Health Canada Will Review Request for Masitinib as Add-on Therapy

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Health Canada has authorized a new drug submission for masitinib — AB Science’s add-on therapy for amyotrophic lateral sclerosis (ALS) — under the notice of compliance with conditions policy (NOC/c), designed to get new medications to patients more quickly.

If granted, this would allow conditional marketing of the therapy in Canada.

“We welcome the decision of Health Canada to authorize filing of the masitinib registration dossier in ALS. We are committed to work with Health Canada on this new drug submission, which could potentially lead to the rapid availability of masitinib to ALS patients through the NOC/c policy and ultimately to full registration,” Alain Moussy, co-founder and CEO of AB Science, said in a press release.

NOC/c authorization allows sooner market access to eligible therapies that show significant promise for the treatment of severe, life-threatening diseases, such as ALS.

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To receive permission to apply for NOC/c authorization, AB Science submitted safety and efficacy data, which were reviewed by a Health Canada committee. According to the company, masitinib was authorized for filing based on meeting two important criteria under the NOC/c policy.

First, masitinib is a treatment for a serious, life-threatening, and severely debilitating disease. Second, there is sufficient evidence that masitinib is clinically effective, safe, and presents a more favorable treatment profile than existing ALS therapies.

Specifically, AB Science submitted data from the AB10015 Phase 2/3 clinical trial (NCT02588677), which showed that 4.5 milligrams per kilogram (mg/kg) per day of masitinib given in combination with the approved therapy Rilutek (riluzole) safely and significantly slowed disease progression and extended survival by more than two years in patients with moderate ALS, showing a marked improvement over Rilutek alone.

Furthermore, according to the company, this extended survival has not been seen with Radicava (edaravone), an ALS therapy developed by Mitsubishi Tanabe Pharma that is already approved in Canada. Those findings, by a Health Canada Adjudicating Committee that assessed the therapy, suggest masitinib fills an unmet treatment need.

AB Science now has 60 days to file the application, which Health Canada will aim to review within 200 days, or less than seven months. If granted, the NOC/c authorization will allow for marketing of masitinib in Canada for use in ALS, though its availability would be subject to certain conditions, which would be determined during the review process.

“We thank all investigators and researchers who have supported the masitinib development program in ALS over several years. Above all, we are very pleased with this decision for the ALS patients and community, for whom masitinib may offer a new therapeutic hope,” Moussy said.

Masitinib is an oral medication that blocks the activity of tyrosine kinase enzymes, which subsequently reduces the activity of immune cells that drive inflammation in ALS.

“As a scientist and as the director of the scientific strategy of AB Science, I am very happy with this news. Masitinib’s selective targeting of the innate immune system … has proven to be the right strategy in neurodegenerative diseases and in particular for ALS,” said Olivier Hermine, president of AB Science’s scientific committee.

“For me, it is the unique mechanism of action of masitinib that explains the efficacy observed … on the slowing of functional decline … and the extended survival,” Hermine added.

A Phase 3 trial called AB19001 (NCT03127267) is seeking to expand on these previous findings, and will assess whether masitinib in combination with Rilutek leads to greater functional recovery, measured using the ALS Functional Rating Scale-revised (ALSFRS-R), than Rilutek alone.

The trial, which was originally paused due to safety concerns, has been cleared to resume enrollment. It is now recruiting up to 495 adults with mild to moderate functional impairments who were diagnosed with ALS within the last two years.

The company will host a webcast on Monday, Feb. 28, at 12 p.m. EST to provide more information about this application in Canada, and on its masitinib development program.