ClinicaSpace

After last year’s ‘stampede’ for FGF21 assets, the focus for the metabolic dysfunction-associated steatohepatitis space has shifted toward differentiated approaches, such as THR-β agonists and combination treatments, that seek to mirror the commercial success of Madrigal’s Rezdiffra.

Following the FDA’s refusal to review Moderna’s investigational mRNA flu vaccine last week, Commissioner Marty Makary faced questions from the U.S. president about the agency’s handling of vaccines. It’s a clear signal that the tension long brewing at the drug regulator has now gone all the way to the top.

The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is “part of a disturbing pattern” of moving regulatory goalposts, according to Clay Alspach, executive director of the Alliance for mRNA Medicines. Meanwhile, streamlined communications with regulators in other countries pave the way for rapid uptake of novel modalities.

The FDA’s refusal to review Moderna’s mRNA-based flu vaccine is part of a larger communications crisis unfolding at the agency over the past nine months that has also ensnarled Sarepta, Capricor, uniQure and many more.

With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne muscular dystrophy pipeline, as Sarepta’s Elevidys leaves the door open.

Biopharma’s obsession with the weight loss sector—projected to exceed $130 billion in revenue by 2035—shows no signs of slowing down as analysts gear up for a year that will test the oral market and provide key data on novel mechanisms.

AstraZeneca’s $15 billion pledge to its China operations highlights the country’s advantages. But other regions are also hoping to host more clinical studies.

With Lykos’ regulatory failure now squarely in the rearview mirror, Compass Pathways and Definium are leading what one analyst suspects will be “a very big year for psychedelics.”

Phacilitate’s annual event dawns as cell and gene therapies reach a new tipping point: the science has hit new heights just as regulatory and government policies spark momentum and frustration.

Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.