Four therapies have reshaped the outlook across oncology and metabolic disease in recent months. Two have already won FDA approval: a first-of-its-kind breast cancer drug guided by blood-based mutation detection and the first targeted pill for pancreatic cancer. A third, an mRNA melanoma vaccine, met its phase 3 primary endpoint and is moving toward regulatory review. A fourth, Eli Lilly’s next-generation obesity drug, is moving toward a 2027 filing while already reaching select patients through expanded access. Here’s what pharmacy leaders need to know about each.
1. Etcamah (camizestrant) — first-of-its-kind breast cancer drug
The FDA granted accelerated approval Sept. 4 to AstraZeneca’s Etcamah, marking the first cancer therapy approval guided by the detection of a resistance mutation in circulating tumor DNA before imaging shows disease progression. Used with a CDK4/6 inhibitor, it treats HR-positive, HER2-negative breast cancer in patients who develop an ESR1 mutation while receiving an aromatase inhibitor — a mutation present in fewer than 5% of patients at diagnosis but in nearly 40% after disease progression. In trials, it extended median progression-free survival to 16 months compared with 9.2 months with standard aromatase inhibitor therapy.
2. Rasonque (daraxonrasib) — first targeted pancreatic cancer pill
The FDA approved Rasonque on Aug. 26 for adults with advanced pancreatic cancer that stopped responding to prior treatment. In a 500-patient trial, median overall survival nearly doubled to 13.2 months compared with 6.7 months with chemotherapy, with a lower discontinuation rate. It’s the first drug to target multiple forms of RAS, a mutation long considered undruggable. A phase 3 trial is underway at more than 30 U.S. cancer centers and could expand its use earlier in treatment.
3. Intismeran + Keytruda — first mRNA cancer vaccine to hit a phase 3 endpoint
Merck and Moderna announced Aug. 19 that their personalized mRNA melanoma vaccine, paired with Keytruda, met its primary endpoint in the phase 3 INTerpath-001 trial, reducing the risk of recurrence or metastasis in patients with surgically removed stage IIB-IV melanoma. It’s the first randomized late-stage trial to demonstrate efficacy for neoantigen cancer vaccines. The results come as mRNA technology faces a mixed regulatory backdrop: an FDA panel backed the first mRNA flu vaccine in June even as HHS pulled nearly $500 million in mRNA vaccine contracts last year. The companies have not released full melanoma trial data yet but plan to seek regulatory approval.
4. Retatrutide — first triple-hormone obesity drug approaching the finish line
Eli Lilly’s investigational triple agonist, targeting GLP-1, GIP and glucagon receptors, helped adults with type 2 diabetes lower A1C by roughly 2% and lose an average of 33.3 pounds in a phase 3 trial reported March 19. Other TRIUMPH program readouts have shown weight loss of up to 30.3% at two years among patients with a BMI above 35. Lilly confirmed Aug. 4 that it established a formal expanded access program allowing select patients to receive the drug before approval. The program is available to patients 18 and older with refractory obesity and at least two serious weight-related complications. Lilly plans to file for FDA approval in the first quarter of 2027.
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