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The First-Ever Treatment for Sanfilippo Syndrome Type A Costs $3.95 Million
Until this week, parents of children with Sanfilippo syndrome type A could only watch as the disease erased their kids' speech, motor skills, and personality. Now there's Fayuvi, a one-time gene therapy from Ultragenyx that just received full FDA approval. Treated children scored 23.5 points higher on cognitive tests than untreated peers, with benefits lasting nearly eight years. The catch: at $3.95 million per dose, it's one of the most expensive therapies in the U.S. Ultragenyx desperately needed the win after its stock cratered 45% two weeks ago following a separate clinical failure.
Why it matters: Each new multi-million-dollar gene therapy forces the same uncomfortable question: how do health systems pay for one-time cures that cost more than a house, especially when the patient population numbers in the hundreds? Fayuvi won't be the last to test that math.
Read more →Clinical Wins and Stumbles
Zero Patients Progressed: Trutakna's Kidney Data Defies Belief
A 76% reduction in kidney disease progression. Zero patients needing dialysis or transplant (versus eight on placebo). Kidney function essentially frozen in place for two years. Vera Therapeutics' Trutakna posted the kind of IgA nephropathy results that make clinicians squint. The drug targets the upstream immune drivers of the disease rather than managing downstream damage, and every prespecified endpoint was met.
Read more →Xenon's Depression Drug Triggered Psychosis in Patients It Was Meant to Help
A depression drug causing psychosis is about as bad as it sounds. Xenon paused enrollment in two Phase 3 trials for azetukalner after a small number of patients experienced psychotic episodes (roughly 1% or under). The stock dropped 25%, analysts split on whether dose adjustments can save the program, and the company insists it's temporary. The side effect never appeared in Phase 2, which is its own red flag.
Read more →ALS Breaks Another Heart as Novartis Kills Lifonebart
Add another headstone to the ALS graveyard. Novartis terminated lifonebart after its Phase 2 trial missed every single endpoint, continuing a decades-long streak of failures against a disease that has defeated over 60 drug candidates. The company is doubling down on brain-delivery platforms, though, spending hundreds of millions to solve the blood-brain barrier problem that keeps tripping up neurology drugs.
Read more →England Finally Says Yes to the Cancer Drug It Rejected Two Years Ago
After a two-year standoff over price, England's NHS approved Enhertu for HER2-low metastatic breast cancer. About 1,000 patients a year had watched Scottish patients access the drug while their own system said it cost too much. A confidential discount and updated evaluation methods broke the logjam. The drug is approaching $5 billion in global sales.
Read more →Deals and Dealmaking
Novo Nordisk Bets $1.4B on Ring-Shaped Molecules Most Pharma Gave Up On
Novo committed up to $1.4 billion to tiny Danish biotech Orbis Medicines for its macrocycle platform, which aims to turn injectable blockbusters into pills. Orbis claims oral bioavailability of up to 18% in preclinical work (versus the approximately 1-2% typical of leading oral peptide macrocycles in the clinic). The deal is one of several billion-dollar-plus external bets Novo has placed recently as it races to own oral cardiometabolic medicine.
Read more →Genentech Drops $190M Upfront on a Drug That's Never Been in a Human
Genentech paid $190 million upfront (up to $2.3 billion total) for Hanmi's HM17321, a preclinical obesity drug that works through an entirely different mechanism than GLP-1s. The molecule targets a receptor that, in animal studies, burns fat while building muscle simultaneously. If it translates to humans, it could solve the biggest complaint about current weight-loss drugs: patients lose muscle along with fat.
Read more →SK Biopharma Pays $315M for a Parkinson's Drug That Hasn't Picked Its Final Molecule
SK Biopharmaceuticals signed a deal worth up to $315 million for 1ST-104, a preclinical Parkinson's candidate designed to hit two targets (LRRK2 and c-Abl) with one pill. The timing is bold: the field's most advanced LRRK2 drug just failed Phase 2b in May. SK is betting that dual inhibition succeeds where single-target approaches couldn't.
Read more →The Expensive Lessons
BMS Torches $100M on a Drug Class That Just Lost Its Only Clinical Proof Point
Bristol Myers Squibb killed BMS-986497, the world's only degrader-antibody conjugate in clinical trials, after disappointing Phase 1 data in blood cancers. The $100 million upfront payment to Orum Therapeutics is gone. More importantly, the entire DAC field just lost its only shot at proving the concept works in humans, which will make partnering and fundraising significantly harder for every company building in this space.
Read more →Electra Therapeutics Raises $350M in One of 2026's Biggest Biotech IPOs
Electra Therapeutics pulled in $350 million in an upsized IPO, betting on antibodies that target a protein family called SIRPs. Unlike most newly public biotechs, Electra already has a registrational trial running for a rare, life-threatening inflammatory condition. The deal adds to a resurgent 2026 IPO market that has now topped $6.5 billion across 21 biotech debuts.
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