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Thrombocytopenia

Rare Disease 4 original drugs
4
Novel Drugs
10
Reformulations
0
Active Trials
3
Drug Targets

Rare Disease indication. Thrombocytopenia has 4 FDA-approved novel treatments, led by Amgen across 3 drug targets. Explore market leaders, treatments by mechanism, the clinical pipeline, and drug targets below.

Thrombocytopenia Market Leaders

Companies with the most FDA-approved novel Thrombocytopenia drugs.

Phase 3 Readouts Pro

12 active Phase 3 trials with confidence-graded completion dates.

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Q3 2026
QL0911
Qilu Pharmaceutical Co., Ltd.
Estimated · fresh NCT06456528
Q3 2026
Kedrion IVIG 10%
Kedrion S.p.A.
Estimated · fresh NCT07059000
Q4 2026
efgartigimod PH20 SC
argenx
Estimated · fresh NCT04812925
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Anchored on CT.gov primary completion date. Topline announcements typically precede this by 3–9 months. Confidence labels combine date type (ACTUAL/ESTIMATED) with last-update freshness.

Thrombocytopenia Treatments by Mechanism

Top 4 mechanisms across 13 industry trials with a known mechanism of action.

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Thrombopoietin Receptor Agonist
8 trials 1 active 2 sponsors
Kinase Inhibitor
1 trials 1 active 1 sponsor
Anti-coagulant
2 trials 2 sponsors
TPO receptor
2 trials 1 sponsor

MoA derived from FDA pharmClassEpc when intervention matches an approved drug. Codenamed clinical-stage assets without an approved counterpart show "—" and aren't grouped here — they're still in the phase tables below.

Thrombocytopenia Clinical Pipeline by Phase

132 industry-sponsored trials across 66 sponsors

Precision Medicine Biomarkers

All biomarkers →

Biomarkers used for patient selection in Thrombocytopenia

Novel Thrombocytopenia Drugs by Company (New molecular entities)

4 companies have an FDA-approved novel drug for Thrombocytopenia.

Reformulations (10 drugs) Click to expand

Thrombocytopenia Drug Targets

Molecular targets of approved and investigational Thrombocytopenia drugs — 3 targets tracked.

Generic Drugs (3 ANDA approved) Click to expand

Generic drugs contain the same active ingredient as the brand-name drug and are approved via ANDA (Abbreviated New Drug Application).

Related Indications

Other Rare Disease indications

Drug Categories:

  • Novel Drugs: NDA Type 1/2 (new molecular entity) or original BLA
  • Reformulations: NDA Type 3/5 (new dosage form of existing molecule)
  • Biosimilars: BLA-approved biologics highly similar to reference products
  • Generics: ANDA-approved copies of small molecule drugs

View data sources and methodology