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The biopharma world – and markets – spent Thursday evening reacting as the FDA gave the green light to only its third-ever drug for ALS, Amylyx’s AMX0035. 

Amylyx Pharmaceuticals Inc. said on Friday it has set the list price of its newly approved drug to slow the progression of amyotrophic lateral sclerosis (ALS) at about $158,000 per year in the United States.

The U.S. Food and Drug Administration has approved Fennec Pharmaceuticals Inc.’s intravenous therapy to prevent chemotherapy-induced hearing loss in children, according to the health regulator’s website.

The FDA approved Mallinckrodt Pharmaceuticals’ Terlivaz (terlipressin) in adults with hepatorenal syndrome (HRS), making it the only FDA-approved drug for that indication in the U.S.

In the latest win for Regeneron Pharmaceuticals and Sanofi’s Dupixent (dupilumab), the partners announced positive results from a Phase III trial of the blockbuster in kids ages one to 11 years with eosinophilic esophagitis (EoE).

One of the tenets of “personalized medicine” is that medications will be tailored to individuals based on their personalized genetics. Last year, the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research approved 50 drugs. A new study found that 33, or 66% of them, were supported with genomic data.

The U.S. Food and Drug Administration has awarded its first approval in the specific interleukin-23 inhibitor space for Crohn’s disease to AbbVie’s Skyrizi I (risakizumab-rzaa). 

Despite the Biden administration’s pledge to end formula shortages, slow responses and requests for more information from the U.S. Food and Drug Administration (FDA) have led to weeks-long delays for baby formula makers seeking U.S. approval, the two companies told Reuters.

Amylyx Pharmaceuticals Inc.’s experimental drug for the neurodegenerative disease amyotrophic lateral sclerosis (ALS) would be more cost effective than other U.S.-approved treatments if priced around $170,000, an independent U.S. pricing watchdog said on Tuesday.

Mallinckrodt plc today announced the company has resubmitted the New Drug Application (NDA) to the U.S. Food and Drug Administration for the investigational agent terlipressin to treat adults with hepatorenal syndrome (HRS) involving rapid reduction in kidney function.