ClinicaSpace

A year of significant policy change at the FDA brought momentum and scrutiny into the new year. As 2026 gets underway, biopharma companies are responding to sweeping vaccine changes while concerns surface about the politicization of the agency.

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.

Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.

From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.

The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.