Otsuka Pharmaceutical Netherlands B.V. has officially retracted its application to extend the use of Inaqovi for treating specific blood disorders. The company withdrew its request for chronic myelomonocytic leukaemia (CMML) on August 5, 2024, and for myelodysplastic syndromes (MDS) on November 6, 2024, following concerns raised by the European Medicines Agency (EMA).
Details of the Withdrawal
Inaqovi, containing cedazuridine and decitabine, is currently approved for adult patients with newly diagnosed acute myeloid leukaemia (AML) who are unsuitable for standard chemotherapy. The expansion aimed to include patients with MDS and CMML, conditions where the bone marrow fails to produce sufficient healthy blood cells. However, during the assessment process, Otsuka limited the MDS indication to higher-risk cases, ultimately deciding to withdraw both applications due to unresolved issues.
EMA’s Concerns and Company’s Response
The EMA expressed doubts about the clinical benefits of Inaqovi for MDS and CMML, questioning whether the advantages outweighed the associated risks. In response, Otsuka acknowledged these concerns but stated it could not address the EMA’s major objections at that time, leading to the withdrawal of both applications. This decision does not impact ongoing clinical trials involving Inaqovi.
- The withdrawal highlights the stringent evaluation criteria for expanding drug indications.
- Otsuka’s decision may influence future applications for similar therapies.
- Patients currently in clinical trials remain unaffected by this withdrawal.
Ongoing studies showed varying responses, but the EMA remained unconvinced of sufficient clinical benefits for the new indications. As a result, Inaqovi remains authorized solely for AML treatment within the EU.
The company’s strategic withdrawal underscores the challenges pharmaceutical companies face in meeting regulatory standards for broader drug applications. It also emphasizes the importance of robust clinical data to support efficacy and safety claims in new therapeutic areas.
Inaqovi’s continued authorization for AML offers a vital treatment option for patients not eligible for standard chemotherapy, maintaining its role in the current therapeutic landscape. Healthcare professionals and patients are advised to stay informed about any future developments regarding Inaqovi’s applications.
This move by Otsuka may prompt a re-evaluation of strategies when seeking approvals for additional indications, ensuring that future applications address regulatory concerns more comprehensively to achieve successful authorization.
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