New hope for rare sun allergy: bitopertin offered to patients with no options

NCT ID NCT07603401

AVAILABLE Disease control Sponsor: Disc Medicine, Inc Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Aug 04, 2026 · Updated 7 times

Summary

This expanded access program provides bitopertin (DISC-1459) to patients aged 12 and older with erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP) who have no satisfactory treatment options in the US. The goal is to offer access and gather safety information. Participants take a 60 mg oral dose once daily.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: bitopertin (DISC-1459)
What this could lead to: If successful, this could provide a treatment option for people with EPP and XLP who currently have no satisfactory therapies.
What could go wrong: This is an expanded access program, not a formal trial, so data on effectiveness is limited. It may not work for everyone, and side effects are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

erythropoietic protoporphyria porphyria X-linked erythropoietic protoporphyria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ERYTHROPOIETIC PROTOPORPHYRIA (EPP) X-LINKED PROTOPORPHYRIA (XLP)

Contacts and locations

Locations

Massachusetts General Hospital
AVAILABLE

Boston, Massachusetts, 02115, United States
MetroBoston Clinical Partners
AVAILABLE

Boston, Massachusetts, 02135, United States
Mount Sinai Hospital
AVAILABLE

New York, New York, 10029, United States
University of Miami Miller School of Medicine
AVAILABLE

Miami, Florida, 33146, United States
Wake Forest University
AVAILABLE

Winston-Salem, North Carolina, 27157, United States

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