Latest news with #orphanDrug
Yahoo
3 days ago
- Business
- Yahoo
Press Release: Rilzabrutinib granted orphan drug designation in the US for sickle cell disease
Rilzabrutinib granted orphan drug designation in the US for sickle cell disease Fourth orphan drug designation for rilzabrutinib in rare diseases Under regulatory review in the US, the EU, and China in immune thrombocytopenia Paris, June 3, 2025. The US Food and Drug Administration (FDA) has granted orphan drug designation to rilzabrutinib, a novel, advanced, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor that works via multi-immune modulation, to target a reduction in vaso-occlusive crises, which may occur via inflammation, in sickle cell disease. The FDA grants orphan drug designation to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the US. Global Head of Development, Rare Diseases'Receiving our fourth orphan drug designation for rilzabrutinib reinforces our continued dedication to developing medicines to address the unmet medical needs of people living with rare diseases. People with sickle cell disease often live with severe episodes of pain from vaso-occlusive crises and other complications that can significantly impact both quality of life and life expectancy. There remains a need for novel treatment approaches to address these experiences by modulating the immune system responses that can contribute to sickle cell disease pathogenesis.' In addition to sickle cell disease, rilzabrutinib has received orphan drug designation for immune thrombocytopenia (ITP) in the US, the EU, and Japan, for warm autoimmune hemolytic anemia (wAIHA) in the US and the EU, and for IgG4-related disease (IgG4-RD) in the US. The safety and efficacy of rilzabrutinib have not been determined by any regulatory authority. Rilzabrutinib is currently under regulatory review in the US, the EU, and in China for its potential use in ITP. The target action date for the FDA regulatory decision for ITP, which was granted fast track designation, is August 29, 2025. Sickle cell disease supporting dataPreclinical data on sickle cell disease was presented at ASH 2024 showing that rilzabrutinib helped reduce vaso-occlusion –blockage of blood vessels – and inflammation in transgenic mice with sickle cell disease. About rilzabrutinib Rilzabrutinib is a novel, advanced, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor that has the potential to be an effective new medicine for several rare immune-mediated or inflammatory diseases by working to restore immune balance via multi-immune modulation. BTK, expressed in B cells, macrophages, and other innate immune cells, plays a critical role in multiple immune-mediated disease processes and inflammatory pathways. With the application of Sanofi's TAILORED COVALENCY® technology, rilzabrutinib can selectively inhibit the BTK target while potentially reducing the risk of off-target side effects. About sickle cell diseaseSickle cell disease is a group of rare, genetic blood disorders in which red blood cells are misshapen, typically in a sickle or crescent shape, causing them to get stuck in small blood vessels, blocking blood flow. This can lead to episodes of severe pain as well as other health complications including infections, stroke, lung, eye, and kidney disease. Sickle cell disease affects more than 100,000 people in the US, approximately 90% of whom are African American and 3-9% of whom are Hispanic or Latino. Approximately 1 in 365 babies of African American descent born in the US have sickle cell disease, while an estimated 1 in 13 are carriers of the disease. People in the US with sickle cell disease have an estimated life expectancy that is 20 years shorter than average. About Sanofi Sanofi is an R&D driven, AI-powered biopharma company committed to improving people's lives and creating compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people's lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY Media RelationsSandrine Guendoul | +33 6 25 09 14 25 | Berland | +1 215 432 0234 | Léo Le Bourhis | +33 6 75 06 43 81 | Victor Rouault | +33 6 70 93 71 40 | Timothy Gilbert | +1 516 521 2929 | Investor RelationsThomas Kudsk Larsen |+44 7545 513 693 | Alizé Kaisserian | +33 6 47 04 12 11 | Lauscher | +1 908 612 7239 | Browne | +1 781 249 1766 | Nathalie Pham | +33 7 85 93 30 17 | Elgoutni | +1 617 710 3587 | Thibaud Châtelet | +33 6 80 80 89 90 | Li | +33 6 84 00 90 72 | Sanofi forward-looking statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words 'expects', 'anticipates', 'believes', 'intends', 'estimates', 'plans' and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi's ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under 'Risk Factors' and 'Cautionary Statement Regarding Forward-Looking Statements' in Sanofi's annual report on Form 20-F for the year ended December 31, 2024. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. All trademarks mentioned in this press release are the property of the Sanofi group. Attachment Press Release
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.'
Medscape
23-05-2025
- Health
- Medscape
EMA Recommends Treatment for Maple Syrup Urine Disease
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has granted an exceptional circumstances positive opinion for Maapliv, a solution of amino acids intended for the treatment of maple syrup urine disease (MSUD) in patients presenting with an acute decompensation episode from birth and who are not eligible for an oral and enteral branched-chain amino acids (BCAA)-free formulation. MSUD is a rare genetic disorder of BCAA metabolism. In this condition, buildup of toxic metabolites from BCAAs produced from protein breakdown leads to significant and potentially irreversible developmental effects. It may cause metabolic derangements, cerebral edema, seizures, coma, and respiratory failure, and it may be fatal. It affects fewer than 0.1 in 10,000 people in the EU, equivalent to fewer than 5000 people. This is below the ceiling for orphan drug designation (5 in 10,000). In 2020, the EMA granted orphan designation to a solution of amino acids not containing any BCAAs, intended to be given by infusion to replace other sources of amino acids. At the time, the EMA said, there were no suitable treatments authorized in the EU for MSUD, and patients were managed with strict diets to control the amount of BCAAs taken in from proteins. Some forms of the disorder also responded to vitamin B supplements. Some patients needed hospitalization for enteral feeding or procedures to filter BCAAs directly from the blood. Other patients were judged suitable for liver transplantation, which restores the ability to break down BCAAs. Medicine Reduces Harmful Amino Acids Maapliv, manufactured by Recordati Rare Diseases, is a combination of amino acids free of BCAA. It is used as a solution for infusion in combination with carbohydrate and lipid supplementation to prevent or reverse protein catabolism and promote anabolism in patients with MSUD decompensation, thereby reducing harmful alpha-keto acid levels. The CHMP said that leucine normalization had been shown in patients with MSUD decompensation who are given Maapliv in five scientific publications that reported on parenteral use of BCAA-free solutions with the same formulation as Maapliv. Treatment with Maapliv should be initiated under the supervision of a physician experienced in the management of MSUD disease. Detailed recommendations for the use of Maapliv will be described in the summary of product characteristics, which will be published on the EMA website in all official European Union languages after the marketing authorization has been granted by the European Commission.
