The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending an indication extension of arsenic trioxide.

The indication extension is for use in newly diagnosed low to intermediate risk acute promyelocytic leukaemia (APL) in combination with retinoic acid.

Arsenic trioxide, in combination with retinoic acid, has shown a very high overall survival rate with almost no relapses after more than four years (50 months) of median follow-up.

If the European Commission approves this label extension, it would mark the first time that a form of acute leukaemia can be effectively treated with a regimen that is entirely chemotherapy-free.

APL is a life-threatening type of leukaemia as it can cause uncontrollable bleeding and can kill within hours or days if left untreated. 

In Europe, approximately 1,500 to 2,000 people are diagnosed with APL each year.

In light of its rarity, and because most cases present with low blood cell count and low leukaemic cells in the blood, diagnosis can be difficult.

However, the rapid progression of APL leading to early mortality is a substantial problem, affecting up to 30% of patients.

Rapid diagnosis and commencement of treatment is essential to avoid early mortality.

Arsenic trioxide is currently indicated for second line treatment of patients, who have not responded to treatment with retinoids and chemotherapy, or when their disease has returned after this type of treatment.

Commenting on the announcement, Francesco Lo-Coco, Professor of Haematology and Head of the Laboratory of Integrated Diagnosis of Oncohematologic Diseases, Department of Biomedicine and Prevention, University of Rome Tor Vergata, Italy said, “This CHMP opinion is very encouraging. Considering it was based on existing published academic data only, this opinion points to a recognition by the EMA that treating low to intermediate risk APL with a chemo-free regimen of arsenic trioxide plus retinoic acid can increase survival rates and dramatically reduce the risk of relapse and chemotherapy-related side effects in patients suffering from this rare and aggressive form of leukaemia. In particular, avoiding the risk of life-threatening infection and that of developing secondary leukaemias due to chemotherapy is a great gain for patients. The success of this regimen represents a major breakthrough and a paradigm of targeted therapy in oncology and medicine. This is therefore good news, not only for APL patients, but also for the whole medical community.”

The CHMP positive opinion is a formal recommendation to grant marketing authorization for an extended indication for first line treatment for arsenic trioxide.

The recommendation will now be reviewed by the European Commission, which has authority to approve medicines for use in the 28 countries of the European Union along with Norway, Liechtenstein and Iceland.

A final decision by the European Commission is expected by the end of the year.

In commenting on the CHMP positive opinion, Rob Koremans, President & CEO, Teva Global Specialty Medicines (who market arsenic trioxide as Trisenox®) said, “As a company committed to providing medicines and solutions that really make a difference in patients’ lives, we’re pleased to reach this important milestone, and hope soon to be able to offer a chemotherapy-free treatment regimen for APL patients at the point of diagnosis. Recognizing the high unmet patient need in this orphan disease, we’ve put everything in place to obtain the label extension for this life-saving treatment. We look forward to receiving an approval from the European Commission for Trisenox® as a first line treatment.”

Source: CHMP