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Kalydeco is a brand name for Ivacaftor. The medicine, its uses, side effects and dosage are the same regardless of brand.
Used for: 1 INDICATIONS AND USAGE KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data [see Clinical Pharmacology (12.1) and Clinical Studies…
Verbatim from this product's FDA label. Tap a section to expand.
4 mg packet every 12 hours 4 months to less than 6 months 5 kg or greater One 25 mg packet every 12 hours 6 months to less than 6 years 5 kg to less than 7 kg One 25 mg packet every 12 hours 7 kg to less than 14 kg One 50 mg packet every 12 hours 14 kg or greater One 75 mg packet every 12 hours 6 years and older - One 150 mg tablet every 12 hours Taken orally with fat-containing food See full prescribing information for the recommended dosage in patients aged 6 months and older with moderate or severe hepatic impairment.
6 ) See full prescribing information for dosage modifications due to drug interactions with KALYDECO. 1 ) Not recommended in pediatric patients less than 1 month of age. 4 ) Not recommended in patients 1 month to less than 6 months of age with any level of hepatic impairment and/or taking concomitant moderate or strong CYP3A inhibitors.
5) ]. 2 Recommended Dosage in Pediatric Patients Aged 1 Month to Less than 6 Years The recommended dosage of KALYDECO (oral granules) for pediatric patients aged 1 month to less than 6 years is weight-based provided in Table 1. 5) ] .
Table 1:
Recommended Dosage of KALYDECO Oral Granules by Body Weight in Pediatric Patients Aged 1 Month to Less than 6 Years Age Body Weight (kg) KALYDECO Dosage 1 month to less than 2 months KALYDECO is not recommended for use in pediatric patients under 1 month of age.
Use of KALYDECO in pediatric patients aged 1 to less than 6 months born at a gestational age less than 37 weeks has not been evaluated. 3 Recommended Dosage for Patients with Hepatic Impairment KALYDECO is not recommended in patients less than 6 months of age with any level of hepatic impairment.
5) ] for patients aged 6 months and older with hepatic impairment: Mild Hepatic Impairment (Child-Pugh Class A): Less than 6 months of age: KALYDECO is not recommended. 3) ] .
Moderate Hepatic Impairment (Child-Pugh Class B):
Less than 6 months of age: KALYDECO is not recommended. 2) ]. 6 years of age and older: 150 mg orally once daily.
Severe Hepatic Impairment (Child-Pugh Class C):
Should not be used in patients less than 6 months of age. In patients aged 6 months and older should be used with caution. KALYDECO has not been studied in patients with severe hepatic impairment (Child-Pugh Class C), but exposure is expected to be higher than in patients with moderate hepatic impairment.
6) ] The most common adverse drug reactions to KALYDECO (≥8% of patients with CF who have a G551D mutation in the CFTR gene) were headache, oropharyngeal pain, upper respiratory tract infection, nasal congestion, abdominal pain, nasopharyngitis, diarrhea, rash, nausea, and dizziness.
gov/medwatch . 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 clinical practice.
The overall safety profile of KALYDECO is based on pooled data from three placebo-controlled clinical trials conducted in 353 patients 6 years of age and older with CF who had a G551D mutation in the CFTR gene (Trials 1 and 2) or were homozygous for the F508del mutation (Trial 3).
In addition, the following clinical trials have also been conducted [ see Clinical Pharmacology (12) and Clinical Studies (14) ]: An 8-week, crossover design trial (Trial 4) involving 39 patients between the ages of 6 and 57 years with a G1244E , G1349D , G178R , G551S , G970R , S1251N , S1255P , S549N , or S549R mutation in the CFTR gene.
A 24-week, placebo-controlled trial (Trial 5) involving 69 patients between the ages of 6 and 68 years with an R117H mutation in the CFTR gene. A 24-week, open-label trial (Trial 6) in 34 patients 2 to less than 6 years of age. Patients eligible for Trial 6 were those with the G551D, G1244E , G1349D , G178R , G551S , G970R , S1251N , S1255P , S549N , or S549R mutation in the CFTR gene.
Of 34 patients enrolled, 32 had the G551D mutation and 2 had the S549N mutation. An 8-week, crossover design trial (Trial 7) involving patients between the ages of 12 and 72 years who were heterozygous for the F508del mutation and a second CFTR mutation predicted to be responsive to ivacaftor.
5 WARNINGS AND PRECAUTIONS Elevated transaminases (ALT or AST): Transaminases (ALT and AST) should be assessed prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter. In patients with a history of transaminase elevations, more frequent monitoring of liver function tests should be considered.
Patients who develop increased transaminase levels should be closely monitored until the abnormalities resolve. Interrupt dosing in patients with ALT or AST of greater than 5 times the upper limit of normal (ULN). Following resolution of transaminase elevations, consider the benefits and risks of resuming KALYDECO dosing.
1 , 6 ) Hypersensitivity reactions: Anaphylaxis has been reported with KALYDECO in the postmarketing setting. Initiate appropriate therapy in the event of a hypersensitivity reaction. 2 ) Intracranial hypertension : Intracranial hypertension (IH) has been reported in the postmarketing setting with use of drugs containing the same or similar active ingredients as KALYDECO.
If an unusual headache or visual disturbances occur during treatment, and IH is suspected, interrupt KALYDECO and refer for prompt medical evaluation. 3 ) Neuropsychiatric events, including suicidal thoughts and behaviors : Serious neuropsychiatric events, including symptoms of anxiety, depression, suicidal ideation and behavior, and sleep disturbances, have been reported in the postmarketing setting for KALYDECO or drugs containing the same or similar active ingredient.
Monitor patients closely for new or worsening symptoms. Consider the risks and benefits for the individual patient to determine if therapy with KALYDECO should be interrupted at the occurrence of neuropsychiatric symptoms. , rifampin, St.
John's wort) substantially decreases exposure of ivacaftor, which may diminish effectiveness. Therefore, co-administration is not recommended. 3 ) Cataracts: Non-congenital lens opacities/cataracts have been reported in pediatric patients treated with KALYDECO.
4 CONTRAINDICATIONS None. None ( 4 )
Not medical advice. Always read the patient information leaflet and follow your prescriber or pharmacist.
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3) ] .
Less than 6 months of age:
KALYDECO is not recommended. 2) ]. 6 years of age and older: 150 mg orally once daily or less frequently. 4 Dosage Modification for Patients Taking Drugs that are CYP3A Inhibitors Concomitant use of moderate or strong CYP3A inhibitors is not recommended in patients below 6 months of age.
3) ]. 5) ]. Dosage modification for patients 6 months of age and older taking CYP3A inhibitors : Moderate CYP3A inhibitors: Less than 6 months of age: KALYDECO is not recommended. 2) ]. 6 years of age and older: 150 mg orally once daily.
Strong CYP3A inhibitors :
Less than 6 months of age: KALYDECO is not recommended. 2) ]. 6 years of age and older: 150 mg orally twice weekly. 5 Administration Information Administer KALYDECO tablets or oral granules with fat-containing food. Examples include eggs, butter, peanut butter, cheese pizza, whole-milk dairy products (such as whole milk, cheese, yogurt, breast milk, or infant formula), etc.
3) ]. Instruction for Administration of Tablets Swallow tablets whole. Instruction for Administration of Oral Granules Administer each dose of KALYDECO oral granules immediately before or after ingestion of fat-containing food. Mix the entire contents of each packet of oral granules with one teaspoon (5 mL) of age-appropriate soft food or liquid that is at or below room temperature.
Some examples of soft foods or liquids may include puréed fruits or vegetables, yogurt, applesauce, water, breast milk, infant formula, milk, or juice. Food or liquid should be at or below room temperature. Once mixed, the product should be completely consumed within one hour.
A total of 156 patients were randomized to and received KALYDECO. A 24-week open-label clinical trial in patients with CF aged less than 24 months (Trial 8) including a cohort of 19 patients aged 12 months to less than 24 months, a cohort of 11 patients aged 6 months to less than 12 months, a cohort of 6 patients aged 4 months to less than 6 months, and a cohort of 7 patients aged 1 month to less than 4 months.
Patients with a gating mutation or R117H mutation were eligible for the first three cohorts of this study. Patients with any ivacaftor-responsive mutation were eligible for the cohort aged 1 to less than 4 months. Of the 353 patients included in the pooled analyses of patients with CF who had either a G551D mutation or were homozygous for the F508del mutation in the CFTR gene, 50% of patients were female and 97% were Caucasian; 221 received KALYDECO, and 132 received placebo for 16 to 48 weeks.
The proportion of patients who prematurely discontinued study drug due to adverse reactions was 2% for KALYDECO-treated patients and 5% for placebo-treated patients. Serious adverse reactions, whether considered drug-related or not by the investigators, that occurred more frequently in KALYDECO-treated patients, included abdominal pain, increased hepatic enzymes, and hypoglycemia.
The most common adverse reactions in the 221 patients treated with KALYDECO were headache (17%), upper respiratory tract infection (16%), nasal congestion (16%), nausea (10%), rash (10%), rhinitis (6%), dizziness (5%), arthralgia (5%), and bacteria in sputum (5%).
The incidence of adverse reactions below is based upon two double-blind, placebo-controlled, 48-week clinical trials (Trials 1 and 2) in a total of 213 patients with CF ages 6 to 53 who have a G551D mutation in the CFTR gene and who were treated with KALYDECO 150 mg orally or placebo twice daily.
Table 2 shows adverse reactions occurring in ≥8% of KALYDECO-treated patients with CF who have a G551D mutation in the CFTR gene that also occurred at a higher rate than in the placebo-treated patients in the two double-blind, placebo-controlled trials.
Table 2:
Incidence of Adverse Drug Reactions in ≥8% of KALYDECO-Treated Patients with a G551D Mutation in the CFTR Gene and Greater than Placebo in 2 Placebo-Controlled Phase 3 Clinical Trials of 48 Weeks Duration Adverse Reaction (Preferred Term) Incidence: Pooled 48-Week Trials KALYDECO N=109 n (%) Placebo N=104 n (%) Headache 26 (24) 17 (16) Oropharyngeal pain 24 (22) 19 (18) Upper respiratory tract infection 24 (22) 14 (14) Nasal congestion 22 (20) 16 (15) Abdominal pain 17 (16) 13 (13) Nasopharyngitis 16 (15) 12 (12) Diarrhea 14 (13) 10 (10) Rash 14 (13) 7 (7) Nausea 13 (12) 11 (11) Dizziness 10 (9) 1 (1) Adverse reactions in the 48-week clinical trials that occurred in the KALYDECO group at a frequency of 4 to 7% where rates exceeded that in the placebo group include: Infections and infestations : rhinitis Investigations: aspartate aminotransferase increased, bacteria in sputum, blood glucose increased, hepatic enzyme increased Musculoskeletal and connective tissue disorders: arthralgia, musculoskeletal chest pain, myalgia Nervous system disorders: sinus headache Respiratory, thoracic and mediastinal disorders: pharyngeal erythema, pleuritic pain, sinus congestion, wheezing Skin and subcutaneous tissue disorders: acne The safety profile for the CF patients enrolled in the other clinical trials (Trials 3-8) was similar to that observed in the 48-week, placebo-controlled trials (Trials 1 and 2).
Laboratory Abnormalities Transaminase Elevations:
In Trials 1, 2, and 3 the incidence of maximum transaminase (ALT or AST) >8, >5, or >3 × ULN was 2%, 2%, and 6% in KALYDECO-treated patients and 2%, 2%, and 8% in placebo-treated patients, respectively. 5%) on KALYDECO permanently discontinued treatment for elevated transaminases, all >8 × ULN.
Two patients treated with KALYDECO were reported to have serious adverse reactions of elevated liver transaminases compared to none on placebo. 1) ]. 7% (5/34). All 5 patients had maximum ALT or AST levels >8 × ULN, which returned to baseline levels following interruption of KALYDECO dosing.
Transaminase elevations were more common in patients who had abnormal transaminases at baseline. 1) ]. 1% (2/18), respectively. 1%) had elevated ALT of >3 to ≤5 × ULN. In the cohort of patients aged 4 months to less than 6 months (N=6), no patients had elevated ALT or AST (>3× ULN).
1) ] . 2 Postmarketing Experience The following adverse reactions have been identified during post approval use of KALYDECO or drugs containing the same or similar active ingredient as KALYDECO. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.
Immune System Disorders : anaphylaxis Nervous System Disorders : intracranial hypertension Psychiatric Disorders: anxiety, depression, suicidal ideation and behavior, insomnia
Baseline and follow-up examinations are recommended in pediatric patients initiating KALYDECO treatment. 1 Transaminase (ALT or AST) Elevations Elevated transaminases have been reported in patients with CF receiving KALYDECO. ALT and AST should be assessed prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter.
For patients with a history of transaminase elevations, consider more frequent monitoring of liver function tests. Patients who develop increased transaminase levels should be closely monitored until the abnormalities resolve. Dosing should be interrupted in patients with ALT or AST of greater than 5 times the upper limit of normal (ULN).
6) ] . 2) ] . If signs or symptoms of serious hypersensitivity reactions develop during treatment, discontinue KALYDECO and institute appropriate therapy. Consider the benefits and risks for the individual patient to determine whether to resume treatment with KALYDECO .
2) ] . Clinical manifestations of IH include headache, blurred vision, diplopia, and potential vision loss; papilledema can be found on fundoscopy. If an unusual headache or visual disturbances occur during treatment, and IH is suspected, interrupt KALYDECO and refer for prompt medical evaluation.
Consider the benefits and risks for the individual patient to determine whether to resume treatment with KALYDECO. Patients should be monitored until IH resolution and for recurrence. Patients with elevated vitamin A levels may be at increased risk.
2) ] . The events were reported in adult and pediatric patients with and without a previous history of neuropsychiatric symptoms. Symptoms may occur within the first three months of treatment initiation. Assess patients for baseline neuropsychiatric symptoms and monitor for new or worsening symptoms of anxiety, depression, suicidal ideation or behavior, or sleep disturbances.
Consider the benefits and risks for the individual patient to determine if therapy with KALYDECO should be interrupted at the occurrence of neuropsychiatric symptoms and whether to resume therapy with symptom improvement. 5 Concomitant Use with CYP3A Inducers Use of KALYDECO with strong CYP3A inducers, such as rifampin, substantially decreases the exposure of ivacaftor, which may reduce the therapeutic effectiveness of KALYDECO.
, rifampin, St. 3) ]. 6 Cataracts Cases of non-congenital lens opacities/cataracts have been reported in pediatric patients treated with KALYDECO. Although other risk factors were present in some cases (such as corticosteroid use and/or exposure to radiation), a possible risk attributable to KALYDECO cannot be excluded.
Baseline and follow-up ophthalmological examinations are recommended in pediatric patients initiating KALYDECO treatment.