharma's Type 1 Diabetes Drug 11-3

On Thursday, the independent advisory committee of the U.S. Food and Drug Administration (FDA) decided against recommending Lexicon Pharmaceuticals' drug, sotagliflozin, for additional treatment beyond insulin therapy for adults with Type 1 diabetes and chronic kidney disease. This marks another obstacle in the company's ongoing journey to establish itself in the market.

The panel's vote concluded with 11 members opposing and only 3 supporting the drug, indicating significant concern over its risk-benefit profile. The rejection follows a pattern set in early 2019 when the same advisory committee was divided on whether to endorse sotagliflozin for treating Type 1 diabetes. The FDA ultimately chose not to approve the drug for market release at that time.

Lexicon Pharmaceuticals did not give up and resubmitted the drug for market approval in June 2024. They aimed to get the drug approved for patients with Type 1 diabetes and chronic kidney disease, particularly those whose kidney function scores or estimated glomerular filtration rates (eGFR) ranged from 45 to less than 60, 60 to less than 90, and greater than 90.

Despite these efforts, some committee members suggested that the drug's benefits might outweigh its risks for specific patient groups, particularly those with a kidney function score of less than 60 to 90 or those with mild to moderate kidney disease. However, concerns were raised about the lack of sufficient clinical data to back these claims. The committee members pointed out the absence of prospective studies targeting the patient subgroups that might benefit the most from the drug.

One of the committee members, Cecilia Wang, who voted against the drug, expressed her reluctance but felt compelled by the nature of the issue and the unique population involved. "I wished I could have voted for it. I didn't vote for it because the nature of the issue is very special, and the population involved is extremely special... If the issue involved people aged 60 to 90, my position might have been different," Wang stated, highlighting her skepticism about the data concerning people aged 60 to less than 90.

She emphasized the limited number of relevant data points, stating, "The numbers are very limited. With the scarcity of relevant data, it's really hard for me to vote in favor of approving a new drug." This sentiment was echoed by other committee members who expressed disappointment over the lack of robust clinical evidence to support the drug's approval.

The committee's decision deals a significant blow to Lexicon Pharmaceuticals' aspirations of entering the market with sotagliflozin as an additional treatment option for adults with Type 1 diabetes and chronic kidney disease. Despite the setback, the company may need to gather more comprehensive clinical data to address the concerns raised by the FDA's advisory committee and possibly consider resubmitting the drug for approval in the future.