EMA turns down approval of AB Science’s ALS drug

| By | Drug Development, EMA, GCP
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AB Science, a pharmaceutical company specialized in research, development and marketing of protein kinase inhibitors (PKIs), announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicine Agency (EMA) has adopted a negative opinion for the marketing authorization of masitinib in the treatment of adult patients with Amyotrophic Lateral Sclerosis (ALS).

Amyotrophic lateral sclerosis is a rare degenerative disorder that results in wasting and progressive paralysis of muscles. There are approximately 50,000 people with ALS in the European Union and in the US, with more than 16,000 new cases diagnosed each year in Europe and in the US. This disease has a fatal outcome for most patients within 5 years from diagnosis.

The CHMP considered, based on a Good Clinical Practice inspection carried out on two of the main clinical investigation centers of the study, that the reliability of the data was not robust enough to support a registration. The CHMP did not recognize the clinical relevance of the distinction made by AB Science between patients with “normal” progression and for whom an improvement on the primary endpoint – ALS Functional Rating Scale (ALSFRS) score – has been demonstrated, and those with “rapid” progression. The CHMP considered that the primary analysis of the ALSFRS score for patients who stopped the study prematurely, based on the LOCF method (last observation carried forward), could introduce a bias in the analysis of the results.

In order to address these grounds for refusal, AB Science will provide further analyses on each of these points as part of the re-examination procedure. The re-examination will lead the CHMP to deliver a second opinion in July 2018.

Headquartered in Paris, France, AB Science reaffirms its commitment to carry out the development of masitinib in amyotrophic lateral sclerosis.

SOURCE: ab science
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