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Dosing and administration of POMBILITI + OPFOLDA1,2

POMBILITI™ (cipaglucosidase alfa-atga) must be administered in combination with OPFOLDA™ (miglustat).1,2

Treatment day schedule1,2

Pombiliti + Opfolda dosing timeline

POMBILITI + OPFOLDA dosing is based on actual body weight

  • Recommended dosage for POMBILITI: 20 mg/kg of actual body weight administered intravenously every other week
  • Recommended dosage for OPFOLDA:
    • 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 Prescribing Information, or see the OPFOLDA recommended dosage section below.

Switch without delay

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

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

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

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

Dosing, administration, and storage for POMBILITI

Calculating the POMBILITI dose

Recommended dosage for POMBILITI 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 reconstituted1

Step 1
  • Patient's actual body weight (kg)
  • X
  • Recommended dosage (20 mg/kg)
  • =
  • Patient's dose (mg)
Step 2
  • 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
  • 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)

Using the dosage 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.

Enter patient’s weight
OR
Weight should be between 40-200 kg (88-441 lb)

*Rounded to the nearest whole number.

Dosage
000 mg
Total 0 vials
0 vials
Withdraw
0 mL
0 vials
Withdraw
0 mL
Total dose 0 mL of reconstituted POMBILITI
to be withdrawn

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.

Questions about dosing?

Contact a Patient Education Liaison at 1-833-AMICUS-A (1-833-264-2872)

The infusion will take approximately 4 hours

Administering the infusion1,2

  • Infusion of POMBILITI should start approximately 1 hour after oral administration of OPFOLDA
  • 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.
Recommended POMBILITI infusion volumes and rates by weight1
Patient
Weight
Range
(kg) 		Total
Infusion
Volume
(mL)
Step 1
1 mg/kg/hr
Step 2
3 mg/kg/hr
Step 3
5 mg/kg/hr
Step 4
7 mg/kg/hr
Infusion rate in mL/hr
40–50 250 13 38 63 88
50.1–60 300 15 45 75 105
60.1–100 500 25 75 125 175
100.1–120 600 30 90 150 210
120.1–140 700 35 105 175 245

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 dilution1

  • 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 body weight

Reconstituting the lyophilized powder1

Step 1

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

Step 2

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

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

Step 4

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

Diluting the solution1

Step 1

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

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

Slowly inject the reconstituted POMBILITI directly into the infusion bag.

Step 4

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

Questions about the reconstitution or dilution process?

Contact a Patient Education Liaison at 1-833-AMICUS-A (1-833-264-2872)

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.

Store at 2 °C to 8 °C
(36 °F to 46 °F)
Do not freeze
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.

Infusion Preparation Infusion-use stability
Refrigerated storage
2 °C to 8 °C
(36 °F to 46 °F)
Room temperature storage
20 °C to 25 °C
(68 °F to 77 °F)
Once POMBILITI vial is reconstituted with sterile water for injection 24 hours Not recommended
Once reconstituted vial is diluted with sodium chloride 9 mg/mL (0.9%) solution for injection in the infusion bag 16 hours* Not recommended

*After removal of the diluted solution from the refrigerator:

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

Dosing and storage for OPFOLDA

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.

  • 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

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

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; 2023.
  2.  OPFOLDA. Prescribing information. Amicus Therapeutics US, LLC; 2023.