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Upcoming rare disease clinical trial readouts

Updated ·next FDA decision on the calendar
8 upcoming rare disease readouts, September 2026 to May 2031. Each row links its own trial record or company release. Dates shown at the precision the source gave.
ARDX · Sep 2026 (est.)
Tenapanor · company release
RYTM · Oct 2026 (est.)
Setmelanotide · company release
SLDB · Oct 2026 (est.)
SGT-003 · company release
SRPT · Oct 2026 (est.)
Eteplirsen · company release
CORT · Dec 2026 (est.)
Relacorilant · company release
IONS · Jun 2027 (est.)
Olezarsen · no trial record on file
IONS · Aug 2027 (est.)
ION582 · no trial record on file
AKBA · May 2031 (est.)
Vadadustat · company release
What is counted: every rare disease readout in this dataset whose date has not yet passed, taken from the row's own therapeutic-area field rather than guessed from the drug name. 0 of these 8 dates come from company guidance and 8 are estimated primary-completion windows from ClinicalTrials.gov, which shift; each row shows the precision its source actually gave, so a row reading a month or a quarter is one where nobody published a day. 6 of 8 link a trial record or company release. This is a list of dates and sources: it carries no approval odds, no probability of success and no ranking.

This is a floor, not a complete census. 157 of the 269 upcoming readouts we track (58%) carry no therapeutic area, or are tagged only “Other”, and are therefore not counted on this page. Some of them are almost certainly rare disease. We would rather under-count against a field we can check than pad the number by guessing a therapeutic area from a drug name, which is how this site once published a discontinued 2017 compound as an upcoming event.

See also upcoming oncology readouts, the full clinical trial readout calendar, and the FDA decision calendar.

Questions

How many rare disease readouts are expected?
This page lists 8 upcoming rare disease clinical-trial readouts, covering September 2026 to May 2031. 0 of the dates are company-guided and 8 are estimated primary-completion windows from ClinicalTrials.gov. That number is a floor: 157 of the 269 upcoming readouts we track carry no therapeutic area or are tagged only “Other”, and are not counted here.
Are these rare disease readout dates exact?
Mostly no, and the page says which are which. A company-guided date is what the sponsor stated. An estimated date is the trial's primary-completion window from ClinicalTrials.gov, which is an estimate that moves. Where a source gave only a month or a quarter, this page shows a month or a quarter rather than inventing a day.
Does this page predict which readouts will be positive?
No. pdufa.bio publishes dates, sources and historical statistics. It does not publish approval odds or probability of success, and nothing here is investment advice.