PDUFA date set for
“We know that living with a rare kidney disorder like cystinuria can be a challenge so we have worked with the patient, caregiver and medical communities over the past several years to identify ways we can further enhance Thiola,” said
The NDA was filed by the Company’s partner,
About Thiola® (tiopronin)
Thiola (tiopronin) tablets are indicated for the prevention of cystine (kidney) stone formation in patients with severe homozygous cystinuria with urinary cystine >500 mg/day, who are resistant to treatment with conservative measures of high fluid intake, alkali and diet modification, or who have adverse reactions to d-penicillamine.
Important Safety Information
Warnings & Precautions:
While no deaths have been reported with Thiola treatment, and despite the apparent reduced toxicity of Thiola relative to d-penicillamine, Thiola can potentially cause all the serious adverse reactions reported for d-penicillamine, including death. Hematologic abnormalities requiring drug discontinuance may occur. Proteinuria, may sometimes occur, severe enough to cause nephrotic syndrome. Thiola therapy should be discontinued if there are findings suggestive of Goodpasture syndrome, myasthenic syndrome, or myasthenia gravis. To help reduce the risk of serious complications, the following tests are recommended: peripheral blood counts, direct platelet count, hemoglobin, serum albumin, liver function tests, 24-hour urinary protein and routine urinalysis. The safety and efficacy for Thiola in children under 9 years of age have not been established.
Thiola is contraindicated during pregnancy (except where the benefits clearly outweigh the risks), in nursing mothers, and in patients who have previously developed agranulocytosis, aplastic anemia or thrombocytopenia while on this medication.
Some patients may develop drug fever, usually during the first month of therapy. Thiola treatment should be discontinued until the fever subsides. Other potential associated adverse reactions include: gastrointestinal side effects, impairment in taste and smell, hypersensitivity reactions, hematologic abnormalities, renal complications, pulmonary manifestations, wrinkling and friability of skin, and neurological complications. The most common adverse reactions in clinical trials include: diarrhea (2%), and the following at 1%: reflux esophagitis, malaise, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy.
Please see the full prescribing information including the complete indications and usage, contraindications, warnings, precautions and adverse reactions at thiola.com.
This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words "may", "might", "believes", "thinks", "anticipates", "plans", "expects", "intends" or similar expressions. In addition, expressions of our strategies, intentions or plans are also forward-looking statements. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with the Company’s business and finances in general, success of its commercial products, as well as risks and uncertainties associated with the Company's preclinical and clinical stage pipeline. Specifically, the Company faces risks associated with market acceptance of its products (including the new formulation of Thiola if approved for marketing by the
Vice President, Investor Relations & Corporate Communications
Source: Retrophin, Inc.