Following a negative opinion from the EMA’s human medicines committee, the CHMP, for its orphan drug Albrioza (sodium phenylbutyrate/ursodoxicoltaurine) for amyotrophic lateral sclerosis (ALS), Amylyx Pharmaceuticals, Inc. told the Pink Sheet that regulators do not yet fully understand the best outcomes measures for neurodegenerative diseases.
Regulators Still To Get To Grips With Neurodegenerative Outcome Measures
Outcomes measures in the neurodegenerative disease space are not yet well understood by regulators, according to Amylyx, which recently received a negative opinion from the EMA for its amyotrophic lateral sclerosis drug, Albrioza.
More from Europe
• By
In a historic move for Africa, five products have been backed for approval via a pilot that tested a continental listing process.
• By
Pharmaceutical companies are being encouraged to reach out to NICE in relation to its HTA Innovation Lab, which provides a sandbox environment in which the health technology assessment body can test new methods of evaluating “innovative and disruptive” therapies.
• By
The weight management drug, Mysimba, continues to demonstrate a positive benefit-risk balance but the data available are not sufficient to fully determine the cardiovascular safety beyond 12 months. Meanwhile, Currax this month announced the publication of a peer-reviewed study of Mysimba that followed patients for over 4.7 years and found no evidence of excess cardiovascular risk.
• By
Health technology assessment body NICE said it has taken on feedback about the implications of allowing higher cost-effectiveness thresholds for some medicines after senior health economists offered diverging views on its methods.
More from Geography
• By
One CDRH employee said the cuts already are having a major effect on morale.
• By
Mass FDA layoffs on 1 April were designed to spare product reviewers, but still touched many who are critical to the application review process or drug development, which could mean fewer treatments are brought to the US market first.
• By
Pharmaceutical companies are being encouraged to reach out to NICE in relation to its HTA Innovation Lab, which provides a sandbox environment in which the health technology assessment body can test new methods of evaluating “innovative and disruptive” therapies.