Latest news with #CyclePharmaceuticals
Business Wire
19-06-2025
- Health
- Business Wire
Cycle Pharmaceuticals' HARLIKU™ (nitisinone) Tablets Receive First FDA-Approval as Treatment for Alkaptonuria (AKU)
BOSTON & CAMBRIDGE, England--(BUSINESS WIRE)--Cycle Pharmaceuticals announced that the FDA has approved HARLIKU (nitisinone) Tablets for the reduction of urine homogentisic acid (HGA) in adult patients with AKU. 1 Launching in July 2025, HARLIKU will be the first and only FDA-approved treatment for AKU, 1 an ultra-rare genetic metabolic disorder in which patients have a buildup of HGA that leads to osteoarthritis, ochronosis, and complications in the kidneys, and heart. 2 Patients with AKU often develop pain, reduced joint mobility, and require large joint replacements; the symptoms impede their physical functionality, emotional well-being, and quality of life. 2, 3 The approval of HARLIKU is based on data from a randomized, no-treatment controlled study of 40 patients with AKU. As part of the intramural research program of the National Human Genome Research Institute at the National Institutes of Health (NIH), Dr. Wendy J. Introne, MD and her team showed that nitisinone helped patients improve pain, energy levels, and physical functioning after three years of treatment, assessed using the 36-Item Short-Form Survey. 4 Steve Fuller, Chief Strategy Officer of Cycle Pharmaceuticals commented, "We are deeply grateful for Cycle's collaboration with Dr. Wendy Introne, Dr. Bill Gahl, and the broader team at the NIH, whose pioneering work laid the foundation for this FDA approval. We look forward to making HARLIKU available to U.S. AKU patients as soon as possible and remain committed to supporting the AKU community to the fullest extent of our capabilities." 'The approval of HARLIKU is an important advance for the AKU community. Our scientific team has translated decades of research into launching nitisinone as a new treatment option, and we stand hopeful that it can ease the significant burden of AKU,' said Dr. Wendy J. Introne, MD, of NIH's National Human Genome Research Institute (NHGRI). Building on the company's previous success in rare diseases, HARLIKU will be Cycle Pharmaceuticals' eighth commercial product in the US. Indications HARLIKU™ is indicated for the reduction of urine homogentisic acid (HGA) in adult patients with alkaptonuria (AKU). Important Safety Information Do not prescribe HARLIKU to patients allergic to nitisinone or any other contained ingredients. Warnings and Precautions: Ocular Symptoms and Hyperkeratotic Plaques Due to Elevated Plasma Tyrosine Level: Ocular signs and symptoms including keratitis, corneal opacities, corneal irritation, corneal ulcers, conjunctivitis, eye pain, and photophobia, have been reported in patients. Perform slit-lamp examination prior to treatment and regularly thereafter. Reexamine patients if symptoms develop or tyrosine levels are > 500 micromole/L. Assess plasma tyrosine levels in patients with an abrupt change in neurological status. Perform a clinical laboratory assessment, including plasma tyrosine levels, in patients with an abrupt change in neurological status. Leukopenia and Severe Thrombocytopenia In clinical trials, patients with hereditary tyrosinemia type 1 (HT-1) treated with another oral formulation of nitisinone and dietary restriction, developed reversible leukopenia, thrombocytopenia, or both. No patients developed infections or bleeding as a result of the episodes of leukopenia and thrombocytopenia. Monitor platelet and white blood cell counts during HARLIKU therapy. Adverse Reactions: The most common adverse reactions (≥1%) reported in patients with AKU taking nitisinone in clinical trials are elevated tyrosine levels, thrombocytopenia and keratitis. Drug Interactions: Nitisinone is a moderate CYP2C9 inhibitor and a weak CYP2E1 inducer. Potential clinical impact of Harliku administration with CYP2C9 substrates. Reduce the dosage of the co-administered drugs metabolized by CYP2C9 by half. Additional dosage adjustments may be needed. Nitisinone is an inhibitor of OAT1/OAT3. Potential clinical impact of administration with OAT1/OAT3 substrates. Patients should be monitored for potential adverse reactions. Use in Specific Populations: Pregnancy: Limited available data with nitisinone use in pregnant women are not sufficient to determine a drug-associated risk of adverse developmental outcomes. The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. Lactation: The developmental and health benefits of breastfeeding should be considered against the mother's clinical need for HARLIKU, along with potential adverse effects on the breastfed infant from HARLIKU or from the underlying maternal condition. Pediatric Use: The safety and effectiveness of HARLIKU have not been established in pediatric patients with AKU. Geriatric Use: Insufficient data from clinical studies of HARLIKU to determine if patients ≥ 65 years of age respond differently. Elderly patients should be cautious reflecting any decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy. For more detailed information, please refer to the full Prescribing Information at 1-855-831-5413, or the FDA at: 1-800-FDA-1088 or US-HKU-2500032 | May 2025 References HARLIKU (nitisinone) Tablets. Prescribing Information. Cycle Pharmaceuticals Ltd Introne WJ, Perry M, Chen M. 2003 (updated 2021). Alkaptonuria. Available at: [Accessed May 29, 2025] Rudebeck M, Scott C, Sireau N, Ranganath L. 2020. A patient survey on the impact of alkaptonuria symptoms as perceived by the patients and their experiences of receiving diagnosis and care. JIMD Rep. 53(1):71-79. doi:10.1002/jmd2.12101 Spears KR, Rossignol F, Perry MB, et al. 2024 Patient-reported outcomes and functional assessments of patients with Alkaptonuria in a 3-year Nitisinone treatment trial. Mol Genet Metab. 143(1-2),108562. doi:10.1016/ ©2025 Cycle Pharmaceuticals Limited. All rights reserved. HARLIKU™ and Cycle Vita™ are trademarks of Cycle Pharmaceuticals Limited in the United States. About Cycle Pharmaceuticals Cycle Pharmaceuticals was founded in 2012 with the sole aim of delivering drug treatments and product support to the underserved rare disease community. Cycle Pharma focuses on rare genetic conditions in metabolic, immunology, urology, and oncology. In neurology, we focus on multiple sclerosis. Cycle Pharma is headquartered in Cambridge, UK and has offices in Boston, Massachusetts. For more information, please visit and follow us on X, LinkedIn and Facebook.
The Independent
14-05-2025
- Business
- The Independent
E2E International 100 list for 2025 revealed
In collaboration with The Independent, E2E is delighted to present the E2E International 100 list, showcasing the outstanding accomplishments of the top 100 global businesses headquartered in the UK. These companies have demonstrated successful expansion into international markets, achieved the strongest revenue growth over the past two years, and created a successful business model that has put them in the top echelon of international businesses. The list was carefully compiled using comprehensive reliable data from Experian and Creditsafe, ensuring a meticulous and credible selection process that recognises only those companies with proven contributions to the international business landscape. Our headline partner for the E2E 100 Series 2025 is Universal Partners. Spanning various sectors and industries, from manufacturing, retail, hospitality, IT services, food and beverage, recruitment and import and export, these league tables serve as a testament to the incredible achievements of UK entrepreneurs driving global success. Featured in the list and demonstrating extraordinary growth are: Charlotte Tilbury Beauty Limited – A powerhouse in the global cosmetics industry, Charlotte Tilbury has scaled rapidly through its strong D2C model, celebrity-driven campaigns, and presence in key international markets such as the US and Middle East. Cycle Pharmaceuticals Ltd – Specialising in treatments for rare diseases, Cycle Pharma has expanded its footprint across Europe and North America, using digital innovation and regulatory expertise to drive growth. Vivienne Westwood Limited – An iconic fashion brand with a renewed global momentum, Vivienne Westwood has leveraged heritage with sustainability-focused strategies, expanding its retail and online presence worldwide. This initiative stands as a powerful testament to the exceptional calibre of UK enterprises, highlighting their commitment to excellence, impressive revenue growth, and successful expansion into international markets. Their groundbreaking business strategies not only set them apart within their sectors but also resonate nationwide and globally. Highlighting talent from every corner of the UK, this list highlights the rich diversity of global businesses and the monumental successes they have achieved despite navigating through the challenges of an uncertain economic landscape. As the Founder and CEO of E2E: 'The E2E International 100 list celebrates the phenomenal achievements of UK businesses that have not only expanded successfully into international markets but have also shown exceptional revenue growth and innovative business models. This recognition underscores the resilience and ingenuity of our entrepreneurs, highlighting their pivotal role in driving the global success of the UK's business landscape. It's truly inspiring to see such a diverse array of talent and leadership navigating and excelling in an unpredictable economic climate. Congratulations to all the remarkable businesses featured in this year's list!' Andrew Morley, CRO at The Independent, added: 'We are incredibly proud to partner with E2E in celebrating the remarkable achievements of these top 100 businesses. Their success stories reflect the dynamic and resilient nature of the UK's business community, and their ability to thrive on the international stage is a testament to their innovative spirit and dedication. This list not only honours their hard work and success but also serves as an inspiration for other businesses striving to make their mark globally. Congratulations to all the honourees on this well-deserved recognition.' The tracks are independently compiled by Creditsafe, and Experian according to specific criteria and official data, ensuring a trusted and transparent process. Each track is supported by our partners: Universal Partners (Headline Partner), Lioncroft, Creditsafe and Experian. click here.
Associated Press
22-04-2025
- Health
- Associated Press
Cycle Pharmaceuticals Launches Cycle Vita PKU™, an AI-Powered Smartphone App for On-The-Go Management of Phenylketonuria (PKU)
BOSTON--(BUSINESS WIRE)--Apr 22, 2025-- Cycle Pharmaceuticals (Cycle) has launched Cycle Vita PKU, an AI-powered app to help patients with PKU manage every aspect of their treatment plan. PKU is an inherited disorder caused by a deficiency of the phenylalanine hydroxylase (PAH) enzyme needed to process phenylalanine (Phe), which is found in all foods containing protein. 1 Nearly all cases of PKU are diagnosed through newborn screenings, 2 and require lifelong adherence to a strict PKU diet. 3 It can be challenging for patients with PKU, or their caregivers, to keep on top of everything, like medication, medical formula, lab results and diet. The app is designed to offer flexible and timely support with daily tracking, reminders and diet management tools. Users can plan their meals with a searchable food database of over 9,000 foods, as well as an AI-powered meal scanner that estimates Phe levels with a photo snap of the meal. For adolescents and young adults, who are gaining independence and navigating the transition to adulthood, Cycle Vita PKU may be a helpful aid for adhering to their medication and a Phe-restricted diet on-the-go. 'The launch of Cycle Vita PKU marks an exciting milestone as the first app from Cycle Vita™, Cycle's dedicated patient support platform. As part of Cycle's commitment to the PKU community, Cycle will continue to innovate and find new ways to provide support tailored to patients' needs. Cycle Vita PKU is just one example of that commitment.' says Jamie Ray, Director - Patient Support Program, Cycle. Cycle Vita PKU does not provide medical advice, diagnosis or treatment. Always seek the advice of your healthcare provider with any questions you may have. References Cycle Vita™ and Cycle Vita PKU™ are trademarks of Cycle Pharmaceuticals Ltd. ©2025 Cycle Pharmaceuticals Limited. All rights reserved. US-VITA-2500003. February 2025 About Cycle Pharmaceuticals Cycle Pharmaceuticals was founded in 2012 with the sole aim of delivering drug treatments and product support to the underserved rare disease patient community. Cycle focuses on rare metabolic, immunological, and neurological genetic conditions. Within neurological conditions, we focus on multiple sclerosis. Cycle is headquartered in Cambridge, UK and has offices in Boston, Massachusetts. For more information, please visit and follow us on X, LinkedIn and Facebook. View source version on CONTACT: [email protected] Cycle Pharmaceuticals Tel: +44 1223 354 118 KEYWORD: UNITED STATES NORTH AMERICA MASSACHUSETTS INDUSTRY KEYWORD: BIOTECHNOLOGY FDA OTHER HEALTH HEALTH GENERAL HEALTH PHARMACEUTICAL NEUROLOGY RESEARCH GENETICS SCIENCE CLINICAL TRIALS SOURCE: Cycle Pharmaceuticals Copyright Business Wire 2025. PUB: 04/22/2025 09:48 AM/DISC: 04/22/2025 09:48 AM
