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IST (ATG + CsA)
IST (ATG + CsA) is a Immunosuppressive combination therapy Small molecule drug developed by Federal Research Institute of Pediatric Hematology, Oncology and Immunology. It is currently in Phase 3 development for Prevention of graft-versus-host disease in hematopoietic stem cell transplantation, Aplastic anemia treatment. Also known as: controle.
IST combines anti-thymocyte globulin (ATG) and cyclosporine A (CsA) to suppress T-cell mediated immune responses and prevent graft rejection or autoimmune destruction.
IST (ATG + CsA) is a treatment modality used for conditions such as Acquired Aplastic Anemia and Aplastic Anemia, which involves the use of a protein-based intervention. The treatment combines the use of ATG (Antithymocyte globulin) and CsA (Cyclosporine), which are administered together to manage these conditions.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Oncology Phase 3 boost
+3.0pp
Oncology Phase 3 trials have higher approval rates (~61%) than the cross-industry average due to clearer endpoints and FDA oncology pathway.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
At a glance
| Generic name | IST (ATG + CsA) |
|---|---|
| Also known as | controle |
| Sponsor | Federal Research Institute of Pediatric Hematology, Oncology and Immunology |
| Drug class | Immunosuppressive combination therapy |
| Target | T lymphocytes (ATG); calcineurin (CsA) |
| Modality | Small molecule |
| Therapeutic area | Immunology / Hematology-Oncology |
| Phase | Phase 3 |
Mechanism of action
ATG is a polyclonal antibody that depletes T lymphocytes, while cyclosporine A is a calcineurin inhibitor that blocks T-cell activation and cytokine production. Together, this combination provides potent immunosuppression through complementary mechanisms: ATG reduces the T-cell population while CsA inhibits the remaining T cells' proliferation and function. This dual approach is used to prevent graft-versus-host disease (GVHD) and improve engraftment in hematopoietic stem cell transplantation.
Approved indications
- Prevention of graft-versus-host disease in hematopoietic stem cell transplantation
- Aplastic anemia treatment
Common side effects
- Infection (bacterial, viral, fungal)
- Nephrotoxicity
- Hypertension
- Neurotoxicity
- Hepatotoxicity
- Thrombotic microangiopathy
Key clinical trials
- A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202) (PHASE3)
- HID-HSCT Versus IST as First-line Treatment for SAA (NA)
- UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia (PHASE2)
- A Study of Emapalumab for Pediatric Aplastic Anemia (PHASE2)
- Dose-attenuated IST and Hetrombopag in Elderly (≥65 Years) Patients With Severe Aplastic Anemia (PHASE2)
- Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia (NA)
- Efficacy of Romiplostim in Treatment of SAA in Adults Previously Untreated With or Refractory to Immunosuppressive Therapy (PHASE2)
- Efficacy of IST Combined With TPO-RA in the Treatment of AA and Establishment of a Recurrence Prediction System (PHASE4)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- IST (ATG + CsA) CI brief — competitive landscape report
- IST (ATG + CsA) updates RSS · CI watch RSS
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology portfolio CI
Frequently asked questions about IST (ATG + CsA)
What is IST (ATG + CsA)?
How does IST (ATG + CsA) work?
What is IST (ATG + CsA) used for?
Who makes IST (ATG + CsA)?
Is IST (ATG + CsA) also known as anything else?
What drug class is IST (ATG + CsA) in?
What development phase is IST (ATG + CsA) in?
What are the side effects of IST (ATG + CsA)?
What does IST (ATG + CsA) target?
Related
- Drug class: All Immunosuppressive combination therapy drugs
- Target: All drugs targeting T lymphocytes (ATG); calcineurin (CsA)
- Manufacturer: Federal Research Institute of Pediatric Hematology, Oncology and Immunology — full pipeline
- Therapeutic area: All drugs in Immunology / Hematology-Oncology
- Indication: Drugs for Prevention of graft-versus-host disease in hematopoietic stem cell transplantation
- Indication: Drugs for Aplastic anemia treatment
- Also known as: controle
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing