Deferasirox oral

Deferasirox oral is an FDA-registered drug product whose active ingredient is DEFERASIROX ORAL. It is labelled by Ascend Laboratories, LLC taken by oral and marketed as a human prescription drug product. Identifiers on file: NDC 67877-675, application ANDA213374, label effective 20260618. This page reproduces the FDA-approved label text so you can check what a product is, who makes it and how it is supplied before discussing it with a pharmacist or prescriber.

Across FDA labelling, products containing DEFERASIROX ORAL are most often described in connection with: iron overload, treatment chronic, patients years, due blood, non-transfusion-dependent thalassemia, ntdt syndromes, concentration lic, dry weight. Wording differs between labels, and the full indications section on this page takes precedence when it is present.

1 INDICATIONS AND USAGE Deferasirox is an iron chelator indicated for the treatment of chronic iron overload due to blood transfusions in patients 2 years of age and older. ( 1.1 ) Deferasirox is indicated for the treatment of chronic iron overload in patients 10 years of age and older with non-transfusion-dependent thalassemia (NTDT) syndromes, and with a liver iron (Fe) concentration (LIC) of at least 5 mg Fe per gram of dry weight (Fe/g dw) and a serum ferritin greater than 300 mcg/L. ( 1.2 ) Limitations of Use: The safety and efficacy of deferasirox when administered with other iron chelation therapy have not been established. ( 1.3 ) 1.1 Treatment of Chronic Iron Overload Due to Blood Transfusions (Transfusional Iron Overload) Deferasirox oral granules are indicated for the treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients 2 years of age and older. 1.2 Treatment of Chronic Iron Overload in Non-Transfusion-Dependent Thalassemia Syndromes Deferasirox oral granules is indicated for the treatment of chronic iron overload in patients 10 years of age and older with non-transfusion-dependent thalassemia (NTDT) syndromes and with a liver iron concentration (LIC) of at least 5 milligrams of iron per gram of liver dry weight (mg Fe/g dw) and a serum ferritin greater than 300 mcg/L. 1.3 Limitations of Use The safety and efficacy of deferasirox when administered with other iron chelation therapy have not been established.

2 DOSAGE AND ADMINISTRATION Transfusional Iron Overload: Initial dose for patients with estimated glomerular filtration rate (eGFR) greater than 60 mL/min/1.73 m 2 is 14 mg per kg (calculated to nearest whole sachet content for granules) once daily. ( 2.1 ) NTDT Syndromes: Initial dose for patients with eGFR greater than 60 mL/min/1.73 m2 is 7 mg per kg (calculated to nearest whole sachet content for granules) once daily. ( 2.2 ) See full prescribing information for information regarding monitoring, administration, and dose-reductions for organ impairment. ( 2.1 , 2.2 , 2.3 , 2.4 ) 2.1 Transfusional Iron Overload Deferasirox therapy should only be considered when a patient has evidence of chronic transfusional iron overload. The evidence should include the transfusion of at least 100 mL/kg of packed red blood cells (e.g., at least 20 units of packed red blood cells for a 40 kg person or more in individuals weighing more than 40 kg), and a serum ferritin consistently greater than 1,000 mcg/L. Prior to starting therapy, or increasing dose, evaluate: Serum ferritin level Baseline renal function: Obtain serum creatinine in duplicate (due to variations in measurements). Calculate the estimated glomerular filtration rate (eGFR). Use a prediction equation appropriate for adult patients (e.g., CKD-EPI, MDRD method) and in pediatric patients (e.g., Schwartz equations). Obtain urinalyses and serum electrolytes to evaluate renal tubular function [see Dosage and Administration ( 2.4 ), Warnings and Precautions ( 5.1 )]. Serum transaminases and bilirubin [see Dosage and Administration ( 2.4 ), Warnings and Precautions ( 5.2 )] Baseline auditory and ophthalmic examinations [see Warnings and Precautions ( 5.10 )] Initiating Therapy: The recommended initial dose of deferasirox for patients 2 years of age and older with eGFR greater than 60 mL/min/1.73 m 2 is 14 mg per kg body weight orally, once daily. Calculate doses (mg per kg per day) to the nearest whole sachet content for granules. Changes in weight of pediatric patients over time must be taken into account when calculating the dose. During Therapy: Monitor serum ferritin monthly and adjust the dose of deferasirox, if necessary, every 3 to 6 months based on serum ferritin trends. Use the minimum effective dose to achieve a trend of decreasing ferritin. Make dose adjustments in steps of 3.5 or 7 mg per kg and tailor adjustments to the individual patient’s response and therapeutic goals. In patients not adequately controlled with doses of 21 mg per kg (e.g., serum ferritin levels persistently above 2,500 mcg/L and not showing a decreasing trend over time), doses of up to 28 mg per kg may be considered. Doses above 28 mg per kg are not recommended [see Warnings and Precautions ( 5.6 )]. Adjust dose based on serum ferritin levels If the serum ferritin falls below 1,000 mcg/L at 2 consecutive visits, consider dose reduction especially if the deferasirox dose is greater than 17.5 mg/kg/day [see Adverse Reactions ( 6.1 )]. If the serum ferritin falls below 500 mcg/L, interrupt deferasirox therapy to minimize the risk of overchelation, and continue monthly monitoring [see Warnings and Precautions ( 5.6 )]. Evaluate the need for ongoing chelation therapy for patients whose conditions no longer require regular blood transfusions. Use the minimum effective dose to maintain iron burden in the target range [see Warnings and Precautions ( 5.6 )]. Monitor blood counts, liver function, renal function and ferritin monthly [see Warnings and Precautions ( 5.1 , 5.2 , 5.4 )]. Interrupt deferasirox for pediatric patients who have acute illnesses, which can cause volume depletion, such as vomiting, diarrhea, or prolonged decreased oral intake, and monitor more frequently. Resume therapy as appropriate, based on assessments of renal function, when oral intake and volume status are normal [see Dosage and Administration ( 2.4 , 2.5 ), Warnings and Precautions ( 5.1 ), Use in Specific Populations ( 8.4 ), Clinical Pharmacology ( 12.3 )]. 2.2 Iron Overload in Non-Transfusion-Dependent Thalassemia Syndromes Deferasirox therapy should only be considered when a patient with NTDT syndrome has an LIC of at least 5 mg Fe/g dw and a serum ferritin greater than 300 mcg/L. Prior to starting therapy, obtain: LIC by liver biopsy or by an FDA-cleared or approved method for identifying patients for treatment with deferasirox therapy Serum ferritin level on at least 2 measurements 1-month apart [see Clinical Studies ( 14 )] Baseline renal function: Obtain serum creatinine in duplicate (due to variations in measurements). Calculate the estimated glomerular filtration rate (eGFR). Use a prediction equation appropriate for adult patients (e.g., CKD-EPI, MDRD method) and in pediatric patients (e.g., Schwartz equations). Obtain urinalyses and serum electrolytes to evaluate renal tubular function [see Dosage and Administration ( 2.4 ), Warnings and Precautions ( 5.1 )] . Serum transaminases and bilirubin [see Dosage and Administration ( 2.4 ), Warnings and Precautions ( 5.2 )] Baseline auditory and ophthalmic examinations [see Warnings and Precautions ( 5.10 )] Initiating Therapy: The recommended initial dose of deferasirox for patients with eGFR greater than 60 mL/min/1.73 m 2 is 7 mg per kg body weight orally once daily. Calculate doses (mg per kg per day) to the nearest whole sachet content for granules. If the baseline LIC is greater than 15 mg Fe/g dw, consider increasing the dose to 14 mg/kg/day after 4 weeks. During Therapy: Monitor serum ferritin monthly to assess the patient's response to therapy and to minimize the risk of overchelation [see Warnings and Precautions (5.6)]. Interrupt treatment when serum ferritin is less than 300 mcg/L and obtain an LIC to determine whether the LIC has fallen to less than 3 mg Fe/g dw. Use the minimum effective dose to achieve a trend of decreasing ferritin. Monitor LIC every 6 months. After 6 months of therapy, if the LIC remains greater than 7 mg Fe/g dw, increase the dose of deferasirox to a maximum of 14 mg/kg/day. Do not exceed a maximum of 14 mg/kg/day. If after 6 months of therapy, the LIC is 3 to 7 mg Fe/g dw, continue treatment with deferasirox at no more than 7 mg/kg/day. When the LIC is less than 3 mg Fe/g dw, interrupt treatment with deferasirox and continue to monitor the LIC. Monitor blood counts, liver function, renal function and ferritin monthly [see Warnings and Precautions ( 5.1 , 5.2 , 5.4 )] . Increase monitoring frequency for pediatric patients who have acute illness, which can cause volume depletion, such as vomiting, diarrhea, or prolonged decreased oral intake. Consider dose interruption until oral intake and volume status are normal [see Dosage and Administration ( 2.4 , 2.5 ), Warnings and Precautions ( 5.1 ), Use in Specific Populations ( 8.4 ), Clinical Pharmacology ( 12.3 )] . Restart treatment when the LIC rises again to more than 5 mg Fe/g dw. 2.3 Administration Take deferasirox oral granules on an empty stomach or with a light meal (contains less than 7% fat content and approximately 250 calories). Examples of light meals include 1 whole wheat English muffin, 1 packet jelly (0.5 ounces), and skim milk (8 fluid ounces) or a turkey sandwich (2 oz. turkey on whole wheat bread w/ lettuce, tomato, and 1 packet mustard) [see Clinical Pharmacology ( 12.3 )]. Administer deferasirox oral granules by sprinkling the full dose on soft food (e.g., yogurt or applesauce) immediately prior to use and administered orally. Deferasirox oral granules should be taken once a day, preferably at the same time each day. Do not take deferasirox oral granules with aluminum-containing antacid products [see Drug Interactions ( 7.1 )]. For patients who are currently on chelation therapy with deferasirox tablets for oral suspension and converting to deferasirox oral granules, the dose should be about 30% lower, rounded to the nearest whole sachet content for granules. The table below provides additional information on dosing conversion to deferasirox oral granules. Deferasirox Tablets for oral suspension Deferasirox Oral Granules Transfusion-Dependent Iron Overload Starting Dose 20 mg/kg/day 14 mg/kg/day Titration Increments 5–10 mg/kg 3.5–7 mg/kg Maximum Dose 40 mg/kg/day 28 mg/kg/day Non-Transfusion-Dependent Thalassemia Syndromes Starting Dose 10 mg/kg/day 7 mg/kg/day Titration Increments 5–10 mg/kg 3.5–7 mg/kg Maximum Dose 20 mg/kg/day 14 mg/kg/day 2.4 Use in Patients With Baseline Hepatic or Renal Impairment Patients with Baseline Hepatic Impairment Mild (Child-Pugh A) Hepatic Impairment: No dose adjustment is necessary. Moderate (Child-Pugh B) Hepatic Impairment: Reduce the starting dose by 50%. Severe (Child-Pugh C) Hepatic Impairment: Avoid deferasirox oral granules [see Warnings and Precautions ( 5.2 ), Use in Specific Populations ( 8.7 )]. Patients with Baseline Renal Impairment Do not use deferasirox in adult or pediatric patients with eGFR less than 40 mL/min/1.73 m 2 [see Dosage and Administration ( 2.5 ), Contraindications ( 4 )]. For patients with renal impairment (eGFR 40-60 mL/min/1.73 m 2 ), reduce the starting dose by 50% [see Use in Specific Populations ( 8.6 )]. Exercise caution in pediatric patients with eGFR between 40 and 60 mL/minute/1.73 m 2 . If treatment is needed, use the minimum effective dose and monitor renal function frequently. Individualize dose titration based on improvement in renal injury [see Use in Specific Populations ( 8.6 )]. 2.5 Dose Modifications for Decreases in Renal Function While on Deferasirox Deferasirox is contraindicated in patients with eGFR less than 40 mL/min/1.73 m 2 [see Contraindications ( 4 )]. For decreases in renal function while receiving deferasirox [see Warnings and Precautions ( 5.1 )], modify the dose as follows: Transfusional Iron Overload Adults: If the serum creatinine increases by 33% or more above the average baseline measurement, repeat the serum creatinine within 1 week, and if still elevated by 33% or more, reduce the dose by 7 mg per kg. Pediatric Patients (ages 2 years-17 years): Reduce the dose by 7 mg per kg if eGFR decreases by greater than 33% below the average baseline measurement and repeat eGFR within 1 week. Interrupt deferasirox for acute illnesses, which can cause volume depletion, such as vomiting, diarrhea, or prolonged decreased oral intake, and monitor more frequently. Resume therapy as appropriate, based on assessments of renal function, when oral intake and volume status are normal. Avoid use of other nephrotoxic drugs [see Warnings and Precautions (5.1)]. In the setting of decreased renal function, evaluate the risk benefit profile of continued deferasirox use. Use the minimum effective deferasirox dose and monitor renal function more frequently, by evaluating tubular and glomerular function. Titrate dosing based on renal injury. Consider dose reduction or interruption and less nephrotoxic therapies until improvement of renal function. If signs of renal tubular or glomerular injury occur in the presence of other risk factors such as volume depletion, reduce or interrupt deferasirox to prevent severe and irreversible renal injury [see Warnings and Precautions ( 5.1 )]. All Patients (regardless of age): Discontinue therapy for eGFR less than 40 mL/min/1.73 m 2 [see Contraindications ( 4 )]. Non-Transfusion-Dependent Thalassemia Syndromes Adults: If the serum creatinine increases by 33% or more above the average baseline measurement, repeat the serum creatinine within 1 week, and if still elevated by 33% or more, interrupt therapy if the dose is 3.5 mg per kg, or reduce by 50% if the dose is 7 or 14 mg per kg. Pediatric Patients (ages 10 years -17 years): Reduce the dose by 3.5 mg per kg if eGFR decreases by greater than 33% below the average baseline measurement and repeat the eGFR within 1 week. Increase monitoring frequency for pediatric patients who have acute illnesses, which can cause volume depletion, such as vomiting, diarrhea, or prolonged decreased oral intake. Consider dose interruption until oral intake and volume status are normal. Avoid use of other nephrotoxic drugs [see Warnings and Precautions ( 5.1 ) ]. In the setting of decreased renal function, evaluate the risk benefit profile of continued deferasirox use. Use the minimum effective deferasirox dose and monitor renal function more frequently, by evaluating tubular and glomerular function. Titrate dosing based on renal injury. Consider dose reduction or interruption and less nephrotoxic therapies until improvement of renal function. If signs of renal tubular or glomerular injury occur in the presence of other risk factors such as volume depletion, reduce or interrupt deferasirox to prevent severe and irreversible renal injury [ see Warnings and Precautions ( 5.1 ) ]. All Patients (regardless of age): Discontinue therapy for eGFR less than 40 mL/min/1.73 m 2 [see Contraindications ( 4 )] . 2.6 Dose Modifications Based on Concomitant Medications UDP-glucuronosyltransferases (UGT) Inducers Concomitant use of UGT inducers decreases systemic exposure. Avoid the concomitant use of strong UGT inducers (e.g., rifampicin, phenytoin, phenobarbital, ritonavir). If you must administer deferasirox oral granules with a strong UGT inducer, consider increasing the initial dose by 50%, and monitor serum ferritin levels and clinical responses for further dose modification [see Dosage and Administration ( 2.1 , 2.2 ), Drug Interactions ( 7.5 )]. Bile Acid Sequestrants Concomitant use of bile acid sequestrants decreases systemic exposure. Avoid the concomitant use of bile acid sequestrants (e.g., cholestyramine, colesevelam, colestipol). If you must administer deferasirox oral granules with a bile acid sequestrant, consider increasing the initial dose by 50%, and monitor serum ferritin levels and clinical responses for further dose modification [see Dosage and Administration ( 2.1 , 2.2 ), Drug Interactions ( 7.6 )].

4 CONTRAINDICATIONS Deferasirox is contraindicated in patients with: Estimated GFR less than 40 mL/min/1.73 m 2 [see Dosage and Administration ( 2.5 ), Warnings and Precautions ( 5.1 )]; Poor performance status [see Warnings and Precautions ( 5.1 , 5.3 )] ; High-risk myelodysplastic syndromes (this patient population was not studied and is not expected to benefit from chelation therapy); Advanced malignancies [see Warnings and Precautions ( 5.1 , 5.3 )]; Platelet counts less than 50 x 109/L [see Warnings and Precautions ( 5.3 , 5.4 )]; Known hypersensitivity to deferasirox or any component of deferasirox [see Warnings and Precautions ( 5.7 ), Adverse Reactions ( 6.2 )]. Estimated GFR less than 40 mL/min/1.73 m 2 . ( 4 ) Patients with poor performance status. ( 4 ) Patients with high-risk myelodysplastic syndrome (MDS). ( 4 ) Patients with advanced malignancies. ( 4 ) Patients with platelet counts less than 50 x 10 9 /L. ( 4 ) Known hypersensitivity to deferasirox or any component of deferasirox oral granules. ( 4 )

6 ADVERSE REACTIONS The following clinically significant adverse reactions are also discussed in other sections of the labeling: Acute Kidney Injury, Including Acute Renal Failure Requiring Dialysis, and Renal Tubular Toxicity Including Fanconi Syndrome [see Warnings and Precautions ( 5.1 , 5.6)] Hepatic Toxicity and Failure [see Warnings and Precautions ( 5.2 , 5.6)] GI Hemorrhage [see Warnings and Precautions ( 5.3 )] Bone Marrow Suppression [see Warnings and Precautions ( 5.4 )] Hypersensitivity [see Warnings and Precautions ( 5.7 )] Severe Skin Reactions [see Warnings and Precautions ( 5.8 )] Skin Rash [see Warnings and Precautions ( 5.9 )] Auditory and Ocular Abnormalities [see Warnings and Precautions ( 5.10 )] In patients with transfusional iron overload, the most frequently occurring (greater than 5%) adverse reactions are diarrhea, vomiting, nausea, abdominal pain, skin rashes, and increases in serum creatinine. In deferasirox-treated patients with NTDT syndromes, the most frequently occurring (greater than 5%) adverse reactions are diarrhea, rash, and nausea. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Ascend Laboratories, LLC at 1-877-272-7901 or FDA at 1-800-FDA-1088 o r www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Deferasirox was evaluated in healthy volunteer trials. Currently, there are no clinical data in patients with deferasirox oral granules. Deferasirox contains the same active ingredient as deferasirox tablets for oral suspension. The following adverse reactions have been reported with deferasirox tablets for oral suspension. Transfusional Iron Overload A total of 700 adult and pediatric patients were treated with deferasirox for 48 weeks in premarketing studies. These included 469 patients with beta-thalassemia, 99 with rare anemias, and 132 with sickle cell disease. Of these patients, 45% were male, 70% were Caucasian, and 292 patients were less than 16 years of age. In the sickle cell disease population, 89% of patients were black. Median treatment duration among the sickle cell patients was 51 weeks. Of the 700 patients treated, 469 (403 beta-thalassemia and 66 rare anemias) were entered into extensions of the original clinical protocols. In ongoing extension studies, median durations of treatment were 88 to 205 weeks. Six hundred twenty-seven (627) patients with myelodysplastic syndrome (MDS) were enrolled across 5 uncontrolled trials. These studies varied in duration from 1 to 5 years. The discontinuation rate across studies in the first year was 46% (Adverse Events (AEs) 20%, withdrawal of consent 10%, death 8%, other 4%, lab abnormalities 3%, and lack of efficacy 1%). Among 47 patients enrolled in the study of 5-year duration, 10 remained on deferasirox at the completion of the study. Table 1 displays adverse reactions occurring in greater than 5% of deferasirox-treated beta-thalassemia patients (Study 1), sickle cell disease patients (Study 3), and patients with MDS (MDS pool). Abdominal pain, nausea, vomiting, diarrhea, skin rashes, and increases in serum creatinine were the most frequent adverse reactions reported with a suspected relationship to deferasirox. Gastrointestinal symptoms, increases in serum creatinine, and skin rash were dose related. Table 1. Adverse Reactions a Occurring in greater than 5% of Deferasirox-treated Patients in Study 1, Study 3, and MDS Pool Study 1 (Beta-thalassemia) Study 3 (Sickle Cell Disease) MDS Pool Adverse Reactions Deferasirox N=296 n (%) Deferoxamine N=290 n (%) Deferasirox N=132 n (%) Deferoxamine N=63 n (%) Deferasirox N=627 n (%) Abdominal Pain b 63 (21) 41 (14) 37 (28) 9 (14) 145 (23) Diarrhea 35 (12) 21 (7) 26 (20) 3 (5) 297 (47) Creatinine Increased c 33 (11) 0 (0) 9 (7) 0 89 (14) Nausea 31 (11) 14 (5) 30 (23) 7 (11) 161 (26) Vomiting 30 (10) 28 (10) 28 (21) 10 (16) 83 (13) Rash 25 (8) 9 (3) 14 (11) 3 (5) 83 (13) Abbreviation: MDS, myelodysplastic syndrome. a Adverse reaction frequencies are based on AEs reported regardless of relationship to study drug. b Includes ‘abdominal pain’, ‘abdominal pain lower’, and ‘abdominal pain upper’. c Includes ‘blood creatinine increased’ and ‘blood creatinine abnormal’. See also Table 2. In Study 1, a total of 113 (38%) patients treated with deferasirox had increases in serum creatinine greater than 33% above baseline on 2 separate occasions (Table 2) and 25 (8%) patients required dose reductions. Increases in serum creatinine appeared to be dose related [see Warnings and Precautions (5.1)]. In this study, 17 (6%) patients treated with deferasirox developed elevations in serum glutamic-pyruvic transaminase (SGPT)/ALT levels greater than 5 times the upper limit of normal (ULN) at 2 consecutive visits. Of these, 2 patients had liver biopsy proven drug-induced hepatitis and both discontinued deferasirox therapy [see Warnings and Precautions (5.2)]. An additional 2 patients, who did not have elevations in SGPT/ALT greater than 5 times the ULN, discontinued deferasirox because of increased SGPT/ALT. Increases in transaminases did not appear to be dose related. Adverse reactions that led to discontinuations included abnormal liver function tests (2 patients) and drug-induced hepatitis (2 patients), skin rash, glycosuria/proteinuria, Henoch Schönlein purpura, hyperactivity/insomnia, drug fever, and cataract (1 patient each). In Study 3, a total of 48 (36%) patients treated with deferasirox had increases in serum creatinine greater than 33% above baseline on 2 separate occasions (Table 2) [see Warnings and Precautions (5.1)]. Of the patients who experienced creatinine increases in Study 3, 8 deferasirox-treated patients required dose reductions. In this study, 5 patients in the deferasirox group developed elevations in SGPT/ALT levels greater than 5 times the ULN at 2 consecutive visits and 1 patient subsequently had deferasirox permanently discontinued. Four additional patients discontinued due to adverse reactions with a suspected relationship to study drug, including diarrhea, pancreatitis associated with gallstones, atypical tuberculosis, and skin rash. In the MDS pool, in the first year, a total of 229 (37%) patients treated with deferasirox had increases in serum creatinine greater than 33% above baseline on 2 consecutive occasions (Table 2) and 8 (3.5%) patients permanently discontinued [see Warnings and Precautions (5.1)]. A total of 5 (0.8%) patients developed SGPT/ALT levels greater than 5 times the ULN at 2 consecutive visits. The most frequent adverse reactions that led to discontinuation included increases in serum creatinine, diarrhea, nausea, rash, and vomiting. Death was reported in the first year in 52 (8%) of patients [see Clinical Studies (14)]. Table 2. Number (%) of Patients with Increases in Serum Creatinine or SGPT/ALT in Study 1, Study 3, and MDS Pool Study 1 (Beta-thalassemia) Study 3 (Sickle Cell Disease) MDS Pool Laboratory Parameter Deferasirox N=296 n (%) Deferoxamine N=290 n (%) Deferasirox N=132 n (%) Deferoxamine N=63 n (%) Deferasirox N=627 n (%) Serum Creatinine Creatinine increase greater than 33% at 2 consecutive post-baseline visits 113 (38) 41 (14) 48 (36) 14 (22) 229 (37) Creatinine increase greater than 33% and greater than ULN at 2 consecutive post-baseline visits 7 (2) 1 (0) 3 (2) 2 (3) 126 (20) SGPT/ALT SGPT/ALT greater than 5 x ULN at 2 post-baseline visits 25 (8) 7 (2) 2 (2) 0 9 (1) SGPT/ALT greater than 5 x ULN at 2 consecutive post-baseline visits 17 (6) 5 (2) 5 (4) 0 5 (1) Abbreviations: ALT, alanine transaminase; MDS, myelodysplastic syndrome; SGPT, serum glutamic-pyruvic transaminase; ULN, upper limit of normal. Non-Transfusion-Dependent Thalassemia Syndromes In Study 5, 110 patients with NTDT received 1 year of treatment with deferasirox 5 or 10 mg/kg/ day and 56 patients received placebo in a double-blind, randomized trial. In Study 6, 130 of the patients who completed Study 5 were treated with open-label deferasirox at 5, 10, or 20 mg/kg/ day (depending on the baseline LIC) for 1 year [see Clinical Studies (14)]. In Study 7, 134 patients with NTDT of 10 years of age or older with iron overload, received deferasirox tablets for oral suspension for up to 5 years, at a starting dose of 10 mg/kg/day followed by dose adjustment at Week 4, and then approximately every 6 months thereafter based on LIC levels. Table 3 and 4 display the frequency of adverse reactions in patients with NTDT. Adverse reactions with a suspected relationship to study drug were included in Table 3 if they occurred at ≥ 5% of patients in Study 5. Table 3. Adverse Reactions Occurring in Greater Than 5% ‎Patients with NTDT Any adverse reaction Study 5 Study 6 Study 7 Deferasirox N=110 n (%) Placebo N=56 n (%) Deferasirox N=130 n (%) Deferasirox N = 134 n (%) 31 (28) 9 (16) 27 (21) 50 (37) Nausea 7 (6) 4 (7) 2 (2) a 7 (5) Rash 7 (6) 1 (2) 2 (2) a 3 (2) a Diarrhea 5 (5) 1 (2) 7 (5) 8 (6) Abbreviation: NTDT, non-transfusion-dependent thalassemia. a The occurrence of nausea, and rash are included for Study 6 and rash for Study 7 for consistency. There were no additional adverse reactions with a suspected relationship to study drug occurring in >5% of patients in Study 6 and Study 7. In Study 5, 1 patient in the placebo 10 mg/kg/day group experienced an ALT increase to greater than 5 times ULN and greater than 2 times baseline (Table 4). Three deferasirox-treated patients (all in the 10 mg/kg/day group) had 2 consecutive serum creatinine level increases greater than 33% from baseline and greater than ULN. Serum creatinine returned to normal in all 3 patients (in 1 spontaneously and in the other 2 after drug interruption). Two additional cases of ALT increase and 2 additional cases of serum creatinine increase were observed in the 1-year extension of Study 5. The number (%) of patients with NTDT with increase in serum creatinine or SGPT/ALT in Study 5, Study 6, and Study 7 are presented in Table 4 below. Table 4. Number (%) of Patients with NTDT with Increases in Serum Creatinine or SGPT/ALT Laboratory Parameter Study 5 Study 6 Study 7 Deferasirox N=110 n (%) Placebo N=56 n (%) Deferasirox N=130 n (%) Deferasirox N=134 n (%) Serum creatinine (>33% increase from baseline and >ULN at ≥2 consecutive postbaseline values) 3 (3) 0 2 (2) 2 (2) SGPT/ALT (>5 x ULN and >2 x baseline) 1 (1) 1 (2) 2 (2) 1 (1) Abbreviations: ALT, alanine transaminase; NTDT, non-transfusion-dependent thalassemia; SGPT, serum glutamic-pyruvic transaminase; ULN, upper limit of normal. Proteinuria In clinical studies, urine protein was measured monthly. Intermittent proteinuria (urine protein/creatinine ratio greater than 0.6 mg/mg) occurred in 18.6% of deferasirox-treated patients compared to 7.2% of deferoxamine-treated patients in Study 1 [see Warnings and Precautions (5.1)]. Other Adverse Reactions In the population of more than 5,000 patients with transfusional iron overload, who have been treated with deferasirox during clinical trials, adverse reactions occurring in 0.1% to 1% of patients included gastritis, edema, sleep disorder, pigmentation disorder, dizziness, anxiety, maculopathy, cholelithiasis, pyrexia, fatigue, laryngeal pain, cataract, hearing loss, GI hemorrhage, gastric ulcer (including multiple ulcers), duodenal ulcer, renal tubular disorder (Fanconi syndrome), and acute pancreatitis (with and without underlying biliary conditions). Adverse reactions occurring in 0.01% to 0.1% of patients included optic neuritis, esophagitis, erythema multiforme, and drug reaction with eosinophilia and systemic symptoms (DRESS). Adverse reactions, which most frequently led to dose interruption or dose adjustment during clinical trials were rash, GI disorders, infections, increased serum creatinine, and increased serum transaminases. Pooled Analysis of Pediatric Clinical Trial Data A nested case control analysis was conducted within a deferasirox tablets for oral suspension pediatric-pooled clinical trial dataset to evaluate the effects of dose and serum ferritin level, separately and combined, on kidney function. Among 1213 children (aged 2 to 15 years) with transfusion-dependent thalassemia, 162 cases of acute kidney injury (eGFR ≤ 90 mL/min/1.73 m 2 ) and 621 matched-controls with normal kidney function (eGFR ≥ 120 mL/min/1.73 m 2) were identified. The primary findings were: - A 26% increased risk of acute kidney injury was observed with each 5 mg/kg increase in daily deferasirox tablets for oral suspension dosage equivalent to 3.5 mg/kg deferasirox, starting at 20 mg/kg/day equivalent to 14 mg/kg/day deferasirox (95% confidence interval (CI): 1.08-1.48). - A 25% increased risk for acute kidney injury was observed with each 250 mcg/L decrease in serum ferritin starting at 1250 mcg/L (95%CI: 1.01-1.56). - Among pediatric patients with a serum ferritin less than 1,000 mcg/L, those who received deferasirox tablets for oral suspension dosage greater than 30 mg/kg/day, equivalent to 21 mg/kg/day deferasirox compared to those who received lower dosages, had a higher risk for acute kidney injury (Odds ratio (OR)=4.47, 95% CI: 1.25-15. 95), consistent with overchelation. In addition, a cohort-based analysis of ARs was conducted in the deferasirox tablets for oral suspension pediatric pooled clinical trial data. Pediatric patients who received deferasirox tablets for oral suspension dose greater than 25 mg/kg/day equivalent to 17.5 mg/kg/day deferasirox when their serum ferritin was less than 1,000 mcg/L (n=158), had a 6-fold greater rate of renal adverse reactions (Incidence Rate Ratio (IRR) = 6.00, 95% CI: 1.75-21.36), and a 2-fold greater rate of dose interruptions (IRR= 2.06, 95% CI: 1.33-3.17) compared to the time-period prior to meeting these simultaneous criteria. Adverse reactions of special interest (cytopenia, renal, hearing, and GI disorders) occurred 1.9-fold more frequently when these simultaneous criteria were met, compared to preceding time-periods (IRR=1.91, 95% CI: 1.05-3.48) [see Warnings and Precautions (5.6)]. 6.2 Postmarketing Experience The following adverse reactions have been spontaneously reported during post-approval use of deferasirox in the transfusional iron overload setting. Because these reactions are reported voluntarily from a population of uncertain size, in which patients may have received concomitant medication, it is not always possible to reliably estimate frequency or establish a causal relationship to drug exposure. Skin and Subcutaneous Tissue Disorders: Stevens-Johnson syndrome (SJS), leukocytoclastic vasculitis, urticaria, alopecia, toxic epidermal necrolysis (TEN) Immune System Disorders: hypersensitivity reactions (including anaphylactic reaction and angioedema) Renal and Urinary Disorders: acute renal failure, tubulointerstitial nephritis Hepatobiliary Disorders: hepatic failure GI Disorders: GI perforation Blood and Lymphatic System Disorders: worsening anemia 5-Year Pediatric Registry In a 5-year observational study, 267 pediatric patients 2 to less than 6 years of age (at enrollment) with transfusional hemosiderosis received deferasirox. Of the 242 patients who had pre-and post-baseline eGFR measurements, 116 (48%) patients had a decrease in eGFR of ≥33% observed at least once. Twenty-one (18%) of these 116 patients with decreased eGFR had a dose interruption, and 15 (13%) of these 116 patients had a dose decrease within 30 days. Adverse reactions leading to permanent discontinuation from the study included liver injury (n=11), vomiting (n=2), renal tubular disorder (n=1), proteinuria (n=1), hematuria (n=1), upper GI hemorrhage (n=1), abdominal pain (n=1), and hypokalemia (n=1).

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What is Deferasirox oral used for?
1 INDICATIONS AND USAGE Deferasirox is an iron chelator indicated for the treatment of chronic iron overload due to blood transfusions in patients 2 years of age and older. ( 1.1 ) Deferasirox is indicated for the treatment of chronic iron overload in patients 10 years of age and older with non-transfusion-dependent thalassemia (NTDT) syndromes, and with a liver iron (Fe) concentration (LIC) of …

Who makes Deferasirox oral?
Ascend Laboratories, LLC is the labeler of record for Deferasirox oral.

What form does Deferasirox oral come in?
administered by oral. Strengths and pack sizes vary by label, so compare the NDC on your pack with the identifier listed on this page.

Is Deferasirox oral prescription or over-the-counter?
The FDA label for this product is recorded as human prescription drug. Marketing status can differ between strengths and countries, so check the dispensing rules where you live.

Has Deferasirox oral been recalled?
5 FDA recall record(s) match this product name or manufacturer. Review the recall and safety notices section on this page and confirm lot numbers against the official FDA enforcement report.

Where does this information come from?
All content is reproduced from FDA Structured Product Labelling data via openFDA. It is provided for identification and reference only and is not medical advice.