Tag Archive for: amyotrophic lateral sclerosis (ALS)

Europe’s medicines regulator on Friday recommended granting a marketing authorization to Biogen’s drug for the deadly and progressive neurodegenerative disease known as amyotrophic lateral sclerosis (ALS).

Amylyx Pharmaceuticals Inc. said on Tuesday that a European regulatory panel had taken a negative view on its amyotrophic lateral sclerosis (ALS) treatment, stalling the company’s plans on introducing its first commercial drug in the region.

ALS is the 5,000-piece jigsaw puzzle of the therapeutic world. As the regulatory edges come together, new research pieces are also connecting. 

2022 was a big year for uniQure with the U.S. and European approval of world’s first gene therapy for hemophilia B. In its 2022 financial report Monday, the Lexington, Mass. and Amsterdam-based company provided updates on the progress of its Huntington’s disease and ALS programs. 

uniQure inked a deal with Apic Bio Tuesday to gain development and commercialization rights for APB-102, a therapeutic intended to treat a rare, genetic form of ALS. 

BrainStorm Cell Therapeutics Inc., a leading developer of adult stem cell therapeutics for neurodegenerative diseases, announced that the U.S. Food and Drug Administration (FDA) has granted a Type A meeting to discuss the contents of a refusal to file letter previously issued regarding the company’s New Biologics License Application (BLA) for NurOwn for the treatment of ALS. The Type A Meeting has been scheduled to occur on January 11, 2023.

BrainStorm Cell Therapeutics ran into another stumbling block with ALS hopeful NurOwn Thursday as the FDA issued a Refusal to File Letter for its Biologics License Application.

The U.S. Food and Drug Administration has extended the review of Biogen Inc.’s (BIIB.O) experimental treatment for an inherited form of amyotrophic lateral sclerosis (ALS) by three months, the company said on Monday.

Biogen may have found its CEO in Mathai Mammen – former head of research and development in pharmaceuticals at Johnson & Johnson – following the pending departure of current company head Michel Vounatsos.

Amylyx is dipping back into the well to raise funds to launch its recently approved ALS drug Relyvrio. The company plans to offer 6 million shares in a public offering to bring in between $167 to $192.2 million.