For the use of Registered Medical Practitioners (Specialist in Medicine) or a Hospital or a Laboratory only

Infusion Protocol
Indication: Myozyme is indicated for long-term enzyme
replacement therapy (ERT) in patients with a confirmed
diagnosis of Pompe disease (acid α-glucosidase deficiency)
(Alglucosidase alfa for injection 50mg)

Myozyme Dosage: Reconstitution and Dilution

 Dose - 20 mg/kg every 2 weeks as an IV infusion  Allow vials to reach room temperature (30 minutes)
1
 Total volume of Infusion determined by the patient’s weight  Reconstitute each vial with 10 ml Sterile water

 Each vial contains 50 mg Myozyme ( lyophilized powder)

 Slow drop wise injection down the inside wall of the vial (not directly onto the lyophilized cake)
 Round up the number if no. of vials in fraction 2
 Tilt and roll vial gently (Don’t invert and shake)

 Check for any particulate matter or discoloration

Patient weight in kg × 20 3
 Protect from direct sunlight
mg/kg = number of mg of 4 Vials
Myozyme required for patient
4  Slowly withdraw the reconstituted solution from vial
 Dilution in 0.9% NaCl, refer dosing guide for final volume

10 kg patient weight × 20  Prepare the infusion and label it

5
mg/kg = 200 mg patient dose  Use immediately ( if not, then it can be stored up to 24 hours in 2° to 8° C)
200mg/ 50mg vial = 4 vials
6  Follow the infusion protocol and monitor.

Administration Monitoring
 Don’t infuse in same IV line for other products  Infusion should be given by experienced medical personnel

 Check for Vitals

 Starting Initial infusion rate not more than 1mg/kg/hr
 Check for infusion reactions during and up to 2 hours post infusion

 Total time of infusion around 4 hours  Infusion reaction occurs generally with Higher rates

 Every 30 minutes, rate can be increased by 2 mg/kg/hr Refer the chart for final Volume to be used
 If patient can tolerate, go up to 7 mg/kg/hr
Recommended Infusion Volumes and Rates
Step 1 Step 2 Step 3 Step 4
Patient Weight Total Infusion
1 mg/kg/hr 3 mg/kg/hr 5 mg/kg/hr 7 mg/kg/hr
Range (kg) Volume (mL)
 Decrease rate or stop the infusion if infusion reaction (mLhr) (mL/hr) (mL/hr) (mL/hr)

1.25 - 10 50 3 8 13 18
Safety profile 10.1 - 20 100 5 15 25 35

 Anaphylaxis or hypersensitivity may occur in <1 % but Emergency 20.1 - 30 150 8 23 38 53

anaphylaxis management including cardiorespiratory resuscitation should 30.1 - 35 200 10 30 50 70
be accessible always.
35.1 - 50 250 13 38 63 88
 Patients who have experienced IARs (infusion associated reactions) should 50.1 - 60 300 15 45 75 105
be treated with caution when re-administering the drug
60.1 - 100 500 25 75 125 175
 Mild and transient IARs may not require medical treatment or 100.1 - 120 600 30 90 150 210
discontinuation of the infusion. Reduction of the infusion rate, temporary
interruption of the infusion, or pre-treatment, generally with oral 120.1 - 140 700 35 105 175 245
antihistamine and/or antipyretics and/or corticosteroids, has effectively 140.1 - 160 800 40 120 200 280
managed most reactions.
160.1 - 180 900 45 135 225 315
 IARs may occur at any time during the infusion of Myozyme or generally 180.1 - 200 1000 50 150 250 350
up to 2 hours after and are more likely with higher infusion rates.

 For reporting adverse events: PV.india@sanofi.com

Abridged Prescribing Information For the use only of a Registered Medical Practitioners (Specialist in Medicine) or a Hospital or a Laboratory
MYOZYME®
Alglucosidase alfa for injection (r-DNA origin) 50mg Lyophilized Powder for concentrate for solution for infusion
MAT-IN-2302721-0.1-12/23

COMPOSITION Immunomodulation: Immunogenicity data from clinical trials and published literature in CRIM-negative infantile-onset patients (IOPD) suggests that the administration of immune tolerance induction (ITI)
One vial contains 50 mg of alglucosidase alfa) After reconstitution, the solution contains 5 mg of alglucosidase alfa per ml and after dilution, the concentration varies from 0.5 mg to 4 mg/ml. regimen given to alglucosidase alfa naive patients (prophylactic ITI) may be effective in preventing or reducing the development of High Sustained Antibody Titer (HSAT) against alglucosidase alfa. Patients with
Pompe disease are at risk of respiratory infections due to the progressive effects of the disease on the respiratory muscles. Fatal and life-threatening respiratory infections have been observed in some of these
Alglucosidase alfa is a recombinant form of human acid α-glucosidase and is produced in Chinese hamster ovary cells (CHO) by recombinant DNA technology. patients
THERAPEUTIC INDICATION: Myozyme is indicated for long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid α-glucosidase deficiency). Pregnancy: Myozyme should not be used during pregnancy unless clearly necessary.
DOSAGE & ADMINISTRATION: Myozyme treatment should be supervised by a physician experienced in the management of patients with Pompe disease or other inherited metabolic or neuromuscular Lactation: Alglucosidase alfa may be excreted in breast milk. Because there are no data available on effects in neonates exposed to alglucosidase alfa via breast milk, it is recommended to stop breast-feeding
diseases. The recommended dose regimen of alglucosidase alfa is 20 mg/kg of body weight administered as intravenous infusion once every 2 weeks. Myozyme should be administered as an intravenous when Myozyme is used.
infusion. Myozyme has to be reconstituted with water for injections, then diluted with sodium chloride 9 mg/ml (0.9%) solution for injection and then administered by intravenous infusion using aseptic
techniques. A 0.2 micron low protein binding in-line filter should be used for administration. Infusions should be administered incrementally. It is recommended that the infusion begin at an initial rate of 1 ADVERSE REACTIONS :Very Common: Tachycardia, Tachypnoea, Cough, Vomiting, Urticaria, Rash, Pyrexia, Oxygen saturation decreased. Common: Agitation, Hypersensitivity, Tremor, Dizziness, Paraesthesia,
mg/kg/h and be gradually increased by 2 mg/kg/h every 30 minutes if there are no signs of infusion associated reactions (IARs) until a maximum rate of 7 mg/kg/h is reached. Headache, Cyanosis, Hypertension, Pallor, Flushing, Throat tightness, Retching , Nausea, Diarrhoea,, Vomiting, Nausea, Erythema, Rash maculopapular, Rash macular, Rash popular, Pruritus, Urticaria, Rash popular,
Hyperhidrosis, Muscle spasms, Muscle twitching, Myalgia, Pyrexia, Chest discomfort , Peripheral oedema, Local swelling, Fatigue, Feeling hot, Blood pressure increased. Not Known: Agitation, Restlessness, Tremor,
SAFETY RELATED INFORMATION Headache, Conjunctivitis, Cardiac arrest, Bradycardia, Tachycardia, Cyanosis, Hypertension, Hypotension, Vasoconstriction, Pallor, Respiratory arrest, Apnea, Respiratory distress, Bronchospasm, Wheezing,
Contraindications: Life threatening hypersensitivity (anaphylactic reaction) to the active substance or to any of the excipients, when rechallenge was unsuccessful. Pharyngeal oedema, Dyspnoea, Tachypnoea, Throat tightness, Stridor, Cough, Abdominal pain, Retching, Periorbital edema, Livedo reticularis, Lacrimation increased, Rash, Erythema, Hyperhidrosis,
Warnings & Precautions: Arthralgia, Nephrotic syndrome, Proteinuria, Chest pain, Face edema, Feeling hot, Pyrexia, Chills, Chest discomfort, Irritability, Peripheral coldness, Infusion site pain , Infusion site reaction, Oxygen saturation
decreased, Heart rate increased.
Hypersensitivity/Anaphylactic reactions: Serious and life-threatening anaphylactic reactions, including anaphylactic shock, have been reported in infantile- and late-onset patients during Myozyme infusions.
If severe hypersensitivity or anaphylactic reactions occur, immediate discontinuation of Myozyme infusion should be considered and appropriate medical treatment should be initiated The current medical For full prescribing information please contact: Sanofi Healthcare India Pvt Ltd, Sanofi House, CT Survey No 117-B, L& T Business Park, Saki Vihar Road, Powai, Mumbai-400072
standards for emergency treatment of anaphylactic reactions are to be observed.
Source: EU summary of Product Characteristics (SmPC) for CCDS V9 dated 25-April-2019 Date: April 2022.
Infusion Associated Reactions: Approximately half of the patients treated with Myozyme in infantile-onset clinical studies and 28% of the patients treated with Myozyme in a late-onset clinical study
developed infusion associated reactions (IARs. IARs are defined as any related adverse event occurring during the infusion or during the hours following infusion. Patients with an acute illness (e.g. pneumonia,
sepsis) at the time of Myozyme infusion appear to be at greater risk for IARs. Patients with advanced Pompe disease may have compromised cardiac and respiratory function, which may predispose them to
a higher risk of severe complications from infusion associated reactions. Therefore, these patients should be monitored more closely during administration of Myozyme
Immunogenicity: Patients who experience hypersensitivity reactions may also be tested for IgE antibodies to alglucosidase alfa and other mediators of anaphylaxis. Patients who develop IgE antibodies to
alglucosidase alfa appear to be at a higher risk for the occurrence of IARs when Myozyme is re-administered
Immune-mediated reactions: Severe cutaneous reactions, possibly immune mediated, have been reported with alglucosidase alfa, including ulcerative and necrotizing skin lesions. Nephrotic syndrome was
observed in a few patients with Pompe disease treated with alglucosidase alfa and who had high IgG antibody titres (≥ 102,400) Patients should be monitored for signs and symptoms of systemic
immune-mediated reactions involving skin and other organs while receiving alglucosidase alfa.

Sanofi Healthcare India Pvt. Ltd., Sanofi House, CT Survey No. 117-B,
Adverse events should be reported at : PV.India@sanofi.com L&T Business Park, Saki Vihar Road, Powai, Mumbai – 400 072