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The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended that ixazomib (NinlaroTM) receive conditional marketing authorization.
The recommendation is for ixazomib in combination with lenalidomide and dexamethasone as a treatment for adults with multiple myeloma (MM) who have received at least 1 prior therapy.
The CHMP’s recommendation will be reviewed by the European Commission (EC).
If the EC grants the authorization, ixazomib will be the first oral proteasome inhibitor approved for use across the European Economic Area.
Ixazomib is being developed by Takeda Pharmaceutical Company Limited.
Phase 3 trial
The CHMP’s positive opinion of ixazomib is based on results from the phase 3 TOURMALINE-MM1 trial, which were presented at the 2015 ASH Annual Meeting.
The trial included 722 patients with relapsed or refractory MM. The patients were randomized to receive ixazomib, lenalidomide, and dexamethasone (IRd, n=360) or placebo, lenalidomide, and dexamethasone (Rd, n=362).
Baseline patient characteristics were similar between the treatment arms. Fifty-nine percent of patients in both arms had received 1 prior line of therapy, and 41% in both arms had 2 or 3 prior lines of therapy.
Seventy-eight percent of patients responded to IRd, and 72% responded to Rd (P=0.035). The rates of complete response were 12% and 7%, respectively (P=0.019).
At a median follow-up of about 15 months, the median progression-free survival was 20.6 months in the IRd arm and 14.7 months in the Rd arm. The hazard ratio was 0.742 (P=0.012).
At a median follow-up of about 23 months, the median overall survival had not been reached in either treatment arm.
The incidence of adverse events (AEs) was 98% in the IRd arm and 99% in the Rd arm. The incidence of grade 3 or higher AEs was 74% and 69%, respectively. The incidence of serious AEs was 47% and 49%, respectively.
Common AEs in the IRd and Rd arms, respectively, were diarrhea (45% vs 39%), constipation (35% vs 26%), nausea (29% vs 22%), vomiting (23% vs 12%), rash (36% vs 23%), back pain (24% vs 17%), upper respiratory tract infection (23% vs 19%), thrombocytopenia (31% vs 16%), peripheral neuropathy (27% vs 22%), peripheral edema (28% vs 20%), thromboembolism (8% vs 11%), and neutropenia (33% vs 31%).
About conditional authorization
Conditional marketing authorization represents an expedited path for approval. The EC grants this type of authorization before pivotal registration studies are completed.
Conditional marketing authorization is granted to products whose benefits are thought to outweigh their risks, products that address unmet needs, and products that are expected to provide a significant public health benefit.
If ixazomib receives conditional marketing authorization, Takeda will be required to provide post-approval updates on safety and efficacy analyses for TOURMALINE-MM1 and some other ongoing studies to demonstrate the treatment’s long-term effects. 
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended that ixazomib (NinlaroTM) receive conditional marketing authorization.
The recommendation is for ixazomib in combination with lenalidomide and dexamethasone as a treatment for adults with multiple myeloma (MM) who have received at least 1 prior therapy.
The CHMP’s recommendation will be reviewed by the European Commission (EC).
If the EC grants the authorization, ixazomib will be the first oral proteasome inhibitor approved for use across the European Economic Area.
Ixazomib is being developed by Takeda Pharmaceutical Company Limited.
Phase 3 trial
The CHMP’s positive opinion of ixazomib is based on results from the phase 3 TOURMALINE-MM1 trial, which were presented at the 2015 ASH Annual Meeting.
The trial included 722 patients with relapsed or refractory MM. The patients were randomized to receive ixazomib, lenalidomide, and dexamethasone (IRd, n=360) or placebo, lenalidomide, and dexamethasone (Rd, n=362).
Baseline patient characteristics were similar between the treatment arms. Fifty-nine percent of patients in both arms had received 1 prior line of therapy, and 41% in both arms had 2 or 3 prior lines of therapy.
Seventy-eight percent of patients responded to IRd, and 72% responded to Rd (P=0.035). The rates of complete response were 12% and 7%, respectively (P=0.019).
At a median follow-up of about 15 months, the median progression-free survival was 20.6 months in the IRd arm and 14.7 months in the Rd arm. The hazard ratio was 0.742 (P=0.012).
At a median follow-up of about 23 months, the median overall survival had not been reached in either treatment arm.
The incidence of adverse events (AEs) was 98% in the IRd arm and 99% in the Rd arm. The incidence of grade 3 or higher AEs was 74% and 69%, respectively. The incidence of serious AEs was 47% and 49%, respectively.
Common AEs in the IRd and Rd arms, respectively, were diarrhea (45% vs 39%), constipation (35% vs 26%), nausea (29% vs 22%), vomiting (23% vs 12%), rash (36% vs 23%), back pain (24% vs 17%), upper respiratory tract infection (23% vs 19%), thrombocytopenia (31% vs 16%), peripheral neuropathy (27% vs 22%), peripheral edema (28% vs 20%), thromboembolism (8% vs 11%), and neutropenia (33% vs 31%).
About conditional authorization
Conditional marketing authorization represents an expedited path for approval. The EC grants this type of authorization before pivotal registration studies are completed.
Conditional marketing authorization is granted to products whose benefits are thought to outweigh their risks, products that address unmet needs, and products that are expected to provide a significant public health benefit.
If ixazomib receives conditional marketing authorization, Takeda will be required to provide post-approval updates on safety and efficacy analyses for TOURMALINE-MM1 and some other ongoing studies to demonstrate the treatment’s long-term effects.
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended that ixazomib (NinlaroTM) receive conditional marketing authorization.
The recommendation is for ixazomib in combination with lenalidomide and dexamethasone as a treatment for adults with multiple myeloma (MM) who have received at least 1 prior therapy.
The CHMP’s recommendation will be reviewed by the European Commission (EC).
If the EC grants the authorization, ixazomib will be the first oral proteasome inhibitor approved for use across the European Economic Area.
Ixazomib is being developed by Takeda Pharmaceutical Company Limited.
Phase 3 trial
The CHMP’s positive opinion of ixazomib is based on results from the phase 3 TOURMALINE-MM1 trial, which were presented at the 2015 ASH Annual Meeting.
The trial included 722 patients with relapsed or refractory MM. The patients were randomized to receive ixazomib, lenalidomide, and dexamethasone (IRd, n=360) or placebo, lenalidomide, and dexamethasone (Rd, n=362).
Baseline patient characteristics were similar between the treatment arms. Fifty-nine percent of patients in both arms had received 1 prior line of therapy, and 41% in both arms had 2 or 3 prior lines of therapy.
Seventy-eight percent of patients responded to IRd, and 72% responded to Rd (P=0.035). The rates of complete response were 12% and 7%, respectively (P=0.019).
At a median follow-up of about 15 months, the median progression-free survival was 20.6 months in the IRd arm and 14.7 months in the Rd arm. The hazard ratio was 0.742 (P=0.012).
At a median follow-up of about 23 months, the median overall survival had not been reached in either treatment arm.
The incidence of adverse events (AEs) was 98% in the IRd arm and 99% in the Rd arm. The incidence of grade 3 or higher AEs was 74% and 69%, respectively. The incidence of serious AEs was 47% and 49%, respectively.
Common AEs in the IRd and Rd arms, respectively, were diarrhea (45% vs 39%), constipation (35% vs 26%), nausea (29% vs 22%), vomiting (23% vs 12%), rash (36% vs 23%), back pain (24% vs 17%), upper respiratory tract infection (23% vs 19%), thrombocytopenia (31% vs 16%), peripheral neuropathy (27% vs 22%), peripheral edema (28% vs 20%), thromboembolism (8% vs 11%), and neutropenia (33% vs 31%).
About conditional authorization
Conditional marketing authorization represents an expedited path for approval. The EC grants this type of authorization before pivotal registration studies are completed.
Conditional marketing authorization is granted to products whose benefits are thought to outweigh their risks, products that address unmet needs, and products that are expected to provide a significant public health benefit.
If ixazomib receives conditional marketing authorization, Takeda will be required to provide post-approval updates on safety and efficacy analyses for TOURMALINE-MM1 and some other ongoing studies to demonstrate the treatment’s long-term effects.