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• Egetis Therapeutics has secured US approval of its drug for a rare disease that shortens life expectancy to 35 years.

• The FDA on Monday backed Emcitate (tiratricol) for certain patients with monocarboxylate transporter 8 (MCT8) deficiency, making it the only approved treatment in the US for the X-linked disease, also known as Allan-Herndon-Dudley syndrome. The drug was previously approved in Europe in February 2025.

• Egetis expects to launch the drug in the US eight to 10 weeks after approval. The drug will have an annual list price of about $950,000, according to a company spokesperson. (Endpoints)

• Gazyva just increased its foothold in the autoimmune market with a fresh FDA approval, becoming the first new treatment option in 70 years for patients with idiopathic nephrotic syndrome (INS).

• The regulator cleared the anti-CD20 monoclonal antibody to reduce the risk of relapse in adults and children 2 and older with frequently relapsing or steroid-dependent, childhood-onset INS who are in complete remission.

• The FDA approval, Gazyva’s second autoimmune indication in less than 12 months, cements the 2013 cancer drug’s second act in immune-mediated diseases. (Fierce Pharma)

• Mirum Pharmaceuticals’ Atebrioz is the third FDA-approved drug for fibrodysplasia ossificans progressiva (FOP), but it brings advantages over the two other products for this rare disease that leads to bone growth in soft tissue.

• Mirum acquired Atebrioz (zilurgisertib) from Incyte earlier this year. (MedCity News)

Agentic twins. Phaseless design engine. Fewer trial participants. All these are part of the government’s new efforts to secure global leadership in clinical research.

• Unveiled by the Advanced Research Projects Agency for Health (ARPA-H), the initiative is designed to speed up clinical trials and slash patient burden, according to a release from the US Department of Health and Human Services (HHS).

• The new program will be known as Simulation-augmented, Real-time Platform Adaptive Seamless Trials, or SURPASS.

• The initiative will incorporate advanced computational models, real-time analysis and automation “to evaluate drugs and biologics faster, at lower cost, and with fewer participants,” according to the government release.

• The effort comes the same week that an executive order was signed to establish an AI-powered chatbot to streamline user experience across government services. (BioSpace)

• The FDA issued notices inviting subject-matter experts and consumer representatives to volunteer for its advisory committees. The move comes after the role of FDA's advisory committees was diminished under former FDA leadership.

• FDA said it is initiating a broad, nationwide effort to recruit and expand the range of qualified experts on its independent advisory committees.

• The agency published two notices: one calling for subject matter experts with qualified scientific, technical, and professional backgrounds to volunteer, and another seeking consumer representatives to participate on the committees.

• FDA said it is accepting self-nominations, and those selected will serve up to four years on a committee.

• One expert said the announcement signals renewed interest in the committee system by the agency. (RAPS)

• The FDA has issued a final rule that would remove references to animal studies in its drug safety regulations, as part of a larger effort across HHS to reduce what it calls “unnecessary animal testing” in biomedical research.

• In addition to the new FDA rule, which would take effect Feb. 4, 2027, the NIH simultaneously announced a series of new initiatives intended to develop and promote alternatives to animal testing, including more than $88 million in funding for biomedical infrastructure projects nationwide. (Healio)

• The pace of M&A slowed to a crawl toward the end of the third quarter, but not before Vertex Pharmaceuticals took home one of the largest transactions of the year.

• The July 6 acquisition of Crinetics Pharmaceuticals earned Vertex—a company that rarely buys—the honor of the fourth biggest deal of the year so far.

• The deal was originally tallied at $10 billion by the companies, but with debt and other factors, S&P Capital IQ valued it at $8.8 billion. The deal pushed the cystic fibrosis drugmaker into new territory: the specialty endocrinology market.

• Lilly’s largest buy was Centessa Pharmaceuticals for $8.1 billion, according to S&P Capital IQ.

• The agent chose a relevant tool to look for the answer. Then it fed the tool a molecular string in an invalid format. The tool rejected it.

• The agent then tried to repair the molecule using another tool, but supplied essentially the same malformed input and got another error.

• The answer was wrong.

• The incident was not a story about an AI system going rogue. It was closer to an ordinary software problem: bad input, failed error handling and an improvised workaround that produced a plausible but incorrect scientific conclusion.

• In July, AI agents running an OpenAI cybersecurity evaluation circumvented controls meant to isolate them, reached the public internet and compromised systems on an infrastructure platform called Hugging Face while pursuing the task they had been assigned.

• OpenAI later said the models had resorted to “misaligned strategies” to solve hard tasks, turning the episode into an urgent warning about what can happen when increasingly autonomous agents act beyond their intended boundaries.

• A mundane failure can enter a workflow in which AI systems do far more than simply toss out predictions. They increasingly gather evidence, call specialized tools, interpret outputs and help determine what happens next.