Masitinib Being Reviewed for OK in Canada as Add-on ALS Therapy
The application will be reviewed under the notice of compliance with conditions (NOC/c) policy, agreed upon with the agency in February. This policy allows early market access to therapies showing the potential to fill an unmet medical need in severe, life-threatening diseases, such as ALS.
Health Canada has 200 days, or about seven months, to decide whether to approve masitinib. If granted, the NOC/c authorization will allow it to be used in Canada for ALS, though its availability may be be subject to certain conditions, which will be determined during the review process.
“We look forward to working closely with Health Canada on this New Drug Submission and also continue to discuss masitinib with health authorities around the world to determine the most appropriate path to make masitinib available to ALS patients,” Moussy said.
Masitinib is an oral therapy that works by blocking tyrosine kinase, an enzyme essential for the activity of several immune cells thought to drive neuroinflammation and neurodegeneration. By suppressing these cells, the therapy is expected to slow disease progression in ALS and other neurodegenerative diseases.
To receive permission to apply for NOC/c authorization, AB Science pre-submitted a package of AB10015’s safety and efficacy data — including long-term survival data of about six years — that was reviewed by a Health Canada committee.
According to the company, the authorization was given based on the fact that masitinib meets two important NOC/c policy’s criteria: it’s designed to treat a serious, life-threatening, and severely debilitating disease, and it has sufficient evidence supporting a more favorable benefit/risk profile than existing ALS therapies.
AB Science submitted data from the AB10015 Phase 2/3 clinical trial (NCT02588677), which tested two doses of masitinib against a placebo in 394 ALS patients who were also receiving the approved therapy Rilutek (riluzole).
Results showed that a combination of Rilutek and masitinib’s higher dose (4.5 mg/kg) was generally safe and slowed ALS progression in patients with normal disease progression by 27%, compared with Rilutek alone.
This combination was also associated with a 44% reduced risk of death among patients with mild or moderate disease, extending their lives for more than two years compared with just Rilutek.
According to the company, this extended survival has not been seen with Radicava (edaravone), another therapy approved for ALS in Canada.
These findings suggest masitinib may fill an unmet medical need in ALS.
The results are being confirmed in a larger, international Phase 3 trial, called AB19001 (NCT03127267). The study, which had been paused due to safety concerns, has been cleared to resume enrolling up to 495 adults with mild to moderate functional impairments who were diagnosed with ALS within the last two years. More information on contacts and locations can be found here.