AXL inhibitors(AXL receptor tyrosine kinase inhibitors), RET inhibitors(Tyrosine-protein kinase receptor RET inhibitors), ROS1 inhibitors(Proto-oncogene tyrosine-protein kinase ROS inhibitors)

Therapeutic Areas

NeoplasmsDigestive System DisordersEndocrinology and Metabolic Disease+ [11]

Active Indication

Extra-pancreatic neuroendocrine tumorNeuroendocrine tumor of pancreasDifferentiated Thyroid Gland Carcinoma+ [190]

Inactive Indication

Advanced Bile Duct CarcinomaAdvanced breast cancerAdvanced Malignant Solid Neoplasm+ [46]

Originator Organization

Exelixis, Inc.

Active Organization

Exelixis, Inc.Ipsen Pharma SAS

Ipsen SA

+ [14]

Inactive Organization

Jiangsu Hansoh Pharmaceutical Co., Ltd.

EMD Serono, Inc.

Drug Highest PhaseApproved

First Approval Date

United States (29 Nov 2012),

Thyroid Cancer, Medullary

RegulationFast Track (United States), Orphan Drug (United States), Priority Review (United States), Breakthrough Therapy (United States)

Structure/Sequence

Molecular FormulaC32H30FN3O10

InChIKeyHFCFMRYTXDINDK-WNQIDUERSA-N

CAS Registry1140909-48-3

276

Clinical Trials associated with Cabozantinib (s)-Malate

NCT06502171

/ Not yet recruitingPhase 1IIT

A Phase 1/1b/2 Study of Cabozantinib in Combination With Selumetinib for Plexiform Neurofibroma in Adults and Adolescents With Neurofibromatosis Type 1

Based on the clinical activity of both selumetinib and cabozantinib as monotherapies in clinical trials, the demonstrated activity of these agents in reduced doses in preclinical studies, and the non-overlapping toxicity profiles, the study will assess the tolerability and efficacy of selumetinib and cabozantinib in combination in participants with NF1 ≥16 years old with progressive and/or symptomatic PN in a phase 1/1b/2 clinical trial.
Trial Design Phase 1 This will be an open label, dose escalation phase. Dose level escalation will be determined by a rolling six design. In this design, up to 6 participants can be enrolled at a given dose level and then evaluated for dose limiting toxicity (DLT) within the DLT window. The DLT window is defined as 16 weeks in this study based on the long half-life of cabozantinib and the desire to have maximum confidence about long-term tolerability of the combination prior to proceeding to the next dose level.
Phase 1b Once the recommended phase 2 dose has been determined in phase 1, an expanded cohort of 12 participants will be enrolled in phase 1b portion of the study.
Phase 2 This will be an open label, single-arm phase using the recommended phase 2 dose.

Start Date01 Jul 2025

Sponsor / Collaborator

The University of Alabama at Birmingham

[+2]

NCT06900595

/ Not yet recruitingPhase 2IIT

A Phase II Study of Cabozantinib in Combination With Cemiplimab (Cabo-Cemiplimab) Versus Cabozantinib Alone in Adolescents and Adults With Advanced Adrenocortical Cancer

This phase II trial compares the effect of giving cabozantinib with or without cemiplimab in patients with adrenocortical cancer that has spread to nearby tissue or lymph nodes (locally advanced), and that cannot be removed by surgery (unresectable) or that has come back after a period of improvement (recurrent) or that has spread from where it first started (primary site) to other places in the body (metastatic). Cabozantinib is in a class of medications called tyrosine kinase inhibitors. It works by blocking the action of an abnormal protein that signals cancer cells to multiply, which may help keep cancer cells from growing. Immunotherapy with monoclonal antibodies, such as cemiplimab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving cabozantinib with cemiplimab may kill more tumor cells in patients with locally advanced unresectable or recurrent/metastatic adrenocortical cancer.

Start Date13 Jun 2025

Sponsor / Collaborator

National Cancer Institute

NCT06811116

/ Not yet recruitingPhase 1/2IIT

A Phase I/II Trial of Sapanisertib in Combination With Cabozantinib in β-Catenin-Mutated Hepatocellular Carcinoma

This phase I/II trial studies the side effects and best dose of sapanisertib when given together with cabozantinib, and to see how well they work in treating patients with liver cancer that has spread from where it first started to other places in the body (metastatic) and contains a mutation (change) in the β-catenin gene. Sapanisertib and cabozantinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving sapanisertib and cabozantinib together may work better than giving cabozantinib alone in treating β-catenin-mutated metastatic hepatocellular carcinoma.

Start Date16 May 2025

Sponsor / Collaborator

National Cancer Institute

100 Clinical Results associated with Cabozantinib (s)-Malate

100 Translational Medicine associated with Cabozantinib (s)-Malate

100 Patents (Medical) associated with Cabozantinib (s)-Malate

2,640

Literatures (Medical) associated with Cabozantinib (s)-Malate

01 Jul 2025·BIOCHEMICAL PHARMACOLOGY

Relevance of transportome among the mechanisms of chemoresistance in hepatoblastoma

Article

Author: Macias, Rocio I R ; Martinez-Chantar, Maria Luz ; Asensio, Maitane ; Barranco, María Manuela ; Avila, Matias A ; Briz, Oscar ; Armengol, Carolina ; Cives-Losada, Candela ; Marin, Jose J G ; Cairo, Stefano ; Río-Álvarez, Álvaro Del ; Chinchilla-Tábora, Luis Miguel

Approximately 20 % of hepatoblastomas (HBs) exhibit a poor response to conventional chemotherapy due to mechanisms of chemoresistance (MOCs), such as reduced intracellular drug accumulation. This study evaluated the role of transportome in the multidrug resistance (MDR) of HB. Paired HB and adjacent liver tissue samples (n = 19) and HB-derived cell lines (HepG2, HuH6) were analyzed for their resistome characterization at mRNA (RT-qPCR, Taqman Low-Density Array, sequencing) and protein (western blot, immunohistochemistry, immunofluorescence) levels. Cell viability (MTT test) proliferation and migration (holographic microscopy) were determined. The impact of short-term (72 h) and long-term (>10 months) exposure of HB cells to cisplatin or doxorubicin on the transportome was investigated. Solute carrier (SLC) family of transporters showed minor relevance in HB MDR, while drug export pumps, particularly MRP2, were associated with poor response to chemotherapy. Exposure of HB cells to doxorubicin or cisplatin up-regulated MDR1, MRP1 and MRP2. In cells with induced persistent chemoresistance, the expression of genes involved in other MOCs, and epigenetic machinery was altered. Chemoresistant cells showed cross-resistance to several anticancer drugs but maintained sensitivity to cabozantinib. In conclusion, drug export pumps, but not SLC uptake transporters, are key contributors to HB chemoresistance. Cabozantinib emerges as a potential therapeutic option for HBs resistant to conventional chemotherapy.

01 Jun 2025·Clinical Genitourinary Cancer

Pathological Complete Response in Metastatic Renal Cell Carcinoma Patients Treated With Cabozantinib Plus Nivolumab. Case Series and Literature Review

Article

Author: Fotia, Giuseppe ; Guadalupi, Valentina ; Verzoni, Elena ; Stellato, Marco ; Rota, Simone ; Procopio, Giuseppe ; Barella, Marco ; Claps, Melanie ; Rametta, Alessandro

01 Jun 2025·Clinical Genitourinary Cancer

Characterization of Exceptional Responses in Patients With Metastatic Castration-Resistant Prostate Cancer Treated With Cabozantinib and Immune Checkpoint Inhibitors

Article

Author: Oh, William K ; Joshi, Himanshu ; Tsao, Che-Kai ; Anker, Jonathan F ; Miller, Eric ; Ganta, Teja

455

News (Medical) associated with Cabozantinib (s)-Malate

16 Apr 2025

·www.ipsen.com

Ipsen delivers strong sales in the first quarter 2025 and confirms its full-year guidance

Total sales growth of 11.6% at CER1, or 11.7% as reported, driven by all three therapeutic areas and including an increasing contribution from Iqirvo and Bylvay. Tovorafenib regulatory submission to EMA2 for pediatric low-grade glioma. Confirmation of full-year 2025 financial guidance. PARIS, FRANCE, 16 April 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents sales for the first quarter of 2025. “Ipsen has delivered a strong start to 2025, building further momentum in the transformation of our company,” commented David Loew, Chief Executive Officer, Ipsen. “We continued to execute on our strategy with strong top-line growth and pipeline progression. I am pleased to see the rapid build-up of our Rare Cholestatic Liver disease franchise with two innovate medicines for five indications. 2025 is set to be an important year for Ipsen, with multiple launches underway and several milestones expected across our portfolio.” Full-year guidance Ipsen is confirming financial guidance for full-year 2025: Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in March 2025, a limited effect on total sales from currencies is expected. Core operating margin greater than 30.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities. Guidance includes expected negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. It excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions. Upcoming Milestones Ipsen anticipates several key milestones across its portfolio in 2025, including: Cabometyx (CABINET trial) – Regulatory decision in the European Union for advanced pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors (NETs). fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP). LANT3 (LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics. Q1 pipeline progress The regulatory filing for tovorafenib was accepted by EMA for review in the European Union, marking an important step forward in the development of this potential treatment for pediatric low-grade glioma and reinforcing Ipsen’s commitment to innovation in rare and difficult-to-treat cancers. Ipsen also initiated the entry in Phase I of IPN01195, a RAF inhibitor, complementing IPN01194, an ERK inhibitor, and tovorafenib, two other assets targeting the MAPK pathway. Group refinancing Ipsen announced on March 19th the successful completion of its inaugural Rated Public Bond of €500 million with a coupon of 3.875%, maturing in March 2032. Following the disclosure of the Investment Grade ratings from S&P and Moody’s, this transaction was very well received and largely oversubscribed by a diversified and solid institutional investor base. This transaction is an important component of Ipsen’s refinancing plan which included the successful renewal of €1,5 billion syndicated Revolving Credit Facility, extending Ipsen’s debt maturity profile. Conference call A conference call and webcast for investors and analysts will begin today at 2pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its half-year results on July 31st, 2025. Notes All financial figures are in € millions (€m). The performance shown covers the three-month period to 31 March 2025 (Q1 2025, the quarter), compared to the three-month period to 31 March 2024 (Q1 2024), unless stated otherwise. About Ipsen We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by internal and external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries. Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com. Ipsen contacts InvestorsKhalid Deojee +33 6 69 09 12 96 MediaSally Bain +1 857 320 0517Anne Liontas +33 7 67 34 72 96 Disclaimers and/or forward-looking statements The forward-looking statements, objectives and targets contained herein are based on Ipsen’s management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect Ipsen’s future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words ‘believes’, ‘anticipates’ and ‘expects’ and similar expressions are intended to identify forward-looking statements, including Ipsen’s expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external-growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen’s latest Universal Registration Document, available on ipsen.com. 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.2 EMA: European Medicines Agency 3 Long-acting neurotoxin Attachment Ipsen PR_Q1 2025_15042025 Total sales growth of 11.6% at CER1, or 11.7% as reported, driven by all three therapeutic areas and including an increasing contribution from Iqirvo and Bylvay. Tovorafenib regulatory submission to EMA2 for pediatric low-grade glioma. Confirmation of full-year 2025 financial guidance. PARIS, FRANCE, 16 April 2025 – Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-care biopharmaceutical company, today presents sales for the first quarter of 2025. “Ipsen has delivered a strong start to 2025, building further momentum in the transformation of our company,” commented David Loew, Chief Executive Officer, Ipsen. “We continued to execute on our strategy with strong top-line growth and pipeline progression. I am pleased to see the rapid build-up of our Rare Cholestatic Liver disease franchise with two innovate medicines for five indications. 2025 is set to be an important year for Ipsen, with multiple launches underway and several milestones expected across our portfolio.” Full-year guidance Ipsen is confirming financial guidance for full-year 2025: Total sales growth greater than 5.0%, at constant currency. Based on the average level of exchange rates in March 2025, a limited effect on total sales from currencies is expected. Core operating margin greater than 30.0% of total sales, which includes additional R&D expenses from anticipated early and mid-stage external-innovation opportunities. Guidance includes expected negative impact on Somatuline sales due to increased generic competition in the U.S. and Europe. It excludes any impact from potential late-stage (Phase III clinical development or later) business development transactions. Upcoming Milestones Ipsen anticipates several key milestones across its portfolio in 2025, including: Cabometyx (CABINET trial) – Regulatory decision in the European Union for advanced pancreatic (pNETs) and extra-pancreatic (epNETs) neuroendocrine tumors (NETs). fidrisertib (FALKON trial) – Readout of the pivotal Phase IIb trial in fibrodysplasia ossificans progressiva (FOP). LANT3 (LANTIC trial) – Proof-of-concept data readout, evaluating its potential in aesthetics. Q1 pipeline progress The regulatory filing for tovorafenib was accepted by EMA for review in the European Union, marking an important step forward in the development of this potential treatment for pediatric low-grade glioma and reinforcing Ipsen’s commitment to innovation in rare and difficult-to-treat cancers. Ipsen also initiated the entry in Phase I of IPN01195, a RAF inhibitor, complementing IPN01194, an ERK inhibitor, and tovorafenib, two other assets targeting the MAPK pathway. Group refinancing Ipsen announced on March 19th the successful completion of its inaugural Rated Public Bond of €500 million with a coupon of 3.875%, maturing in March 2032. Following the disclosure of the Investment Grade ratings from S&P and Moody’s, this transaction was very well received and largely oversubscribed by a diversified and solid institutional investor base. This transaction is an important component of Ipsen’s refinancing plan which included the successful renewal of €1,5 billion syndicated Revolving Credit Facility, extending Ipsen’s debt maturity profile. Conference call A conference call and webcast for investors and analysts will begin today at 2pm CET. Participants can access the call and its details by registering here; webcast details can be found here. Calendar Ipsen intends to publish its half-year results on July 31st, 2025. Notes All financial figures are in € millions (€m). The performance shown covers the three-month period to 31 March 2025 (Q1 2025, the quarter), compared to the three-month period to 31 March 2024 (Q1 2024), unless stated otherwise. About Ipsen We are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience. Our pipeline is fueled by internal and external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 100 countries. Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com. Ipsen contacts InvestorsKhalid Deojee +33 6 69 09 12 96 MediaSally Bain +1 857 320 0517Anne Liontas +33 7 67 34 72 96 Disclaimers and/or forward-looking statements The forward-looking statements, objectives and targets contained herein are based on Ipsen’s management strategy, current views and assumptions. Such statements involve known and unknown risks and uncertainties that may cause actual results, performance or events to differ materially from those anticipated herein. All of the above risks could affect Ipsen’s future ability to achieve its financial targets, which were set assuming reasonable macroeconomic conditions based on the information available today. Use of the words ‘believes’, ‘anticipates’ and ‘expects’ and similar expressions are intended to identify forward-looking statements, including Ipsen’s expectations regarding future events, including regulatory filings and determinations. Moreover, the targets described in this document were prepared without taking into account external-growth assumptions and potential future acquisitions, which may alter these parameters. These objectives are based on data and assumptions regarded as reasonable by Ipsen. These targets depend on conditions or facts likely to happen in the future, and not exclusively on historical data. Actual results may depart significantly from these targets given the occurrence of certain risks and uncertainties, notably the fact that a promising medicine in early development phase or clinical trial may end up never being launched on the market or reaching its commercial targets, notably for regulatory or competition reasons. Ipsen must face or might face competition from generic medicine that might translate into a loss of market share. Furthermore, the research and development process involves several stages each of which involves the substantial risk that Ipsen may fail to achieve its objectives and be forced to abandon its efforts with regards to a medicine in which it has invested significant sums. Therefore, Ipsen cannot be certain that favorable results obtained during preclinical trials will be confirmed subsequently during clinical trials, or that the results of clinical trials will be sufficient to demonstrate the safe and effective nature of the medicine concerned. There can be no guarantees a medicine will receive the necessary regulatory approvals or that the medicine will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Other risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and healthcare legislation; global trends toward healthcare cost containment; technological advances, new medicine and patents attained by competitors; challenges inherent in new-medicine development, including obtaining regulatory approval; Ipsen’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of Ipsen’s patents and other protections for innovative medicines; and the exposure to litigation, including patent litigation, and/or regulatory actions. Ipsen also depends on third parties to develop and market some of its medicines which could potentially generate substantial royalties; these partners could behave in such ways which could cause damage to Ipsen’s activities and financial results. Ipsen cannot be certain that its partners will fulfil their obligations. It might be unable to obtain any benefit from those agreements. A default by any of Ipsen’s partners could generate lower revenues than expected. Such situations could have a negative impact on Ipsen’s business, financial position or performance. Ipsen expressly disclaims any obligation or undertaking to update or revise any forward-looking statements, targets or estimates contained in this press release to reflect any change in events, conditions, assumptions or circumstances on which any such statements are based, unless so required by applicable law. Ipsen’s business is subject to the risk factors outlined in its registration documents filed with the French Autorité des Marchés Financiers. The risks and uncertainties set out are not exhaustive and the reader is advised to refer to Ipsen’s latest Universal Registration Document, available on ipsen.com. 1 At constant exchange rates (CER), which exclude any foreign-exchange impact by recalculating the performance for the relevant period by applying the exchange rates used for the prior period.2 EMA: European Medicines Agency 3 Long-acting neurotoxin Attachment Ipsen PR_Q1 2025_15042025

Phase 2Phase 1Financial Statement

13 Apr 2025

·medcitynews.com

BMS Immunotherapy Combo Gets Two FDA Nods for GI Cancers in Span of a Week - MedCity News

The pairing of two Bristol Myers Squibb immunotherapies is now permitted for the first-line treatment of two types of gastrointestinal cancers, after regulatory decisions handed out this past week that convert the accelerated approval status of the drug combination to full FDA approval in both indications. The drugs, Opdivo and Yervoy, belong to the class of medicines called checkpoint inhibitors. On April 8, the FDA approved use of this drug combination for adults and children age 12 and older whose colorectal cancer has metastasized or cannot be removed by surgery. The cancer must also be microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), references to biomarkers that indicate the stability of DNA in a tumor. The regulatory submission was based on the results of an open-label Phase 3 test that compared the Opdivo/Yervoy combination to either Opdivo alone or chemotherapy. Results showed the immunotherapy combination led to a 38% reduction in the risk of disease progression or death compared to Opdivo monotherapy, and by 79% compared to chemo, both in the first-line setting. The study is ongoing to assess secondary measures, including overall survival. BMS said it will continue working with study investigators to present these data and longer-term follow-up results in the future. Sponsored Post Changes in Nurse Staffing Answer Clinician Demands The ongoing nursing shortage facilitates high turnover rates since nurses know they won’t have difficulties finding new jobs. In order to retain and attract staff, it’s in a facility’s best interest to understand what nurses want. By Tomasz Rezik On April 11, the FDA approved the combination of Opdivo and Yervoy for treating advanced cases of hepatocellular carcinoma. In the pivotal test in this indication, the drug combination led to median overall survival of 23.7 months. The comparator arm, in which patients received standard of care drugs sorafenib or levantinib, median overall survival was 20.6 months. At three years, the drug combination showed an overall survival rate of 38% versus 24% in the comparator arm. FDA approval of Opdivo plus Yervoy in hepatocellular carcinoma follows the European Commission’s early March approval of the drug combination in this indication. There’s plenty of news on the regulatory front. Here’s a recap of other recent highlights: New and Expanded Regulatory Approvals —Argenx drug Vyvgart Hytrulo received an additional approval for dosing of the product via a prefilled syringe. The drug, supplied in vials, previously received FDA approvals for the autoimmune conditions chronic inflammatory demyelinating polyneuropathy and generalized myasthenia gravis. Analysts say prefilled syringes bring patients a more convenient dosing option while giving Argenx the opportunity to get more market share by penetrating into earlier lines of therapy. Sponsored Post Understanding EGPA: The Role of Eosinophils and Advancements in Treatment Options FASENRA® (benralizumab) injection, for subcutaneous use, 30 mg is indicated for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA). FASENRA provides a treatment option for HCPs to consider when managing this challenging disease. By FASENRA, Sponsored Content —Amgen’s Uplizna expanded its label to include the treatment of immunoglobulin G4-related disease (IgG4-RD), an immune-mediated inflammatory disorder that can affect multiple organs. The regulatory decision makes the intravenously infused antibody the first FDA-approved treatment for this rare and chronic disease. Uplizna was first approved in 2020 as a treatment for neuromyelitis optica spectrum disorder. —Bayer’s Viktrakvi now has full FDA approval for the treatment of NTRK gene fusion-positive solid tumors in adults and children without a known acquired resistance mutation. The drug received accelerated approval in this indication in 2018. —AstraZeneca and Daiichi Sankyo welcomed European Union approval of Datroway as a treatment for metastatic breast cancer that is HR positive and HER2 negative. Datroway received FDA approval in the same indication in January. The drug is an antibody drug conjugate designed to target the cancer protein TROP2. —Novartis drug atrasentan, brand name Vanrafia, was awarded accelerated approval as a new treatment for the kidney disorder immunoglobulin A nephropathy (IgAN). The drug is an oral small molecule designed to block the endothelin A receptor. It’s one of two IgAN nephropathy drugs that came via the $3.2 billion acquisition of Chinook Therapeutics in 2023. Novartis’s internal IgAN research yielded the complement inhibitor Fabhalta, which received accelerated approval in this indication last summer. —Speaking of Fabhalta, that Novartis drug expanded its label to include C3 glomerulopathy, making the twice-daily pill the first approved treatment for this rare kidney disorder. Fabhalta is a small molecule designed to block the complement protein C3. It was first approved in 2023 as a treatment for the rare blood disorder paroxysmal nocturnal hemoglobinuria. —Sanofi received FDA approval for fitusiran, brand name Qfitlia, as a treatment for both hemophilia A and B. The drug leverages RNA interference to lower levels of AT, a protein that inhibits blood clotting. The approval covers the treatment of adults and children age 12 and older with or without inhibitors, which are antibodies that can develop in hemophilia patients, making it more difficult to control bleeding. By contrast, new Pfizer drug Hympavzi is approved only for hemophilia A and B patients without inhibitors while Sanofi’s Alhemo is approved only for hemophilia A and B patients with inhibitors. —Blockbuster AstraZeneca cancer immunotherapy Imfinzi landed European Union approval for the treatment of limited-stage small cell lung cancer. This new approval is based on Phase 3 results showing a 27% reduction in the risk of death compared to a placebo. The FDA approved Imfinzi for this indication last December. —Imfinzi also added perioperative treatment of muscle invasive bladder cancer to its list of FDA-approved indications. The newest FDA nod covers use of the drug in combination with chemotherapy before surgery to remove the bladder, followed by Imfinzi as an adjuvant monotherapy. In the pivotal study supporting this new indication, results showed a 32% reduction in the risk of cancer recurrence and a 25% reduction in the risk of death compared to neoadjuvant chemotherapy alone. —The European Commission approved Pfizer vaccine Abrysvo for protection against respiratory syncytial virus (RSV). The regulatory decision expands the vaccine’s approval to adults ages 18 to 59. European regulators initially approved the vaccine in 2023 for preventing lower respiratory tract disease in adults age 60 and older. Abrysvo won FDA approval the same year. —The FDA expanded approval of Novartis’s Pluvicto to include use as an earlier line of treatment — after anti-androgen drugs and before chemotherapy — for PSMA-positive metastatic castration-resistant prostate cancer (mCRPC). Pluvicto was initially approved in 2022 for PSMA-positive mCRPC that has already been treated with an anti-androgen drug and chemotherapy. Novartis said this expansion is important because about half of patients do not live long enough to receive a second-line treatment. —Exelixis cancer drug Cabometyx expanded its FDA-approved uses to advanced cases of pancreatic neuroendocrine tumors (pNET) and extra-pancreatic neuroendocrine tumors (epNET). The approval covers adults and children age 12 and older. Cabometyx is a small molecule designed to block enzymes that support cancer growth. The drug was first approved in 2016 for advanced renal cell carcinoma. —GSK landed FDA approval for Blujepa, an oral antibiotic developed to treat uncomplicated urinary tract infections. While drugs are already available for these infections, they can become resistant to treatment. Blujepa is designed to block two enzyme targets key to bacterial DNA replication, an approach intended to lower the potential for drug resistance. —The European Commission approved Capvaxive, a pneumococcal vaccine developed by Merck to protect against the 21 Streptococcus pneumoniae serotypes that lead to the majority of cases of invasive pneumococcal disease including eight that are not covered by any other available pneumococcal vaccine. The European regulatory decision covers adults age 18 and older. Capvaxive received FDA approval last July. —Alynlam Pharmaceuticals drug Amvuttra received FDA approval for treating cardiomyopathy caused by transthyretin amyloidosis (ATTR). The RNA interference drug was first approved in 2022 for the treatment of polyneuropathy caused by ATTR. In the cardiomyopathy indication, it will compete with Pfizer’s Vyndaqel and Vyndamax, which are established as the standard of care, as well as Attruby, a BridgeBio Pharma drug that received FDA approval last fall. —Bristol Myers Squibb’s Breyanzi received European Commission approval for relapsed or refractory follicular lymphoma. The CAR T-therapy received accelerated FDA approval in this indication last May. —Geron Corporation’s Rytelo received European Commission approval for the treatment of transfusion-dependent anemia caused by lower-risk myelodysplastic syndrome, a progressive type of blood cancer. The intravenously infused drug is the first telomerase inhibitor to reach the market. Rytelo landed FDA approval last June. —Neffy, an epinephrine nasal spray that ARS Pharmaceuticals developed to treat allergic reactions to insect bites, medications, and food, expanded its FDA approval to include patients weighing 15 to 30 kilograms (about 33 to 60 pounds). The product’s initial approval last year covered its use in patients weighing 30 kg or more. —Roche subsidiary Genentech received FDA approval for tenecteplase, brand name TNKase, as a treatment for acute ischemic stroke in adults. Genentech said TNKase is the first stroke medicine approved by the FDA in nearly 30 years. It’s the second approved indication for the clot-busting drug. The FDA approved it in 2000 for treating acute myocardial infarction. —A Neurotech Pharmaceuticals cell and gene therapy administered via a surgical implant received FDA approval for the treatment of macular telangectasia type 2. The therapy, brand name Encelto, is the first approved treatment for this rare vision-loss disorder. Regulatory Setbacks —The FDA missed the April 1 regulatory decision target date for Novavax’s Covid-19 vaccine. Politico reported the holdup was due to the intervention of Sara Brenner, the FDA’s principal deputy commissioner, who asked for more data. Novavax said it has responded to all of the FDA’s information requests and believes the application is ready for approval based on the submitted Phase 3 data. Novavax’s protein-based vaccine initially received FDA emergency use authorization in 2022, a status that it retained under amended authorizations in 2023 and last year, both times for new formulas that covered the prevalent variants at the time. Full FDA approval would put the vaccine on par with the approved messenger RNA vaccines marketed by Pfizer and Moderna. Novavax’s Covid-19 vaccine will be co-commercialized with Sanofi under a partnership announced last year. —Roche paused European clinical tests of the Duchenne muscular dystrophy gene therapy Elevidys, the pharmaceutical giant said in a letter to the World Duchenne Organization. The move follows Sarepta Therapeutics’ recent report of a liver failure-associated patient death. Roche said the European Medicines Agency asked for a clinical hold on four studies until the inquiry into the cause of death is complete. Under a 2019 deal with Sarepta, Roche holds rights to Elevidys outside of the U.S. —For the second time, the FDA has rejected Aldeyra Therapeutics drug reproxalap as a treatment for dry eye disease. The agency turned down an application for the drug in 2023. According to Aldeyra, the FDA’s latest complete response letter said the new drug application failed to demonstrate efficacy. The regulator asked the company to run at least one more clinical trial. Among the concerns raised by the FDA is differences in baseline scores, which may have affected interpretation of trial results. Aldeyra said results from two ongoing studies are expected later in the current quarter, potentially paving the way for a mid-year resubmission of the application. —The European Medicines Agency refused marketing authorization for Eli Lilly Alzheimer’s disease drug Kisunla. Safety was the main reason cited, particularly the brain inflammation and bleeding that is a known complication risk associated with all drugs in the same class of Alzheimer’s medicines. The agency said the drug’s benefits were not enough to outweigh the potentially fatal safety risks. The FDA approved Kisunla last July. —In other Alzheimer’s drug news, Eisai again failed to persuade Australia’s drugs regulator to register Leqembi. The pharma company had asked the Therapeutics Goods Administration (TGA) to reconsider its October 2024 decision to not register the drug in Australia. Eisai’s request for reconsideration covered a narrower indication, but Eisai said the TGA did not agree that safety has been established for these patients.

Drug ApprovalClinical ResultPhase 3ImmunotherapyVaccine

31 Mar 2025

·www.drugs.com

FDA Approves Cabozantinib for Previously Treated Advanced Neuroendocrine Tumors

MONDAY, March 31, 2025 -- The U.S. Food and Drug Administration has approved cabozantinib (Cabometyx), an oral tyrosine kinase inhibitor, for patients with previously treated advanced neuroendocrine tumors, offering a new standard of care for this patient group, according to a press release from the Dana-Farber Cancer Institute. The approval was based on the results of the phase 3 CABINET study, which compared cabozantinib to a placebo in patients with advanced pancreatic neuroendocrine tumors and advanced extrapancreatic neuroendocrine tumors who had previously undergone treatment. The results of the trial were published last September in the New England Journal of Medicine . Patients treated with cabozantinib survived significantly longer with no worsening of their disease compared with patients who received placebo. Given the improved efficacy observed in the interim analysis, the trial was terminated early and unblinded in August 2023. The reported side effects of cabozantinib were similar to those found in other studies of the drug, including hypertension, fatigue, and diarrhea. "Patients with neuroendocrine tumors often face a difficult journey," lead author of the CABINET study, Jennifer Chan, M.D., M.P.H., of the Dana-Farber Cancer Institute, said in a statement. "Despite advances in recent years, there has remained a critical need for new and effective therapies for patients whose cancer has grown or spread. Cabozantinib significantly improved outcomes in this patient population and this FDA approval provides new hope." Approval of cabozantinib was granted to Exelixis. More Information Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

Drug ApprovalPhase 3Clinical ResultAccelerated Approval

100 Deals associated with Cabozantinib (s)-Malate

External Link

KEGG	Wiki	ATC	Drug Bank
D10095	Cabozantinib (s)-Malate	L01XE26	DB08875

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Extra-pancreatic neuroendocrine tumor	United States	Exelixis, Inc.	26 Mar 2025
Neuroendocrine tumor of pancreas	United States	Exelixis, Inc.	26 Mar 2025
Differentiated Thyroid Gland Carcinoma	South Korea	Ipsen Ltd.	26 Sep 2017
Renal Cell Carcinoma	South Korea	Ipsen Ltd.	26 Sep 2017
Hepatocellular Carcinoma	European Union	Ipsen Pharma SAS	09 Sep 2016
Hepatocellular Carcinoma	Iceland	Ipsen Pharma SAS	09 Sep 2016
Hepatocellular Carcinoma	Liechtenstein	Ipsen Pharma SAS	09 Sep 2016
Hepatocellular Carcinoma	Norway	Ipsen Pharma SAS	09 Sep 2016
Advanced Renal Cell Carcinoma	United States	Exelixis, Inc.	25 Apr 2016
Thyroid Cancer, Medullary	United States	Exelixis, Inc.	29 Nov 2012

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Solid tumor	NDA/BLA	China	Jiangsu Hansoh Pharmaceutical Co., Ltd.	28 Sep 2020
Refractory Differentiated Thyroid Gland Carcinoma	Phase 3	United States	National Cancer Institute	22 Aug 2024
Metastatic osteosarcoma	Phase 3	-	National Cancer Institute	13 Jan 2023
Unresectable Bone Sarcoma	Phase 3	-	National Cancer Institute	13 Jan 2023
Advanced Urothelial Carcinoma	Phase 3	United States	National Cancer Institute	03 Jun 2022
Advanced Urothelial Carcinoma	Phase 3	Canada	National Cancer Institute	03 Jun 2022
Bladder Cancer	Phase 3	United States	National Cancer Institute	03 Jun 2022
Bladder Cancer	Phase 3	Canada	National Cancer Institute	03 Jun 2022
Metastatic Urethral Urothelial Carcinoma	Phase 3	United States	National Cancer Institute	03 Jun 2022
Metastatic Urethral Urothelial Carcinoma	Phase 3	Canada	National Cancer Institute	03 Jun 2022

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06026410 (FIT-001, AACR2025)	Phase 1	-	-	Farnesyl transferase inhibitor KO-2806	jdjayewpxn(nuwtaqfjxs) = lfvyicmfix jzqeswjjvh (tujztzdkti )	Positive	28 Apr 2025
				Cabozantinib	lexxmwbaaj(pnotvpmgnd) = gtzabkoocp gbculsnizg (vzivbssibd )
NCT04316182 (ACTION, CTgov)	Phase 2	Hepatocellular Carcinoma	24	Cabozantinib	ecouhefhxq = kshrvtgnuv igmiiwantx (thwmohyoju, zzhthsmazn - cbwoehxuns) View more	-	30 Mar 2025
NCT03375320 (CABINET, FDA_CDER) Manual	Phase 3	Neuroendocrine tumor of pancreas	-	CABOMETYX (pNET)	csylfscqjg(juhmrpxuwc) = htggashhuw vuwzatkaks (hkijwqnccw, 8.9 - 17.0) View more	Positive	26 Mar 2025
				Placebo (pNET)	csylfscqjg(juhmrpxuwc) = uwbjwfochi vuwzatkaks (hkijwqnccw, 2.8 - 5.7) View more
NCT03945773 (CaboPoint, CTgov)	Phase 2	Metastatic Renal Cell Carcinoma \| Locally Advanced Renal Cell Carcinoma \| Metastatic Clear Cell Renal Cell Carcinoma	127	Cabozantinib (Cohort A)	agassxldwr = gmpjafikvt kdqtoguwjc (xjslijfsyz, otsixmiaxl - fjrjbtdrhw) View more	-	25 Mar 2025
				Cabozantinib (Cohort B)	hdobjyaeli(eqspagbbru) = mhsnhhoado pwwogmyzvp (oppgbxoiud, aglhezjpsa - mmununrkpm) View more
NCT03170960 (COSMIC-021, Pubmed \| J Clin Oncol)	Phase 1	Metastatic urothelial carcinoma First line \| First line	121	Cabozantinib 40 mg + Atezolizumab 1,200 mg	wsjcnfxwtm(xucfxixowu) = obzuoubyck zztzfewgag (hgeictigsi, 15 - 49) View more	Positive	18 Feb 2025
NCT03937219 (COSMIC-313, ASCO_GU2025) Manual	Phase 3	Advanced Renal Cell Carcinoma First line	855	Cabozantinib 40 mg QD + Nivolumab + Ipilimumab	tmphtkmaiu(srqerjppuo) = qadsoqpych hmqsikvbby (dexilxtzrm, 14.0 - 22.6) View more	Positive	13 Feb 2025
				Placebo + Nivolumab + Ipilimumab	tmphtkmaiu(srqerjppuo) = fjfgtexjjt hmqsikvbby (dexilxtzrm, 9.3 - 14.0) View more
NCT04714697 (AT ASIA, KCSG GU21-02, ASCO_GU2025) Manual	Phase 2	Renal Cell Carcinoma Second line	88	Cabozantinib	anhmmzoovx(jodtmcdbib) = gymahdpdkj stoafddgfq (wmibdphdjk ) View more	Positive	13 Feb 2025
				Cabozantinib (first-line nivolumab plus ipilimumab)	wxexpnpxek(zwevqwbody) = whtjhrcfpy syxnfturds (pfcteusjox, 40.1–61.5) View more
METEOR (ASCO_GU2025) Manual	Not Applicable	Metastatic Renal Cell Carcinoma Second line	603	Cabozantinibipilimumabnivolumab (1L ipilimumab/nivolumab)	vmxpgajtua(kxnprcwifg) = xybawwzovg xpixjytktn (djfysheljw ) View more	Positive	13 Feb 2025
				Cabozantinib (1L anti-PD1 + TKI)	vmxpgajtua(kxnprcwifg) = orhzsoumzp xpixjytktn (djfysheljw ) View more
JPRN-jRCTs031210220 (ASCO_GU2025) Manual	Phase 2	Advanced Renal Cell Carcinoma	31	Cabozantinib 60 mg	xvgflwllcd(qbliudzetn) = tsxqdltyew jdwrbmptew (tgkndywbwz, 1.2 - 18.9) View more	Positive	13 Feb 2025
NCT03634540 (LITESPARK-003, ASCO_GU2025) Manual	Phase 2	Renal Cell Carcinoma	102	Belzutifan 120 mg + Cabozantinib 60 mg (patients with no prior systemic therapy)	cvxyendcqe(ygesycsbat) = roncwquewv ffuxqkfjbb (vsrvvqccjw, 55 - 82) View more	Positive	13 Feb 2025
				Belzutifan 120 mgBelzutifan 120 mg + CabozantinibCabozantinib 60 mg (patients who had received prior immunotherapy and ≤2 systemic regimens)	cvxyendcqe(ygesycsbat) = ghazcnpmjk ffuxqkfjbb (vsrvvqccjw, 19 - 45) View more

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis