On August 26, 2025 Oxcia reported its drug candidate OXC-101 received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of acute myeloid leukemia (Press release, Oxcia, AUG 26, 2025, View Source;utm_medium=rss&utm_campaign=ema-approves-odd-for-oxc-101-in-aml [SID1234655482]). Now, the European Medicines Agency (EMA) has also granted OXC-101 ODD approval for European markets.
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This approval significantly strengthens Oxcia’s position, both from a strategic, regulatory and commercial standpoint, according to Oxcia’s CEO Ulrika Warpman Berglund:
-These dual approvals give us up to 10 years of market exclusivity in the EU and 7 years in the US, accelerated regulatory processes and significant cost savings. EMA’s approval also provides support and scientific advice from EMA’s expert committee (COMP) for orphan drugs which is a large advantage.
EMA is also evaluating whether the drug candidate can offer significant benefit compared to existing treatments. Obtaining ODD from both the FDA and EMA, two of the world’s largest drug regulatory authorities, strengthens scientific credibility and differentiation from competitors. It facilitates the work of raising additional venture capital and signing licensing agreements with pharmaceutical companies.
Briefly about OXC-101 in AML
Oxcia’s lead drug candidate OXC-101 is a so-called "first-in-class" mitotic MTH1 inhibitor. It has a dual mechanism of action that allows it to target a weakness of cancer cells – their high levels of oxidative stress and their propensity to develop DNA damage. In short, OXC-101 induces further oxidative stress and prevents the cancer cells from repairing the DNA damage.
Preclinical studies have shown that OXC-101 significantly reduces tumor growth and prolongs survival in models of acute myeloid leukemia (AML). In addition, there is support for further development from clinical results from a phase I study in patients with advanced hematological cancers. In addition to OXC-101 having potential as a monotherapy with better efficacy than cytarabine (which is one of the standard treatments for AML) in AML disease models, OXC-101 may also further improve the efficacy of treatment with various standard combinations (various chemotherapies).
Oxcia is currently conducting an expansion study, a combined phase I/II study, in a selected group of patients with relapsed/recurrent AML. The treatment is given in combination with one of the standard treatments (idarubicin). Oxcia has obtained a Vinnova grant for the expansion phase of the study. The aim of the study is to confirm the promising preliminary effects previously observed, and to lay the foundation for a pivotal phase II study that could form the basis for a regulatory accelerated approval. In several of the patients in the MAATEO study, clinical benefits have been observed with a partial response and some stable disease up to 5 months, which is a long time for this aggressive disease in this phase. Some improvement in quality of life has also been reported.
In collaboration with Dr. Tom Erkers and Nona Struyf, Scilifelab/Karolinska Institute, so-called precision medicine studies are being carried out on bone marrow samples from patients in the MAATEO study. These studies aim to identify the patients with the best response. The method may be used to stratify patients in future studies.