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The FDA’s Peripheral and Central Nervous System Drugs advisory committee will discuss the merits of Biogen and Ionis’s ALS candidate tofersen on March 22nd.

Last year CDER approved 37 new drugs never before approved or marketed in the U.S., known as “novel” drugs, as noted in its annual New Drug Therapy Approvals report. The agency also approved drugs in new settings, such as for new uses and patient populations.

As we move into 2023, BioSpace takes a pause to reflect on the year gone by and some of the most remarkable news bits, looking at the top five noteworthy FDA drug and biologic therapy approvals in 2022.  

Biohaven Pharmaceutical Holding Company Ltd said its experimental drug to treat amyotrophic lateral sclerosis (ALS) failed a clinical study, the second therapy by the drugmaker to fail trials in recent months.

The agencies announced the launch of the Critical Path for Rare Neurodegenerative Diseases (CP-RND) – a public-private partnership aimed at advancing the understanding of neurodegenerative diseases and fostering the development of treatments for amyotrophic lateral sclerosis (ALS) and other rare neurodegenerative diseases.

In what has been a long time coming, Tel Aviv and New York-based BrainStorm announced Monday that it would submit a Biologics License Application (BLA) to the FDA for NurOwn as a potential treatment for ALS. Simultaneously, BrainStorm announced corrected analyses it says strengthen its original conclusions.

“Our work shows that terazosin is protective of motor neuron cell death in multiple models of MND [motor neuron disease], making it an exciting new potential therapy,” says Helena Chaytow, the study’s first author and senior postdoctoral researcher at the University of Edinburgh.

The agency will reconvene a meeting of its outside experts to review the company’s application seeking approval for its amyotrophic lateral sclerosis (ALS) treatment.

Canada has approved Amylyx Pharmaceuticals’ treatment for amyotrophic lateral sclerosis (ALS), a neurodegenerative disease, the first such approval for the oral drug. The decision came with certain conditions, including the release of data from its global late-stage study of about 600 patients, which is expected in 2024, as well as additional studies.