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hATG

Phase 3

Severe Aplastic Anemia | Small molecule | Hematology |Novartis AG|Last Updated: Apr 24, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment202
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02099747hATG+CsA vs hATG+CsA+Eltrombopag for SAAPHASE3 COMPLETED 202Jul 1, 2015Dec 1, 2020Apr 24, 202629 France, Italy +4
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Study Endpoints
Primary Endpoints
CR rate
3 months

The primary objective of this trial is to investigate whether Eltrombopag added to standard immunosuppressive treatment increases the rate of early (at three months) complete response in untreated AA patient.

Secondary Endpoints
Time to best heamatological response
2 year
Heamatological Response at 6, 12, 18 and 24 months
2 year
Cumulative incidence of response
2 year
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
hATG + CsAACTIVE_COMPARATORControl Arm
hATG + CsA + EltrombopagEXPERIMENTALExperimental
Interventions
NameTypeDescription
hATGDRUG -
CsADRUG -
EltrombopagDRUG -
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Eligibility Criteria
Age Range15 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites29

Inclusion Criteria: 1. Diagnosis of severe or very severe aplastic anemia, defined by \[29\]: * At least two of the following: * Absolute neutrophil counts \<0.5 x 109/L (severe) or \<0.2 x 109/L (very severe) * Platelet counts \<20 x 109/L * Reticulocyte counts \<60 x 109/L ...

Countries:FranceItalyNetherlandsSpainSwitzerlandUnited Kingdom
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