The US Food and Drug Administration (FDA) has approved an enteric-coated delayed-release formulation of tiopronin (Thiola EC) for treatment of cystinuria.
Cystinuria is a rare inherited disorder that causes an increase in cystine levels in the urine, leading to recurring cystine kidney stones.
Thiola EC is indicated for the prevention of cystine stone formation in adults and pediatric paatients weighing at least 20 kg who have severe homozygous cystinuria.
“Thiola’s utility as the treatment of choice for cystinuria is well established. However, for certain patients, the challenges of administration one hour before or two hours after meals three times a day, coupled with a high pill burden, have been challenging,” said David Goldfarb, MD, clinical chief, Division of Nephrology, NYU Langone Health, New York City. in a news release.
However, with advancement over the original formulation Thiola EC tablets have limited food restrictions and may be taken with or without food. It also provides the potential to reduce the number of tablets necessary to manage cystinuria, explains Eric Dube, PhD, chief executive officer of Retrophin, the maker of the drug.
Thiola EC delayed-release tablets will be made available in doses of 100 mg and 300 mg from next month, announced the company.
The FDA approved Thiola EC tablets through the 505(b)(2) regulatory pathway, which allows the agency to reference previous findings of safety and efficacy for an already-approved product, as well as review findings from further studies of the product.