Drug intelligence / Profile preview

busulfan + melphalan + fludarabine + anti-thymocyte globulin + palifermin

Development stage
Unknown
Lead developer
GSK
Modality
Recombinant Proteins and Enzymes, Antibody-Based Therapeutics, Small Molecules
Administration
Intravenous
01

Overview

This is a **combination myeloablative conditioning regimen** used prior to allogeneic hematopoietic stem cell transplantation (allo-HSCT), especially for patients with hematologic malignancies such as acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)[3][7]. The regimen includes the following agents: - **Busulfan:** An alkylating agent that cross-links DNA, leading to inhibition of DNA replication and cell death. - **Melphalan:** Another alkylating agent that also cross-links DNA, providing potent anti-tumor activity. - **Fludarabine:** A purine analog antimetabolite inhibiting DNA synthesis, leading to immunosuppression and anti-leukemic effects[9]. - **Anti-thymocyte globulin (ATG):** A polyclonal antibody preparation that depletes T lymphocytes, providing additional immunosuppression to prevent graft rejection. - **Palifermin:** A recombinant human keratinocyte growth factor (KGF) used to reduce oral mucositis by stimulating epithelial cell proliferation and healing in the oral mucosa[2][10]. This combination is used to maximize anti-leukemic effect, ensure immunosuppression for graft acceptance, and reduce regimen-related toxicity such as severe oral mucositis. Palifermin is included specifically for its protective effects against mucositis, which can be a significant side effect of high-dose chemotherapy[2][6][7].

02

Targets

POLA1 (DNA polymerase alpha)RNR (Ribonucleotide reductase)DNADNA-directed primase/polymerase protein (PrimPol)FGFR2b (Fibroblast growth factor receptor 2 IIIb isoform)

Beyond the preview

Go deeper on busulfan + melphalan + fludarabine + anti-thymocyte globulin + palifermin.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Full profile access

Follow clinical development from study design and recruitment through results.

  • Trial phase
  • Status
  • Readouts

Indications & development

Full profile access

Explore development by indication, patient population, and geography.

  • Indications
  • Development status
  • Countries

Licensing & deals

Full profile access

Trace asset ownership, licensing agreements, and commercial partnerships.

  • Partners
  • Deal terms
  • Milestones

Patents & exclusivity

Full profile access

Explore the patent landscape and regulatory exclusivity around an asset.

  • Patents
  • Expiration dates
  • Exclusivity

Competitive landscape

Full profile access

Compare development programs by target, modality, and indication.

  • Competing assets
  • Targets
  • Development stage

Research & analysis

Full profile access

Connect source evidence and development news to your research questions.

  • Publications
  • News
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on busulfan + melphalan + fludarabine + anti-thymocyte globulin + palifermin.

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call