Biogen receives FDA approval for breakthrough ALS treatment: ‘Pivotal moment in ALS research’

BOSTON — Cambridge, Massachusetts-based Biogen has gotten the green light for a breakthrough ALS treatment that targets a devastating, ultra-rare genetic form of the progressive neurodegenerative disease.

The U.S. Food and Drug Administration on Tuesday granted approval of Qalsody for the treatment of ALS in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. Biogen’s treatment is the first approved treatment to target a genetic cause of ALS.

“For more than a decade, Biogen has been steadfast in our commitment to pursuing treatments for ALS, and I want to thank the scientists as well as the entire ALS community who have all worked tirelessly to bring this first-of-its-kind treatment to people with SOD1-ALS,” said Christopher Viehbacher, Biogen’s president and CEO.

About 30,000 people in the U.S. have ALS, according to estimates. SOD1-ALS is diagnosed in about 2% of all ALS cases, with about 330 people in the U.S. living with the disease.

The FDA approval for the treatment was based on the reduction in plasma neurofilament light chain seen in patients treated with Qalsody. Neurofilaments are proteins that are released from neurons when they are damaged, making them a marker of neurodegeneration.

“Today also marks a pivotal moment in ALS research as we gained, for the first time, consensus that neurofilament can be used as a surrogate marker reasonably likely to predict clinical benefit in SOD1-ALS,” Viehbacher added. “We believe this important scientific advancement will further accelerate innovative drug development for ALS.”

Qalsody is administered through a spinal injection by healthcare professionals who are experienced in performing lumbar punctures.

SOD1-ALS is a progressive neurodegenerative disease that attacks and kills the nerve cells that control voluntary muscles. Voluntary muscles produce movements such as chewing, walking, breathing and talking. ALS causes the nerves to lose the ability to activate specific muscles, which causes the muscles to become weak and leads to paralysis.

“Since SOD1 mutations were first identified as a cause of ALS 30 years ago, the familial ALS community has been searching for genetically targeted treatments,” said Jean Swidler, chair of Genetic ALS & FTD: End the Legacy.

“Qalsody offers families who have lost generation after generation in the prime of their life to this devastating disease a therapy targeting the underlying cause of SOD1-ALS,” Swidler added.

This FDA approval for Biogen comes after a different Cambridge company’s ALS treatment drug — boosted by funding from the Ice Bucket Challenge — received approval from the FDA.

Amylyx Pharmaceuticals got the OK from the FDA in September, giving the green light for the local firm’s ALS drug treatment for the relentlessly progressive and fatal neurodegenerative disorder. The drug Relyvrio was the first FDA-approved treatment funded by ALS Ice Bucket Challenge donations.


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