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• Ionis Pharmaceuticals received FDA approval for its rare neurodegenerative disease treatment Zanvastro (zilganersen), paving the way for the company’s first wholly owned neurology product.

• Ionis’ drug is an RNA-based therapy for children and adults with Alexander disease, a progressive disorder that usually appears in childhood and causes problems with mobility and cognition, including difficulty swallowing and making intentional movements.

• The treatment, which is given as a spinal injection every three months, has a list price of $285,000 per dose, Ionis said.

• Alexander disease occurs when genetic mutations cause the body to make too much of a protein called glial fibrillary acidic protein (GFAP), which leads a type of brain cells called astrocytes to be dysfunctional.

• Zanvastro is designed to stop the excess accumulation of GFAP, targeting the underlying cause, according to Ionis. (Endpoints)

• The FDA overruled the advice of its own expert advisors and approved Etcamah as a frontline treatment for breast cancer.

• Oral selective oestrogen receptor degrader (SERD) Etcamah (camizestrant) is now approved for use in combination with a CDK4/6 inhibitor as a first-line treatment for locally advanced or metastatic HR-positive, HER2-negative breast cancer with a mutation in the ESR1 gene.

• The decision came after the FDA's Oncologic Drugs Advisory Committee (ODAC) voted in May that data from the pivotal SERENA-6 trial supporting AZ's marketing application for Etcamah was not strong enough to support approval. Shortly afterwards, the FDA extended its review by three months.

• The FDA also approved a companion diagnostic test developed by Guardant to detect emerging ESR1 resistance mutations in ctDNA, Guardant360 CDx, to support the rollout of Etcamah in this indication. (Pharmaphorum)

• Bayer has landed an accelerated US approval for its non-small cell lung cancer (NSCLC) therapy, Hyrnuo (sevabertinib), expanding the drug’s role into the frontline treatment setting within an indication linked to poor patient prognosis.

• Hyrnuo will now become available as a frontline treatment option for adults with locally advanced or metastatic, non-squamous NSCLC – specifically if they have HER2 (ERBB2) tyrosine kinase domain-activating mutations, as determined by an FDA-approved test.

• This accelerated approval marks a move up the treatment paradigm for Hyrnuo, an oral tyrosine kinase inhibitor (TKI), which was previously indicated for use in patients who had relapsed after systemic therapy. (Pharmaceutical Technology)

• Spying a “golden decade” beyond the branded drug industry’s looming patent cliff, Sandoz is making big bets on its biosimilar capabilities in the coming years.

• Under the company’s so-called Bio100 plan, Sandoz is setting the ambitious goal to establish a portfolio of more than 100 biosimilars by 2040, up substantially from the 13 it currently markets.

• Meanwhile, the company also aims to expand the overall biosimilar loss of exclusivity (LOE) value it holds from some 50% today to around 80% from 2035 onwards.

• Part and parcel to that, Sandoz hopes to double its net sales from 2025 through the middle of the next decade, according to a release outlining the strategy.

• For all of last year, the Swiss drugmaker reported 5% net sales growth at constant currencies to $11.1 billion.

• The company’s apparent urgency comes as brand-name drugmakers face a period of heavy patent losses toward the back half of the 2020s and early into the next decade. (Fierce Pharma)

• Novartis’ drug designed to lower levels of Lp(a), a known cardiovascular risk factor, failed to reduce patients’ risk of a heart attack, stroke, or other major cardiovascular events in the first late-stage test for a drug meant to tackle the mysterious molecule.

• The phase 3 Lp(a)Horizon trial enrolled 8,323 patients with elevated Lp(a) who had previously experienced a cardiovascular event or had established cardiovascular disease (CVD).

• The goal of the trial was to see if lowering Lp(a) with pelacarsen, an antisense oligonucleotide, could reduce the chance that these high-risk patients would experience a second event.

• An estimated 20% of the global population has levels of Lp(a), short for lipoprotein(a) and read as “L-P-little A,” that put them at risk.

• Given this large population of potential patients, numerous experts have said that drugs lowering the “diabolical” molecule could become blockbusters.

• But what exactly the molecule does in the body remains unknown, and studying it was long difficult due to its repeating structure. (Fierce Biotech)

• With over $400 million in funding and big pharma backing behind its experimental lupus treatment, the stakes were high for Beeline Medicines' lead candidate to succeed in mid-stage testing.

• Now, it looks like afimetoran has buzzed through the critical study with ease, and the biotech is making a beeline for pivotal lupus trials.

• Beeline on Wednesday reported that afimetoran met its primary response endpoint, as measured by the SLE Responder Index-4 (SRI-4), in a global Phase II study involving patients with systemic lupus erythematosus (SLE). The once-daily oral small molecule is an equipotent inhibitor of toll-like receptors 7 and 8 (TLR7/8).

• The clinical win comes about five months after the startup emerged from stealth with $300 million in funding and five immunology assets in-licensed from Bristol Myers Squibb, which teamed up with Bain Capital in 2025 to form Beeline.

• Then in June, the partners pumped an additional $126.3 million into the biotech in anticipation of a positive readout for afimetoran in SLE. (FirstWord)

• Michael Davis, the acting director of the Center for Drug Evaluation and Research, will now take on the role permanently, the Health and Human Services Department announced.

• Likewise, Karim Mikhail will become the full-time director of the Center for Biologics Evaluation and Research after leading the division in an interim role.

• The administration also created a new post for Jared Seehafer, who will be the first deputy commissioner for technology and artificial intelligence at the FDA. And Bret Koplow will serve as director of the Center for Tobacco Products.

• Mass layoffs, turnover in key positions and what appeared to be erratic decision making left drugmakers frustrated and investors on edge.

“The appointment of the current interim directors is likely to be seen as a sign of continuity and stability that should help ease concerns that the FDA will see major shifts in policy,” RBC Capital Markets analyst Brian Abrahams wrote in a note to clients.

• His special interest in psychedelics may also be a positive for companies such as Eli Lilly looking to bring new products to the market.

• Mikhail has fewer scientific credentials, but his decades of leadership at both large and small drugmakers and his push for accelerated drug development suggests he will be industry-friendly, Abrahams said.

• Trump tapped policy aide Heidi Overton for the role in August, but she may face a rocky confirmation.

• If she does get the job, Overton is likely to be more of a delegator to the division directors, Abrahams wrote. “We do not expect another `larger-than-life’ commissioner looking to remake the agency,” he said. (BioPharma Dive)

“The direction was a little counterintuitive. Clinically, we tend to watch for high liver enzymes, so seeing that low AST and ALT were associated with a higher likelihood of early-onset colorectal cancer stood out,” said Kersten Bartelt, a clinician and researcher at Epic Research who worked on the new analysis.

• The new research highlights an association but does not mean that low levels cause cancer or that people should rush to get an early colonoscopy due to low levels alone.

• Rather, the findings – which have not been published in a peer-reviewed journal – suggest an area to investigate to better understand the potential underlying factors in the rise in colorectal cancer among younger adults.