April 29, 2026

The need-to-know this morning

The Italian pharma company Chiesi is buying KalVista Therapeutics for $1.9 billion, adding an approved treatment for a genetic swelling disorder to its rare-disease portfolio. The deal values KalVista at $27 per share, or a 40% premium to its Tuesday closing stock price. The KalVista medicine, called Ekterly, is a pill used to treat acute swelling attacks in people with hereditary angioedema.
Biogen, AstraZeneca, Regeneron Pharma, Abbvie and GSK reported first-quarter earnings.

regulation

FDA plans to review AZ, Amgen trials in real time

In an effort to get drugs on the market faster, the FDA announced yesterday it will start reviewing trial data in real time, starting with oncology studies conducted by AstraZeneca and Amgen.

The trials will use a real-time data platform built by Paradigm Health, through which reviewers will be able to view safety signals and clinical endpoints, Commissioner Marty Makary said.

In a STAT opinion piece, Makary said the agency will launch a broader real-time clinical trials pilot program with more companies this summer.

With this approach, “FDA scientists can detect adverse reactions sooner, potentially enhancing patient safety. Conversely, when a new therapy is showing strong efficacy, we can identify that success rapidly. Real-time trials are not just technically possible; they are transformative for the entire clinical ecosystem,” he wrote.

drug pricing

SCOTUS will hear a key case on generic drugs

Today, the Supreme Court will hear an oral argument about a wonky but important tactic used by generic companies called “skinny labeling,” which allows them to introduce drugs to the market faster.

Essentially, generic makers seek approval to market a medicine for a specific use, but not other patented uses for which a brand-name drug is prescribed.

This court case will be a critical test for whether skinny labeling can survive. If the Supreme Court does not back the tactic, “we could see delayed generic entry for much longer periods of time, which would mean higher prices for patients and worse health outcomes,” one law professor said.

biotech

A patent win for Pfizer and BridgeBio

Pfizer said yesterday that it's settled patent lawsuits with three generic manufacturers, extending the patent expiration of its heart drug Vyndamax from 2028 to 2031.

The drug, a significant revenue generator for Pfizer, treats transthyretin amyloid cardiomyopathy (ATTR-CM), a disease estimated to affect more than 400,000 people globally.

The patent extension also provides some relief for for BridgeBio, which has a similar drug on the market called Attruby. (Some investors were disappointed, though, since they hoped the patent would extend into 2032 or 2033.)

In ATTR-CM, patients’ livers produce unstable proteins that misfold and clump into amyloid deposits in the heart. Pfizer and BridgeBio's drugs work by stabilizing the proteins. Alnylam also has a newly launched ATTR-CM drug, but it works differently by silencing messenger RNA involved in the production of the unstable protein, so it wasn't expected to be as threatened by generic competition.

rare disease

Rocket sells voucher as it advances gene therapies

From my colleague Drew Joseph: Rocket Pharmaceuticals said yesterday it had sold its priority review voucher (PRV), which it earned with the approval of its gene therapy Kresladi for the ultra-rare disease LAD-1 earlier this year, for $180 million, extending the company's cash runway from one to two years.

“We're not just trying to catch up and execute so we can raise money. We can just execute,” Rocket CEO Gaurav Shah told STAT in an interview in Rome at the Alliance for Regenerative Medicine's Meeting on the Med.

It was not disclosed who bought the voucher, which provides a speedier regulatory review for a medicine.

The sales price of PRVs has yo-yoed over the years, sometimes reaching $200 million or more, though analysts at Leerink wrote in a note that $180 million is above the average from fiscal years 2024 and 2025.

Shah said that the additional funding will help the company as it advances its three clinical stage cardiovascular gene therapies.

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