Aplastic Anemia

Hematology
6
Pipeline Programs
5
Companies
6
Clinical Trials
1 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
3
0
1
1
Early DiscoveryClinical DevelopmentMarket

On Market (1)

Approved therapies currently available

Novartis
PROMACTA KITApproved
eltrombopag olamine
Novartis
oral2015

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Novartis
NovartisBASEL, Switzerland
1 program
1
1
PROMACTA KIT(Eltrombopag)Phase 21 trial
Active Trials
NCT03988608Completed20Est. May 2023
Pfizer
PfizerNEW YORK, NY
2 programs
1
PF-06462700Phase 31 trial
Anti-human thymocyte immunoglobulin, equineN/A1 trial
Active Trials
NCT06039020Active Not Recruiting1Est. Jan 2027
NCT04350606Completed3Est. Apr 2021
BioLineRx
BioLineRxIsrael - Modi’in
1 program
1
BL-8040Phase 21 trial
Active Trials
NCT02462252CompletedEst. Nov 2020
Qilu Pharmaceutical
Qilu PharmaceuticalChina - Jinan
1 program
1
standard IST combined with Romiplostim N01Phase 21 trial
Active Trials
NCT06613880RecruitingEst. Sep 2026
Astellas
AstellasChina - Shenyang
1 program
1
AlefaceptPhase 11 trial
Active Trials
NCT01267643Terminated4Est. Apr 2013

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
PfizerPF-06462700
Qilu Pharmaceuticalstandard IST combined with Romiplostim N01
NovartisEltrombopag
BioLineRxBL-8040
AstellasAlefacept
PfizerAnti-human thymocyte immunoglobulin, equine

Clinical Trials (6)

Total enrollment: 28 patients across 6 trials

NCT04350606PfizerPF-06462700

A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia

Start: Jul 2020Est. completion: Apr 20213 patients
Phase 3Completed
NCT06613880Qilu Pharmaceuticalstandard IST combined with Romiplostim N01

Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia

Start: Oct 2024Est. completion: Sep 2026
Phase 2Recruiting
NCT03988608NovartisEltrombopag

Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.

Start: Dec 2019Est. completion: May 202320 patients
Phase 2Completed
NCT02462252BioLineRxBL-8040

Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome

Start: Oct 2015Est. completion: Nov 2020
Phase 2Completed
NCT01267643AstellasAlefacept

Alefacept in Patients With Relapsed/Refractory Aplastic Anemia

Start: May 2011Est. completion: Apr 20134 patients
Phase 1Terminated
NCT06039020PfizerAnti-human thymocyte immunoglobulin, equine

ATGAM General Investigation

Start: Feb 2024Est. completion: Jan 20271 patients
N/AActive Not Recruiting

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 28 patients
5 companies competing in this space

Related Indications

Anemia(92)Myelofibrosis(21)Polycythemia Vera(16)Primary Immune Thrombocytopenia(15)Primary Myelofibrosis(14)Immune Thrombocytopenia(12)Thrombocytopenia(11)Beta-Thalassemia(10)Anemia of Chronic Kidney Disease(7)Iron Deficiency Anemia(6)