Summary: President Trump has announced the dismissal of FDA Commissioner Robert Makary, citing policy disagreements with the White House.

According to Reuters, U.S. President Trump has announced the dismissal of Food and Drug Administration (FDA) Commissioner Robert Makary.

Makary, a prominent surgeon and public health researcher formerly at Johns Hopkins University, was nominated to lead the FDA in 2025. During his tenure, he advanced several drug approval reforms.

The White House did not provide detailed reasons for the dismissal, but sources close to the administration said Makary had disagreements with the White House team on multiple regulatory policies, particularly regarding drug pricing and approval timelines.

The leadership change has drawn attention from the medical community and the pharmaceutical industry. Analysts say the transition could impact major drug approval decisions in the coming months.

According to The Washington Post, the White House is preparing to replace FDA Commissioner Marty Makary. The agency has been rocked by internal turmoil in recent months, with several policy decisions sparking controversy and eroding public trust. Makary’s reform agenda has drawn intense debate from both the scientific community and political circles.

Analysts suggest this leadership change could further impact the FDA’s regulatory direction and policy continuity.

Source: The Washington Post

The White House is preparing to replace FDA Commissioner Marty Makary, according to The Washington Post. President Trump has signed off on the ouster, senior officials confirmed. Makary’s tenure has been marked by internal policy disputes and personnel turmoil at the agency. The leadership change signals a potential shift in the FDA’s regulatory direction.

Source: The Washington Post, Google News

The New York Times reports that Trump has approved the dismissal of FDA Commissioner Marty Makary. This personnel change could shift the direction of US Food and Drug Administration policy. Makary has played a key role in drug approval and public health policy since taking office. A senior official confirmed Trump has signed off on the decision.

Source: NYT

According to a report by Ars Technica, the Trump administration reportedly blocked the U.S. Food and Drug Administration (FDA) from publishing research findings that demonstrated the benefits of vaccines – a move that has sparked strong concerns in the public health community.

What Happened

The report alleges that FDA researchers completed multiple studies showing positive vaccine outcomes, but these findings were subject to government-level intervention and censorship before publication.

This could prevent the public from accessing complete scientific information about vaccine safety and effectiveness, undermining transparency in public health decision-making.

Academic Response

Public health experts and medical researchers have expressed serious concern. They argue that transparency of scientific data is essential for maintaining public health and trust.

Several experts have called on the administration to stop interfering with the publication of scientific research results and to ensure the public has access to complete, objective health information.

The free flow of scientific information is the foundation of the public health system; any form of censorship harms the public interest.

Source: Ars Technica, compiled report

The U.S. Food and Drug Administration has withdrawn multiple studies that had found Covid and shingles vaccines to be safe. The move has drawn significant attention from the public health community.

The FDA has not provided detailed reasons for the withdrawal, but the decision could impact public confidence in vaccination programs. Health experts urge the public to continue following existing vaccination guidelines until the agency provides a clear explanation.

On May 5, 2026, the US Food and Drug Administration commissioner publicly defended the agency’s drug approval decisions, responding to criticism from the pharmaceutical industry and some members of Congress.

The Controversy

Several pharmaceutical companies have expressed dissatisfaction with the FDA’s approval pace and standards, arguing the agency has been overly cautious on certain medications. Some lawmakers have also raised concerns.

Commissioner Response

The FDA commissioner emphasized that approval standards will not be lowered under political or commercial pressure, and that protecting public health remains the agency’s top priority. The agency will continue to base approval decisions on scientific evidence.

May 2, 2026 — The U.S. Food and Drug Administration (FDA) has approved expanded access to daraxonrasib, a targeted pancreatic cancer drug, allowing more patients to use the medication before its formal market approval. This is the world’s first RAS gene mutation inhibitor, and clinical trials have demonstrated its potential to significantly extend survival in metastatic pancreatic cancer patients.

A Breakthrough for the “King of Cancers”

Pancreatic cancer is known as the “king of cancers” — one of the most aggressive malignancies with a five-year survival rate of less than 10%. Over 90% of pancreatic cancer patients carry KRAS gene mutations, a target long considered “undruggable.”

Daraxonrasib, developed by Revolution Medicines, is the first drug to demonstrate in clinical trials that it can effectively inhibit KRAS mutations and extend survival in pancreatic cancer patients. In Phase II trials, patients receiving the drug showed significantly improved median survival compared to historical controls.

Expanded Access Program

The FDA’s expanded access approval allows critically ill patients who cannot benefit from other treatments to use the drug before formal market approval. This decision means eligible pancreatic cancer patients can gain earlier access to this innovative therapy.

Notably, the wife of former Senator Ben Sasse is among the early beneficiaries of the drug. The Sasse family has publicly supported the drug’s development and shared positive responses on social media.

Scientific Breakthrough

RAS proteins play a critical role in cell signaling, and their mutations drive uncontrolled cancer cell growth. For decades, scientists struggled to develop RAS-targeting drugs because the protein’s smooth structure lacked traditional drug-binding sites, making it seem nearly impossible to target.

In recent years, advances in structural biology and drug design have finally enabled strategies to target specific RAS mutations (such as KRAS G12C and G12D). Daraxonrasib is the product of this scientific breakthrough.

Next Steps in Approval

Revolution Medicines is currently advancing daraxonrasib through Phase III clinical trials. If results meet expectations, the company expects to submit a New Drug Application (NDA) to the FDA within 12-18 months.

Wall Street analysts are optimistic about the drug’s commercial prospects. Pancreatic cancer affects over 500,000 new patients globally each year, and effective targeted treatment options remain extremely limited. If approved, daraxonrasib is projected to generate billions in annual revenue.

Medical Community Response

The American Society of Clinical Oncology (ASCO) issued a statement calling daraxonrasib’s progress “one of the most breakthrough developments in pancreatic cancer treatment.” Oncology experts at UW Health expressed enthusiasm for the drug’s potential in interviews.

Sources: The Washington Post, Reuters, CBS News

The U.S. Food and Drug Administration has approved expanded access to daraxonrasib, a targeted pancreatic cancer drug developed by Revolution Medicines, according to The New York Times and Reuters. The decision opens the door for more patients awaiting treatment to receive a therapy that has demonstrated significant efficacy in clinical trials.

A Breakthrough Therapy

Daraxonrasib is a targeted inhibitor directed at the KRAS G12D mutation. KRAS mutations are among the most common driver alterations in pancreatic cancer, found in approximately 35% to 40% of pancreatic ductal adenocarcinoma cases. For decades, the KRAS protein was considered “undruggable” due to its structural characteristics, but a new generation of covalent inhibitors has changed that landscape.

Reuters reports that in early-stage clinical trials, daraxonrasib caused significant tumor shrinkage in some patients with advanced pancreatic cancer. Notably, the spouse of former Senator Ben Sasse has shown encouraging results on the drug, drawing widespread public attention.

What Expanded Access Means

The FDA’s expanded access program — also known as “compassionate use” — allows unapproved drugs to be used by patients with serious or life-threatening conditions who cannot enroll in clinical trials. The approval of this pathway means more patients who are ineligible for trial participation can gain early access to treatment.

The Washington Post notes that the speed of the expanded access approval reflects the FDA’s strong confidence in the drug’s efficacy data. While such approvals typically take several weeks, this one was processed in under two weeks.

The Challenge of Pancreatic Cancer

Pancreatic cancer is often called the “king of cancers” and ranks as the fourth leading cause of cancer-related death in the United States. Due to its subtle early symptoms, difficulty in detection, and limited treatment options, the five-year survival rate has long hovered around just 12%.

Revolution Medicines’ Chief Medical Officer stated: “We are pleased to work with the FDA to give more patients in desperate need the opportunity to receive daraxonrasib. This is an important step toward transforming the pancreatic cancer treatment landscape.”

Market Outlook

Industry analysts project that if daraxonrasib continues to demonstrate positive data in later-stage trials, it could receive full FDA approval by 2027. At that point, the KRAS-targeted therapy market is expected to reach tens of billions of dollars, covering indications in pancreatic cancer, colorectal cancer, and non-small cell lung cancer.

Source: The New York Times · Reuters · USA Today

The U.S. Food and Drug Administration has approved expanded access to daraxonrasib, a breakthrough targeted therapy for patients with previously treated metastatic pancreatic ductal adenocarcinoma (PDAC). Known as the “king of cancers” for its devastating mortality rate, pancreatic cancer has long resisted effective treatment, making this approval a significant milestone for patients and oncologists alike.

The Drug

Daraxonrasib is a targeted therapy designed for pancreatic cancer patients carrying specific genetic mutations. The New York Times reported that the drug has demonstrated encouraging efficacy in clinical trials, showing the ability to significantly extend patient survival.

Expanded Access Program

The FDA’s Expanded Access program allows experimental drugs to be used outside of clinical trials for patients with serious or life-threatening conditions who have no comparable treatment alternatives. USA Today reported that this decision enables a broader pool of pancreatic cancer patients to receive daraxonrasib ahead of full regulatory approval.

Targeted Oncology noted that the approval specifically applies to previously treated metastatic PDAC patients — a population with extremely limited treatment options after first-line therapies fail.

The Pancreatic Cancer Challenge

Pancreatic cancer has long been one of oncology’s greatest challenges. Its early symptoms are subtle, diagnoses typically occur at advanced stages, and the disease responds poorly to conventional chemotherapy. The five-year survival rate remains below 10%, the lowest among all major cancers.

Patient Impact

According to WKOW, doctors at UW Health expressed excitement about the breakthrough developments in pancreatic cancer treatment. The expanded access approval for daraxonrasib means more patients will have a new treatment avenue after standard therapies have failed.

Future Outlook

Daraxonrasib remains in further clinical trials to collect the data needed for full FDA approval. If subsequent trial results confirm its safety and efficacy, the drug could become a standard component of pancreatic cancer treatment protocols.

Analysts note that this approval also reflects the FDA’s continued flexible approach to accelerating innovative drug reviews, particularly for fatal diseases lacking effective treatment options.

Source: New York Times | USA Today | Targeted Oncology

According to The New York Times and Reuters, the US Food and Drug Administration (FDA) has approved expanded access to daraxonrasib, an innovative pancreatic cancer drug, offering new hope for patients facing one of the deadliest forms of cancer.

Pancreatic cancer is one of the most aggressive and lethal cancers, with a five-year survival rate of approximately 12%. Because early symptoms are often unnoticeable, most patients are diagnosed at advanced stages when treatment options are extremely limited. Daraxonrasib, developed by Revolution Medicines, is a targeted drug aimed at KRAS gene mutations—one of the most common oncogenic mutations in pancreatic cancer.

Under the FDA’s expanded access program (also known as “compassionate use”), eligible pancreatic cancer patients can receive the treatment before the drug is formally approved for market. This policy applies to patients who have no other available treatment options and are not eligible for clinical trials.

Reuters reported that the drug has shown encouraging efficacy data in early clinical trials. Among treated patients, some demonstrated significant tumor shrinkage, and the drug was well-tolerated. Revolution Medicines plans to submit a New Drug Application (NDA) to the FDA later this year, seeking formal approval.

The New York Times analysis noted that the field of pancreatic cancer treatment has long lacked major breakthroughs, and daraxonrasib represents a significant advance. KRAS mutations were once considered “undruggable,” but in recent years, advances in drug development technology have led to remarkable progress in KRAS-targeted therapeutic strategies.

According to American Cancer Society data, pancreatic cancer causes approximately 50,000 deaths annually in the United States, making it one of the deadliest cancers globally. The expanded access approval means some patients can obtain this potentially life-extending treatment sooner.

An oncologist at UW Health described the development as “exciting” but also cautioned patients and doctors to maintain realistic expectations, as long-term efficacy and safety data still require further validation.

Sources: The New York Times, Reuters, USA Today

According to reports from The Washington Post, BBC, and CNN, a US federal appeals court ruled on May 1 to temporarily block the FDA’s rules allowing mail-order access to the abortion drug mifepristone. The decision has significant implications for reproductive healthcare access for millions of women across the country.

The ruling marks the latest development in a long-running legal challenge by anti-abortion groups against FDA policy. The appeals court determined that the FDA had not sufficiently evaluated safety implications when expanding access to mifepristone, and therefore temporarily suspended regulations that permitted mail-order prescriptions and telehealth prescriptions for the drug.

Following the ruling, reproductive rights advocacy groups responded swiftly, announcing plans for an immediate emergency appeal to the Supreme Court. “This ruling will directly harm women living in states where abortion clinics are scarce or where abortion is banned,” the ACLU’s Reproductive Freedom Project director said in a statement.

The FDA had previously allowed mail-order access to mifepristone, which enabled women living far from abortion clinics—particularly in rural areas—to more easily obtain safe medication abortion services. It is estimated that approximately one-third of medication abortions were completed via mail under the previous regulations.

The Guardian reported that this ruling comes amid ongoing state-level legislative challenges to abortion rights in the United States. Since the Supreme Court overturned Roe v. Wade in 2022, multiple states have implemented or attempted to implement strict abortion restrictions.

Legal experts suggest the case will likely be appealed to the Supreme Court once again. The Court had previously ruled in favor of preserving mifepristone access in similar cases, but the current composition of the Court introduces uncertainty about the potential outcome.

A White House spokesperson said the administration was “deeply disappointed” by the ruling and stated it would pursue all available legal avenues to protect women’s healthcare access.

Sources: The Washington Post, BBC, CNN

A US federal appeals court issued a temporary order on Thursday blocking the Food and Drug Administration’s (FDA) rule that allowed women to obtain the abortion drug mifepristone by mail, CNN and The New York Times reported. The ruling will have significant implications for medication abortion accessibility across the country.

The Ruling

The appeals court’s temporary order effectively suspends the FDA’s expanded policy that permitted certified healthcare providers to mail mifepristone to patients. Mifepristone is the primary drug used in medication abortions, which currently represent the most common method for the procedure in the United States.

The ruling is the latest development in an ongoing legal campaign by anti-abortion organizations and state governments challenging the FDA’s regulatory decisions on the drug. Opponents argue that mailing prescription abortion drugs poses safety risks and should be subject to stricter oversight.

Impact

The mifepristone-misoprostol medication abortion regimen is currently the most prevalent method for terminating early pregnancies in the US. The FDA’s previous rule allowing mail distribution of the drug significantly expanded access for women in remote areas and states with restrictive abortion laws.

The temporary order will reintroduce barriers to medication access for this population, particularly in states that have implemented strict abortion restrictions.

Reactions

Pro-choice organizations strongly condemned the ruling, arguing it infringes on personal medical autonomy. Related legal groups have indicated they will continue pursuing legal avenues to defend the FDA’s original policy.

Conversely, anti-abortion groups welcomed the ruling as an interim victory and called on the court to permanently overturn the FDA’s mail distribution rule.

Legal Background

The FDA’s regulatory approach to mifepristone has undergone several changes in recent years. In 2021, the agency eliminated the in-person dispensing requirement, allowing the drug to be distributed by mail. Since then, anti-abortion states and organizations have filed multiple lawsuits seeking to reverse this policy change.

It remains unclear whether the appeals court’s temporary order will persist and how the case will proceed through higher levels of the judicial system.

Source: CNN, The New York Times

Drug Background

Daraxonrasib is a selective inhibitor targeting the KRAS G12D mutation, developed by Revolution Medicines. KRAS gene mutations are one of the most common drivers of pancreatic cancer, accounting for approximately 35% to 40% of cases. For decades, KRAS mutations were considered “undruggable,” but recent technological advances have made it possible to selectively target specific KRAS mutations.

Clinical Trial Data

In early-stage clinical trials, daraxonrasib has demonstrated encouraging efficacy data. Some patients experienced significant tumor shrinkage, with disease control rates surpassing existing standard-of-care treatments. Perhaps most importantly, the drug has shown a favorable tolerability profile, with manageable side effects.

According to The New York Times, former U.S. Senator Ben Sasse is among the beneficiaries of the drug. After being diagnosed with pancreatic cancer, Sasse participated in the clinical trial and has seen his condition effectively controlled.

Expanded Access Program

The FDA’s approval of the Expanded Access Program, also known as “compassionate use,” allows patients with serious or life-threatening conditions to access investigational drugs before they receive formal approval.

This mechanism is particularly important for patients who are ineligible for ongoing clinical trials and have no other effective treatment options. Pancreatic cancer, often called the “king of cancers,” has a five-year survival rate of only about 12%, making it one of the cancer types with the most urgent need for new therapies.

Industry Significance

Analysts note that daraxonrasib’s progress is significant not only for pancreatic cancer patients but also opens new possibilities for treating other KRAS-mutated solid tumors, including colorectal cancer and non-small cell lung cancer.

Revolution Medicines’ shares rose following the announcement, reflecting market optimism about the drug’s commercial prospects. If daraxonrasib ultimately receives full FDA approval, it would represent a major milestone in pancreatic cancer treatment.

Next Steps

Revolution Medicines plans to continue advancing daraxonrasib through Phase III clinical trials to gather more comprehensive efficacy and safety data in support of full marketing approval. The company expects to report interim results from the pivotal trial within the next year.

Source: The New York Times, Reuters

The U.S. Food and Drug Administration (FDA) approved expanded early access for the experimental pancreatic cancer drug daraxonrasib on May 1, 2026. This decision brings new hope for late-stage pancreatic ductal adenocarcinoma (PDAC) patients and has sparked enthusiastic discussion in the medical community about the broad potential of targeted therapy in oncology.

Drug Background

Daraxonrasib is an experimental drug targeting the KRAS G12D mutation. KRAS gene mutations are extremely common in pancreatic cancer, with approximately 30-40% of PDAC patients carrying this mutation. For decades, KRAS was considered an “undruggable” target until recent breakthroughs in targeted therapy technology made substantive progress possible.

According to the New York Times, the drug has shown encouraging efficacy in early clinical trials — some late-stage pancreatic cancer patients experienced significant tumor shrinkage, with a few cases achieving complete remission. This result represents a milestone in pancreatic cancer treatment, as the disease has long been called the “king of cancers” with a five-year survival rate of less than 13%.

Expanded Access Program

The FDA’s approval is for an “Expanded Access” protocol, also known as “Compassionate Use.” This mechanism allows patients with serious or life-threatening diseases to access experimental drugs before they receive full regulatory approval.

According to the Washington Post, CBS News reported that former Senator Ben Sasse is one of the beneficiaries of this drug, sharing his treatment experience publicly. This high-profile political figure’s involvement has further elevated public awareness of the drug.

Professional medical journals including OncLive and Targeted Oncology have both covered this development in detail, noting that the approval of the expanded access protocol means the drug has received the FDA’s basic safety endorsement, paving the way for large-scale clinical trials and eventual market approval.

The Pancreatic Cancer Treatment Challenge

Pancreatic cancer is one of the deadliest cancers globally, characterized by difficulties in early diagnosis, poor response to traditional chemotherapy, and low surgical resection rates. According to the American Cancer Society, approximately 66,000 people in the United States are expected to be diagnosed with pancreatic cancer in 2026, with about 51,000 dying from the disease.

Mayo Clinic’s recently developed REDMOD AI system has demonstrated the ability to identify pancreatic cancer signatures from CT scans an average of 16 months before diagnosis (as reported in today’s AI & Tech section), while daraxonrasib’s progress represents an important breakthrough on the treatment side. The combination of AI-assisted diagnosis and targeted therapy may be reshaping the entire pancreatic cancer care ecosystem.

Looking Ahead

Analysts note that if daraxonrasib can confirm its efficacy in large-scale Phase III clinical trials, it would represent a major breakthrough in pancreatic cancer targeted therapy — the first in decades. Currently, multiple pharmaceutical companies are competing in this space, including other targeted drugs aimed at KRAS G12C mutations.

The expanded access approval means eligible patients can access treatment before the drug’s formal market approval, but it also requires doctors and patients to fully understand the drug’s experimental nature and potential risks.

Source: New York Times | USA Today | Washington Post

The US Food and Drug Administration (FDA) announced a major initiative in late April 2026 to introduce artificial intelligence and real-time data collection systems to accelerate the drug clinical trial approval process, according to the Wall Street Journal and STAT News. This move is seen as a fundamental transformation of the drug development regulatory model.

Core Elements of the Plan

The FDA’s initiative aims to leverage AI technology for real-time analysis and evaluation of clinical trial data, rather than the traditional post-hoc review model. By deploying advanced AI algorithms, the FDA will be able to continuously monitor trial progress, identify potential safety signals and efficacy trends earlier, and thereby shorten the cycle from clinical trials to drug approval.

According to the WSJ, the plan includes the following key components:

1. Real-Time Data Pipeline: Establishing direct data transmission channels from clinical trial sites to the FDA, enabling real-time data collection and analysis.
2. AI-Assisted Review: Using machine learning models to automatically identify patterns and anomalies in trial data, assisting reviewers in making more efficient decisions.
3. Adaptive Trial Design: Encouraging pharmaceutical companies to adopt more flexible trial designs that allow protocol adjustments based on interim data during the trial.

Industry Impact

This initiative has profound implications for the pharmaceutical industry. Traditional drug development cycles typically take over 10 years and cost billions of dollars. By introducing AI and real-time data technology, the FDA could shorten the approval cycle by months or even years, significantly reducing R&D costs.

According to TipRanks analysis, this policy change is particularly beneficial for small and mid-sized biotech companies, which typically have limited resources and rely more heavily on fast and efficient approval processes.

Historical Context and Challenges

The FDA is not new to AI applications. Previously, the FDA had deployed an AI tool called “Elsa” for routine regulatory operations. However, deeply integrating AI into the clinical trial approval process still faces numerous challenges, including data privacy protection, algorithm transparency, and the need for explainability of AI-driven decisions.

Previous reports noted that the FDA experienced some errors when using AI tools internally, raising questions about the reliability of AI-assisted regulatory decisions. The FDA needs to find the right balance between innovation and prudence.

Future Outlook

The FDA’s initiative aligns with the broader trend of accelerating AI applications in healthcare globally. If successfully implemented, it could not only transform the US drug approval process but also serve as a reference model for regulatory agencies in other countries.

Source: STAT News, Wall Street Journal, TipRanks

On April 28, 2026, the U.S. Food and Drug Administration approved Otarmeni (lunsotogene parvec-cwha), the world’s first dual adeno-associated virus (AAV) vector-based gene therapy for treating severe-to-profound sensorineural hearing loss associated with biallelic OTOF gene variants. The therapy was developed by Regeneron Pharmaceuticals.

The approval marks several milestones: it was granted just 61 days after the Biologics License Application (BLA) was filed, tying for the fastest BLA approval in modern FDA history. It is also the sixth product approved under the Commissioner’s National Priority Voucher (CNPV) pilot program and the first gene therapy product to receive approval under the program.

Genetic mutations cause approximately half of all congenital hearing loss. OTOF gene variants account for 2% to 8% of inherited, non-syndromic cases. Patients with two non-functional copies of the gene cannot produce otoferlin, a protein essential for sound signal transmission. Prior to this approval, no disease-modifying treatments existed for OTOF-related deafness.

Otarmeni is a one-time biologic-device combination product administered surgically as a single dose per ear into the cochlea. It delivers a functional copy of the OTOF gene to inner hair cells, restoring otoferlin production and auditory signaling.

In a clinical trial involving 24 patients aged 10 months to 16 years, 80% of the 20 evaluable patients (16 individuals) experienced improved hearing — an outcome not expected in the natural history of the disease without intervention.

FDA Commissioner Marty Makary, M.D., M.P.H., said: “Today’s approval is a significant milestone in the treatment of genetic hearing loss. Through the national priority voucher pilot program, we are accelerating therapies for rare diseases with unmet medical needs while proving we can successfully review even the most complex submissions — such as novel dual vector gene therapies and combination products requiring coordination across multiple offices and centers — in significantly shortened timeframes.”

Common side effects included middle ear infection, nausea, dizziness, and procedural pain. The therapy has received orphan drug, rare pediatric disease, fast track, and regenerative medicine advanced therapy (RMAT) designations.

Regeneron will continue to collect data on the durability of hearing improvement and verification of treatment effects on clinical measures of speech development and quality of life ahead of the FDA’s public meeting on the CNPV program scheduled for June 4.

Source: FDA, New England Journal of Medicine