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Upcoming Rare Disease FDA decisions & readouts

Updated August 7, 2026·next 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 & Oncology Obesity & Metabolic CNS & Neurology Immunology & Inflammation Cardiovascular Infectious Disease & Vaccines Hematology Ophthalmology & Eye

Trial readouts (24)

WVE · Jun 2026 (est.)

WVE-210201: Duchenne Muscular Dystrophy

ARDX · Jun 2026 (est.)

Tenapanor: Cystic Fibrosis

SION · Jul 2026 (est.)

SION-719: Cystic Fibrosis (CF)

PHVS · Aug 2026 (est.)

deucrictibant: Hereditary Angioedema (HAE)

MSLE · Aug 2026 (est.)

SAT-3247: Duchenne Muscular Dystrophy (DMD)

DSGN · Sep 2026 (est.)

DT-168: Fuchs Endothelial Corneal Dystrophy

SLDB · Oct 2026 (est.)

SGT-003: Duchenne Muscular Dystrophy

SNY · Oct 2026 (est.)

Venglustat: Gaucher Disease Type 1

SRPT · Oct 2026 (est.)

Eteplirsen: Muscular Dystrophy, Duchenne

DSGN · Dec 2026 (est.)

DT-168: Friedreich Ataxia

ARWR · Dec 2026 (est.)

Plozasiran: Myotonic Dystrophy 1

IMMX · Dec 2026 (est.)

NXC-201 CAR-T: Light Chain (AL) Amyloidosis

IONS · Dec 2026 (est.)

Donidalorsen: Hereditary Angioedema

NTLA · 2026-H2

NTLA-2002: Hereditary Angioedema

PHVS · 2026-H2

deucrictibant: Hereditary Angioedema

SION · 2026-H2

SION-719: Cystic Fibrosis (CF)

LRMR · Jan 2027 (est.)

CTI-1601: Friedreich Ataxia

PEPG · Mar 2027 (est.)

PGN-EDODM1: Myotonic Dystrophy 1

MSLE · Mar 2027 (est.)

SAT-3247: Duchenne Muscular Dystrophy

SLDB · May 2027 (est.)

SGT-003: Duchenne Muscular Dystrophy

PHVS · Jun 2027 (est.)

deucrictibant: Hereditary Angioedema

ICLR · Oct 2027 (est.)

PRX-102: Fabry Disease

NTLA · Dec 2027 (est.)

NTLA-2002: Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

QURE · Dec 2027 (est.)

AMT-191: Fabry Disease

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