Is Grafapex Worth It in 2025? Pros, Cons, and Clinical Trial

In January 2025, the FDA approved Grafapex™ (treosulfan) in combination with fludarabine as a conditioning regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT).

This approval applies to adult and pediatric patients aged one year and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

What Is The Use Of Grafapex?

Grafapex (treosulfan) is a drug used to prepare patients for stem cell transplantation (allogeneic hematopoiesis) in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

It is used in combination with fludarabine. Treosulfan helps to destroy the remaining cancer cells in the body and weakens the patient’s immune system so that rejection of the donor stem cells is avoided.

Allogeneic Hematopoietic Stem Cell Transplantation (alloHSCT) is the process of receiving stem cells from blood or bone marrow from a donor who is genetically similar to you.

How Does Grafapex Work?

The mechanism of action of Treosulfan is an alkylating agent that kills cells by adding alkyl groups to the DNA structure, leading to depletion of hematopoietic stem cells, antitumor activity and its immunosuppressive effects.

Pros And Cons Of Grafapex

Pros of Grafapex

✅FDA Approval and Orphan Drug Designation
Treosulfan received FDA approval in January 2025 for use in combination with fludarabine as a preparative regimen for alloHSCT in patients with AML or MDS.

Its Orphan Drug designation provides up to seven years of marketing exclusivity, potentially leading to significant market share.

✅Potential Market Impact
Medexus plans to release Treosulfan in the U.S. market during the first half of 2025, with expectations of generating over $100 million in annual revenue within five years of launching.

Cons and Considerations

❗Safety Profile
Treosulfan carries a boxed warning for severe and prolonged myelosuppression, necessitating careful monitoring of hematologic parameters and supportive care.

❗Limited Indications
The FDA approval is specific to its use in combination with fludarabine for conditioning in alloHSCT for AML and MDS patients aged one year and older.

Its applicability outside these indications remains to be established.

How Much Does Grafapex Cost?

The price of Treosulfan, 1 gram of intravenous powder for injection, is approximately $661 (for reference).

Clinical Trial Overview

The pivotal study supporting Grafapex’s approval was the phase 3 MC-FludT.14/L trial (NCT00822393), a randomized, active-controlled trial comparing treosulfan plus fludarabine to busulfan plus fludarabine as preparative regimens for alloHSCT.

The study enrolled 570 patients aged 18 to 70 years with AML or MDS, who had a Karnofsky performance status ≥60% and either were aged ≥50 years or had a hematopoietic cell transplantation comorbidity index (HCT-CI) score >2.

The primary efficacy endpoint was overall survival (OS), which is the time from randomization to death from any cause. Trexaphan significantly improved overall survival compared to buthionine.

Trexaban was generally well tolerated. The most common adverse reactions (≥20%) included musculoskeletal pain, stomatitis, fever, nausea, edema, infection, and vomiting.

Clinical trial experience with Grafapex has demonstrated its efficacy and safety as a modifier for allogeneic organ transplantation in patients with acute myeloid leukemia and myelodysplastic syndromes, potentially replacing traditional sodium bisulfate-based regimens.

Grafapex Side Effect

The common Treosulfan side effects are:

⭕Abdominal pain

⭕Muscle and joint pain

⭕Inflammation of mouth or lips stomatitis 

⭕Fever

⭕Nausea

⭕Edema

⭕Infection

⭕Vomiting

⭕Rash

⭕Diarrhea

⭕Headache

⭕Febrile neutropenia

Drug Interactions

Effects of GRAFAPEX on Other Drugs (Certain CYP2C19 and CYP3A Substrates): Based on knowledge of the metabolism mechanism of trimethoprim, it is anticipated that concomitant use of Treosulfan will increase exposure to CYP2C19 and CYP3A4 substrates, which may increase the risk of adverse reactions.

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