The past two years have seen a pair of new treatments approved for a particularly intractable neurodegenerative disease —amyotrophic lateral sclerosis. But while Amylyx’s Relyvrio and Biogen’s Qalsody have crossed the FDA finish line, others have stumbled in a regulatory space that experts say is still evolving.
Tag Archive for: neurodegenerative disease
The Massachusetts–based startup will use the funds to support the development of its oral small molecule drugs it claims have neurorestorative properties, including a GPR17 antagonist that could promote the remyelination of neurons, as well as a CSF1R inhibitor that could target the demyelination process and neuroinflammation.
The two companies are preparing to launch the drug in the second quarter of Eisai’s 2024/25 business year starting in April.
The agency found data from the company’s mid-stage study for the treatment of a type of neurodegenerative disease to be insufficient to support accelerated approval.
The blood-brain barrier: It’s neuroscience’s next frontier, and a presentation on Roche’s trontinemab at the Clinical Trials on Alzheimer’s Disease conference last month sparked new optimism that researchers might be getting close to breaking through it.
The decision comes after Amylyx in June requested a formal re-examination of the initial negative opinion adopted by the Committee for Medicinal Products for Human Use of the European Medicines Agency.
Although the profile is not precise enough to definitively diagnose Alzheimer’s, it offers a convenient way for doctors to determine which patients need advanced testing, according to the company.
The Japanese multinational pharma is pledging up to $580 million in a development and commercialization deal with AcuraStem for the latter’s PIKFYVE program for amyotrophic lateral sclerosis.
The development comes after a health ministry panel recommended the drug’s approval in August, followed by standard approval from the U.S. Food and Drug Administration (FDA) in July.
Seeking to deepen its neurology and rare disease pipelines, AstraZeneca’s Alexion has joined forces with Verge Genomics to leverage its artificial intelligence platform in drug discovery and development.
