An influential U.S. drug pricing watchdog raised the price estimate of two experimental gene therapies from Vertex Pharmaceuticals /CRISPR Therapeutics and bluebird bio to as much as $2.05 mln, saying the new price can be cost effective to treat sickle cell disease.
Clene said on Thursday its experimental amyotrophic lateral sclerosis (ALS) drug showed reduction in levels of an indicator believed to be tied with the disease progression in a mid-stage trial.
Expected FDA approval would make Leqembi the first in its class of drugs that work by removing sticky amyloid plaques from the brain to achieve the regulatory milestone.
The U.S. Medicare health plan said on Thursday it would limit reimbursement for Eisai Co Ltd and Biogen Inc's Alzheimer's drug Leqembi to patients whose doctors participate in a health agency database should the drug win full approval, a move advocates say will hinder its use.
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.