AB Science Not Pursuing Re-examination of Masitinib after EMA’s Negative Opinion on Marketing Approval

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by Diogo Pinto |

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AB Science has decided not to seek the re-examination it initially requested after the European Medicines Agency (EMA) issued a negative opinion on marketing authorization for masitinib, the company’s investigational amyotrophic lateral sclerosis (ALS) therapy.

The company announced in a press release that the re-examination procedure would not properly address all the issues raised by the EMA’s Committee for Human Medicinal Products (CHMP).

In April 2018, CHMP issued a negative opinion on the conditional marketing authorization of masitinib based on three factors: data reliability, robustness, and possible bias.

Masitinib’s application was accepted by the EMA in October 2016, based on interim results from the Phase 2/3 AB10015 clinical trial (NCT02588677).

Because the final safety data from the trial were not generated until February 2018, these results could not be included in the evaluation process, and new data cannot be presented during the re-examination procedure.

The randomized AB10015 trial investigated the effectiveness and safety of masitinib, in combination with Rilutek (riluzole) for the treatment of ALS patients.

The study’s primary objective was to measure changes in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score, an assessment of patients’ abilities such as walking, speech, hand control, and respiration. Secondary outcomes were changes in function, survival and quality of life.

AB Science is now considering the possibility of resubmitting the application using the final results from the AB10015 study, which met its main goals. New data will be included in the application, such as:

  • Final safety data.
  • New sensitivity analyses of the study’s primary endpoint, the ALSFRS-R score, for patients who stopped the study before the study ended, as recommended by EMA’s guidelines, in order to confirm the results obtained using the last observation carried forward (LOCF) method, a method used to overcome missing data.
  • New preclinical data demonstrating the detailed mechanisms of action of masitinib, a crucial consideration for applications based on single, pivotal studies.

AB Science will discuss these and other details required to obtain marketing authorization for masitinib with the EMA.

“AB Science reaffirms its commitment to carry out the development of masitinib in ALS in order to provide a new treatment option to ALS patients,” the company said in the release.