Upcoming Rare Disease FDA decisions & readouts

PDUFA target dates and trial readouts in rare-disease disease, each linked to its primary source. Updated from FDA/SEC/ClinicalTrials.gov; dates can shift.

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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.