Quick answer · last reviewed 1 November 2026
Grafapex cost at its US list price is US$610 per 1 g vial and US$3,050 per 5 g vial — the same US$610 per gram either way.[3] A full course of 10 g/m² a day for 3 days uses 30 g per m² of body surface area, so about US$32,940 at list price for a 1.8 m² adult, before discounts and excluding fludarabine and hospital charges.
Grafapex (treosulfan) is an alkylating agent the US FDA approved on 22 January 2025, with fludarabine, as a preparative (conditioning) regimen before allogeneic haematopoietic stem cell transplant in adults and children aged 1 year and older with acute myeloid leukaemia (AML) or myelodysplastic syndrome (MDS).[1][2]
Grafapex cost: list price per vial and per course
Medexus set the wholesale acquisition cost (WAC) at launch on 24 February 2025: US$610 per 1 g vial and US$3,050 per 5 g vial, and said the price may change over the product’s life cycle.[3] Its later quarterly and annual results releases, up to June 2026, did not publish a revised price.[9]
Because the price works out to US$610 per gram for both vial sizes, the drug cost of a course depends only on body surface area (BSA). The label dose is 10 g/m² a day on three days, so 30 g/m² per course.[1]
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| Body surface area | Daily dose | Course (3 days) | List-price drug cost |
|---|---|---|---|
| 1.5 m² | 15 g | 45 g | US$27,450 |
| 1.8 m² | 18 g | 54 g | US$32,940 |
| 2.0 m² | 20 g | 60 g | US$36,600 |
WAC is the manufacturer’s list price to wholesalers. It is not what a hospital, insurer or patient finally pays. The figures above are arithmetic from the published WAC, not a quoted price.
Grafapex coupon, insurance and prior authorization
- No manufacturer coupon or copay card was listed for Grafapex on the Drugs.com price guide when checked on 1 November 2026. That page lists third-party foundation programmes instead.[6] Grafapex is given by IV infusion in the transplant unit as part of conditioning, so it is supplied through the hospital rather than collected from a retail pharmacy.[1]
- Medicare: CMS approved a New Technology Add-On Payment (NTAP) for eligible inpatient cases using Grafapex from 1 October 2025, worth up to US$21,411 per case in CMS fiscal year 2026 (to 30 September 2026).[5] Check the current IPPS final rule for its status in fiscal year 2027.
- Commercial insurers: expect prior authorization. Cigna’s coverage policy IP0727 (effective 1 May 2026), for example, requires it and approves Grafapex for 30 days when the patient has AML or MDS, is aged 1 year or older, receives it with fludarabine before allogeneic transplant, and it is prescribed by or with a haematologist, oncologist or transplant physician.[4] Patients new to the process can read our step-by-step guide to prior authorization.
How Grafapex (treosulfan) works
Treosulfan is an alkylating agent; DNA alkylation is thought to be responsible for its cytotoxic activity. It is a prodrug: under physiological conditions it converts spontaneously into an active monoepoxide intermediate and then into diepoxybutane, which alkylate DNA.[1] In a conditioning regimen this kills the patient’s existing marrow cells and suppresses the immune system so that donor stem cells can engraft. That is why transplant is mandatory after dosing.
Grafapex dosage and administration
The label schedule counts days relative to the stem cell infusion on Day 0, so the dosing days are negative numbers.[1]
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| Day | Treosulfan (Grafapex) | Fludarabine |
|---|---|---|
| −6 | — | 30 mg/m² IV |
| −5 | — | 30 mg/m² IV |
| −4 | 10 g/m² IV over 2 h | 30 mg/m² IV |
| −3 | 10 g/m² IV over 2 h | 30 mg/m² IV |
| −2 | 10 g/m² IV over 2 h | 30 mg/m² IV |
| 0 | Allogeneic stem cell infusion | |
- Premedication: give antiemetics before the first dose and continue them on a fixed schedule through treosulfan dosing.[1]
- Reconstitution: 0.45% or 0.9% sodium chloride, 5% dextrose or Sterile Water for Injection — 20 mL for the 1 g vial and 100 mL for the 5 g vial (about 0.05 g/mL). Sterile Water alone is not recommended in children aged 12 or younger because of hypo-osmolarity. The reconstituted solution keeps up to 24 hours at room temperature; do not refrigerate.[1]
- Do not exceed 10 g/m² a day. A trial using 14 g/m² a day was stopped early because of more early fatal or serious adverse reactions.[1]
- Renal and hepatic impairment: mild impairment showed no clinically significant change in exposure; the effect of moderate or severe impairment is unknown.[1]
Grafapex vs busulfan: what the MC-FludT.14/L trial showed
Approval rests on MC-FludT.14/L (NCT00822393), a randomised, active-controlled trial of treosulfan plus fludarabine against busulfan plus fludarabine. It enrolled adults aged 18 to 70 who were at increased risk with standard myeloablative conditioning, because they were aged 50 or older, had an HSCT comorbidity index above 2, or both. The comparator was a reduced-intensity busulfan regimen.[7]
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| Measure | Treosulfan | Busulfan | Source |
|---|---|---|---|
| Patients randomised (FDA label) | 280 | 290 | Label[1] |
| Overall survival, all randomised | HR 0.67 (95% CI 0.51–0.90) | Label[1] | |
| Overall survival, AML / MDS subgroups | AML HR 0.73 (0.51–1.06); MDS HR 0.64 (0.40–1.02) | Label[1] | |
| 2-year event-free survival (published primary endpoint) | 64.0% | 50.4% | Lancet Haematol[7] |
| Grade 3–4 infections to Day +30 | 12% | 6% | Label[1] |
| Hepatotoxicity, all grades | 10% | 8% | Label[1] |
The published confirmatory analysis covered 476 patients enrolled between June 2013 and May 2016. It met non-inferiority and superiority on event-free survival (HR 0.65, 95% CI 0.47–0.90).[7] The FDA label reports overall survival for the full 570 randomised patients. Both subgroup confidence intervals cross 1, so the AML-only and MDS-only results are not individually conclusive.[1]
Correction to the earlier version of this page: it said Grafapex caused fewer severe infections than busulfan. The label shows the opposite: grade 3–4 infections occurred in 12% of Grafapex patients against 6% on busulfan through Day +30.[1] The earlier claim of “reduced hepatotoxicity” is also not supported by the label figures above.
Grafapex side effects and boxed warning
Boxed warning — myelosuppression. Grafapex causes severe and prolonged myelosuppression at the recommended dose. Haematopoietic stem cell transplant is required to prevent potentially fatal complications, and blood counts must be monitored.[1]
Common adverse reactions (20% or more, to Day +30)
Musculoskeletal pain 39%, stomatitis 38%, pyrexia 34%, nausea 33%, oedema 29%, infection 23% and vomiting 22%.[1]
Warnings and precautions on the label
- Seizures have been reported. Clonazepam prophylaxis may be considered for patients at higher risk, including infants.[1]
- Skin disorders, including diaper dermatitis from treosulfan excreted in urine. The label advises frequent diaper changes for 12 hours after each infusion.[1]
- Injection-site reactions and tissue necrosis if the infusion extravasates.[1]
- Secondary malignancies: treosulfan is carcinogenic and genotoxic. Patients with Fanconi anaemia or other DNA-breakage disorders may be at higher risk; they were not studied.[1]
- Increased early morbidity and mortality at doses above the recommended 10 g/m² a day.[1]
- Embryo-fetal toxicity (see below).[1]
Precautions: pregnancy, children, older adults and interactions
- Pregnancy: not a listed contraindication (the only contraindication is hypersensitivity), but treosulfan can harm a fetus. Females should use contraception during treatment and for 6 months after the last dose; males with partners of reproductive potential, for 3 months.[1]
- Breastfeeding: not during treatment or for 1 week after the last dose.[1]
- Children: safety and efficacy are established from age 1 year, not below it. Hepatic and gastrointestinal adverse reactions were more frequent in children than in adults. The dose is the same 10 g/m² a day.[1]
- Older adults: 27% of trial patients were aged 65 to 74 and none were 75 or older; no significant differences in safety or effectiveness were seen.[1]
- Drug interactions: treosulfan inhibits CYP2C19 and CYP3A4. The label gives no strength rating, but advises monitoring for toxicity of sensitive substrates of these enzymes and reducing their dose as needed.[1]
Who makes Grafapex, and how it is supplied
- Developer and licence: treosulfan was developed by medac GmbH (Germany); Medexus Pharmaceuticals holds exclusive US commercial rights under a February 2021 licence.[2]
- Manufacture and distribution: manufactured by Oncotec Pharma Produktion GmbH, Dessau-Roßlau, Germany; distributed by Medexus Pharma, Inc., Chicago.[1]
- Presentations: single-dose vials of lyophilised powder, 1 g (NDC 59137-335-01) and 5 g (NDC 59137-365-01). Store at 20–25 °C. Treosulfan is a hazardous drug, so follow special handling and disposal procedures.[1]
- Launch: commercially available in the US from 24 February 2025.[3]
- Exclusivity: Grafapex holds orphan drug designation, which Medexus says gives seven years of regulatory exclusivity in the approved indication.[8] Medexus’s January 2025 approval release had also used the phrase “up to seven-and-a-half years”.[2]
- Uptake (company-reported): as of 25 June 2026, 74 of the 180 US transplant centres had ordered Grafapex. Product-level net revenue was US$11.6 million in the year to 31 March 2026, and Medexus guided US$30–32 million for fiscal 2027.[9]
Side-effect reporting and product enquiries
Report suspected adverse reactions to Medexus on 1-855-336-3322, or to the FDA on 1-800-FDA-1088 or at fda.gov/medwatch.[1] Patients should ask their transplant team about access and costs. Hospitals source Grafapex through Medexus’s US distribution channel, which used a single wholesaler as of March 2026.[9] Corporate information is at medexus.com.
Grafapex was one of the FDA’s novel approvals of 2025. See it alongside the others in our FDA novel drug approvals 2025 tracker.
Grafapex FAQ
At launch in February 2025 Medexus set the US wholesale acquisition cost at US$610 per 1 g vial and US$3,050 per 5 g vial, which is US$610 per gram. A course of 10 g/m² a day for three days therefore costs about US$18,300 per square metre of body surface area at list price, or about US$32,940 for a 1.8 m² adult, before discounts and excluding fludarabine and hospital charges.
No manufacturer coupon or copay card was listed on the Drugs.com price guide when checked on 1 November 2026. Grafapex is infused in hospital during transplant conditioning, so its cost is normally handled through the transplant admission and the patient’s insurance rather than a pharmacy coupon. Patients should ask the transplant centre’s financial team about coverage and any foundation assistance.
Commercial insurers commonly require it. Cigna’s policy IP0727, effective 1 May 2026, requires prior authorization and approves Grafapex for 30 days when the patient has AML or MDS, is aged 1 year or older, receives it with fludarabine before an allogeneic stem cell transplant, and it is prescribed by or with a haematologist, oncologist or transplant physician. Other insurers set their own criteria.
In the MC-FludT.14/L trial, treosulfan plus fludarabine improved overall survival against reduced-intensity busulfan plus fludarabine (HR 0.67, 95% CI 0.51 to 0.90), and the published 2-year event-free survival was 64.0% against 50.4%. Grade 3 to 4 infections through Day +30 were more common with treosulfan (12% against 6%). The trial enrolled adults aged 18 to 70 at increased risk with standard conditioning.
Grafapex is approved from age 1 year at the same 10 g/m² daily dose as adults. Children had more hepatic and gastrointestinal adverse reactions than adults. Sterile Water alone should not be used for reconstitution in children aged 12 or younger, diapers should be changed frequently for 12 hours after each infusion, and clonazepam prophylaxis may be considered for those at higher seizure risk, including infants.
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- Medexus Pharma, Inc. GRAFAPEX (treosulfan) for injection, for intravenous use: prescribing information. Revised 1/2025. Bethesda (MD): National Library of Medicine, DailyMed; 2025. Available from: dailymed.nlm.nih.gov. Accessed Nov 2026.
- Medexus Pharmaceuticals. Medexus announces FDA approval of GRAFAPEX (treosulfan) for injection and provides business update [press release]. 22 Jan 2025. Available from: newsfilecorp.com. Accessed Nov 2026.
- Medexus Pharmaceuticals. Medexus announces commercial availability of GRAFAPEX (treosulfan) for injection [press release]. 24 Feb 2025. Available from: medexus.com. Accessed Nov 2026.
- Cigna Healthcare. Transplantation – Grafapex. Coverage policy IP0727, effective 1 May 2026. Available from: static.cigna.com. Accessed Nov 2026.
- Medexus Pharmaceuticals. GRAFAPEX (treosulfan) for injection receives CMS approval of New Technology Add-On Payment (NTAP) for eligible cases in CMS’s fiscal year 2026 [press release]. 5 Aug 2025. Available from: newsfilecorp.com. Accessed Nov 2026.
- Drugs.com. Grafapex prices, coupons, copay cards and patient assistance [secondary source]. Available from: drugs.com. Accessed 1 Nov 2026.
- Beelen DW, Trenschel R, Stelljes M, et al. Treosulfan or busulfan plus fludarabine as conditioning treatment before allogeneic haemopoietic stem cell transplantation for older patients with acute myeloid leukaemia or myelodysplastic syndrome (MC-FludT.14/L): a randomised, non-inferiority, phase 3 trial. Lancet Haematol. 2020;7(1):e28–e39. doi:10.1016/S2352-3026(19)30157-7. Available from: PubMed 31606445.
- Medexus Pharmaceuticals. Medexus provides business update on GRAFAPEX (treosulfan) for injection, capital allocation and investor conference participation [press release]. 15 Apr 2026. Available from: newsfilecorp.com. Accessed Nov 2026.
- Medexus Pharmaceuticals. Medexus announces fiscal year 2026 results, demonstrating continued momentum and strong product-level performance of GRAFAPEX (treosulfan) for injection [press release]. 25 Jun 2026. Available from: newsfilecorp.com. Accessed Nov 2026.
Disclaimer: technical and educational content for healthcare and pharmaceutical professionals. It is not medical, reimbursement or investment advice. Prices, coverage policies and labels change; confirm against the current FDA label and your payer before acting. Reviewed by Darshan Singh, pharmaceutical QA/QC and regulatory professional (23+ years), on 1 November 2026.
