U.S. Food and Drug Administration (FDA)

The U.S. Food and Drug Administration (FDA) has signed off on the chemistry, manufacturing, and controls (CMC) clinical development plan for NeuroSense Therapeutics‘ lead candidate for amyotrophic lateral sclerosis (ALS) ahead of a planned Phase 3 study. The Phase 3 trial of PrimeC is due to start…

Akava Therapeutics will soon launch a Phase 1 clinical trial to test AKV9, its investigational therapy for amyotrophic lateral sclerosis (ALS), in healthy people. The trial follows the clearance of Akava’s investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA). It will be AKV9’s…

Cognixion’s flagship device Cognixion ONE Axon, a product designed to improve communication in people with severe motor impairments, has beed granted breakthrough device designation by the U.S. Food and Drug Administration (FDA). The wireless device is specifically designed for people with people with amyotrophic lateral sclerosis (ALS)…

The U.S. Food and Drug Administration (FDA) will hold an advisory committee meeting to discuss BrainStorm Cell Therapeutics‘ application to approve NurOwn for amyotrophic lateral sclerosis (ALS). Such a meeting, called an ADCOM, is normally for when a therapy is under regulatory review so experts can discuss its…

Kadimastem is planning a Phase 2a clinical trial to test if repeat dosing of AstroRx, its investigational therapy for amyotrophic lateral sclerosis (ALS), can continuously delay the disease’s progression. The study will investigate every three-month dosing after findings from an earlier Phase 1/2 trial (NCT03482050) showed a…

The U.S. Food and Drug Administration (FDA) is extending by three months its review of tofersen, Biogen’s investigational treatment for forms of amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene. Earlier this summer, the FDA granted the application priority review, with a decision expected no…

Kadimastem has received a U.S. patent covering its investigational cell therapy AstroRx for treating amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The patent, granted by the U.S. Patent and Trademark Office, supports an upcoming Phase 2a clinical trial that’s planned to start in the U.S. next year.

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…