FDA Rejects Disc Medicine's Porphyria Drug Bitopertin in First Test of Makary Fast-Track Program

The rejection highlights the challenges of balancing speed and thoroughness in drug approvals for rare diseases.

Published on Feb. 14, 2026

Got story updates? Submit your updates here. ›

The Food and Drug Administration (FDA) has rejected bitopertin, a therapy developed by Disc Medicine for the treatment of porphyria, a rare blood disorder. This decision marks the first time the agency has declined a drug submitted through FDA Commissioner Marty Makary's newly implemented program designed to accelerate drug reviews. The FDA cited 'uncertainties' regarding the link between the blood-based biomarker used in Disc Medicine's clinical trials and actual clinical improvement for patients.

Why it matters

The rejection of bitopertin, despite receiving expedited review, underscores the inherent challenges in balancing speed with thoroughness in the drug approval process, particularly for rare diseases where identifying reliable biomarkers can be difficult. While the FDA's fast-track program aims to bring potentially life-saving treatments to patients more quickly, the agency's primary responsibility is to ensure the safety and efficacy of all drugs before they are made available to the public.

The details

Porphyria is a group of genetic disorders that affect the body's ability to produce heme, a crucial component of hemoglobin. Deficiencies in specific enzymes involved in heme production lead to a buildup of porphyrins, chemicals that can cause a variety of symptoms, including painful abdominal cramps, neurological problems, and severe skin reactions triggered by sunlight exposure. Currently, treatment options for porphyria are largely supportive, focusing on managing symptoms and avoiding triggers like sunlight. Bitopertin was developed by Disc Medicine as a potential new therapy for the condition.

  • The FDA rejected bitopertin on Friday, February 13, 2026.
  • In December, the FDA had previously approved a generic antibiotic through the same fast-track program.

The players

Disc Medicine

A pharmaceutical company that developed bitopertin, a therapy intended for the treatment of porphyria.

Marty Makary

The FDA Commissioner who implemented a new program designed to accelerate drug reviews.

Food and Drug Administration (FDA)

The federal agency responsible for regulating and supervising the safety and efficacy of drugs and medical devices in the United States.

Got photos? Submit your photos here. ›

What’s next

Disc Medicine will likely review the FDA's feedback and determine the next steps for bitopertin's development, which could involve conducting additional clinical trials to further investigate the relationship between the biomarker and clinical outcomes.

The takeaway

The rejection of bitopertin, despite receiving expedited review, highlights the complexities involved in drug development for rare diseases and the importance of establishing a strong correlation between biomarker changes and clinical benefit before a drug can be approved. While the FDA's fast-track program aims to accelerate the review process, the agency's commitment to ensuring patient safety and efficacy remains paramount.