Cycle Pharmaceuticals Limited announced today the US launch of VENXXIVA (tiopronin) Delayed-Release Tablets for cystinuria treatment, following FDA approval. The product launch represents a collaborative effort between Cycle, Torrent Pharma Inc., and exclusive distribution partner Vanscoy Rare Pharmacy.
Cystinuria affects approximately 1 in 7,000 people worldwide and is characterized by the formation of cystine stones in the urinary system. The condition can significantly impact patients' quality of life and requires consistent management to prevent stone formation.
Treatment Approach and Patient Support
VENXXIVA is indicated for patients aged 9 and older with severe homozygous cystinuria who don't respond adequately to high fluid intake, alkali, and diet modifications alone. The medication is available in 100mg and 300mg delayed-release tablets, with the higher dose potentially reducing pill burden for patients.
A key component of the VENXXIVA launch is the Cycle Vita™ patient support program, which provides eligible patients with financial assistance, product information, and clinical education tailored to their needs.
"One in five patients taking tiopronin have reported missing doses on a daily basis. If improvements in tiopronin adherence can be achieved, along with diet modifications for low salt and high fluid intake, there is a possibility for a greater health-related quality of life," said Jamie Ray, Director of Patient Support Program at Cycle. "At Cycle, we understand that rare disease patients need additional support to manage their condition. That's why we're providing individualized support for patients with cystinuria, including dietary and nutritional support."
Building on Previous Success
VENXXIVA follows Cycle and Torrent's successful launch of generic tiopronin delayed-release tablets in March 2024. The companies plan to work with patients and clinics to transition those currently using the generic version to VENXXIVA, ensuring continued access to Cycle Vita's support services.
Administration and Safety Considerations
VENXXIVA must be taken without food, an important administration detail for patients and healthcare providers. The medication carries several warnings and precautions, including risks of proteinuria, nephrotic syndrome, membranous nephropathy, and hypersensitivity reactions.
The most common adverse reactions (≥10%) include nausea, diarrhea or soft stools, oral ulcers, rash, fatigue, fever, arthralgia, proteinuria, and emesis. Healthcare providers should be aware that alcohol consumption may affect the release rate of tiopronin from VENXXIVA, and patients are advised to avoid alcohol 2 hours before and 3 hours after taking the medication.
Special Population Considerations
For pregnant patients, available data have not identified drug-associated risks for major birth defects or adverse maternal outcomes, though renal stones during pregnancy may lead to complications. Breastfeeding is not recommended during treatment due to potential serious adverse reactions.
The medication is not approved for pediatric patients weighing less than 20kg or those unable to swallow tablets. For elderly patients, dose adjustments may be necessary due to the drug's renal excretion pathway and the higher likelihood of decreased renal function in this population.
About the Companies
Cycle Pharmaceuticals, founded in 2012, focuses on delivering treatments for rare metabolic, immunological, and neurological genetic conditions. The company is headquartered in Cambridge, UK, with offices in Boston, Massachusetts.
Torrent Pharma Inc. is a leading pharmaceutical company from India with a significant global presence in over 40 countries, ranking first among Indian companies for market share in Brazil and Germany.
Vanscoy Rare Pharmacy, based in Pittsburgh, Pennsylvania, specializes in pharmacy services for patients with rare and complex medical conditions, emphasizing personalized care and patient education.
Healthcare providers and patients seeking more information about VENXXIVA can visit www.venxxiva.com, where the full prescribing information is available.
