Last reviewed · How we verify
Cladribine Low Dose
Cladribine Low Dose is a Purine nucleoside analog Small molecule drug developed by Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany. It is currently in Phase 3 development for Multiple sclerosis (relapsing-remitting and progressive forms), Lymphoproliferative disorders.
Cladribine is a purine nucleoside analog that depletes lymphocytes by inhibiting ribonucleotide reductase and inducing apoptosis in dividing cells.
Cladribine is a small molecule DNA inhibitor used in the treatment of various conditions, including Acute Myeloid Leukemia (AML) and Refractory Hematologic Cancer. It is typically administered as part of a combination therapy, such as in the study NCT07423104, which investigated its use in conjunction with low dose cytarabine and venetoclax for relapsed or refractory AML.
-
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). -
Immunology slight uplift
+1.0pp
Mature endpoint landscape (ACR, DAS28, PASI) makes immunology approvals slightly more predictable. -
Big-pharma sponsor
+3.0pp
Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany is a top-20 pharma sponsor — historical approval rates run ~3pp above average due to scale, regulatory experience, and trial-design quality.
| 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 | Cladribine Low Dose |
|---|---|
| Sponsor | Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany |
| Drug class | Purine nucleoside analog |
| Target | Ribonucleotide reductase; DNA synthesis |
| Modality | Small molecule |
| Therapeutic area | Immunology |
| Phase | Phase 3 |
Mechanism of action
Cladribine is a deoxyadenosine analog that is phosphorylated intracellularly and incorporated into DNA, leading to strand breaks and cell death. It preferentially affects lymphocytes due to their high deoxycytidine kinase activity and low 5'-nucleotidase activity. The low-dose formulation is designed to achieve sustained lymphocyte depletion while minimizing toxicity to other cell populations.
Approved indications
- Multiple sclerosis (relapsing-remitting and progressive forms)
- Lymphoproliferative disorders
Common side effects
- Lymphopenia
- Infections
- Anemia
- Thrombocytopenia
- Nausea
- Headache
Key clinical trials
- Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad) (PHASE3)
- IMPACT-AML: A Randomized Pragmatic Clinical Trial for Relapsed or Refractory Acute Myeloid Leukemia. (PHASE3)
- A Study of Cladribine, Low Dose Cytarabine, and Venetoclax in Treatment of Relapsed/Refractory or Secondary Acute Myeloid Leukemia (PHASE2)
- Cladribine Plus Low Dose Cytarabine (LDAC) Alternating With Decitabine in Patients With Acute Myeloid Leukemia (AML) or High-Risk Myelodysplastic Syndrome (MDS) (PHASE2)
- Alternating Regimen of VA and Low-dose CHA in the Treatment of Unfit Newly Diagnosed AML (PHASE2)
- A Phase II Study of Cladribine and Low Dose Cytarabine in Combination With Venetoclax, Alternating With Azacitidine and Venetoclax, in Patients With Higher-risk Myeloproliferative Chronic Myelomonocytic Leukemia or Higher-risk Myelodysplastic Syndromes With Excess Blasts (PHASE2)
- CAV Regimen for R/R AML (PHASE2)
- CAV Regimen for R/R Ph- B-ALL (NA)
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:
- Cladribine Low Dose CI brief — competitive landscape report
- Cladribine Low Dose updates RSS · CI watch RSS
- Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany portfolio CI
Frequently asked questions about Cladribine Low Dose
What is Cladribine Low Dose?
How does Cladribine Low Dose work?
What is Cladribine Low Dose used for?
Who makes Cladribine Low Dose?
What drug class is Cladribine Low Dose in?
What development phase is Cladribine Low Dose in?
What are the side effects of Cladribine Low Dose?
What does Cladribine Low Dose target?
Related
- Drug class: All Purine nucleoside analog drugs
- Target: All drugs targeting Ribonucleotide reductase; DNA synthesis
- Manufacturer: Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany — full pipeline
- Therapeutic area: All drugs in Immunology
- Indication: Drugs for Multiple sclerosis (relapsing-remitting and progressive forms)
- Indication: Drugs for Lymphoproliferative disorders
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing