Heather McKenzie

UniQure’s planned third-quarter submission for its Huntington’s disease gene therapy may be a harbinger of a more flexible FDA under acting commissioner Kyle Diamantas—but how long will it last? And how can companies be sure these positive decisions won’t just be reversed?

AbbVie scooped up immunology player Apogee Therapeutics for nearly $11 billion in one of the year’s top deals to-date, while Sanofi made a big play to survive its upcoming Dupixent patent cliff; FDA uncertainty continues as the agency changes direction on gene therapies by uniQure and REGENXBIO;…

A new report from RBC Capital Markets lists Biotechnology Innovation Organization chief executive John Crowley and military physician Heidi Overton as potential candidates for the role of FDA commissioner—as is the agency's current acting leader, top food regulator Kyle Diamantas.

Linda Marbán was “surprised” when the FDA informed Capricor Therapeutics it would be holding an advisory committee meeting for its Duchenne muscular dystrophy cardiomyopathy cell therapy prior to its August target action date.

The vibe at BIO 2026 in San Diego last week was overwhelmingly positive, with attendees observing noticeable changes at the FDA and an uptick in dealmaking and IPOs. Plus, a top medical journal this week retracted a pivotal study for Amgen's rare disease drug Tavneos, which has been in the FDA's crosshairs since January.

A surprising deal from Vertex Pharmaceuticals adds to Big Pharma's acquisitive streak as Crinetics folds into the cystic fibrosis drugmaker. Meanwhile, IPOs and venture capital raises trend upward, but mostly for derisked companies. Plus, FDA decisions slow only slightly as the hunt for a permanent leader drags on.

Even as FDA approvals for biologic therapies fell in the first half of 2026, regulatory experts are optimistic about a turnaround in the rare disease space after the departure of key leaders at the agency. Still, there will continue to be tension between science and politics.

Saol Therapeutics is the latest biotech to resubmit for approval of a drug rejected under former FDA Commissioner Marty Makary, following REGENXBIO and Replimune.

The FDA greenlit 26 novel therapies in the first half of 2026, including four for cancer and six for orphan indications. Meanwhile, AstraZeneca and Johnson and Johnson took home a combined 11 of the agency's 79 total approvals, including supplemental nods.

The failure of Roche's Ionis-partnered tominersen in Huntington's disease may indicate that Wave Life Sciences' allele-specific antisense oligonucleotide candidate WVE-003 is on the right track, according to analysts at Rodman & Renshaw.