The U.S. Food and Drug Administration (FDA) has postponed its decision on a potential treatment for erythropoietic protoporphyria, a rare blood disorder, developed by Disc Medicine Inc. (NASDAQ:IRON). The new target date for the decision regarding the drug, bitopertin, is now set for February 10, 2024, pushing the timeline back by approximately two weeks.
Disc Medicine’s stock has shown positive movement, increasing by 2.59% to reach $72.88 at publication. The interest surrounding the stock is driven by the anticipation of FDA approval, but uncertainty remains regarding the agency’s evaluation processes. Analysts have noted concerns about the reliability of a secondary endpoint in clinical trials, which measures “pain-free time in the sun.” The FDA has raised questions about whether this endpoint is a statistically valid measure of effectiveness, suggesting that biomarker data may be more crucial for supporting approval.
Compounding the situation, according to a report from Reuters, FDA staff assigned to evaluate drugs with potential abuse risks have been asked to investigate whether bitopertin poses any risk of misuse or addiction. This additional scrutiny introduces further complexity into the review process.
In response to the recent developments, Douglas Tsao, an analyst at H.C. Wainwright, expressed confidence in the eventual approval of bitopertin. Tsao indicated that the company was not aware of a specific FDA action date prior to the media coverage, although the implied timing of January 27 aligns with prior FDA guidance concerning the Compassionate Use Program (CNPV) and the management’s expectations for a decision by early February.
Disc Medicine received CNPV designation for bitopertin in October 2023. However, a report from STAT News in December highlighted skepticism from Vinay Prasad, the head of the FDA’s Center for Biologics Evaluation and Research, regarding the drug’s efficacy under the CNPV program. Despite this, the company maintains that its interactions with the FDA have followed a “regular” review process, albeit with increased frequency due to the expedited timeline.
Tsao remarked, “Another leak belies internal controversies within the FDA; we still remain confident that approval will occur. Even in the case of a Complete Response Letter — an outcome we continue to see as unlikely — the delay would be relatively modest.” H.C. Wainwright has reiterated a Buy rating for Disc Medicine, projecting a price target of $118.
As the FDA’s decision date approaches, the pharmaceutical sector is closely monitoring this situation, given the potential implications for patients suffering from erythropoietic protoporphyria and the broader market for rare disease treatments.
