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Kalydeco

Last reviewed on RxList: 5/30/2017
Kalydeco Side Effects Center

Medical Editor: John P. Cunha, DO, FACOEP

Last reviewed on RxList 05/25/2017

Kalydeco (ivacaftor) film-coated tablets are a CFTR potentiator indicated for the treatment of a rare form of cystic fibrosis (CF) in patients ages 6 years and older who have the specific G551D mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene. Common side effects of Kalydeco include:

  • upper respiratory tract infection,
  • headache,
  • stomach pain,
  • nausea,
  • rash,
  • acne,
  • diarrhea,
  • dizziness,
  • joint or muscle pain, or
  • cold symptoms (stuffy nose, sneezing, sore throat).

Serious side effects of Kalydeco include:

  • liver problems,
  • severe stomach pain,
  • chest pain,
  • wheezing,
  • shortness of breath,
  • itching,
  • loss of appetite,
  • dark urine,
  • clay-colored stools,
  • jaundice (yellowing of the skin or eyes), or
  • low blood sugar (headache, hunger, weakness, sweating, confusion, irritability, dizziness, fast heart rate, or feeling jittery).

In adults and pediatric patients age 6 years and older, one 150 mg Kalydeco tablet should be given orally every 12 hours with fat-containing food. Dosage of Kalydeco should be reduced when co-administered with moderate and severe hepatic impairment. Serious side effects include liver problems. There are no adequate and well-controlled studies of Kalydeco in pregnant women. Kalydeco should be used during pregnancy only if clearly needed. Caution should be exercised when Kalydeco is administered to a nursing woman. Consult your doctor before breastfeeding The safety and efficacy of Kalydeco with CF patients younger than age 6 years have not been established.

Our Kalydeco Side Effects Drug Center provides a comprehensive view of available drug information on the potential side effects when taking this medication.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Kalydeco Consumer Information

Get emergency medical help if you have any of these signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Call your doctor at once if you have a serious side effect such as:

  • severe stomach pain;
  • chest pain, wheezing, feeling short of breath;
  • nausea, upper stomach pain, itching, loss of appetite, dark urine, clay-colored stools, jaundice (yellowing of the skin or eyes); or
  • low blood sugar (headache, hunger, weakness, sweating, confusion, irritability, dizziness, fast heart rate, or feeling jittery).

Less serious side effects may include:

  • headache;
  • nausea, diarrhea, stomach pain;
  • dizziness;
  • acne or mild rash;
  • joint or muscle pain; or
  • cold symptoms such as stuffy nose, sneezing, sore throat.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Read the entire detailed patient monograph for Kalydeco (Ivacaftor)

Kalydeco Professional Information

SIDE EFFECTS

The following adverse reaction is discussed in greater detail in other sections of the label:

  • Transaminase Elevations [see WARNINGS AND PRECAUTIONS]

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 and Clinical Studies]:

  • 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.

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 from 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

  Incidence: Pooled 48-Week Trials
Adverse Reaction
(Preferred Term)
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 3-6) 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 x ULN was 2%, 2%, and 6% in KALYDECO-treated patients and 2%, 2%, and 8% in placebo-treated patients, respectively. Two patients (2%) on placebo and 1 patient (0.5%) on KALYDECO permanently discontinued treatment for elevated transaminases, all >8 x ULN. Two patients treated with KALYDECO were reported to have serious adverse reactions of elevated liver transaminases compared to none on placebo. Transaminase elevations were more common in patients with a history of transaminase elevations [see WARNINGS AND PRECAUTIONS].

During the 24-week, open-label, clinical trial in 34 patients ages 2 to less than 6 years (Trial 6), where patients received either 50 mg (less than 14 kg) or 75 mg (14 kg or greater) ivacaftor granules twice daily, the incidence of patients experiencing transaminase elevations (ALT or AST) >3 x ULN was 14.7% (5/34). All 5 patients had maximum ALT or AST levels >8 x ULN, which returned to baseline levels following interruption of KALYDECO dosing. Transaminase elevations were more common in patients who had abnormal transaminases at baseline. KALYDECO was permanently discontinued in one patient [see WARNINGS AND PRECAUTIONS].

Read the entire FDA prescribing information for Kalydeco (Ivacaftor)

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© Kalydeco Patient Information is supplied by Cerner Multum, Inc. and Kalydeco Consumer information is supplied by First Databank, Inc., used under license and subject to their respective copyrights.

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