The US Food and Drug Administration has approved a new drug for amyotrophic lateral sclerosis, a rare disease that attacks and kills the nerve cells that control voluntary muscles. In a Phase 2 trial, the drug Relyvrio (sodium phenylbutyrate and /taurursodiol), showed an ability to slow the rate of neuromuscular decline. Moreover in a follow-up study, it extended patient survival compared with a placebo.
The European Medicines Agency has launched a new project to help academic scientists and those working for non-profit organisations understand the regulatory requirements for developing and eventually commercialising cell, gene and tissue engineered therapies. The project complements the agency’s priority medicines scheme, PRIME, which was launched in 2016 to provide support to developers of medicines that meet a major public health need.
Lecanemab, an antibody therapy targeting amyloid-beta protofibrils in patients with early Alzheimer’s disease, achieved positive results in a Phase 3 study, paving the way for regulatory reviews in the US, EU and Japan, the developer Eisai Inc announced on 28 September. The study, called Clarity AD, met the primary endpoint as well as key secondary targets, confirming that lecanemab’s mechanism of action is able to reduce cognitive clinical decline in Alzheimer's patients. Lecanemab works by binding to soluble amyloid-beta protofibrils.
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