Darwin's Our Take 8.31.26: FDA grants momentous approval in pancreatic cancer

FDA grants momentous approval in pancreatic cancer

The FDA granted two notable approvals in the past week — including one that could ultimately change the standard of care for pancreatic cancer — and cleared another blood test for use in helping to diagnose Alzheimer’s disease.

The approval with the most impact was that of Redwood City, Calif.-based Revolution Medicine (RevMed)’s Rasonque (daraxonrasib) as a once-daily oral treatment for adults with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy or who are not candidates for a multi-agent systemic therapy.

Rasonque targets RAS, a protein that regulates cell growth and other processes. The drug is the first targeted cancer medicine to gain approval from a “groundbreaking” new class of RAS(ON) multi-selective and mutant-selective inhibitors, RevMed said in a news release.

The earliest RAS inhibitors to receive FDA approval only block a specific mutation and target the protein in its “off,” or inactive, state, MedCity News reported. Rasonque blocks multiple forms of RAS in the “on” state.

Dr. Mark Goldsmith, RevMed’s CEO, said the approval “is a monumental step forward for patients with pancreatic cancer and for the oncology field. For the first time, patients have an approved targeted medicine designed to directly inhibit the main cause of pancreatic cancer, RAS, which has been one of the most intractable disease targets since its discovery decades ago.”

Dr. Anna Berkenblit, chief scientific and medical officer of the Pancreatic Cancer Action Network, said the “landmark approval is the most significant advance we have seen in the fight against pancreatic cancer.

“I believe this drug will transform how pancreatic cancer is treated, giving people the opportunity for more time with loved ones, the possibility of a better quality of life, and optimism that continued research may lead to even greater advances,” she added.

Dr. Brian Wolpin, director of the Hale Family Center for Pancreatic Cancer Research at Dana-Farber Cancer Institute and principal investigator for the Phase III trial used to support Rasonque’s approval, said he believes the drug is well positioned to become a new standard of care.

“For decades, despite RAS being the main drive and potential drug target for pancreatic cancer, physicians have largely relied on intravenous cytotoxic chemotherapy to treat this aggressive disease,” Dr. Wolpin said. “This approval gives physicians the confidence that directly inhibiting RAS can make a striking difference for patients and provides a critically needed new approach to treating patients with metastatic pancreatic cancer.”

In the randomized, open-label, Phase III RASolute 302 trial, Rasonque reduced the risk of death by 60%, RevMed said, and demonstrated “an unprecedented overall survival benefit compared with chemotherapy.”

Based on preliminary results from the trial, median overall survival was 13.2 months in the group that received Rasonque versus 6.7 months in the group that received standard-of-care chemotherapy.

Median progression-free survival was 7.2 months with Rasonque versus 3.6 months with standard chemotherapy. The study was published May 31 in The New England Journal of Medicine.

RevMed noted that Rasonque is approved for patients with or without an identified RAS tumor mutation, and that it does not require the use of a companion diagnostic test.

RevMed’s New Drug Application for Rasonque was reviewed under the FDA’s voucher pilot program; the approval was granted 35 days after the NDA was accepted — more than six months ahead of the user fee deadline.

The drug has a list price of $39,800 a month, multiple news outlets reported.

In other oncology-related news, the FDA approved Jazz Pharmaceuticals’ Ziihera (zanidatamab-hrii) as a first-line treatment for adults with certain types of HER2-positive gastric, gastroesophageal junction, and esophageal adenocarcinoma.

The newly approved indications are for Ziihera in combination with chemotherapy — with or without BeOne Medicines’ Tevimbra (tislelizumab-jsgr) — for adults with HER2-positive, unresectable, locally advanced, or metastatic forms of the cancers.

Dr. Geoffrey Ku, who is with Memorial Sloan Kettering Cancer Center and a co-author of the Phase III clinical trial that supported the newly approved indications, said in a news release, “This approval represents an important advancement in the treatment of HER2+ GEA [gastroesophageal adenocarcinoma].

”Importantly, similar outcomes were seen in patients whose tumors were PD-L1 positive or PD-L1 negative, suggesting that this regimen has the potential to benefit a broad range of patients,” Dr. Ku noted.

Dr. Rachna Shroff, cancer center chief of the division of hematology/oncology at University of Arizona’s College of Medicine, said results from the trial were “truly practice changing … and a landmark for the world of upper GI malignancies,” MedPage Today reported.

The FDA also approved two companion diagnostic devices for identifying patients who are candidates for treatment with Ziihera in accordance with the new indications.

Ziihera initially received accelerated approval in 2024 for adults with previously treated, unresectable, or metastatic HER2-positive biliary tract cancer.

The expanded approval of Ziihera triggered a $250 million milestone payment to Zymeworks under an exclusive strategic collaboration and license agreement the company has with Jazz. Zymeworks, the original developer of zanidatamab, is eligible for up to an additional $1.3 billion in Ziihera-related milestone payments.  

And in other FDA news, the agency cleared Roche and Eli Lilly’s blood test for Alzheimer’s disease.

The Elecsys pTau217 test is the first FDA-cleared single-biomarker blood test that supports both “rule-in and rule-out assessment” of amyloid pathology associated with Alzheimer’s disease, Roche said in a press release.

The test can be used in primary and specialty care settings to assess adults ages 55 and older who have signs, symptoms, or complaints of cognitive decline, according to Roche.

In May 2025, the FDA cleared Fujirebio Diagnostics’ Lumipulse G pTau217/Beta-Amyloid 1-42 Plasma Ratio, making it the first FDA-cleared in vitro diagnostic blood test for use in detecting amyloid plaques associated with Alzheimer’s disease. That blood test measures two proteins, phosphorylated tau 217 and beta-amyloid 1-42, and calculates their ratio.

Earlier this month, the FDA cleared C2N Diagnostics’ PrecivityAD2 for use in adults ages 40 years and older who present with signs or symptoms of cognitive impairment. This test uses mass spectrometry to measure ratios of amyloid and two forms of tau protein in the blood, BioPharma Dive reported.

OUR TAKE: Rasonque, the first drug in Revolution Medicines’ pipeline to receive FDA approval, has the potential to quickly become a blockbuster, according to analysts.

Some had expected the drug to have a list price of $25,000 to $30,000 for a month’s supply, so the reported list price of $39,800 per month is considerably higher than anticipated. Annually, that’s a wholesale acquisition cost of $477,600.

It’s also more than double the list price of Merck’s Keytruda (pembrolizumab), the top-selling cancer drug globally, Biopharma Dive noted.

Granted, Rasonque is only indicated for pancreatic cancer, but RevMed is evaluating the drug as a potential treatment for other cancers as well, including non-small cell lung cancer (NSCLC) and colorectal cancer. The company has additional promising RAS(ON) inhibitors in its pipeline, too, including zoldonrasib, which has demonstrated encouraging results in NSCLC.

According to Biopharma Dive, analysts at Evercore ISI estimate that sales of Rasonque could reach $2.4 billion next year and $15.1 billion by 2034 for the pancreatic cancer indication alone. They believe peak annual revenue for all potential indications could hit $20.8 billion.

Analysts at RBC Capital Markets weren’t quite as optimistic, placing peak annual sales for the pancreatic cancer indication at $11.5 billion.

Meanwhile, Jazz earned $24.8 million in revenue last year for Ziihera in the biliary tract cancer setting. According to an analyst with RBC Capital Markets, Biopharma Dive reported, the newly approved indications provide a market opportunity exceeding $1.5 billion.  

What else you need to know

Courts recently rejected several challenges to Medicare drug price negotiations established under the Inflation Reduction Act. In the latest development, the U.S. Court of Appeals for the Fifth Circuit on Thursday upheld a lower court’s ruling that rejected a challenge by PhRMA and two co-plaintiffs on the grounds the price negotiation program is unconstitutional.

Last Monday, the U.S. Court of Appeals for the Third Circuit ruled against Merck in a case that also challenged the constitutionality of the program. On Aug. 19, the U.S. District Court for the District of Maryland dismissed a lawsuit filed by AstraZeneca, which claimed CMS improperly grouped multiple products together as a “qualifying single source drug” for negotiation eligibility.‍

Roche’s Genentech will license a novel obesity therapy from Hanmi Pharm for up to $2.3 billion. The agreement is for HM17321, a proprietary injectable urocortin-2 analog that has a non-incretin mechanism of action and could be a first-in-class treatment designed to promote weight loss while preserving lean body mass.

South Korea-based Hanmi Pharm noted in the news release that GLP-1-based incretin therapies are associated with a loss of lean body mass. As a peptide-based therapy, HM17321 could be used in combination with incretin-based treatments, the company said.

Hanmi Pharm will complete an ongoing Phase I clinical trial before Genentech takes over development of HM17321. Genentech will pay Hanmi Pharm $190 million up front, as well as potential milestone payments and tiered royalties on net sales.  

McKesson agreed to pay approximately $2.25 billion for Precision Medicine Group, a company based in Bethesda, Md., that provides clinical research and biopharma commercialization services. According to the press release, Precision Medicine Group offers integrated services and technology-enable products such as biomarker intelligence, lab services, an international clinical research organization, market access, and commercialization support.

The transaction is subject to customary closing conditions, including regulatory approvals. Although McKesson did not provide an estimate for completing the acquisition, the company said Precision Medicine Group would become part of McKesson’s oncology and multispecialty unit when the deal has been finalized. ‍

Over 30 health systems and hospitals asked CMS not to finalize proposed limits on remote monitoring services. They are among more than 200 healthcare organizations that signed a letter to CMS Administrator Dr. Mehmet Oz urging the agency not to finalize remote monitoring policies in the 2027 Medicare Physician Fee Schedule proposed rule. They said finalizing the policies “would cause immediate and significant disruption for approximately 1 million Medicare beneficiaries who rely on remote monitoring to manage chronic conditions, avoid preventable complications, and remain connected to their care teams.”‍

The organizations added that finalizing the proposed policies would force providers to reduce enrollment, terminate programs, and potentially stop offering remote monitoring, noting that small practices, rural providers, and safety-net organizations would be hardest hit. Consequently, patients would likely experience increased emergency department visits, hospitalizations, and institutional care, they said.

The proposed rule includes a provision that would allow medical practices to bill Medicare for specified remote monitoring services only when the clinical staff delivering the services is directly employed by the practice. Roughly 60% to 70% of hospitals and health systems that provide remote monitoring rely on outsourced vendor models, Becker’s Hospital Review reported.

Dr. Peter Pisters will retire as president of UT MD Anderson Cancer Center on Sept. 30. Dr. Pisters joined MD Anderson 32 years ago and has served as president since 2017. Dr. Jeffrey Lee, a surgical oncologist, has been named interim president and will lead the organization “over the next several years,” according to the announcement. Dr. Lee has been with MD Anderson 35 years and currently serves as executive vice president, chief physician executive, and chief cancer network officer.

What we’re reading

Prescription to Over-the-Counter Switching as a Policy Tool. JAMA, 8.28.26

340B Could Inflate Spending In The CMS Bridge Program By More Than A Billion Dollars. Health Affairs, 8.27.26

Crew Resource Management — Navigating AI’s Automation Paradox. NEJM, 7.22.26 (subscription required)