Masitinib

The Phase 2/3 trial examined the safety and effectiveness of masitinib in combination with riluzole in 394 people with MND, when taken for nearly a year. The results showed that masitinib potentially slows disease progression and increases survival in a subgroup of people with MND. 

Researchers grouped participants from the trial based on their rate of disease progression and disease severity, using the ALSFRS-R. They defined normal progression as people living with ALS who had a change of less than 1.1 points per month on the ALSFRS-R scale. Moderate ALS was defined as people living with ALS with a starting ALSFRS-R score of greater than or equal to 2 in all categories. Participants who had both normal progression and moderate ALS were found to have a potential increase in survival of approximately 25 months when taking 4.5mg/kg of masitinib alongside riluzole, when compared to those who took placebo and riluzole. Results also suggested a potential slower decline in ALSFRS-R score per month for this subgroup. You can read more about the trial here or read the results here.

October 2024- Upon re-examination, the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency, have confirmed their recommendation of refusing conditional marketing authorisation for Masitinib for MND in Europe. AB Science are continuing a Phase 3 trial of the drug to collect more data.

June 2024- The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has announced a negative opinion for the conditional approval of Masitinib for the treatment of MND. This means that the CHMP felt that the data from the Phase 2b/3 trial did not provide sufficient evidence that Masitinib is beneficial for people with MND and that any benefits seen did not outweigh the risks of taking it. AB Science have asked for this decision to be re-examined.

January 2024 - The Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency, has requested AB Science to submit a written response to the List of Outstanding Issues. This has pushed back the timeline to a decision on whether to approve masitinib for the treament of MND in Europe. A decision is now expected before mid-2024.

Dec 2022 - Health Canada has asked for more information about Masitinib before continuing to review the request for its approval. Find out more here. 

May 2022 - AB Science confirmed that Health Canada is now reviewing the new drug submission application for Masitinib. You can read more here.

Feb 2022 - AB Science confirmed that Health Canada granted authorisation to file a new drug submission for Masitinib. You can read more here.

Sept 2021 - AB Science confirmed that the study was allowed to resume, with some enhanced measures in place to ensure participant safety. You can read more here.

June 2021 - AB Science filed for a temporary hold on all ongoing studies until concluding an investigation of a potential risk of ischemic heart disease associated with masitinib use. You can read more here.

• Update on clinical trials - part 2 - MND Research Blog