Data from the open-label extension trial of Radicava (edaravone) in patients with amyotrophic lateral sclerosis (ALS) demonstrated that the treatment continued to provide benefits after 48 weeks of treatment.
These findings — presented by researchers from MT Pharma America at the May 18-20 European Network for the Cure of ALS (ENCALS) Annual Meeting in Slovenia — strengthen the drug’s profile as a therapy capable of significantly slowing disease progression in ALS.
“The 12-month data presented at ENCALS suggest that early intervention with Radicava may lead to a meaningful clinical benefit when promptly initiated in people with ALS, as opposed to being delayed by six months,” Dr. Jean Hubble, vice-president for medical affairs at MT Pharma America, said in a press release. “We strongly believe in the potential of Radicava to help people with this devastating disease, and are committed to continuing to advance clinical evidence for this treatment option.”
The trial extension compared patients who had taken Radicava for 48 weeks to those treated with placebo for six months in the original Phase 3 trial (NCT01492686), before switching to Radicava for another 24 weeks in the open-label study.
Measurements of the ALS Functional Rating Scale-Revised (ALSFRS-R) showed that those who received Radicava for the full 48 weeks had significantly less physical decline and an about 58 percent lower relative risk of death or certain disease progression events. For instance, loss of the ability to use one’s arms was classified as a disease progression event.
Both groups had similar rates of new adverse events, except for bruising — which was a common side effect reported in Radicava-treated patients in the original Phase 3 trial.
The U.S. Food and Drug Administration (FDA) approved Radicava for ALS on May 5 as data from the six-month Japanese trial showed significantly slowed rates of physical decline among Radicava-treated patients. The approval is rare, as the FDA commonly requests trials also be performed in the United States before considering approval.
Radicava is an intravenous treatment, administered in 28-day cycles as a one-hour infusion. The first cycle is composed of infusions for two weeks, followed by two weeks off the drug. All following cycles contain only 10 infusion days — nevertheless making up an intense treatment schedule.
Radicava costs nearly $1,100 per infusion, or about $146,000 annually. The treatment should be available to patients by August.