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Upcoming Rare Disease FDA decisions & readouts

Updated ยทnext FDA decision on the calendar
PDUFA target dates and trial readouts in rare disease, each linked to its primary source. Updated from FDA/SEC/ClinicalTrials.gov; dates can shift.
Cancer & OncologyObesity & MetabolicCNS & NeurologyImmunology & InflammationCardiovascularInfectious Disease & VaccinesHematologyOphthalmology & Eye

PDUFA decisions (3)

CAPR · 2026-11-22
Deramiocel (CAP-1002) - (HOPE-3): Duchenne muscular dystrophy; refined proposed indication focused on upper limb function
BBIO · 2026-11-27
BBP-418 (ribitol) - (FORTIFY): Limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)
PHVS · 2027-04-23
Deucrictibant IR - (RAPIDe-3): Hereditary angioedema, on-demand

Trial readouts (8)

ARDX · Sep 2026 (est.)
Tenapanor
RYTM · Oct 2026 (est.)
Setmelanotide
SLDB · Oct 2026 (est.)
SGT-003
SRPT · Oct 2026 (est.)
Eteplirsen
CORT · Dec 2026 (est.)
Relacorilant
IONS · Jun 2027 (est.)
Olezarsen
IONS · Aug 2027 (est.)
ION582
AKBA · May 2031 (est.)
Vadadustat

Every upcoming FDA decision date, all therapeutic areas, on the 2026 PDUFA calendar · all conditions

We don't show an approval percentage for these rare disease catalysts: here's why.