BioSpace News Archive

Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like MPS II moving into the spotlight.

Pfizer announces the first data from its Metsera-acquired pipeline just ahead of its earnings call, where analysts pressed execs for more details; Merck and Roche also released Q4 and full year earnings, with Eli Lilly, Novo Nordisk and others reporting Wednesday; REGENXBIO hits a regulatory snag ahead of its upcoming PDUFA; more.

Novartis will still be on the lookout for early-stage deals under $2 billion, and later-stage agreements around a product that could reach the market within five years, CEO Vas Narasimhan said Wednesday.

Rep. Jake Auchincloss of Massachusetts said the Commissioner’s National Priority Voucher program did not receive congressional backing. The FDA has also not yet made disclosures for eight senior reviewers, according to Auchincloss.

Instead of joining the increasingly crowded GLP-1 arena, GSK will focus its efforts downstream of obesity—a push currently anchored by its Phase III-ready FGF21 analog efimosfermin alfa for liver fibrosis.

Amgen believes that it can transcend the expected tradeoff between convenience and efficacy, anticipating that its investigational obesity drug MariTide will continue to provide competitive weight loss even at monthly or longer schedules.

After review, Amgen is certain that Tavneos is effective and has a favorable benefit-risk profile. The company informed the FDA on January 28 that they would not pull the drug.

Novo Nordisk CEO Maziar Mike Doustdar acknowledged the market pressure facing the company’s GLP-1 products but sought to assure investors that Novo has the situation under control.

Mounjaro and Zepbound combined for $11.7 billion in the fourth quarter, which beat analyst consensus of $10.6 billion.

On its fourth quarter earnings call Wednesday, AbbVie CEO Robert Michael called oncology and neuroscience “underappreciated” areas of focus for the pharma.