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TI

tiopronin (Thiola EC)

✓ Approved

Mission Pharmacal Company · Small Molecule · Small Molecule

What is tiopronin?

tiopronin is a small molecule developed by Mission Pharmacal Company. It is approved for therapeutic indications via oral (po).

Drug Profile

Brand NamesThiola EC
CompanyMission Pharmacal Company
Drug ClassSmall Molecule
RouteOral (PO)
StatusApproved
Sources: ClinicalTrials.gov, Mission Pharmacal Company

Therapeutic Indications

tiopronin is developed for 5 unique indications across 5 therapeutic areas.

Therapeutic AreaConditionPhase
Congenital, familial and genetic disordersCystinuriaBLA/NDA
Renal and urinary disordersCalculus urinaryPreclinical
Eye disordersCataractPreclinical
Injury, poisoning and procedural complicationsMetal poisoningPreclinical
Musculoskeletal and connective tissue disordersRheumatoid arthritisPreclinical

Related Research Articles

PubMedKidney & blood pressure research2026-06-30

Cystine-binding medication is poorly accessible for the treatment of cystinuria.

Peng Xuemi X, Penninx Bart B, Marsman Roos F RF, Capolongo Giovanna G et al.

Introduction Cystinuria is a rare inherited disorder characterized by recurrent cystine stone formation. When lifestyle modification and urine alkalinization fail, cystine-binding medication such as tiopronin and D-penicillamine are indicated. Despite proven benefit, their accessibility across Europe appears limited. We hypothesized that access to this medication is restricted and varies substantially between European countries. Methods We surveyed physicians affiliated with the European Reference Network for Rare Kidney Diseases (ERKNet) regarding access barriers and prescribing practices. The online questionnaire was completed by 40 clinicians from 15 countries. Results Eighty-five percent of respondents had prescribed cystine-binding medication in the past two years, with tiopronin being the preferred agent. However, 30% reported having no access to tiopronin at all, and 83% of recent prescribers experienced accessibility problems, including high costs and unfilled prescriptions. Substantial differences were observed both between and within countries. Prescription rates correlated with physicians' cystinuria caseload. Discussion Cystine-binding medication is poorly and inconsistently accessible across Europe. Coordinated efforts are urgently needed to stabilize supply and ensure equitable, affordable access.

PMID 42378189
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PubMedTalanta2026-05-22

Development of two flow analysis methods for the determination of tiopronin in pharmaceuticals.

Kukoc Lea L, Biocic Maja M, Spassov Tony G TG, Kolev Spas D SD

A flow injection analysis (FIA) method and the first sequential injection analysis (SIA) method for the determination of tiopronin in pharmaceuticals have been developed. Both utilize the one-step colour reaction involving the reduction of the Cu(II)-bathocuproine complex by tiopronin to the orange-coloured Cu(I)-bathocuproine complex with maximum absorbance at 483 nm. The FIA and SIA methods exhibit linear calibration ranges of 4.0 × 10-7 - 4.0 × 10-5 and 4.0 × 10-7 - 6.0 × 10-5 mol L-1, repeatability (expressed as relative standard deviation) of 1.4 and 2.9 % and sampling rates of 180 and 36 h-1, respectively. Both methods are characterised by limits of detection and quantitation of 1.4 × 10-7 and 4.6 × 10-7 mol L-1, respectively. They have been successfully applied to the determination of tiopronin in a pharmaceutical. No statistically significant difference has been observed between the two methods, and between each one of them and the standard Pharmacopoeia method on one hand and the amount of tiopronin per tablet quoted by the tablet manufacturer. Good recovery results for both flow methods have been also obtained with spiked tablet samples. Based on the sampling rate and reagent consumption results, it has been concluded that the FIA method, which offers a higher sampling rate than existing FIA methods, is more suitable for the analysis of large numbers of samples while the SIA method minimises analysis costs and the generation of chemical waste.

PMID 42167033
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PubMedOrphanet journal of rare diseases2026-04-23

A rapid HPLC method to simultaneously quantify therapeutic thiols and monitor their disulfide exchange reactions with cystine.

Hadjipour Azhidhack A, Gayatri Gayatri G, Rioux Patrice P, Kavanagh Oisín N ON

This study introduces a rapid and environmentally sustainable High Performance Liquid Chromatography method to simultaneously quantify free thiols and monitor their disulfide exchange kinetics with cystine. This method is based on the derivatisation of thiols with 5,5’-dithiobis(2-nitrobenzoic acid) (DTNB, also known as Ellman’s reagent). The method utilises a mobile phase consisting of 0.1% v/v Trifluoroacetic acid in water and methanol, with Ultraviolet detection at 248 nm. The method exhibited high sensitivity (Limit of Quantification 0.78 µM; Limit of Detection 0.39 µM) and selectivity for biologically relevant thiols (cysteamine, cysteine, tiopronin, glutathione, captopril and penicillamine) in an aqueous environment. Simultaneous quantification of these thiols was successfully achieved in phosphate-buffered saline as well as in biological samples such as human saliva and urine (from a vegetarian subject). Furthermore, it facilitated kinetic analysis of thiol-related reactions, with potential applications in both clinical and pharmacological research.

PMID 42021294
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PubMedJournal of fluorescence2026-04-16

N-Doped CQDs as a Fluorescence Probe for Tiopronin Detection in Pharmaceutical Formulations via Static Quenching.

Liu Tianhui T, Yan Yiling Y, Chen Zhenni Z, Zhao Chengfei C

In recent years, fluorescent sensors based on carbon quantum dots (CQDs) have been widely reported for pharmaceutical detection owing to their numerous advantages. In this study, a Fe³⁺-mediated fluorescence “off–on” sensor based on nitrogen-doped carbon quantum dots (N-CQDs) was developed for the selective detection of tiopronin (TPN). Briefly, highly fluorescent N-CQDs were synthesized via a one-step hydrothermal method using citric acid monohydrate and diethylenetriamine. XPS, FT-IR, UV-Vis, fluorescence spectroscopy, and DLS confirmed that the N-CQDs exhibit uniform size, aqueous stability, excitation-independent fluorescence, and a high quantum yield of 62.99%. Fe³⁺ selectively bound to N-CQDs to form a complex, resulting in fluorescence quenching. Subsequently, upon the addition of TPN, the fluorescence of N-CQDs showed a proportional recovery. During this process, the competitive coordination between TPN and Fe³⁺ led to the release of Fe³⁺ from the N-CQDs, thereby restoring the quenched fluorescence. The limit of detection (LOD) of TPN was 80 μmol/L, with a linear range of 0.2–0.7 mmol/L and an R² value of 0.9993. The content of TPN in enteric-coated tablets was quantified using this method, yielding recovery rates ranging from 92.59% to 104.22%. This method is simple, rapid, and reliable, offering good sensitivity without the need for expensive reagents or complex equipment.

PMID 41986572
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PubMedThe Turkish journal of pediatrics2026-03-24

Comparative analysis of surgical costs vs. theoretical tiopronin therapy in pediatric cystinuria: a single-center experience from Türkiye.

Sözduyar Sümeyye S, Ulman Hilmican H, Tekin Ali A, Ulman İbrahim İ et al.

Cystinuria is a rare autosomal recessive disorder leading to recurrent cystine stone formation, often necessitating repeated surgical interventions. In Türkiye, tiopronin-a proven medical therapy-has become inaccessible since 2019, raising concerns about its economic and clinical consequences. This study aimed to compare the annual hospital costs of surgical management with the theoretical cost of tiopronin therapy in pediatric cystinuria patients. This single-center, retrospective study included 10 consecutive pediatric patients (median age: 12 years; range: 3-17 years) with genetically or biochemically confirmed cystinuria who underwent surgery for cystine stones in 2023. The annual cost of surgical management was calculated by summing all direct medical costs (operating room, anesthesia, devices, hospitalization, and related diagnostics) obtained from the hospital billing database. The theoretical annual cost of tiopronin therapy was calculated based on the last accessible market price of the drug in Türkiye, converted to Turkish Lira at the 2023 average exchange rate, and adjusted per patient using a standard dosing regimen of 15 mg/kg/day. Costs were compared using the Mann-Whitney U test. The median annual surgical cost per patient was 49,936 TL (range: 14,791-84,576 TL), compared to a theoretical tiopronin cost of 27,923 TL (range: 9,307-27,925 TL). Surgical management was significantly more expensive than tiopronin therapy (p=0.001) in 9 of 10 cases. Six patients had a history of tiopronin use, and five of them experienced a surgery-free interval during medication (median duration: 3 years; range: 2-6 years). Preliminary follow-up data for 2024 revealed that 70% of patients required further surgical interventions (median: 2 surgeries per patient), demonstrating the persistent and recurrent nature of the disease when managed solely with surgery. Surgical treatment of cystinuria poses a substantially higher economic burden compared to tiopronin therapy. Our findings support the reintroduction of tiopronin into the Turkish healthcare system, particularly for pediatric patients with frequent stone recurrence.

PMID 41871560
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PubMedEcotoxicology and environmental safety2026-01-28

Molecular mechanisms and clinical insights of benzo[a]pyrene-related lung adenocarcinoma: New discoveries in multi-scale approaches.

Xu Zhenyu Z, Luo Guolu G, Cao Xuchen X, Huang Chongbiao C

While the carcinogenicity of Benzo[a]pyrene (BaP) in lung adenocarcinoma (LUAD) is well-documented, the molecular mechanisms underlying BaP-driven tumorigenesis remain not fully clear. We first identified BaP-related prognostic genes for LUAD by analyzing online data and constructed prognostic models. Then diagnostic genes were screened from the aforementioned genes, and machine learning algorithms were employed to develop diagnostic models. Subsequently, single-cell and spatial transcriptomics were applied to characterize the cellular and spatial distribution of target genes, along with their gene co-localization. Molecular docking and dynamics were conducted to assess the binding affinities and stability between BaP and target proteins. In addition, we conducted some other analyses such as the correlation analysis between the expression of target genes (as well as the key genes of some pathways) and the patients' smoking status. During the construction of prognostic and diagnostic models, we identified five genes (SOD1, HK2, ACSS1, ANGPTL4, and CTBP2) that serve as core targets for BaP in the occurrence and progression of LUAD. Single-cell RNA sequencing and spatial transcriptomic analysis further validated these targets, and explained possible pathways how BaP causes LUAD, such as immunity and metabolism together with other analyses. Molecular docking and dynamics collectively revealed strong binding affinities and dynamic interactions between BaP and these targets, while the correlation analysis has also shown good results. Drug enrichment analysis highlighted tiopronin as promising therapeutic candidate for BaP-exposed populations. This study bridges BaP carcinogenesis and LUAD pathogenesis, offering translational insights for risk assessment, early diagnosis, and targeted therapy of BaP-related LUAD.

PMID 41601038
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