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The Disease That Turns You to Bone Just Got a Once-Daily Pill
The FDA approved Mirum's Atebrioz (zilurgisertib), a once-daily pill for fibrodysplasia ossificans progressiva, a condition where muscles, tendons, and ligaments slowly turn into bone. In trials, the drug cut new bone formation by over 99% compared to placebo. FOP affects roughly one in a million people, and most patients need a wheelchair by early adulthood. Atebrioz works by blocking ALK2, the broken genetic switch that constantly tells the body to build bone in the wrong places. Analysts estimate the drug could be priced around $750,000 per year, with launch expected in October.
Why it matters: For a community that spent decades with zero approved therapies, a second treatment option (after palovarotene in 2023) with a completely different mechanism means real flexibility in managing an unpredictable, lifelong disease.
Read more →Clinical and Regulatory
A $3.95 Million Gene Therapy Rewrites the Future for Kids With Sanfilippo Syndrome
Ultragenyx's FAYUVI is the first FDA-approved treatment for Sanfilippo syndrome, a fatal childhood brain disease that previously had zero options. The one-time gene therapy reduced toxic brain sugar buildup by 64% and showed meaningful cognitive improvements in treated kids. The price: $3.95 million per patient. For families who were once told to simply "take their kids home and love them," there's finally an action plan.
Read more →A Child Died in a Chinese CRISPR Trial. The World Found Out a Year Later.
A boy with Duchenne muscular dystrophy died after receiving a high-dose CRISPR therapy in Shanghai in 2025, but the death wasn't disclosed until STAT News pressed the company roughly a year later. Preclinical data had already flagged serious liver toxicity in primates. A second child died in a separate Chinese gene-editing trial around the same period, raising urgent questions about whether China's post-He Jiankui regulations are actually being enforced.
Read more →Parkinson's Gets Its First New Type of Dopamine Drug in Decades
The FDA approved AbbVie's Juvmo (tavapadon), the first selective D1/D5 dopamine receptor agonist for Parkinson's, targeting receptors that existing drugs mostly ignore. In trials, patients on levodopa gained an extra 1.1 hours of daily "on" time without troublesome involuntary movements. AbbVie paid $8.7 billion for this drug when it acquired Cerevel; now it needs the launch to deliver.
Read more →Merck and Daiichi Sankyo Pull Their Second FDA Application Under a $22B Partnership
The companies withdrew their U.S. application for ifinatamab deruxtecan in small cell lung cancer after the FDA signaled that Phase 2 response data wasn't enough for accelerated approval. That's two pulled applications under one mega-deal, with no approved products yet from Merck's side of the collaboration. The FDA's tightening standards for accelerated approval are catching the entire ADC sector off guard.
Read more →Deals and Strategy
Novo Nordisk Pays $1.3B for a Coating Thinner Than a Human Cell
Novo licensed Nanexa's PharmaShell technology for up to 1.165 billion euros, gaining the ability to coat drug particles in a 30-nanometer ceramic shell that could turn weekly obesity shots into monthly ones. With Amgen, Pfizer, and Lilly all chasing longer-acting injectables, Novo is betting that convenience (not just efficacy) will decide who wins the GLP-1 wars.
Read more →Boehringer Commits Over $1B to an AI Startup Hunting Cancer Targets in RNA Splicing
Boehringer Ingelheim signed a billion-dollar-plus framework deal with Envisagenics, whose SpliceCore platform scans over 14 million RNA splicing events to find tumor-specific targets invisible to traditional genomics. No approved cancer drug based on splicing modulation exists yet, which means whoever finds the right targets first has a wide-open field.
Read more →Eli Lilly Loses $90M Verdict for Breaking a Rule That Was Never Written Down
A California jury hit Lilly with a $90 million award after finding it violated the implied covenant of good faith in its partnership with Nektar, allegedly deprioritizing a co-developed drug after acquiring a competing asset. The verdict could reshape how pharma collaboration deals are drafted, negotiated, and dissolved going forward.
Read more →AbbVie Signs Multi-Year AI Drug Discovery Deal With Nvidia-Backed Iambic
AbbVie partnered with Iambic Therapeutics (which filed for its IPO the same day, naturally) to use its AI platform for small-molecule discovery across immunology, neuroscience, and oncology. Iambic's system connects AI models directly to robotic labs that build and test compounds in real time, and it already has a drug candidate in Phase 1 trials.
Read more →Science and Emerging Platforms
A Triple-Mechanism Cancer Drug Gets Its FDA Ticket to Human Testing
Shanghai-based Nutshell Therapeutics received FDA clearance to test NTS231, a molecular glue degrader that combines covalent binding, allosteric modulation, and targeted protein degradation in a single molecule. The target is NRF2, a cancer defense protein that nobody's been able to drug. If the chemistry works in humans, the implications extend far beyond one protein.
Read more →China's Hottest Biotech Deal Model Just Got Its First Trust Crisis
A New York lawsuit alleges a former Hengrui dealmaker secretly advised two competing Chinese biotechs while serving as exclusive consultant to one of them. With Chinese cross-border licensing deals topping $110 billion in the first half of 2026, the case exposes governance risks in the intermediary-dependent NewCo licensing model that's reshaping global pharma dealmaking.
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