Overview

The European Medicines Agency has recommended the refusal of the marketing authorisation for Masitinib AB Science, a medicine intended for the treatment of amyotrophic lateral sclerosis (ALS).

The Agency issued its opinion on 27 June 2024. The company that applied for authorisation, AB Science, may ask for re-examination of the opinion within 15 days of receiving the opinion.

Masitinib AB Science was developed as a medicine to treat adults with ALS. It was intended to be used in combination with riluzole (another medicine for ALS). ALS is a progressive disease of the nervous system, where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis.

Masitinib AB Science contains the active substance masitinib mesilate and was to be available as tablets.

Masitinib AB Science was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 29 August 2016 for the treatment of ALS. 

The active substance in Masitinib AB Science, masitinib mesilate, is a protein-kinase inhibitor. This means that it blocks specific enzymes known as protein kinases. Blocking these enzymes affects the activity of certain cells of the immune system (the body’s natural defences) involved in causing inflammation. By blocking these enzymes, masitinib mesilate was expected to reduce inflammation and protect nerve cells from damage, thereby slowing worsening of ALS symptoms. 

The company presented results from a main study involving 394 adults with ALS. Patients were given Masitinib AB Science or placebo (a dummy treatment) twice a day for 48 weeks. All patients also received riluzole. The main measure of effectiveness was the change over 48 weeks in the ALSFRS-R score, a measure of ALS symptoms that affect patient quality of life.

The Agency considered that the study data are not reliable, as findings from good clinical practice (GCP) inspections carried out by EMA and other regulatory authorities identified issues with the conduct of the study that cannot sufficiently be addressed by the company. In addition, the benefits of Masitinib AB Science could not be convincingly demonstrated; the study found no difference between the medicine and placebo in the main measure of effectiveness for the total study population and had several methodological issues.

Therefore, the Agency’s opinion was that the benefits of Masitinib AB Science did not outweigh its risks and it recommended refusing marketing authorisation.

The company informed the Agency that there are no consequences for patients in clinical trials with Masitinib AB Science.

If you are in a clinical trial and need more information about your treatment, speak with your clinical trial doctor.

Questions and answers on the refusal of the marketing authorisation for Masitinib AB Science (masitinib mesilate)

AdoptedReference Number: EMA/293166/2024

English (EN) (104.14 KB - PDF)

First published:
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Product details

Name of medicine
Masitinib AB Science
Active substance
Masitinib mesilate
International non-proprietary name (INN) or common name
masitinib
Therapeutic area (MeSH)
Amyotrophic Lateral Sclerosis
Anatomical therapeutic chemical (ATC) code
L01EX06
EMA product number
EMEA/H/C/005897

Orphan

This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Marketing authorisation applicant
AB Science
Opinion adopted
27/06/2024
Opinion status
Negative
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