Strategic cornerstone for medac: FDA approves treosulfan for alloHSCT

medac has received FDA approval for treosulfan as a new drug application with orphan drug designation prior to allogeneic hematopoietic stem cell transplantation (alloHSCT) in adult and pediatric patients aged 1 year and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

Wedel, Germany – 23 January 2025, The US Food and Drug Administration (FDA) has approved treosulfan, an alkylating agent, in combination with fludarabine as a preparative regimen for alloHSCT in adult and pediatric patients aged 1 year and older with AML or MDS. Treosulfan will be marketed in the USA by Medexus Pharma, Inc. under the trade name “GRAFAPEX (treosulfan) for Injection”.

The FDA’s approval is based on the results of the MC-FludT.14/L Trial II (NCT00822393), a randomized active-controlled trial that compared treosulfan to busulfan with fludarabine as a preparative regimen for allogeneic transplantation. The major efficacy outcome of the trial considered by the FDA was overall survival (OS). Further details of the study can be found in the FDA and Medexus press releases.

“With this approval, medac has achieved a strategic cornerstone for strengthening its global position in the hematology field and is redefining treatment options in alloHSCT due to the toxicity-reduced conditioning regimen with GRAFAPEX,” said Frank Lucaßen, CEO of medac GmbH. “The outstanding experiences gained in Europe using treosulfan-based regimens and the FDA approval form the basis for further growth and geographic expansion of medac.”

Trecondi^® (treosulfan) has been approved by the European Medicines Agency (EMA) since 2019. Following the successful FDA approval, medac is delighted to offer this essential treatment to patients in the US, enhancing their quality of life. The commitment of medac to focus on patients ensures that their needs are at the forefront of all efforts, while medac is simultaneously reinforcing its role as a reliable partner in hematology.

 

About GRAFAPEX (treosulfan) for Injection

The active compound treosulfan is a prodrug and belongs to the group of bifunctional alkylating agents. Despite its structural similarity to busulfan, the treosulfan molecule shows a different mechanism of action due to its two hydroxyl groups. Under physiological conditions, an enzyme-independent intramolecular nucleophilic substitution takes place. Formation of an epoxide ring results in the elimination of two methane sulphonic acid molecules. The resulting molecules (1,2-epoxy-3,4-butanediol-4-methane sulfonate and L-diepoxybutane) are to be regarded as the chemically effective reaction products which bind to biological macromolecules and trigger the antiproliferative and cytotoxic effect.

 

About medac

At medac group, we believe that health is humanity’s most valuable resource. Since 1970, our mission has been to improve patients’ quality of life worldwide by making the best medical treatments available. As a globally operating pharmaceutical company headquartered in Germany, we provide high-quality medical treatments for patients worldwide in 91 countries. With more than 2.000 employees we are committed to improving human health.

Our products are manufactured in Germany and other European countries to the highest standards, utilizing our own logistics center and production sites, and subsequently distributed worldwide.

We are constantly working to improve authorized medicines and to develop innovative therapies in the fields of rheumatology, urology, hematology, and oncology. Part of our mission is to provide safe, high-quality and innovative original products, as well as generics and biosimilars. In this way, we make vital treatments accessible to those affected.

Further information on the company can be found online at www.medac-group.com