Tiopronin Delayed-Release Tablets Launch with Patient Support for Cystinuria in the US

Cycle Pharmaceuticals Limited has announced the release of tiopronin delayed-release tablets, designed to treat patients with cystinuria. These tablets are available in dosages of 100 mg and 300 mg and have received approval from the US Food and Drug Administration (FDA). Importantly, the medication must be taken without food.

Cystinuria, a hereditary condition leading to kidney stones, affects approximately 1 in every 7,000 people globally. This launch marks a collaboration between Cycle Pharmaceuticals, Torrent Pharma Inc., and Anovo Specialty Pharmacy, all of whom are dedicated to providing necessary medication and support services to patients with rare diseases.

The tiopronin delayed-release tablets offer a treatment option for patients with cystinuria, supported by Cycle Vita™, a platform providing tailored product support. Anovo, the exclusive specialty pharmacy for this product, will offer ongoing dispensing and support services.

Chikai Lai, the Senior Vice President and Chief Commercial Officer at Cycle, emphasized the chronic nature of cystinuria and the significant role of dietary control, specifically reducing salt and methionine intake, in managing the condition. Recognizing the challenges faced by patients, Cycle is offering individualized support including a Bridge Program, Co-pay Assistance Program, and a Patient Nutrition Program to complement clinical nutritional plans.

This introduction of tiopronin delayed-release tablets is Cycle’s fifth product launch in the US, following:
- NITYR® (nitisinone) Tablets in 2017,
- SAJAZIR™ (icatibant) Injection in 2021,
- JAVYGTOR™ (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution in 2022,
- and TASCENSO ODT® (fingolimod) in 2023.

Tiopronin delayed-release tablets are indicated for the prevention of cystine stone formation in adults and pediatric patients (aged 9 years and older) with severe homozygous cystinuria who do not respond to high fluid intake, alkali, and diet modification alone. The medication must be taken without food.

Safety information for tiopronin includes warnings about potential proteinuria, including nephrotic syndrome and membranous nephropathy, hypersensitivity reactions, and common adverse reactions such as nausea, diarrhea, oral ulcers, and fatigue. The interaction of alcohol with tiopronin is also noted, advising against alcohol consumption within a specified timeframe around taking the medication.

In terms of specific populations:
- For pregnant women, there is no identified risk from available data, but renal stones during pregnancy might lead to adverse outcomes.
- Breastfeeding is not recommended during treatment due to potential adverse reactions.
- Pediatric patients with severe homozygous cystinuria can use the tablets, but they are not approved for those under 20 kg or those unable to swallow tablets.
- Elderly patients should use the medication with caution due to the increased likelihood of reduced renal function.

Cycle Pharmaceuticals, founded in 2012, focuses on delivering treatments and support for patients with rare diseases, particularly in the areas of metabolic, immunological, and neurological genetic conditions. The company is headquartered in Cambridge, UK, with offices in Boston, Massachusetts.

Torrent Pharma, a leading pharmaceutical company in India, specializes in several therapeutic segments and has a strong presence in more than 40 countries. Anovo, established in 2012, provides comprehensive services for rare disease drug manufacturers and patients, facilitating the management of rare disease medications and associated support services.