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Of all the health care regulations coming out of the current administration, the recent drug rebate proposal presents a new opportunity to significantly lower out-of-pocket prescription drug costs for Medicare patients. But like most things in health care, there is more complexity to the administration’s proposed changes to pharmaceutical company rebates than initially meets the eye.

The Administration's Drug Rebate Proposal: An Opportunity to Put Patients First

The United States Food and Drug Administration (FDA) has approved penpulimab-kcqx with cisplatin or carboplatin and gemcitabine for the first-line treatment of adult patients with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC), the regulatory agency has announced.

The FDA has also approved the drug as a single agent for adults with metastatic non-keratinizing NPC with disease progression on or after platinum-based chemotherapy and at least one other prior line of therapy.

The effectiveness of penpulimab-kcqx plus cisplatin or carboplatin and gemcitabine was evaluated in Study AK105-304, a multicenter trial of 291 patients with recurrent or metastatic NPC who had not been treated with prior systemic chemotherapy for recurrent or metastatic disease. Patients were treated with either penpulimab-kcqx plus cisplatin or carboplatin and gemcitabine followed by penpulimab-kcqx or placebo with cisplatin or carboplatin and gemcitabine followed by placebo.

The median progression-free survival was 9.6 months in the penpulimab-kcqx arm and 7 months in the placebo arm, with 31% and 11% of patients alive and progression-free after 12 months of follow-up, respectively. Overall survival results were immature, with 70% of pre-specified deaths for final analysis reported, but the agency reported that no detrimental trend was observed.

Additionally, the effectiveness of single-agent penpulimab-kcqx was determined in Study AK105-202. The study included 125 patients with unresectable or metastatic non-keratinizing NPC who had disease progression after platinum-based chemotherapy as well as at least one other line of therapy and received penpulimab-kcqx until disease progression or unacceptable toxicity for up to 24 months.

The objective response rate was 28%, and a median duration of response was not reached.

Immune-mediated side effects that occurred with penpulimab-kcqx included pneumonitis, colitis, hepatitis, endocrinopathies, nephritis with renal dysfunction and skin adverse reactions, the FDA reported.

The most common side effects, occurring in at least 20% of patients who received penpulimab-kcqx with cisplatin or carboplatin and gemcitabine were nausea, vomiting, hypothyroidism, constipation, decreased appetite, decreased weight, cough, COVID-19 infection, fatigue, rash and pyrexia.

Furthermore, the most common side effects occurring in at least 20% of patients who received single-agent penpulimab-kcqx were hypothyroidism and musculoskeletal pain. Fatal side effects occured in 1% of patients, including one case each of pneumonitis, septic shock, colitis and hepatitis.

The recommended dosage of penpulimab-kcqx with cisplatin or carboplatin and gemcitabine is 200 milligrams every three weeks until disease progression or unacceptable toxicity for up to 24 months. The recommended dosage of single-agent penpulimab-kcqx is 200 milligrams every two weeks until disease progression or unacceptable toxicity for up to 24 months.

Penpulimab-kcqx, as explained by the National Cancer Institute on its website, is a monoclonal antibody that is intended to target, bind to and inhibit the protein PD-1 and its downstream signaling pathways, which may restore immune function through the activation of T cells and T cell-mediated immune responses against tumor cells. PD-1 functions as an immune checkpoint, negatively regulating T cell activation, and plays an important role in cancer’s evasion from a patient’s immune system, as the National Cancer Institute explained.

Nasopharyngeal cancer, the National Cancer Institute noted on its website, is a type of head and neck cancer that starts in the tissues of the nasopharynx. The nasopharynx, it explained, is the upper part of the pharynx, located behind the nose, and it is connected to the nostrils. In addition, openings on each side of the nasopharynx lead to the ears.

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The FDA has approved penpulimab-kcqx in combination with cisplatin or carboplatin and gemcitabine for first-line treatment of recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC) in adults. It is also approved as a single agent for adults with metastatic NPC after progression on platinum-based chemotherapy and another therapy. Study AK105-304 showed a median progression-free survival of 9.6 months with the combination therapy. Common side effects include nausea, hypothyroidism, and fatigue. Recommended dosages are 200 mg every three weeks or two weeks, depending on the regimen.

The FDA has approved penpulimab-kcqx with chemotherapy and as a single agent for some patients with nasopharyngeal carcinoma.

Tumor

FDA Approves Penpulimab-kcqx For Nasopharyngeal Carcinoma

Professor Richard Scolyer, a top doctor and leading melanoma pathologist, was diagnosed with Glioblastoma (GBM) brain cancer in July of 2023 and told he had only a few months to live. However, Richard didn't give up. Using everything he knew about melanoma science, Richard and his team used pre-surgery combined immunotherapy and a personalized cancer vaccine, the first in the world to do so.

Patients like Richard need your support in order to access the specialists, advanced treatments, and devices they need. Give today to support the End Brain Cancer Initiative's (EBCI’s) mission and help patients and families. Learn more about Richard's story at https://www.youtube.com/watch?app=desktop&v=2D9_mlLZPdI.

Support from individuals is vital for the End Brain Cancer Initiative to continue helping patients and families facing brain cancer, brain tumors, and metastatic disease to the brain. Gifts will support free access to DIRECT support from EBCI, a wealth of resources through the EBCI Website and the EBCI Disease Education Mobile App, and patient disease education online events. People can help EBCI reach their goal of $20,000 dollars raised for GiveBIG 2025 by giving at endbraincancer.org/donate.

Gifts to EBCI for GiveBIG will also fuel the nonprofit’s advocacy efforts, giving EBCI the ability to advocate for policies that increase patient access to specialists, advanced treatments, and devices. One such policy is the Bolstering Research And Innovation Now (BRAIN) Act (H.R.2767/ S.1330), which aims to advance the quest to defeat brain tumors, once and for all, by increasing research funding, fostering collaboration, promoting critical awareness efforts, and supporting innovations in and access to care.

GiveBIG 2025 is all about that good feeling we get when we show up for others, whether it’s a family member, a neighbor, or a local nonprofit we care about. People can make good things happen at the End Brain Cancer Initiative by giving to help patients and families facing brain cancer, brain tumors, and metastatic disease to the brain.

The End Brain Cancer Initiative is a 501(c)3 non-profit patient organization focused on disease education, awareness, outreach and increasing patient access. The End Brain Cancer Initiative, formerly known as the Chris Elliott Fund, is dedicated to ensuring that all patients diagnosed with brain cancer, a brain tumor, or metastatic disease to the brain have equal access to advanced diagnostics, treatments, specialists, and clinical trial participation. We believe that IMMEDIATE ACCESS to these options provides this patient community with the best HOPE for survival and sustained quality of life. We partner with industry, patients, researchers, advocacy groups, medical teams, hospital networks and others to educate patients and their caregivers so they can have empowered conversations with medical teams. Learn more about and support the End Brain Cancer Initiative’s work and mission at EndBrainCancer.org.

Professor Richard Scolyer, a renowned melanoma pathologist, was diagnosed with Glioblastoma in 2023. Despite a grim prognosis, he utilized his expertise to undergo innovative treatments, including pre-surgery combined immunotherapy and a personalized cancer vaccine. The End Brain Cancer Initiative (EBCI) supports patients like Richard by providing access to specialists, treatments, and resources. Donations to EBCI aid in advocacy efforts, such as the BRAIN Act, and help achieve fundraising goals like GiveBIG 2025. EBCI is committed to ensuring equal access to advanced care for brain cancer patients.

People can help EBCI reach their goal of $20,000 dollars raised for GiveBIG 2025 by giving at endbraincancer.org/donate.

NOINDEX

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Support Patient Access and Education with the End Brain Cancer Initiative for GiveBIG 2025

The End Brain Cancer Initiative (EBCI) is currently seeking Primary Central Nervous System Lymphoma (PCNSL) patients and care partners to participate in an online roundtable discussion on April 11, 2025, from 11AM to 12:30PM Pacific Time.

The PCNSL Online Roundtable will help further resources and education provided to this patient population by doing a deep dive into the results of their recent PCNSL patient and care partner survey. The roundtable, sponsored by Ono Pharmaceutical, will be moderated by Dellann Elliott Mydland, President and CEO of the End Brain Cancer Initiative.

Patients and care partners interested in participating in this discussion should fill out the interest form at endbraincancer.org/pcnsl before March 31, 2025. Qualifying participants will be compensated $75 for their time.

Ono Pharmaceutical Co., Ltd., headquartered in Osaka, is an R&D-oriented pharmaceutical company committed to creating innovative medicines in specific areas. Ono focuses its research on oncology, immunology, neurology, and specialty area with high medical needs as priority areas for discovery and development of innovative medicines. For further information, please visit the company’s website at https://www.ono-pharma.com/en.

To support/donate to the End Brain Cancer Initiative’s increased access and health delivery for patients, mission, services and programs, please visit EndBrainCancer.org

The End Brain Cancer Initiative (EBCI) is a 501(c)3 non-profit patient advocacy organization focused on disease education, awareness, outreach, increasing patient access and improving Standard of Care. The End Brain Cancer Initiative, formerly known as the Chris Elliott Fund (CEF), is dedicated to ensuring that all patients diagnosed with brain cancer, a brain tumor, or metastatic disease to the brain have equal access to advanced diagnostics, treatments, specialists, and clinical trial participation. We believe that IMMEDIATE ACCESS to these options provides this patient community with the best HOPE for survival and sustained quality of life. We partner with industry, patients, researchers, advocacy groups, medical teams, hospital networks and others to educate patients and their caregivers so they can have empowered conversations with their medical teams. Learn more about the End Brain Cancer Initiative at EndBrainCancer.org.

The End Brain Cancer Initiative (EBCI) is organizing an online roundtable for Primary Central Nervous System Lymphoma (PCNSL) patients and care partners on April 11, 2025. This event aims to enhance resources and education for this patient group by analyzing recent survey results. Sponsored by Ono Pharmaceutical, the roundtable will be moderated by EBCI's President, Dellann Elliott Mydland. Participants will receive $75 for their involvement. EBCI is a non-profit focused on improving access to advanced diagnostics and treatments for brain cancer patients.

Primary Central Nervous System Lymphoma Patient and Care Partner Roundtable Hosted by the End Brain Cancer Initiative

Registration is now open for the 2025 Know All Your Treatment Options (KAYTO25) online patient disease education event/webinar

Registration is now open for the 2025 Know All Your Treatment Options (KAYTO25) online patient disease education event/webinar, hosted by the End Brain Cancer Initiative (EBCI), on Friday, May 16th, 2025, from 11 AM to 2 PM Pacific Time.

This free annual online event is designed specifically for members of the brain tumor, brain cancer, and metastasized brain tumor community to hear directly from top specialists, including researchers, in the field about advanced and FDA approved treatment options, clinical trials, devices, diagnostics, etc. that they may not have heard about and to “DIRECTLY CONNECT” this patient population to many of these specialists. The event is free to attend due to corporate sponsorships, but pre-registration is required: endbraincancer.org/know-all-your-treatment-options-2025/.

KAYTO attendees will hear from multiple presenters, including a representative from Telix Pharmaceuticals, one of the presenting sponsors of this event. Telix is at the forefront of developing advanced imaging agents to improve the diagnosis and management of brain cancers, particularly gliomas. Telix’s brain cancer program comprises an investigational therapy and an investigational PET imaging agent – both small molecule-based. The company aims to provide clearer and more specific imaging results, aiding in the accurate differentiation between tumor progression and treatment-related changes.

Visit endbraincancer.org/know-all-your-treatment-options-2025/ to learn more about sponsorship opportunities to support this patient disease education online event.

Telix is a biopharmaceutical company focused on the development and commercialization of therapeutic and diagnostic radiopharmaceuticals and associated medical technologies. Telix is developing a portfolio of clinical and commercial stage products that aims to address significant unmet medical needs in oncology and rare diseases. Visit http://www.telixpharma.com/ for further information.

The 2025 Know All Your Treatment Options (KAYTO25) online event, hosted by the End Brain Cancer Initiative, will take place on May 16th, 2025. This free event aims to educate the brain tumor community about advanced treatment options and connect them with specialists. Sponsored by companies like Telix Pharmaceuticals, the event will feature presentations on innovative imaging agents for brain cancer management. The End Brain Cancer Initiative, a non-profit organization, focuses on improving patient access to diagnostics, treatments, and clinical trials, enhancing survival and quality of life.

Registration Open for the 2025 Know All Your Treatment Options Webinar Hosted by the End Brain Cancer Initiative

Each May, for over a decade, the End Brain Cancer Initiative (EBCI) has presented the National HOPE Award to a brain cancer patient who deeply inspires us with their courage and story. EBCI established this patient disease education initiative and award to highlight exceptional people who have done exceptional things during their brain cancer journey, all while inspiring others.

The deadline for submitting HOPE Award nominations is April 16th, 2025. To submit a nomination, people can visit https://endbraincancer.org/hope-award-nomination/. 

Patrick “Paddy” O'Donnell is the 2024 National HOPE Award recipient. Paddy was a 20-year-old collegiate ice hockey goaltender at the University of Utah when he had a seizure. He woke up in the Neuro ICU connected to tubes and monitors wondering what happened. A biopsy revealed he had Glioblastoma (GBM) and three tumors. Paddy was nominated by his mother, Anne O'Donnell. "Paddy had to create his own HOPE and sought answers and second opinions about his treatment," she said. "End Brain Cancer Initiative was vital in our search, at the most critical time, availing us of options and direct contact with experts in the field whom Dellann knew personally."

The 2025 HOPE Award recipient will be announced during the End Brain Cancer Initiative's Know All Your Treatment Options (KAYTO25) online patient disease education event/webinar on May 16th, 2025. This free annual online event is designed specifically for members of the brain tumor, brain cancer, and metastasized brain tumor community to hear directly from top specialists, including researchers, in the field about advanced and FDA approved treatment options, clinical trials, devices, diagnostics, etc. that they may not have heard about and to “DIRECTLY CONNECT” this patient population to many of these specialists. The event is free to attend due to corporate sponsorships, but pre-registration is required: endbraincancer.org/know-all- your-treatment-options-2025/.

To support/donate to the End Brain Cancer Initiative’s increased access and health delivery for patients, mission, services and programs, please visit EndBrainCancer.org. 

The End Brain Cancer Initiative (EBCI) annually presents the National HOPE Award to a brain cancer patient who inspires with their courage and story. The 2024 recipient is Patrick “Paddy” O'Donnell, a collegiate ice hockey goaltender diagnosed with Glioblastoma. The 2025 recipient will be announced at the KAYTO25 event, which connects patients with specialists and provides information on advanced treatments. EBCI, a non-profit organization, focuses on disease education, awareness, and increasing patient access to advanced diagnostics and treatments, aiming to improve survival and quality of life.

Nominations Open for the End Brain Cancer Initiative’s 2025 National Patient Disease Education Initiative and HOPE Award

In March 2025, the FDA approved neoadjuvant treatment with Imfinzi (durvalumab) plus gemcitabine and cisplatin, followed by single-agent, adjuvant Imfinzi following radical cystectomy in muscle invasive bladder cancer, according to a news release from the United States Food and Drug Administration (FDA).

Notably, the efficacy of the now-FDA-approved approach was evaluated in the randomized, open-label, multicenter, 

. In the study, the investigative combination demonstrated a statistically significant improvement in event-free survival and overall survival for patients with muscle invasive bladder cancer who were candidates for radical cystectomy (surgery to remove the urinary bladder).

In an interview with CURE, Dr. Matthew Galsky sat down to discuss the significance 

 and key takeaways for patients within this population.

“The most important takeaway [message] is that there has been a slow but steady improvement in our ability to treat muscle-invasive bladder cancer,” Galsky explained in the interview. “I think we are at a tipping point in the next few years, where the convergence of better therapies and better technologies — specifically, our ability to measure small amounts of cancer with increased precision using better testing — will all come together and potentially completely change the way we treat bladder cancer moving forward.”

Galsky is professor of Medicine (Hematology and Medical Oncology), director of Genitourinary Medical Oncology, co-director of the Center of Excellence for Bladder Cancer, and associate director for Translational Research, all located at The Tisch Cancer Institute, the Mount Sinai Health System, in New York, New York.

CURE: To begin, what makes this approval significant for patients and providers managing muscle-invasive bladder cancer?

: A standard treatment for muscle-invasive bladder cancer is the surgical removal of the bladder. We know that after the bladder is removed, a subset of individuals will experience the cancer reappearing elsewhere in the body, developing metastatic recurrence, typically months to years later.

For decades, doctors attempted to combine chemotherapy with surgery to eradicate any microscopic cancer cells that might be present, aiming to reduce the risk of metastatic recurrence. When chemotherapy is administered before surgery, it's called neoadjuvant therapy. This concept of giving chemotherapy prior to surgery to treat potential microscopic spread of cancer is used in various solid tumors. When chemotherapy is given after surgery, it's known as adjuvant therapy.

For decades, the only neoadjuvant therapy proved beneficial in muscle-invasive bladder cancer was a combination of chemotherapy drugs, including cisplatin. This truly represented the standard treatment approach for many years because nothing had been shown to be superior.

Finally, after several decades, the NIAGARA trial demonstrated that integrating immunotherapy in the pre-surgical and post-surgical periods decreases the risk of metastatic disease development and improves longevity for patients.

The NIAGARA trial demonstrated a 32% reduction in the risk of recurrence and a 25% reduction in the risk of death. How do these results compare with historical outcomes using neoadjuvant chemotherapy alone?

The historical outcomes for patients with muscle-invasive bladder cancer treated with chemotherapy followed by surgery are really mirrored by the control arm of this study, as that was the control arm. Now, patients in this study fared slightly better than what we typically observe historically, likely because our treatment and supportive care have evolved.

However, we know that beyond that standard of care, the addition of immune checkpoint blockade, or [Imfinzi (durvalumab)] — both in the pre-surgical and post-surgical periods — improves our ability to prevent cancer recurrence and allows patients to live longer, even exceeding what we consider the contemporary and historical standard treatment.

What can patients expect during treatment with this combination of immunotherapy and chemotherapy — both in terms of the timeline and potential side effects?

Immune checkpoint blockade involves drugs that we call PD-1 or PDL-1 inhibitors, and Imfinzi is a PDL-1 inhibitor. These drugs have been used alone in various indications and also combined with chemotherapy for a variety of different indications across various solid tumors. So, oncologists are quite accustomed to administering these combinations at this point.

Our extensive experience thus far indicates that when you give immunotherapy with chemotherapy, it does not seem to worsen the side effects of the chemotherapy, nor does the chemotherapy seem to worsen the side effects of the immunotherapy. That said, when you administer two classes of drugs simultaneously, there's a higher overall risk for side effects because you have the potential side effects of each component, but they don't amplify each other.

Regarding side effects, those that can occur with cisplatin-based chemotherapy, in particular, are the ones many people associate with chemotherapy. These can include fatigue, nausea, vomiting, hair loss, an impact on kidney function and neuropathy, such as numbness and tingling in the fingers and toes. Fortunately, many of these side effects are transient and reversible with chemotherapy. Immune checkpoint blockade, or immunotherapy, has a very different side effect profile; many patients experience few to no side effects with it. However, a subset of patients will develop side effects that we refer to as immune-related [side effects].

These involve inflammation of different parts of the body and really mimic what we think of as autoimmune diseases, except usually, when these side effects occur, they affect one organ in an individual rather than multiple organs. For example, one individual might develop inflammation of the skin, called dermatitis, which manifests as a rash or itching. Another person might get inflammation of the bowel, called colitis, which manifests as diarrhea. When these side effects occur, if they're mild, immunotherapy is held. If they're more severe, they are typically treated with steroid medication.

When we give chemotherapy with immunotherapy prior to surgery, in the neoadjuvant setting, it's administered in three-week cycles. During each three-week cycle, treatment is given twice, once in the first week and once in the second week. These three-week cycles are repeated four times, and then surgery is performed. After surgery, the immunotherapy is generally given once a month to complete eight more months of treatment.

What follow-up research or real-world data are needed to further validate the role of this regimen?

There are several other trials testing a similar concept: should we integrate immune checkpoint blockade with cisplatin-based chemotherapy in the perioperative setting, meaning both before and after surgery?

The results of these trials collectively will, of course, enhance our understanding of the full context regarding what is being achieved with these regimens. However, as a standalone trial, NIAGARA led to FDA approval of this regimen and is now an accepted standard of care for use in patients with muscle-invasive bladder cancer.

Transcript has been edited for clarity and conciseness.

The FDA approved a neoadjuvant treatment regimen combining Imfinzi (durvalumab) with gemcitabine and cisplatin, followed by adjuvant Imfinzi, for muscle-invasive bladder cancer. This approval is based on the NIAGARA trial, which showed significant improvements in event-free and overall survival. The integration of immunotherapy in pre- and post-surgical periods reduces metastatic disease risk and enhances patient longevity. The treatment involves immune checkpoint blockade, with manageable side effects. Ongoing trials will further elucidate the regimen's efficacy, but it is now a standard of care.

Dr. Matthew Galsky discusses the FDA approval of Imfinzi and chemo before and after surgery for muscle-invasive bladder cancer, based on the NIAGARA trial.

A. Mark Fendrick & Dan Klein

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