Drug for Rare Genetic Disorder Moves from FDA Priority to Rejection in Four Months

() -- The U.S. Food and Drug Administration's rejection of Disc Medicine Inc.'s experimental drug for a rare and severe condition, only months after the company was granted a voucher meant to expedite U.S. reviews for innovative therapies, highlights the increasing unpredictability at the country's leading drug regulatory agency.

On Friday, the drug company in the development phase announced the FDArejected its applicationRegarding bitopertin, a therapy for the rare genetic condition erythropoietic protoporphyria, which leads to extreme sensitivity to light, it is the most recent medical product to face delays or rejection from the agency due to concerns over its development strategy, which was previously permitted by earlier administrations.

The FDA declined to evaluate Moderna Inc.'s new mRNA flu vaccine this week, stating worries over the study's structure and insufficient control groups. This decision sparked frustration from Moderna's CEO Stephane Bancel, who claimed the agency's growing unpredictability "endangers U.S. leadership in groundbreaking medicines."

The rejection of Disc Medicine's treatment takes things a step further. Only four months ago, its drug bitopertin was among the agency's highest priorities. It was one of the firstnine medicines selectedTo receive a Commissioner’s National Priority Voucher in October. The key initiative promoted by FDA Commissioner Marty Makary is designed to offer faster reviews, with a decision on approval within one to two months for medications that target critical health issues.

Disc Medicine did not receive a decision within the specified timeframe, and the drug was completely denied by the agency, which requires additional trial data. This has led to concerns regarding the structure of the studies and the clinical relevance of the results the company provided to demonstrate the medicine's effectiveness.

The trials were finished, and the Disc Medicine's approval request had already been sent to the agency when it unexpectedly received one of the new vouchers.

Experts noted that the sudden shift indicates that the selection of a product for the commissioner's voucher program might not be as beneficial as initially believed when the program was launched. It also brings up concerns about what is typically a tightly controlled and uniform process involved in the creation of new medical products.

Disc Medicine's stock dropped 22% following the FDA's announcement.

The main concern we are observing is the lack of consistency from the FDA," wrote Evan Seigerman, an analyst at BMO Capital Markets, in a message to clients. Inconsistency is described as "a significant challenge for the industry, which is adversely affecting innovation.

Although the future for Moderna remains uncertain, Disc Medicine stated in a release that the concerns raised by the FDA are "easily resolved."

The organization is seeking a new study that includes additional clinical information. In its statement, Disc Medicine mentioned that it already has a late-stage study in progress, which could yield results by the end of the year.

The medication could serve as the first treatment that alters the course of erythropoietic porphyrias, which encompasses a disorder characterized by intense pain, burning sensations, and skin sensitivity upon exposure to sunlight.

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