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Toremifene

Toremifene is a nonsteroidal (SERM) and triphenylethylene derivative that acts as an estrogen agonist/antagonist, primarily exhibiting antiestrogenic effects in breast tissue to treat in postmenopausal women with estrogen receptor-positive or unknown receptor status tumors. As a chlorinated analogue of , toremifene binds to estrogen receptors, blocking 's ability to stimulate tumor growth while potentially preserving density and lipid profiles in postmenopausal patients. It is administered orally at a standard dose of 60 mg once daily and was approved by the U.S. in 1997 under the brand name Fareston for this indication. Toremifene is metabolized primarily by the enzyme, with a of approximately five days, and its absorption is not significantly affected by food. Key precautions include avoiding use in patients with known QT interval prolongation, uncorrected electrolyte imbalances, or to the drug, due to risks of serious cardiac arrhythmias like . It may also cause hypercalcemia, tumor flare, or endometrial changes, necessitating monitoring, and is contraindicated during due to the potential for fetal harm. Common side effects include hot flashes, sweating, and , while serious adverse effects can involve or irregular heartbeat. Despite these risks, toremifene offers efficacy comparable to in hormone-dependent management.

Medical Uses

Indications

Toremifene is primarily indicated for the treatment of metastatic breast cancer in postmenopausal women with estrogen receptor-positive or receptor-unknown tumors. This approval, granted by the FDA in 1997, targets hormone-dependent advanced disease where the cancer has spread beyond the breast. Phase III clinical trials have established its efficacy as comparable to , the standard (SERM) at the time, with equivalent overall response rates in postmenopausal women with advanced . For instance, randomized studies involving over 1,500 patients showed similar objective response rates (around 20-30%) and median times to progression (approximately 6-7 months) for toremifene 60 mg daily versus tamoxifen 20-40 mg daily. Patient selection emphasizes confirmation of estrogen receptor (ER) status, typically via biopsy of the tumor or metastatic site, to identify those likely to benefit from antiestrogenic therapy. While effective in ER-positive cases, its use in receptor-unknown tumors is based on historical trial data where responses were observed without prior ER testing. Off-label uses include the management of and associated tenderness in men receiving for , where toremifene's antiestrogenic effects in tissue help mitigate these estrogenic side effects. It has also been investigated for preventing bone loss and fractures in this population, demonstrating significant increases in lumbar spine density (up to 3.5%) compared to in phase III trials. Additionally, due to its partial activity on bone, toremifene shows potential benefits for maintaining in postmenopausal women, though this is not an approved indication.

Dosage and Administration

Toremifene is administered orally at a standard dose of 60 mg once daily for the treatment of in postmenopausal women. Treatment should be continued continuously until evidence of disease progression or the development of unacceptable . The tablet may be taken with or without food, as absorption is not significantly affected by meals. No dose adjustments are required for patients with mild hepatic impairment, although caution is advised in those with moderate to severe liver dysfunction due to prolonged elimination . Patients receiving toremifene require regular to assess and , including periodic evaluation of tumor response through clinical and studies such as computed tomography or bone scans. Confirmation of receptor-positive status is essential prior to initiation, typically via or prior diagnostic testing.

Available Forms

Toremifene is formulated as oral tablets containing toremifene citrate as the , with each tablet providing 60 mg of toremifene (equivalent to 88.5 mg of toremifene citrate). The tablets are white to off-white, round, convex, unscored, and uncoated. The brand-name product Fareston was discontinued in 2024. versions of toremifene citrate 60 mg tablets are available following FDA approvals starting in 2020. Storage requirements specify controlled room temperature between 15°C and 30°C (59°F and 86°F), with excursions permitted within this range, and protection from , , and moisture to maintain stability.

Adverse Effects

Common Adverse Effects

The most common adverse effects of toremifene, observed in clinical trials for , are typically mild to moderate and occur at rates comparable to those with , with hot flashes being the most frequent. In a major North American trial involving 865 postmenopausal women, hot flashes affected 35% of patients on toremifene 60 mg daily compared to 30% on 20 mg, while sweating occurred in 20% versus 17%, respectively. These symptoms often emerge early in therapy and may diminish over time as the body adjusts. Nausea is another prevalent effect, reported in 14% of toremifene-treated patients versus 15% with , and is usually transient and self-limiting. , though less common, occurred in 4% of patients on toremifene compared to 2% on . , linked to the drug's partial estrogenic activity on the genital tract, was noted in 13% of cases with toremifene versus 16% with , while affected 2% versus 4%, respectively. Hypercalcemia was reported in approximately 3% of patients in the North American trial (similar to ). Tumor flare, characterized by transient increases in or tumor size often accompanied by hypercalcemia, may occur early in treatment, particularly in patients with bone metastases, but typically resolves without discontinuation. has been reported in clinical use. Management of these effects focuses on supportive measures to improve tolerability without interrupting . For hot flashes and sweating, patients can wear light clothing, maintain a cool environment, apply cool cloths to the skin, and incorporate lifestyle adjustments such as regular exercise and a rich in fruits and ; if symptoms persist, consultation with a healthcare provider for potential medications or alternatives is recommended. and vomiting are often managed with anti-nausea medications as prescribed, eating small frequent meals, and using lozenges or gum to alleviate discomfort. Vaginal discharge or warrants prompt reporting to a for evaluation, while can be addressed through adequate rest, balanced nutrition, and discussion with the healthcare team for supportive interventions. Hypercalcemia and tumor require monitoring, with discontinuation if severe. Overall, these effects contribute to the favorable safety profile of toremifene relative to its benefits in hormone receptor-positive treatment.
Adverse EffectToremifene IncidenceTamoxifen Incidence
Hot Flashes35%30%
Sweating20%17%
14%15%
13%16%
4%2%
2%4%
Hypercalcemia3%3%

Serious Adverse Effects

Toremifene, a selective estrogen receptor modulator (SERM), is associated with an increased risk of thromboembolic events, including deep vein thrombosis (DVT) and pulmonary embolism (PE), with reported incidences of approximately 1-2% in clinical studies, which is comparable to or slightly lower than that observed with tamoxifen. Patients with a history of thromboembolic diseases are at higher risk and generally should not receive toremifene therapy. Warning signs include sudden shortness of breath, chest pain, leg swelling, or pain, and preventive measures involve assessing patient history prior to initiation and prompt discontinuation if events occur. QT interval prolongation is a dose- and concentration-dependent serious adverse effect of toremifene, which can lead to torsades de pointes, ventricular arrhythmias, syncope, seizures, or sudden death. This risk is heightened in patients with congenital or acquired QT prolongation, electrolyte imbalances such as hypokalemia or hypomagnesemia, hepatic impairment, or concurrent use of QT-prolonging drugs. Monitoring includes baseline and periodic electrocardiograms (ECGs) in at-risk patients, along with correction of electrolytes and avoidance of interacting medications. Due to its partial estrogenic effects on the , toremifene can cause and an increased risk of , though the incidence remains low and may be similar to or lower than with . These estrogenic actions in non-target tissues contribute to the uterine risks. Patients with preexisting should avoid long-term treatment, and annual gynecologic examinations are recommended for early detection of abnormalities such as abnormal or . Visual disturbances, including retinal changes, abnormal visual fields, corneal keratopathy, and cataracts, occur in 1% to 10% of patients, with higher rates for cataracts (approximately 10%) compared to controls. Risk factors include preexisting ocular conditions or prolonged use, and preventive measures involve baseline and periodic ophthalmic examinations to monitor for symptoms like or dry eyes. Overall risk factors for serious adverse effects with toremifene include a history of thromboembolic events, cardiac , electrolyte disturbances, hepatic impairment, and preexisting uterine or ocular abnormalities, necessitating careful patient selection and ongoing .

Contraindications and Precautions

Contraindications

Toremifene is contraindicated in patients with known to the drug or any of its excipients, as severe allergic reactions may occur. Toremifene should be avoided in patients with a history of severe thromboembolic disease, including active thrombosis or embolism, due to the increased risk of recurrent events associated with selective estrogen receptor modulators like toremifene. Patients with congenital or acquired QT interval prolongation, uncorrected hypokalemia, or uncorrected hypomagnesemia should not receive toremifene, as the drug can further prolong the QT interval, potentially leading to torsades de pointes and life-threatening arrhythmias. Additionally, clinically relevant bradycardia, heart failure with reduced ejection fraction, or a history of symptomatic cardiac arrhythmias warrant avoidance of toremifene due to compounded risks of QT prolongation. Pre-existing endometrial hyperplasia is a contraindication, particularly for long-term therapy, given the estrogen agonist effects of toremifene on the endometrium that may promote hyperplasia or malignant transformation. Toremifene should be used with caution or avoided in patients with a history of endometrial cancer. Toremifene is contraindicated during because it has demonstrated embryo-fetal in at doses below the recommended human dose of 60 mg daily, indicating significant teratogenic potential; effective contraception is required in women of childbearing potential during treatment. It should not be used during , as it is unknown whether toremifene or its metabolites are excreted in human milk, and the potential for serious adverse reactions in nursing infants exists. In patients with severe hepatic impairment, toremifene is contraindicated for long-term use per guidelines owing to altered metabolism via , which prolongs the elimination and heightens the risk of adverse effects such as QT prolongation; per FDA, use with caution and closely. Mild to moderate hepatic impairment does not constitute an absolute but requires careful monitoring.

Drug Interactions

Toremifene, primarily metabolized by the 3A4 () enzyme, is subject to significant pharmacokinetic interactions with drugs that induce or inhibit this pathway. Strong CYP3A4 inducers, such as rifampin, , , and dexamethasone, can substantially reduce toremifene concentrations by accelerating its metabolism, potentially decreasing its therapeutic efficacy; coadministration of these agents with toremifene should be avoided. Conversely, strong CYP3A4 inhibitors like , , and increase toremifene exposure, with reported increases in maximum concentration (Cmax) by 1.4-fold and area under the curve () by 2.9-fold, heightening the risk of adverse effects; these combinations are also contraindicated. Toremifene can prolong the , leading to pharmacodynamic interactions with other QT-prolonging drugs that may result in additive effects and increase the risk of . Examples include class IA and III antiarrhythmics such as , quinidine, and , as well as certain antipsychotics, antidepressants, antibiotics, and antiemetics; close monitoring of the via electrocardiogram is recommended if coadministration is unavoidable, and toremifene should be interrupted if necessary. Interaction with , a substrate of , may potentiate its effects due to toremifene's influence on factors, necessitating careful of international normalized ratio (INR) levels during concurrent use. Additionally, acts as a inhibitor and should be avoided, as it can elevate toremifene concentrations similarly to pharmaceutical inhibitors. Overall, these interactions underscore the importance of dose adjustments, alternative therapies, or enhanced to mitigate risks and ensure safe administration.

Pharmacology

Pharmacodynamics

Toremifene is a (SERM) that acts as a / at estrogen receptors (ERs), exhibiting tissue-specific effects depending on the target organ and hormonal milieu. It binds to both ERα and ERβ, with a relative binding affinity of approximately 5% that of . In breast tissue, toremifene functions primarily as an by competitively binding to ERα, thereby blocking -induced signaling and inhibiting the of cells. In contrast, toremifene displays estrogenic effects in and on , where it acts as an at ERs to prevent bone loss and improve serum lipid profiles by lowering low-density lipoprotein cholesterol levels. These tissue-selective properties are characteristic of SERMs. Compared to , another first-generation SERM, toremifene shows similar binding affinities for ER. Beyond its primary ER-mediated actions, toremifene exhibits additional mechanisms that may contribute to its anticancer effects, including inhibition of by suppressing endothelial cell tube formation .

Pharmacokinetics

Toremifene is well absorbed following , with near-complete of approximately 100%. Peak concentrations are achieved within 1.5 to 6 hours after dosing, and its are linear over a wide dose range (10–680 mg). Steady-state concentrations are typically reached after 4 to 6 weeks of daily . The drug is extensively distributed throughout the body, with an apparent of approximately 580 L. Toremifene is highly bound to proteins, greater than 99.5%, primarily to . Toremifene undergoes extensive hepatic metabolism, predominantly via the 3A4 () enzyme, to form the N-desmethyltoremifene, which exhibits antiestrogenic activity and achieves serum concentrations 2 to 4 times higher than the parent compound. Other metabolites include 4-hydroxytoremifene and deaminohydroxy-toremifene. The drug may undergo auto-induction of metabolism with prolonged use. Elimination of toremifene follows a biexponential pattern, with a distribution of about 4 hours and a terminal elimination of approximately 5 days for the parent ; the major N-desmethyltoremifene has a of around 6 days. Total clearance is about 5 L/h, and the is primarily excreted in the feces (approximately 90%) as metabolites, with only about 10% appearing in the over one week; enterohepatic recirculation contributes to the prolonged . In special populations, the of toremifene are altered in hepatic impairment, where the elimination may be prolonged by less than twofold in patients with or , while metabolite profiles remain similar. No significant changes occur in renal impairment, and clearance is comparable between elderly and younger patients, though the may be slightly longer in the elderly.

Chemistry

Chemical Structure and Properties

Toremifene is a triphenylethylene characterized by a central moiety substituted with three phenyl rings, one of which bears a and a atom at the terminal position of the . Its molecular formula is C₂₆H₂₈ClNO, and the is 405.96 g/mol. The compound exists predominantly as the (Z)-, with the configuration around the being critical for its selective modulating activity. Key identifiers include the 89778-26-7 and CID 3005573. Toremifene appears as a white to light yellow crystalline powder with a of 108–110 °C. It exhibits very low in (approximately 0.0004 mg/mL) but is soluble in organic solvents such as DMSO (up to 120 mg/mL). Toremifene is synthesized via a multi-step process that constructs its triphenylethylene core and introduces the characteristic on the butenyl . A key route involves the between a substituted , such as 4-(2-dimethylaminoethoxy)phenylmethanone, and 3-hydroxy-1-phenylpropan-1-one to form the central with a pendant on the . This coupling employs low-valent titanium reagents, typically generated from titanium(IV) chloride and in , under conditions to yield the Z-isomer predominantly. Subsequent steps include purification of the core and attachment of the aminoethoxy if not already present. The atom is introduced in a final chlorination step by treating the hydroxy-substituted with in an inert like at elevated temperatures (50–110°C), converting the -CH₂CH₂OH group to -CH₂CH₂Cl while preserving the of the . This method ensures high yield and avoids complications from handling chlorinated precursors during the . An alternative bypasses McMurry by condensing a with , followed by reduction to a 1,4-diol and selective chlorination with . Toremifene's closest structural analog is , which differs only by lacking the substituent on the butenyl side chain (the des-chloro analog), sharing the same triphenylethylene scaffold and aminoethoxy moiety. For comparison, raloxifene represents a distinct class of SERMs based on a core, lacking the linkage but retaining modulating properties through a similar phenolic hydroxy group. Investigational derivatives of toremifene have been developed by modifying the or aryl substituents to enhance selectivity, such as incorporating carbonyl groups or altering the amino functionality to reduce metabolic activation to potentially carcinogenic species. These modifications aim to improve the therapeutic profile while maintaining the core responsible for binding.

History and Development

Discovery and Early Research

Toremifene was developed in the by the pharmaceutical company Orion-Farmos (now part of Orion Corporation) as a of , designed to provide comparable antiestrogenic antitumor effects while potentially offering an improved safety profile compared to , particularly regarding risk. The compound, a triphenylethylene derivative, was first synthesized in 1981 and demonstrated binding to the with an affinity approximately 5% that of . Preclinical research focused on evaluating toremifene's efficacy and safety in animal models of . In DMBA-induced rat mammary tumor models, toremifene exhibited statistically significant antitumor activity similar to , inhibiting tumor growth through antagonism. Safety assessments in female Crl:CD(BR) rats revealed that chronic administration of toremifene did not produce liver tumors or significant DNA adducts, in marked contrast to , which induced and extensive hepatic DNA damage under comparable conditions. These findings supported toremifene's potential as a safer alternative with reduced hepatotoxic potential. Early intellectual property protection included patents filed by for toremifene and related triphenylethylene compounds, with key filings in the mid-1980s leading to US Patent 4,696,949 granted in 1987. The initial detailed descriptions of toremifene's synthesis and pharmacological properties appeared in scientific publications in 1986, including proceedings from a satellite symposium on the compound held during the UICC World Cancer Congress in . Toremifene differs from by a chlorine atom substitution on the side chain, which preclinical data suggested contributed to its more favorable toxicity profile.

Regulatory Approvals

Toremifene, developed by the Finnish pharmaceutical company Orion Corporation, received its initial regulatory approval in from the () on February 14, 1996, for the first-line hormone treatment of hormone-dependent in postmenopausal women. In the United States, the () approved toremifene citrate under the brand name Fareston on May 29, 1997, for the treatment of in postmenopausal women with receptor-positive or receptor-unknown tumors. Following the expiration of key patents, the FDA approved the first version of toremifene citrate tablets (60 mg) on December 4, 2018, manufactured by EirGen Pharma Limited, enabling broader market availability for the treatment of the same indication. In March 2011, the FDA updated the prescribing information for Fareston to include a regarding the risk of prolongation and , recommending avoidance in patients with congenital or acquired prolongation, uncorrected , or hypomagnesemia, and caution with concomitant use of QT-prolonging drugs. In August 2024, Kyowa Kirin announced discontinuation of Fareston distribution in the US effective September 2024, with generic versions continuing availability. As of 2025, no additional indications beyond have been approved for toremifene by major regulatory agencies, with ongoing focused on cardiovascular risks such as prolongation.

Society and Culture

Generic and Brand Names

Toremifene is the (INN) assigned to the active substance by the . The (USAN) is toremifene citrate, reflecting the formulation commonly used in pharmaceutical products. The primary brand name for toremifene is Fareston, originally developed by Orion Corporation and licensed for marketing in the United States by GTx, Inc. Generic versions are available as toremifene citrate tablets, typically in 60 mg strength equivalent to the base compound. During its development, toremifene was known by the FC 1157a, a research code used prior to its adoption of the . Other developmental synonyms include NK 622 and GTx-006, though these are less commonly referenced in current literature. The citrate salt form is the standard in approved formulations due to its improved and , distinguishing it from the free base or other potential salts. Toremifene is widely available as a prescription-only in the United States, where generic versions have been approved by the FDA since 2018 and are accessible through retail and mail-order pharmacies. The name Fareston was discontinued in August 2024, though generic versions remain available. , it has been authorized since 1996 under the name Fareston and remains available as a prescription-only medicine marketed by Orion Corporation. Legally, toremifene is not classified as a under the U.S. or equivalent schedules in other jurisdictions, but it is strictly prescription-only due to its use in cancer therapy and associated risks such as thromboembolic events. Generics have been available in most major markets since the early , following expiration, though pharmacy stock may vary. In the United States, the of toremifene (60 mg tablets, a typical 30-day supply) ranges from approximately $277 to $422 with discount programs as of 2025, though retail prices without coupons can exceed $1,000. Availability is more limited in some developing countries, where is often preferred due to lower and broader accessibility for treatment.

Research

Completed Clinical Trials

Toremifene's efficacy in treating was established through several phase III trials conducted in the 1990s, primarily comparing it to in postmenopausal women with estrogen receptor-positive or unknown status disease. In the North American trial involving 436 patients, toremifene at 60 mg daily yielded an objective response rate of 21.3%, compared to 19.1% for at 20 mg daily, demonstrating non-inferiority with no significant difference in time to progression or overall . Similarly, the Eastern European trial with 306 patients reported response rates of 20.4% for toremifene 60 mg versus 20.8% for 40 mg, while the Nordic trial with 415 patients showed 31.3% versus 37.3%, respectively; across these studies, toremifene proved equivalent to in efficacy without superior response rates. These results supported toremifene's approval for , highlighting its comparable antitumor activity to the standard therapy at the time. A phase III trial evaluating toremifene for prevention in men with high-grade prostatic intraepithelial neoplasia (PIN), conducted from 2007 to 2013 by GTx Inc., enrolled 1,590 high-risk patients randomized to toremifene 20 mg daily or for three years. The study failed to demonstrate a significant reduction in prostate cancer incidence, with a non-significant 10.2% (hazard ratio approximately 0.90) observed in topline data. This outcome led to discontinuation of further development for this indication, as toremifene did not meet the primary endpoint of lowering cancer development rates based on annual biopsies. In men with receiving (ADT), including antiandrogens like , toremifene has shown effectiveness in preventing and related breast events. A phase III trial of toremifene 80 mg daily versus in 1,382 patients on ADT for localized or advanced reported significant reductions in breast pain and tenderness, with incidence rates dropping by over 60% in the toremifene arm compared to , while maintaining overall tolerability. These findings position toremifene as a viable option for mitigating bicalutamide-induced , as SERMs like toremifene counteract estrogenic effects on tissue without compromising ADT efficacy. Meta-analyses of toremifene's safety profile indicate a potentially lower risk of venous thromboembolism (VTE) compared to . Another analysis of 7,242 patients across multiple phase III studies confirmed no increased VTE risk with toremifene relative to , with odds ratios near 0.9, supporting its favorable cardiovascular safety in settings.

Ongoing and Potential Applications

As of 2025, ongoing on toremifene primarily explores its role in combination therapies for hormone receptor-positive (HR+) , leveraging its (SERM) properties to enhance efficacy in advanced settings. A multicenter, single-arm phase II (NCT06495515) is evaluating the combination of dalpiciclib, a CDK4/6 , with toremifene as first-line treatment for advanced HR+/HER2- in pre- and postmenopausal patients, including those requiring ovarian suppression. Preliminary data from this study, with a follow-up of 7.5 months as of May 2025, reported an objective response rate of 16.7% and a clinical benefit rate of 64%, with manageable adverse events such as , positioning the regimen as a potential option for patients intolerant to (AIs). As of November 2025, the remains ongoing. Similarly, a phase Ia/Ib study of TY-302, another CDK4/6 , combined with toremifene in HR+/HER2- demonstrated a of 8.2 months and a disease control rate of 88.9%, with the maximum tolerated dose established at TY-302 100 mg plus toremifene 60 mg daily; hematologic toxicities like were common but reversible. Emerging indications include toremifene as an adjunct in , where it functions similarly to by blocking receptors, though it is less commonly used due to limited specific efficacy data in men. High-dose toremifene (120 mg daily) has shown promise in reversing resistance to s in postmenopausal women with , achieving a clinical benefit rate of 31.6% in patients who progressed on prior nonsteroidal , outperforming in (7.3 vs. 3.7 months). Recent real-world studies from 2024 comparing toremifene with s in adjuvant settings for premenopausal patients have highlighted its endometrial safety profile, noting higher rates of endometrial thickening but similar incidence of compared to s, supporting its use in patients concerned about joint symptoms. Beyond , toremifene's estrogen-agonist effects on bone tissue suggest potential repurposing for prevention, particularly in patients undergoing deprivation or suppression therapies. In men receiving ADT, toremifene 80 mg daily has demonstrated increases in density (approximately 2% at the lumbar spine after 2 years), contrasting with bone loss seen in groups. Research challenges include limited funding for toremifene expansion, overshadowed by tamoxifen's established dominance and generic availability, which has constrained large-scale trials for novel indications despite its comparable pharmacodynamic versatility.

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