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Dosing and administration of POMBILITI + OPFOLDA1,2

POMBILITI must be administered in combination with OPFOLDA1,2

Treatment Day Schedule1,2

Timeline showing when to start and stop fasting, when to take OPFOLDA, and when POMBILITI infusion begins and ends-graphic

ERT, enzyme replacement therapy.

POMBILITI + OPFOLDA dosing is based on actual body weight1,2

  • Recommended dosage for POMBILITI: 20 mg/kg of actual body weight administered intravenously every other week1
    Confirm the dose using the Dosing Calculator below
  • Recommended dosage for OPFOLDA2*:
    • For patients weighing ≥50 kg, 260 mg (4 capsules of 65 mg) administered orally every other week
    • For patients weighing ≥40 kg to <50 kg, 195 mg (3 capsules of 65 mg) administered orally every other week

* For recommended dosage in patients with renal impairment, see Section 2.3 of the OPFOLDA full Prescribing Information, or see the OPFOLDA recommended dosage section below.

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Start POMBILITI + OPFOLDA 2 weeks after the last ERT dose.1,2

Pretreatment for POMBILITI

Prior to POMBILITI administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids, especially if premedication was used with the patient’s previous ERT.1

Capsule-icon

Taking OPFOLDA1,2

  • OPFOLDA should be taken approximately 1 hour before intravenous infusion of POMBILITI
  • OPFOLDA capsules should be swallowed whole after fasting for 2 hours. Unsweetened beverages (eg, water, tea, or coffee with no cream, sugar, or sweeteners) are permitted while fasting. Patients should not consume other beverages or food for at least 2 hours prior to and 2 hours after administration of OPFOLDA. Patients can stop fasting 2 hours after taking OPFOLDA
  • If the OPFOLDA dosage is missed, POMBILITI should not be administered and treatment should be rescheduled at least 24 hours after OPFOLDA was last taken

For female patients:

  • POMBILITI in combination with OPFOLDA is contraindicated in pregnancy
  • Verify the pregnancy status in females of reproductive potential prior to initiating treatment with OPFOLDA in combination with POMBILITI
  • Advise females of reproductive potential to use effective contraception during treatment with OPFOLDA in combination with POMBILITI and for at least 60 days after the last dose
IV bag-icon

Starting POMBILITI1

  • POMBILITI must be reconstituted and diluted prior to use
  • The POMBILITI infusion should begin approximately 1 hour after oral administration of OPFOLDA
  • If the POMBILITI infusion cannot be started within 3 hours of oral administration of OPFOLDA, reschedule POMBILITI and OPFOLDA at least 24 hours after OPFOLDA was last taken
  • If POMBILITI in combination with OPFOLDA are both missed, restart treatment as soon as possible

POMBILITI recommended dosage1

Calculating the POMBILITI dose

Recommended dosage for POMBILITI is 20 mg/kg of actual body weight administered every other week. Patients should be weighed at every visit to calculate the correct dose.

Determining the number of vials to be reconstituted

Step 1
IV bag-icon
  • Patient's actual body weight (kg)
  • X
  • Recommended dosage (20 mg/kg)
  • =
  • Patient's dose (mg)
Step 2
Two vials-icon
  • Patient's dose (mg)
  • ÷
  • 105 (mg/vial)
  • =
  • Number of vials to reconstitute

If the number of vials include a fraction, round up to the next whole number.
Never round down.

Step 3
Piece of paper with folded corner-icon
  • Record the volume to be extracted from final vial
  • Fraction of final bottle used (from Step 2)
  • X
  • 7.0 mL (extraction volume per vial)
  • =
  • Volume to extract from final vial

Note that each vial requires 7.2 mL of sterile water to reconstitute, but should only yield 7.0 mL of solution for the infusion bag.

Example: A 75 kg patient dosed at 20 mg/kg

  • Step 1: Patient's dose (mg), calculated as 75 kg x 20 mg/kg = 1500 mg total dose
  • Step 2: Number of vials to reconstitute, calculated as 1500 divided by 105 mg/vial = 14.29 and round up to 15 vials
  • Step 3: Extraction volume per vial is 7.0 mL. 14.29 vials x 7.0 mL = 100.0 mL total extraction volume (2 mL from final vial)

Dosing Calculator

Use this calculator to confirm your calculations for patients weighing between 40 and 140 kg (88 to 309 lbs). For more information about dosing and administration, please refer to the Full Prescribing Information for POMBILITI.

By using this tool, you agree to the following: This dosing calculator is provided “as is” and is not intended to be used as a substitute for clinical judgment. All calculations should be confirmed before dosing. This guide is intended for use only by healthcare professionals. Amicus Therapeutics makes no claims as to the accuracy of the information and shall not be liable for any decisions made or actions taken in reliance on this information.

In calculating the dose, the number of vials is always rounded up, not down. A partial dose is withdrawn from the final vial.

This calculator is intended for US HCPs only and should not be used by HCPs outside the US.

OPFOLDA recommended dosage

Determining the OPFOLDA dose1,2

Only use OPFOLDA capsules in conjunction with POMBILITI

OPFOLDA is administered orally every other week. The recommended dosage is based on actual body weight:

  • For patients weighing ≥50 kg, the recommended dose is 260 mg (4 capsules of 65 mg)
  • For patients weighing ≥40 kg to <50 kg, the recommended dose is 195 mg (3 capsules of 65 mg)

Recommended OPFOLDA dosage in patients with moderate or severe renal impairment§:

  • ≥50 kg = 195 mg (3 capsules of 65 mg)
  • ≥40 kg to <50 kg = 130 mg (2 capsules of 65 mg)

For patients with mild renal impairment, the recommended OPFOLDA dosage is the same as for patients with normal renal function.

§ Renal function is classified by creatinine clearance (CLcr) based on the Cockcroft-Gault equation. Mild renal impairment is CLcr 60-89 mL/min, moderate renal impairment is CLcr 30-59 mL/min, and severe renal impairment is CLcr 15-29 mL/min.

POMBILITI administration1

Administering the infusion

  • Infusion of POMBILITI should start approximately 1 hour after oral administration of OPFOLDA
  • The infusion will take approximately 4 hours
  • If the infusion cannot be started within 3 hours of administration of OPFOLDA, reschedule treatment to at least 24 hours after OPFOLDA was last taken. If both medications are missed, restart treatment as soon as possible
  • The infusion solution should be administered at room temperature
  • The initial recommended infusion rate is 1 mg per kg per hour for the first 30 minutes
  • The infusion rate may be gradually increased by 2 mg/kg per hour every 30 minutes, if there are no signs of hypersensitivity or infusion-associated reactions (IARs), until a maximum rate of 7 mg/kg/hour is reached; then, maintain the infusion rate at 7 mg/kg/hour until the infusion is complete
The infusion rate may be slowed or temporarily stopped in the event of mild to moderate IARs. In the event of severe allergic reaction or anaphylaxis, immediately stop the infusion and initiate appropriate medical treatment.

POMBILITI reconstitution and dilution1

Preparing POMBILITI for infusion

POMBILITI is supplied as a sterile, white to slightly yellowish, lyophilized powder with a cake-like appearance.

Items needed for reconstitution and dilution

  • POMBILITI 105-mg vials
  • Sterile water for injection at room temperature
  • Sodium chloride 9 mg/mL (0.9%) solution for injection at room temperature
    • Choose a bag size based on the patient’s actual body weight

Reconstituting the lyophilized powder

Step 1
POMBILITI lyophilized powder-image

Remove vials from the refrigerator and set aside for approximately 30 minutes to allow them to come to room temperature.

Step 2
Reconstituting the vial with sterile water for injection-image

Reconstitute each vial by slowly adding 7.2 mL of sterile water for injection down the inside wall of each vial to avoid foaming. Avoid forceful impact of sterile water for injection on the lyophilized powder and avoid foaming.

Step 3
Rolling and tilting the vial to dissolve-image

Roll and tilt each vial to allow the lyophilized powder to dissolve completely. This typically takes 2 minutes. Do not invert, swirl, or shake.

Step 4
Reconstituted vial of POMBILITI-image

Acceptable:
Clear to opalescent, colorless to yellowish, essentially particle free.

Visually inspect the reconstituted vials for particulate matter and discoloration. The reconstituted solution appears as a clear to opalescent, colorless to yellowish solution, essentially particle free.

  • Discard if foreign matter is observed or the solution is discolored
  • Each reconstituted vial should yield a concentration of 15 mg/mL

If the reconstituted POMBILITI vials are not used immediately, store refrigerated at 2 °C to 8 °C (36 °F to 46 °F) for up to 24 hours. Do not freeze.

Step 5
Repeat steps-icon

Repeat the above steps for the number of vials needed for reconstitution.

Diluting the solution

Step 1
Removing airspace within bag of sodium chloride injection-image

Remove airspace within a bag of 0.9% sodium chloride injection. Remove an equal volume of 0.9% sodium chloride injection that will be replaced by the total volume (mL) of reconstituted POMBILITI.

Step 2
Withdrawing reconstituted solution from vial-image

Slowly withdraw 7 mL of reconstituted solution from each of the vials until the patient’s dose is obtained. Discard any remaining reconstituted solution in the last vial.

Step 3
Injecting reconstituted POMBILITI into infusion bag-image

Slowly inject the reconstituted POMBILITI directly into the infusion bag.

Step 4
Inverting the infusion bag-image

To prevent foaming, gently invert the infusion bag to mix the solution.

  • Avoid vigorous shaking or agitation
  • Do not use a pneumatic tube to transport the infusion bag
  • POMBILITI should be administered as stated in the Prescribing Information
  • An intravenous administration set should be used with an inline, low protein-binding, 0.2-micron filter. If the intravenous line blocks during infusion, change the filter
  • If the diluted solution is not administered immediately, store refrigerated at 2 °C to 8 °C (36 °F to 46 °F) for up to 16 hours. Storage at room temperature is not recommended. Do not freeze
  • Other medicines should not be infused in the same intravenous line as the diluted POMBILITI solution

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POMBILITI storage

Storage for POMBILITI before reconstitution1

POMBILITI is supplied as a sterile, nonpyrogenic, white to slightly yellowish lyophilized cake or powder for reconstitution with sterile water for injection to yield a concentration of 15 mg/mL, then further diluted with 0.9% sodium chloride for injection for intravenous infusion. Single-use vials are available in 105-mg dosage only.

Thermometer-icon
Store at 2 °C to 8 °C
(36 °F to 46 °F)
Freezing temperatures thermometer-icon
Do not freeze
Two vials-icon
Store in the original packaging to protect from light

Storage for reconstituted and diluted solution

If the solution is not administered immediately, it must be refrigerated.

Do not freeze the reconstituted vial or the diluted POMBILITI solution in the infusion bag.

‡After removal of the diluted solution from the refrigerator:

  • Completely infuse within 6 hours
  • Do not restore in the refrigerator

OPFOLDA storage

Storage for OPFOLDA capsules2

  • Do not use if inner seal is missing or broken
  • Keep out of reach of children
  • Store in the original container or equivalent to protect from light
Water glass and capsule-icon

OPFOLDA is supplied as a hard gelatin capsule containing 65 mg of miglustat

Thermometer-icon

Store at 20 °C to 25 °C (68 °F to 77 °F). Excursions are permitted between 15 °C and 30 °F (59 °F and 86 °F)

Help patients get started

References:

  1.  POMBILITI. Prescribing information. Amicus Therapeutics US, LLC; 2024.
  2.  OPFOLDA. Prescribing information. Amicus Therapeutics US, LLC; 2024.