This site is intended for
US Healthcare Professionals only.

Dosing and Administration
ORAL GRANULES AND TABLETS

Recommended Dose for ORKAMBI
 

  • ORKAMBI oral granules and tablets should be taken with fat-containing food1
  • Patients should continue taking all their prescribed CF therapies with ORKAMBI1

 

DOSAGE ADJUSTMENTS FOR ORKAMBI
MISSED DOSE¹
  • If ≤6 hours have passed: Advise patient to take the dose with fat-containing food
  • If >6 hours have passed: Advise patient to skip that dose and resume the normal schedule for the following dose. A double dose should not be taken to make up for the forgotten dose
USE OF ORKAMBI IN SPECIFIC PATIENT POPULATIONS

Pregnancy1

  • There are limited and incomplete human data from clinical trials and postmarketing reports on use of ORKAMBI or its individual components, lumacaftor or ivacaftor, in pregnant women to inform a drug-associated risk

Lactation1

  • There is no information regarding the presence of lumacaftor or ivacaftor in human milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for ORKAMBI and any potential adverse effects on the breastfed child from ORKAMBI or from the underlying maternal condition

Females and Males of Reproductive Potential1

  • ORKAMBI may decrease hormonal contraceptive exposure, reducing the effectiveness. Hormonal contraceptives, including oral, injectable, transdermal, and implantable, should not be relied upon as an effective method of contraception when co-administered with ORKAMBI

Pediatric Use1

  • The safety and efficacy of ORKAMBI in patients with CF younger than age 2 years have not been established. Cases of non-congenital lens opacities have been reported in pediatric patients treated with ORKAMBI and ivacaftor, a component of ORKAMBI. Although other risk factors were present in some cases (such as corticosteroid use and exposure to radiation), a possible risk attributable to ivacaftor cannot be excluded

Geriatric Use1

  • Clinical trials of ORKAMBI did not include sufficient numbers of patients 65 years of age and over to determine whether they respond differently from younger patients

Hepatic Impairment1

  • No dose adjustment is necessary for patients with mild hepatic impairment (Child-Pugh Class A). A dose reduction is recommended for patients with moderate hepatic impairment (Child-Pugh Class B). Studies have not been conducted in patients with severe hepatic impairment (Child-Pugh Class C), but exposure is expected to be higher than in patients with moderate hepatic impairment. A dose reduction is recommended for patients with severe hepatic impairment. Use with caution in patients with severe hepatic impairment after weighing the risks and benefits of treatment. See dose chart above for recommended dose adjustments

Renal Impairment1

  • ORKAMBI has not been studied in patients with mild, moderate, or severe renal impairment or in patients with end-stage renal disease
  • No dose adjustment is necessary for patients with mild to moderate renal impairment. Caution is recommended while using ORKAMBI in patients with severe renal impairment (creatinine clearance ≤30 mL/min) or end-stage renal disease

Degree of Renal Impairment

ORKAMBI

Mild to Moderate

No dosage adjustment

Severe (creatine clearance ≤30 mL/min)

Caution is recommended

End-Stage Renal Disease

Caution is recommended

Patients With Severe Lung Dysfunction1

  • The Phase 3 trials (Trials 1 and 2) included 29 patients receiving ORKAMBI with percent predicted FEV1 (ppFEV1) <40 at baseline. The treatment effect in this subgroup was comparable to that observed in patients with ppFEV1 ≥40. 
  • Respiratory events (eg, chest discomfort, dyspnea, and respiration abnormal) were observed more commonly in patients during initiation of ORKAMBI compared to those who received placebo. These events have led to drug discontinuation and can be serious, particularly in patients with advanced lung disease (ppFEV1 <40). Clinical experience in patients with ppFEV1 <40 is limited, and additional monitoring of these patients is recommended during initiation of therapy

Patients After Organ Transplantation1

  • ORKAMBI has not been studied in patients with CF who have undergone organ transplantation. Use in transplanted patients is not recommended due to potential drug-drug interactions

 

ADMINISTRATION

Oral Granules: Preparation and Administration1

  • Hold the packet with cut line on top
  • Shake the packet gently to settle the ORKAMBI granules
  • Tear or cut packet open along the line
  • Carefully pour all of the ORKAMBI granules in the packet into 1 teaspoon of soft food or liquid in a small container
  • Food or liquid should be at or below room temperature
  • After mixing, caregiver should give within 1 hour
  • It is important that patients consume the entire oral granules mixture with each dose

    Not actual size.
  • Examples of soft foods or liquids include: pureed fruits, applesauce, flavored yogurt, pudding, milk, or juice

Tablets: Administration1

  • Take 2 tablets twice a day, 12 hours apart

    ORKAMBI tablets

    Not actual size.


Give Oral Granules and Tablets with Fat-Containing Food1

  • For patients taking oral granules mixed into a soft food or liquid: dose must be taken just before or after consuming fat-containing food
  • For patients taking tablets: dose must be taken with fat-containing food

Examples of Fat-Containing Foods1

  • Eggs
  • Nuts
  • Peanut butter
  • Avocado
  • Butter
  • Cheese pizza
  • Whole-milk dairy products (eg, whole milk, cheese, and yogurt)

Palatability of ORKAMBI Oral Granules2

  • Children may find the taste of the oral granules to be bitter
  • Mixing the oral granules with soft foods or liquids that are sweet or rich, such as pudding or chocolate sauce, may help with the taste

For more information for caregivers, including tips for taking, please see the Downloadable Materials page.

ppFEV1, percent predicted forced expiratory volume in 1 second.