Rare Disease Phase 3 Readout Calendar
Industry-sponsored rare disease trials with primary completion in the next 12 months or last 12 months.
Data last updated November 12, 2026 · source: ClinicalTrials.gov
What you can answer with this page
A confidence-graded calendar of every industry Phase 3 rare disease trial reading out in the next 12 months — with a per-row staleness flag so you know which completion dates are still credible.
Recently read-out results are free; the upcoming calendar shows a free preview. See the full cross-area calendar → or the Rare Disease landscape →
Recently read out & awaiting topline — Rare Disease
· 4Phase 3 rare disease trials whose primary endpoint completed in the last 12 months — results posted, or completed and awaiting topline. Most recent first.
| Sponsor | Trial / Asset | Indication | Status | Primary completion | Confidence | NCT |
|---|---|---|---|---|---|---|
| argenx | A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP). Asset: efgartigimod | Primary Immune Thrombocytopenia | Completed | Q1 2026 2026-03-11 | Completed · awaiting
Updated 8mo ago
| NCT04225156 |
| Amgen | Study of Romiplostim for Chemotherapy-induced Thrombocytopenia in Adult Subjects With Non-small Cell Lung Cancer (NSCLC), Ovarian Cancer, or Breast Cancer Asset: Romiplostim | Chemotherapy-induced Thrombocytopenia | Active Not Recruiting | Q1 2026 2026-03-10 | Completed · awaiting
Updated 7mo ago
| NCT03937154 |
| Forma Therapeutics, Inc. | A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS) Asset: Etavopivat Tablets Low dose | Sickle Cell Disease | Active Not Recruiting | Q1 2026 2026-02-06 | Completed · awaiting
Updated 7mo ago
| NCT04624659 |
| Sanofi | A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Asset: Venglustat (GZ402671) | Fabry Disease | Active Not Recruiting | Q4 2025 2025-12-17 | Completed · awaiting
Updated 9mo ago
| NCT05206773 |
Upcoming rare disease readouts — next 12 months
· 25Industry Phase 3 rare disease trials with primary completion ahead, soonest first — confidence-graded by date type and CT.gov staleness.
| Sponsor | Trial / Asset | Indication | Status | Primary completion | Confidence | NCT |
|---|---|---|---|---|---|---|
| Gritgen Therapeutics Co., Ltd. | To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A Asset: GS1191-0445 injection | Hemophilia A | Recruiting | Q4 2026 2026-11-30 (est.) | Estimated · aging
Updated 11mo ago
| NCT06833983 |
| Sanofi | Rilzabrutinib for the Adult Participants With Chronic ITP Who Have Completed Phase 3 Study in Japan Asset: rilzabrutinib | Immune Thrombocytopenia | Active Not Recruiting | Q4 2026 2026-11-30 (est.) | Estimated · aging
Updated 12mo ago
| NCT07216079 |
| CSL Behring | Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy Asset: Recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) | Hemophilia B | Completed | Q4 2026 2026-12-01 (est.) | Estimated · aging
Updated 10mo ago
| NCT06399289 |
| Grand Shuyang Life Sciences (Cheng… | An Open-label Study of Intravenous Immunoglobulin (5%) for the Treatment of Primary Immune Thrombocytopenia Asset: Human immunoglobulin (pH4) for intravenous injection | Primary Immune Thrombocytopenia (ITP) | Recruiting | Q4 2026 2026-12 (est.) | Estimated · aging
Updated 7mo ago
| NCT07233213 |
| Amicus Therapeutics | A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease Asset: migalastat HCl 150 mg | Fabry Disease | Active Not Recruiting | Q4 2026 2026-12-31 (est.) | Estimated · aging
Updated 7mo ago
| NCT04020055 |
About this calendar
Dates anchor on the ClinicalTrials.gov primary completion date — the only public, structured signal — and are graded by date type (actual vs sponsor-estimated) and how recently the record was updated. Sponsor-estimated dates can slip silently, so treat each as a current target rather than a guarantee. See the full readout calendar for all areas and filters.