FDA grants accelerated nod to Boehringer’s lung cancer med; reinstates ousted CBER chief Vinay Prasad

By PharmaCompass

2025-08-14

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The US Food and Drug Administration (FDA) has granted an accelerated approval to Boehringer Ingelheim’s Hernexeos (zongertinib) to treat adults with advanced non-small cell lung cancer (NSCLC) with specific HER2 mutations who have received prior treatment. The agency also cleared Insmed’s Brinsupri (brensocatib) as the first therapy for non-cystic fibrosis bronchiectasis, a chronic lung disease.

Within two weeks of his exit, Vinay Prasad is back at the FDA to head its biologics division. The week also saw Bayer sign a US$ 1.3 billion deal with Kumquat Biosciences to develop an oncology drug that targets KRAS mutations.

Meanwhile, FDA has restricted bluebird bio’s Skysona gene therapy, which was approved in 2022, due to increased risk of blood cancer. After the recent deaths of non-ambulatory teenagers, Sarepta Therapeutics has reported new safety data for its gene therapy Elevidys, which shows no deaths among ambulatory patients with Duchenne muscular dystrophy (DMD).

In news from clinical trials, Novartis’ ianalumab became the first drug to show statistically significant phase 3 benefits in Sjögren’s disease, a chronic autoimmune disorder. Pfizer and Astellas’ Padcev (enfortumab vedotin) showed positive results in a phase 3 trial, when used in combination with Merck’s Keytruda (pembrolizumab) in patients with muscle-invasive bladder cancer (MIBC) who cannot receive or refuse cisplatin-based chemotherapy.

Eli Lilly’s oral obesity pill orforglipron met late-stage trial goals. However, it failed to beat the weight loss effect of Novo Nordisk’s semaglutide. And IO Biotech’s melanoma vaccine narrowly missed a trial endpoint.

FDA grants accelerated approval to Boehringer’s med for advanced lung cancer

The US Food and Drug Administration (FDA) has granted an accelerated approval to Hernexeos (zongertinib), a new drug from Boehringer Ingelheim, for adults with advanced non-small cell lung cancer (NSCLC) with specific HER2 mutations who have had prior treatment. In studies, the drug helped 75 percent of patients who had chemotherapy see their cancer shrink or disappear completely.

Okays Insmed’s Brinsupri for chronic lung condition: FDA has approved Insmed’s Brinsupri (brensocatib) as the first treatment for non-cystic fibrosis bronchiectasis, a chronic lung disease. The oral drug blocks overactive inflammatory enzymes in white blood cells, thereby preventing airway damage. Developed after Insmed acquired rights to the drug from AstraZeneca in 2016 for US$ 160 million, the medicine could exceed US$ 4 billion in annual sales by 2031, says a Nature report.

Vinay Prasad returns to FDA as top vaccine regulator after brief ouster

Vinay Prasad is back at the FDA’s Center for Biologics Evaluation and Research (CBER) as its head, overseeing vaccines and gene therapies, the Department of Health and Human Services (HHS) has said. Prasad was pressured to quit FDA on July 29, due to criticism over his handling of safety concerns with Sarepta Therapeutics’ gene therapy for DMD. HHS spokesperson Andrew Nixon said Prasad’s return was requested by the FDA.

FDA limits Skysona use after reports of increased blood cancer risk

FDA has restricted the use of bluebird bio’s gene therapy Skysona (elivaldogene autotemcel) to boys (aged four to 17 years) with the genetic disorder cerebral adrenoleukodystrophy (CALD) who lack alternative treatments or a matched stem cell donor. The change comes after rising incidence of blood cancers, including myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), were observed in patients taking the therapy. FDA now requires updated labelling, strict lifelong monitoring, and post-marketing studies to track long-term safety. At the time of its approval in 2022, 4 percent of patients being treated with Skysona developed MDS. By July 2025, that rate climbed to 15 percent, with the report of one death.

Bayer, Kumquat ink US$ 1.3 bn deal to develop drug that targets KRAS mutations

Bayer has agreed to pay up to US$ 1.3 billion to partner with US-based Kumquat Biosciences in order to develop a cancer drug that targets KRAS G12D mutation. Kumquat will lead initial phase trials, while Bayer will handle later development and commercialization. KRAS mutations occur in almost 25 percent of cancers. The deal gives Bayer access to a promising candidate as it seeks to strengthen its drug pipeline amid growing competition for its top sellers like Xarelto (rivaroxaban) and Eylea (aflibercept).

Novartis’ drug posts encouraging phase 3 results in Sjögren’s disease trial

Novartis reported positive late-stage trial results for ianalumab, a dual-action antibody that treats Sjögren’s disease. In two global studies involving 779 patients, the drug significantly reduced disease activity at 52 weeks compared to placebo, thereby meeting the primary goal. Sjögren’s disease is a serious autoimmune condition with few treatment options.

Padcev-Keytruda combo boosts survival in phase 3 trial on bladder cancer patients

Pfizer and Astellas have reported positive phase 3 results for their cancer therapy Padcev (enfortumab vedotin), when combined with Merck’s Keytruda (pembrolizumab) in patients with muscle-invasive bladder cancer (MIBC) who cannot receive or refuse cisplatin-based chemotherapy. The companies plan to discuss the data with global regulators. MIBC accounts for 30 percent of bladder cancer cases, yet up to half of patients are ineligible for cisplatin. The results highlight the potential of this regimen to become a new standard of care.

Sarepta reports safety data on Elevidys: Sarepta Therapeutics has reported new safety data for its gene therapy Elevidys, showing no deaths among ambulatory patients with DMD. Acute liver injuries occurred in 23 percent of cases, but none in this group were fatal, unlike in non-ambulatory patients where three deaths from acute liver failure have been reported.

IO Biotech’s melanoma vaccine eyes FDA approval, despite trial setback

IO Biotech’s experimental vaccine, Cylembio (imsapepimut and etimupepimut, adjuvant), combined with Merck’s Keytruda, showed promise in a late-stage study for advanced melanoma. However, the drug missed its primary endpoint. In a trial with 407 patients, the combination extended progression-free survival to 19.4 months, compared to 11 months with Keytruda alone. However, the results fell just short of being statistically significant. IO Biotech aims to submit a biologics license application by the end of 2025, seeking approval for Cylembio in treating advanced melanoma.

Lilly’s orforglipron falls short of Novo’s semaglutide: Eli Lilly’s experimental oral obesity drug, orforglipron, helped patients lose 12.4 percent of body weight, on an average, over 72 weeks in a phase 3 trial. The once-daily pill met all primary and secondary endpoints. However, its weight-loss effect was slightly lower than Novo Nordisk’s injectable semaglutide. The company plans to seek regulatory approval by the year-end.

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