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Issue 43: what changed

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REGULATORY

The first broad RAS inhibitor nearly doubles survival in pancreatic cancer ↗

The FDA approved Revolution Medicines' Rasonque, or daraxonrasib, for adults with metastatic pancreatic adenocarcinoma who have had at least one prior therapy. In a randomized open-label trial of 500 patients, median overall survival was 13.2 months against 6.7 months for chemotherapy. RAS has been called undruggable for four decades, and the mutations it carries appear in roughly 90 percent of pancreatic adenocarcinoma, so a drug that targets the family broadly rather than one variant reaches most of the disease at once. Around 67,000 pancreatic cancers are diagnosed in the United States each year, and 90 to 95 percent are adenocarcinoma. The FDA cleared it six and a half months ahead of its deadline.

FDA · 3-min read

REGULATORY

mRNA reaches seasonal flu, with the older-adult claim still unproven ↗

The FDA approved Moderna's mFLUSIVA on August 5 as the first mRNA influenza vaccine in the United States, for adults 50 and older. The two halves of that indication rest on different evidence. For adults 50 to 64 it is a full approval. For those 65 and older, the group with the most to gain from a better flu shot, it is an accelerated approval based on immune response rather than demonstrated protection. Moderna has committed to a randomized study against a high-dose comparator, which began at the end of August and reports in 2029. The platform that shipped covid vaccines in a year now has to prove it beats a shot that already exists.

FDA approval letter · 3-min read

REGULATORY

A gene therapy approved on cornstarch, not blood sugar ↗

Ultragenyx won accelerated approval for Genglycos, a one-time AAV8 gene therapy for glycogen storage disease type Ia, a disorder in which a missing enzyme leaves the liver unable to release stored glucose. It is the first therapy aimed at the underlying cause, and the company's first gene therapy after years of setbacks. The primary endpoint is worth reading closely: a 31 percent mean reduction in daily cornstarch intake, which patients consume around the clock to hold blood sugar up. On glucose itself, treated patients spent slightly more time below 70 milligrams per decilitre than placebo, and had more hypertriglyceridemia, 29 percent against 8 percent. The list price is 2.7 million dollars.

FDA · 3-min read

QUICK HITS

04Takeda's Orzeyful is the first orexin agonist approved for narcolepsy type 1, treating the cause rather than the sleepinessTakeda ↗05GSK takes its own mRNA flu vaccine to phase 3 after it beat standard shots on immune responseFierce Biotech ↗06A protein design model from MIT is scored on stability rather than on how well it imitates natureMIT News ↗

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