Latest news with #CytokineReleaseSyndrome
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.
Irish Times
27-05-2025
- Business
- Irish Times
Poolbeg Pharma treatment gets FDA backing
The US Food and Drug Administration (FDA) has granted orphan drug designation to Poolbeg's preventative therapy for cancer immunotherapy-induced Cytokine Release Syndrome (CRS). The treatment, named POLB 001, is an oral preventative therapy to treat inflammation in blood and tissues. CRS is a severe side-effect that occurs in more than 70 per cent of patients, leading to severe side-effects or death. Cytokines can sweep throughout the body and cause tissue damage and shut down circulation and other essential organs. Orphan status is granted by the FDA for to support the development of treatments for rare disorders that affect under 200,000 people in the US, and makes the development of the drug less risky for Poolbeg, with the potential for seven-year period of US market exclusivity following approval of the treatment, waiver exemption of some fees and tax credits for qualified clinical trials. READ MORE 'POLB 001 is potentially a breakthrough, orally delivered, preventative therapy for cancer immunotherapy-induced CRS which could significantly impact patients' lives,' said Poolbeg chief executive Jeremy Skillington. '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. 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.' There are currently no approved preventative therapies for CRS. The first patients are expected to get the drug in the second half of the year, as part of Poolbeg's Phase 2a trial. 'Orphan Drug Designation from the FDA underscores the urgency and importance of developing innovative therapies for this critical unmet medical need,' said Professor Brendan Buckley, Poolbeg non-executive director and a member of the scientific advisory board. 'We look forward to progressing POLB 001 in our upcoming Phase 2a clinical trial and working closely with prospective partners and regulatory agencies to bring this potential therapy to patients as quickly as possible.'
Associated Press
13-02-2025
- Health
- Associated Press
DATAcc by DiMe Releases New Resources to Support the Development of Digital Health Technologies to Predict and Manage Cancer Immunotherapy Side Effects
New resources will support developers and researchers in developing de-risking digital products to better predict and manage Cytokine Release Syndrome BOSTON, Feb. 13, 2025 /PRNewswire/ -- Today, the Digital Health Measurement Collaborative Community (DATAcc) by the Digital Medicine Society (DiMe), the global home for digital health measures development, released new resources to support the development of digital health technologies (DHTs) for predicting and managing Cytokine Release Syndrome (CRS), a serious side effect of cancer immunotherapies. Over the past few decades, immunotherapies have shown significant promise in treating hematological cancers like leukemia, lymphoma, and multiple myeloma, offering hope to patients with limited treatment options. However, these therapies also bring the risk of CRS, a potentially fatal condition that presents as flu-like symptoms but can quickly escalate to life-threatening complications if not properly managed. The risk of CRS often requires therapies to be administered in a hospital setting or frequent clinical visits, increasing clinical trial costs, especially for patients in underserved or rural areas, and burden on healthcare systems. The lack of standard reporting and a robust, high-frequency collection of clinical measures related to CRS presents barriers to developing high-quality CRS de-risking products. DATAcc's new resources advance the development of CRS de-risking products that may improve care delivery, reduce hospital dependency, and expand immunotherapies for all patients. 'Immunotherapies are lifesaving, but dangerous side effects like CRS create barriers for too many,' said Samantha McClenahan, DiMe. 'The resources launched today will support developers in building the products needed for de-risking CRS outside clinical settings. Leveraging DHTs can help reduce the risk of CRS and get these therapies to patients in need. We're excited to launch resources that will fast-track the development of these new digital products.' 'Immunotherapies are a critical, innovative intervention in the fight against cancer, but today they can be costly and prohibitive for many patients due to the need to monitor for dangerous side effects,' said Cindy Varga, Atrium Health. 'Using CRS de-risking tools during treatment, we could more quickly detect CRS symptoms and intervene earlier to mitigate high grade complications during a cancer patient's treatment journey. Expanding the availability of CRS de-risking tools will help oncologists and researchers detect and manage CRS more effectively, enabling patients to spend less time in inpatient settings and reducing the burden on healthcare systems, patients, and caregivers.' DATAcc is the leader in advancing the use of digital clinical measures by stakeholders across our industry. The new resources launched today accompany existing digital measures resources in physical activity, Alzheimer's disease and related disorders, nocturnal scratch, and sleep. We are tackling some of the biggest challenges and opportunities in the digital medicine ecosystem. To change the digital health landscape as a DiMe or DATAcc partner, click here. About the Digital Medicine Society: DiMe is a global non-profit and the professional home for all members of the digital medicine community. Together, we tackle the toughest digital medicine challenges, develop clinical-quality resources on a technology timeline, and deliver these actionable resources to the field via open-source channels and educational programs. About DATAcc by DiMe: The Digital Health Measurement Collaborative Community ( DATAcc) by DiMe is a collaborative community with the FDA's Center for Devices and Radiological Health. We provide a forum for collaboration where partners and experts from across the digital health field work to advance the use of digital health measures in research to improve lives.
