The Hague, June 23 (EFE).- The European Medicines Agency (EMA) on Friday denied authorizing the marketing of Albrioza, a treatment for amyotrophic lateral sclerosis (ALS), considering that it has not been proven that this drug really delays the worsening of this neurological disease.
At its monthly meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended that the European Commission, which has the final say in drug approval in the European Union (EU), “refuse marketing authorization” for Albrioza, a drug developed to treat the rare neurological disease that affects the nerve cells in the brain and spinal cord that control the movement of voluntary muscles.
“The Agency is concerned that the main study did not convincingly show that Albrioza was effective in delaying the worsening of the disease. The data on survival were also unreliable, given the way they were collected and analysed”, explains the EMA about its reasons for issuing a negative opinion on this treatment.
The company provided the results of one main study involving 137 adult ALS patients who received Albrioza (as a powder to dissolve in water and take by mouth) or a placebo, in addition to their standard treatment, for 24 weeks.
What does the EMA look at to assess the efficacy of this drug for ALS?
The main measure of effectiveness of this drug was the rate at which patients’ symptoms, such as difficulty speaking, breathing, eating, and performing other normal daily activities, worsened during the study. In addition, the company also provided results on overall survival time in its clinical study.
“It is not entirely clear how Albrioza works, but the two active substances, sodium phenylbutyrate and ursodoxycoltaurin, were expected to reduce damage to nerve cells and prevent them from dying. This was expected to help maintain normal muscle function and delay the worsening of the disease,” details the EMA.
Based on these data, in addition to advice from expert groups, patient representatives and experts in neurology, the CHMP concluded that “a positive balance between benefits and risks could not be established” for Albrioza, and recommended that the license be denied. use in the EU that had been requested by the company Amylyx Pharmaceuticals EMEA.
