FDA clears Ionis' therapy for rare swelling disorder

Ionis Pharmaceuticals yesterday won Food and Drug Administration approval for Dawnzera, its antisense drug for patients with hereditary angioedema, a rare and dangerous disorder.

In late-stage trials, the therapy sharply reduced the frequency of swelling attacks in patients, cutting monthly attack rates by more than 80% compared with placebo. The medication showed additional benefit for patients who switched from existing treatments like Takeda's Takhzyro and BioCryst's Orladeyo, STAT's Jonathan Wosen writes.

Priced at $57,462 per dose, Dawnzera will face stiff competition from those blockbusters as well as looming CRISPR-based approaches, but Ionis CEO Brett Monia said its efficacy, safety, and less frequent dosing give it an edge.

manufacturing

Novo plant failed to properly investigate cat hair, contamination, FDA finds

An FDA inspection report paints a troubling picture of a Novo Nordisk manufacturing site in Indiana acquired last year from Catalent. It cites cat hair, pests, bacterial contamination, and critical equipment failures that were not properly investigated, STAT's Ed Silverman and Elaine Chen report.

The lapses have already delayed approvals for Regeneron drugs, including high-dose Eylea, and raised concerns for companies like Scholar Rock that also rely on the plant.

"I believe that we may not be the only [drug application] that's ensnared, because they do — as I said, they do work for virtually all the biopharmaceutical companies," Regeneron CEO Len Schleifer said on a conference call with analysts recently.

podcast

Viking's obesity flop and 'pharm to table' drug sales

On this week's episode of "The Readout LOUD": vikings. No, not the seafaring Norse people of the 10th century, or the Minnesota football team. The gang will discuss obesity drug developer Viking Therapeutics and the investor cult that embraces it, both of which performed a painful belly flop this week.

Then, we'll dish on the growing "pharm to table" movement. That's the clever buzzphrase bandied around by Big Pharma insiders to promote plans to sell drugs directly to consumers — supposedly at lower costs that will make President Trump happy. Except as our cohost Elaine Chen reported this week, these direct-to-consumer plans championed by Eli Lilly, Novo Nordisk, Bristol Myers Squibb, and others are unlikely to make drugs much more affordable. We'll dig into the details.

Listen here.

dei

Supreme Court lets Trump slash NIH research funds

The Supreme Court is allowing the Trump administration to cut $783 million in National Institutes of Health research grants tied to diversity, equity, and inclusion programs, overturning an order by a lower court that had blocked the move.

In the 5-4 vote, Chief Justice John Roberts sided with the three liberals in dissent, warning against the abrupt cancellations. The plaintiffs — who included 16 Democratic attorneys general and public health groups — say these cuts will devastate careers, disrupt ongoing studies, and cost lives.

While the justices left Trump's broader funding guidance on hold, the decision marks a major win for the administration as it seeks to reshape billions in federal science spending.

"I've never seen government racial discrimination like this," U.S. District Judge William Young, an appointee of Republican President Ronald Reagan, said at a hearing in June. He later added: "Have we no shame?"