The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the use of lenalidomide as monotherapy for the maintenance treatment of adult patients with newly diagnosed multiple myeloma (MM) who have undergone autologous stem cell transplantation (ASCT).

Once approved by the European Commission, lenalidomide will be the first and only licensed maintenance treatment available to these patients.

Multiple myeloma is an incurable and life-threatening blood cancer that is characterised by tumour proliferation and suppression of the immune system.

It is a rare but deadly disease—around 39,000 people are diagnosed with MM in Europe, and around 24,000 people die from the disease each year.

The median age at diagnosis in Europe is between 65 and 70 years.

In Europe, patients who are under 65 years, fit and in good clinical condition are typically considered eligible for ASCT.

For newly diagnosed, transplant-eligible MM patients, key treatment goals are to obtain and to maintain a deep response to therapy, with the ultimate objective of delaying disease progression.

These patients typically receive induction therapy and high-dose chemotherapy with melphalan followed by ASCT.

This treatment approach has been an established standard of care for over 20 years.

Considering that over half of patients relapse within 2 to 3 years after ASCT, trials have been conducted to assess whether maintenance therapy following ASCT could enable more durable remissions.

“Studies show that maintenance treatment after ASCT with lenalidomide may help control residual malignant cells and delay tumour growth by enhancing immune function,” says Professor Michel Attal, Executive Director of the Institut Universitaire du Cancer Toulouse Oncopole and Institut Claudius Regaud, France. “Our primary goal is to delay disease progression for as long as possible, and we have seen in several independent studies, that lenalidomide maintenance after ASCT can halve the risk of disease progression by sustaining the response.”

The CHMP recommendation was based on the results of two cooperative group-led studies, CALGB 10010410 and IFM 2005-0211.

CALGB 100104 was a phase III, controlled, double-blind, multi-centre study of 460 patients with newly diagnosed MM undergoing ASCT who received continuous daily treatment with lenalidomIde or placebo until relapse.

IFM 2005-02 was an international, phase III, controlled, double-blind, multi-centre study of 614 patients newly diagnosed with MM who were randomised to receive a 2-month consolidation regimen post-ASCT of lenalidomide monotherapy, followed by continuous daily treatment with either lenalidomide or placebo until relapse.

In the two phase III studies, REVLIMID® monotherapy as maintenance treatment post-ASCT significantly reduced the risk of disease progression or death in patients with MM, leading to the studies being unblinded based on passing their pre-specified boundary for superiority at interim analysis.

In these studies, the safety profile was in line with other clinical data in newly diagnosed non-stem cell transplant (NSCT) and post-approval safety study in relapsed/refractory MM (rrMM).

Across both phase III clinical studies, the most commonly reported adverse events (AE) were haematological and included neutropenia and thrombocytopenia.

The most commonly reported non-haematological AE were infections.

In both trials, an increased incidence rate of haematologic second primary malignancies (SPMs) has been observed in the lenalidomide group compared with the placebo group.

However, the CHMP positive opinion confirms that the benefit-risk ratio for lenalidomide is positive in this expanded indication.

Lenalidomide is marketed by Celgene as Revlimid®.

Tuomo Pätsi, President of Celgene in Europe, the Middle East and Africa (EMEA), said, “Despite substantial progress made so far in multiple myeloma treatment, it remains an incurable disease. We welcome this CHMP opinion as it confirms the important role that REVLIMID® plays in treating multiple myeloma, extending the use of REVLIMID® across the disease continuum. At Celgene, we aspire to turn some of the most challenging diseases, like multiple myeloma, into manageable conditions. Therefore, we will continue to invest more than one-third of our revenues back into research and development.”

Source: BusinessWire