Latest news with #PrescriptionDrugUserFeeAct
Yahoo
a day ago
- Business
- Yahoo
Kura Oncology and Kyowa Kirin Announce FDA Acceptance and Priority Review of New Drug Application for Ziftomenib in Adults with Relapsed or Refractory NPM1-Mutant AML
– New Drug Application based on positive results from the Phase 2 KOMET-001 trial – – FDA assigns a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025 – – Potential first approval of a menin inhibitor for the treatment of adult patients with relapsed or refractory AML with an NPM1 mutation – SAN DIEGO and TOKYO, June 01, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, 'Kura') and Kyowa Kirin Co., Ltd. (TSE: 4151, 'Kyowa Kirin') today announced the U.S. Food and Drug Administration (FDA) has accepted Kura's New Drug Application (NDA) seeking full approval for ziftomenib as a treatment for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025. 'The FDA's acceptance of our New Drug Application marks a significant milestone for Kura and Kyowa Kirin and, more importantly, for patients living with this genetic subset of AML, who face an aggressive form of the disease with few treatment options,' said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. 'This achievement reflects the strength of the clinical data for ziftomenib as well as the incredible commitment of our teams. Along with our partners at Kyowa Kirin, we look forward to continuing to work closely with the FDA throughout the review process and to prepare for the anticipated launch of this treatment, which holds potential to meaningfully impact the lives of patients and their families.' The NDA is based on results from the Phase 2 KOMET-001 registrational trial in R/R NPM1-mutant (NPM1-m) AML (NCT #04067336). The KOMET-001 trial achieved its primary endpoint of complete remission (CR) plus CR with partial hematological recovery (CRh) and the primary endpoint was statistically significant. Ziftomenib was well‑tolerated with limited myelosuppression and 3% ziftomenib-related discontinuations. The safety and tolerability of ziftomenib were consistent with previous reports, and the benefit-risk profile for ziftomenib is highly encouraging. 'Adult R/R NPM1-m AML patients face a significantly poor prognosis, highlighting the urgent need for innovative treatment options that can improve their outcomes,' said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. 'The acceptance of this NDA is a crucial step in our ongoing efforts to explore and evaluate various therapeutic strategies for AML through our comprehensive clinical trials. Our dedicated teams at Kyowa Kirin and Kura are fully committed to working tirelessly to ensure that, once approved, ziftomenib is made available to AML patients as quickly as possible. We recognize the importance of this endeavor and are excited about the possibility of making a meaningful impact on the lives of those affected by this challenging disease.' The KOMET-001 registration-directed trial is designed to assess evidence of clinical activity, safety and tolerability of ziftomenib, the only investigational therapy to receive Breakthrough Therapy Designation (BTD) from the FDA for treatment of R/R NPM1-mutant AML. In addition to BTD, ziftomenib has received Fast Track and Orphan Drug Designations. The full data analyses from the KOMET-001 trial of ziftomenib in R/R NPM1-m AML patients have been selected for oral presentation on Monday, June 2nd at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, and an encore presentation is planned at the 2025 European Hematology Association (EHA) Congress. About -Mutant AML AML is the most common acute leukemia in adults and begins when the bone marrow makes abnormal myeloblasts (white blood cells), red blood cells or platelets. Despite the many available treatments for AML, prognosis for patients remains poor and a high unmet need remains. The menin pathway is considered a driver for multiple genetic alterations of the disease, of which NPM1 mutations are among the most common, representing approximately 30% of AML cases. While patients with NPM1-m AML have high response rates to frontline therapy, relapse rates are high and survival outcomes are poor, with only 30% overall survival at 12 months in the R/R setting. Additionally, NPM1 mutations frequently occur with co-mutations in other disease-associated genes, including FLT3, DNMT3A, and IDH1/2, with prognosis heavily influenced by the presence of such co-occurring mutations. Adult patients with NPM1-m AML and select co-mutations and/or R/R disease have a poor prognosis, with median overall survival of only approximately 7.8 months in 2nd line, 5.3 months in 3rd line, and 3.5 months following the 4th line1. There are currently no FDA-approved therapies targeting NPM1-m AML. About Ziftomenib Ziftomenib is a potent and selective, oral, investigational menin inhibitor currently in development for the treatment of genetically defined AML patients with high unmet need. In April 2024, ziftomenib received BTD from the FDA for the treatment of adult patients with R/R AML with an NPM1 mutation based on data from Kura's KOMET-001 clinical trial. Additional information about clinical trials for ziftomenib can be found at About Kura Oncology Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer. The Company's pipeline consists of small molecule drug candidates designed to target cancer signaling pathways. In November 2024, Kura Oncology entered into a global strategic collaboration agreement with Kyowa Kirin to develop and commercialize ziftomenib, a menin inhibitor, for AML and other hematologic malignancies. Enrollment in KOMET-001, a Phase 2 registration-directed trial of ziftomenib in R/R NPM1-m AML, has been completed, and in the second quarter of 2025, the companies announced submission of an NDA for ziftomenib for the treatment of adult patients with R/R NPM1-m AML. Kura and Kyowa Kirin are conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in newly diagnosed and R/R NPM1-m and KMT2A-rearranged AML. KO-2806, a next-generation farnesyl transferase inhibitor (FTI), is being evaluated in a Phase 1 dose-escalation trial (FIT-001) as a monotherapy and in combination with targeted therapies for patients with various solid tumors. Tipifarnib, a potent and selective FTI, is currently in a Phase 1/2 trial (KURRENT-HN) in combination with alpelisib for patients with PIK3CA-dependent head and neck squamous cell carcinoma. For additional information, please visit Kura's website at and follow us on X and LinkedIn. About Kyowa Kirin Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, Kyowa Kirin has invested in drug discovery and biotechnology innovation for more than 70 years and is currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato-oncology and rare diseases. A shared commitment to Kyowa Kirin's values, to sustainable growth, and to making people smile unites Kyowa Kirin across the globe. You can learn more about the business of Kyowa Kirin at Kura Forward-Looking Statements This news release contains certain forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, the efficacy, safety and therapeutic potential of ziftomenib; the potential for ziftomenib to obtain FDA approval for the treatment of patients with NPM1-m AML, and the anticipated timing of such FDA approval; and the potential launch of ziftomenib. Factors that may cause actual results to differ materially from those indicated by these forward-looking statements include the risk that Kura may not be able to successfully demonstrate the safety and/or efficacy of its product candidates, including ziftomenib; the risk that Kura may not obtain approval to market its product candidates, including ziftomenib, or that such approval may be delayed; the risk that the collaboration with Kyowa Kirin is unsuccessful; and other risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. You are urged to consider statements that include the words 'may,' 'will,' 'would,' 'could,' 'should,' 'believes,' 'estimates,' 'projects,' 'promise,' 'potential,' 'expects,' 'plans,' 'anticipates,' 'intends,' 'continues,' 'designed,' 'goal,' or the negative of those words or other comparable words to be uncertain and forward-looking. For a further list and description of the risks and uncertainties the Company faces, please refer to the Company's periodic and other filings with the Securities and Exchange Commission, which are available at Such forward-looking statements are current only as of the date they are made, and Kura assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. Kura Contacts Investors: Patti Bank Managing Director(415) Media:media@ Kyowa Kirin Contacts Investors: Ryohei Kawaiir@ Media, Global:Wataru Suzuki media@ in to access your portfolio
Business Insider
2 days ago
- Business
- Business Insider
Kura Oncology, Kyowa Kirin announces FDA accepted NDA for ziftomenib
Kura Oncology (KURA) and Kyowa Kirin announced the U.S. Food and Drug Administration, FDA, has accepted Kura's New Drug Application, NDA, seeking full approval for ziftomenib as a treatment for adult patients with relapsed or refractory (R/R) acute myeloid leukemia with a nucleophosmin 1 mutation. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act, PDUFA, target action date of November 30, 2025. 'The FDA's acceptance of our New Drug Application marks a significant milestone for Kura and Kyowa Kirin and, more importantly, for patients living with this genetic subset of AML, who face an aggressive form of the disease with few treatment options,' said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. 'This achievement reflects the strength of the clinical data for ziftomenib as well as the incredible commitment of our teams. Along with our partners at Kyowa Kirin, we look forward to continuing to work closely with the FDA throughout the review process and to prepare for the anticipated launch of this treatment, which holds potential to meaningfully impact the lives of patients and their families.' Confident Investing Starts Here:
RTÉ News
27-05-2025
- Business
- RTÉ News
Poolbeg Pharma gets FDA support for POLB 001 drug
Biopharmaceutical company Poolbeg Pharma said the US Food and Drug Administration has granted Orphan Drug Designation (ODD) to its POLB 001 product as an oral preventative therapy for Cytokine Release Syndrome (CRS). Cancer immunotherapy-induced CRS is a severe side-effect that occurs in more than 70% of patients and which may lead to multi-organ failure and death. There are currently no approved preventative therapies for CRS. Poolbeg focusses on the development of innovative medicines to address unmet medical needs and its programmes target large addressable markets including, cancer immunotherapy-induced Cytokine Release Syndrome (CRS) and metabolic conditions such as obesity. The company said the FDA grants orphan status to support the development of medicines for rare disorders affecting less than 200,000 people in the US. It provides Poolbeg with clinical development and commercialisation benefits including the potential for a seven-year period of US market exclusivity after regulatory approval of POLB 001, potential waiver exemption of Prescription Drug User Fee Act application fees and the potential for tax credits for qualifying clinical trials. Poolbeg expects the first patient to be dosed in its proposed Phase 2a in the second half of this year, with interim analysis expected in the first half of 2026 and Phase 2a topline data in the second half of 2026. Jeremy Skillington, the chief executive of Poolbeg Pharma, said that POLB 001 is potentially a breakthrough, orally delivered, preventative therapy for cancer immunotherapy-induced CRS which could significantly impact patients' lives. "We were delighted to receive Orphan Drug Designation from the FDA, which is a significant development for Poolbeg and for POLB 001, one that we believe will enhance the commercial appeal for prospective partners and help bring POLB 001 to the market faster," he said. "If approved, we believe POLB 001 has the potential to improve quality of life for patients, reduce pressure on healthcare systems, and expand access to cancer immunotherapies," the CEO added.
Yahoo
22-05-2025
- Health
- Yahoo
Trump administration proposes cutting FDA budget by 5.5%
By Ahmed Aboulenein and Mariam Sunny WASHINGTON (Reuters) - The Trump administration is proposing a $6.8 billion budget request for the U.S. Food and Drug Administration for the 2026 fiscal year, its Commissioner Martin Makary said on Thursday, a cut of around 5.5% from its $7.2 billion budget this year. The White House had on May 2 proposed reducing U.S. health spending by more than a quarter next year, with the National Institutes of Health and Centers for Disease Control and Prevention facing the brunt of billions of dollars in cuts. The proposed budget requested $93.8 billion for the Department of Health and Human Services - a cut of $33.3 billion, or 26.2% - from this year's budget of $127 billion, but did not mention the FDA. Makary's comments on Thursday were the first official outlining of the agency's 2026 budget request. "The Trump administration is proposing a $6.8 billion budget for the FDA," Makary told the Senate Appropriations Committee's Subcommittee on Labor, Health and Human Services, Education, and Related Agencies at a hearing on the budget. The FDA in 2025 had a budget of $7.2 billion, including $3.5 billion in user fees, payments made by pharmaceutical and medical device manufacturers to fund the staff resources needed to review their products quickly, conduct inspections, and ensure the safety of clinical trials. Makary said that nearly 1,900 people were laid off at the FDA as part of a massive restructuring of federal health agencies under Secretary of Health and Human Services Robert F. Kennedy Jr. to align with President Donald Trump's goal of dramatically shrinking the federal government. No scientific reviewer or inspector was cut as a part of the reduction in force, he added. Democratic senators pushed back, asking Makary about media reports of the FDA firing and later rehiring scientists. He said the scientists were not fired but rather scheduled for firing before the decision was reversed. A further 1,200 took early retirement packages and the agency is hiring more scientists to replace them, Makary said. The staffing changes have not impacted approval schedules and the agency is on track to meet its treatment review targets under the Prescription Drug User Fee Act, known as PDUFA, he said.
Yahoo
16-05-2025
- Business
- Yahoo
Why Biohaven Stock Dove by Nearly 20% on Thursday
The company announced some discouraging news from a major regulatory body. It will have to wait longer than expected on an application for a very promising developmental drug. 10 stocks we like better than Biohaven › Volatile biotech stock Biohaven (NYSE: BHVN) is prone to sharp movements both up and down. On Thursday, it had one of its down days, with its share price eroding by almost 20% on a development with a top regulator. That was on a day when stocks generally did well, as indicated by the S&P 500 (SNPINDEX: ^GSPC) landing in positive territory with a 0.4% rise. After market close on Wednesday, Biohaven divulged that the U.S. Food and Drug Administration's (FDA) Division of Neurology 1 is extending the due date for its decision on a very promising pipeline drug developed by the company. Specifically, the FDA unit is extending the Prescription Drug User Fee Act (PDUFA) date for Biohaven's troriluzole, a treatment that targets brain disorder spinocerebellar ataxia (SCA), by three months. The biotech has formally submitted troriluzole under the FDA's New Drug Application (NDA) regime, and now expects a decision from the regulator to come in the fourth calendar quarter of this year. According to the company, the FDA said it required the extra time to conduct a full review of recent Biohaven submissions to the regulator's information requests. The FDA division also told the company that it aims to hold an advisory committee meeting to discuss the troriluzole application, but no date has yet been set for this. Many investors consider Biohaven to boast excellent potential with troriluzole, as it targets a rare brain disorder that currently has no treatment. It has been granted fast-track, priority review, and orphan drug designation (ODD) by the FDA, so initially it seemed as if a decision on approval would come soon. It's always disappointing when there's a delay, but until we know more about the regulator's concerns, it's hard to gauge the ultimate future of troriluzole. Much will depend on the FDA's moves in the coming months. Before you buy stock in Biohaven, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and Biohaven wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $620,719!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $829,511!* Now, it's worth noting Stock Advisor's total average return is 959% — a market-crushing outperformance compared to 170% for the S&P 500. Don't miss out on the latest top 10 list, available when you join . See the 10 stocks » *Stock Advisor returns as of May 12, 2025 Eric Volkman has no position in any of the stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy. Why Biohaven Stock Dove by Nearly 20% on Thursday was originally published by The Motley Fool
