Universal reference book for medicines
Product name: MYOZYME

Active substance: alglucosidase alfa

Type: The drug for the treatment of hereditary enzymatic insufficiency

Manufacturer: GENZYME EUROPE (Netherlands) manufactured by GENZYME IRELAND Limited (Ireland)
Composition, form of production and packaging
Lyophilizate for the preparation of a concentrate for the preparation of a solution for infusions
in the form of a compact mass or powder of white or almost white color.
The reconstituted solution is clear or slightly opalescent, colorless or light yellow, white threads and semitransparent fibers are possible.
1 f.

alglucosidase alpha 50 mg (+2.5 mg excess filling)

Auxiliary substances: mannitol 200 mg (+10 mg excess filling), polysorbate 80 0.5 mg, sodium hydrogen phosphate heptahydrate 9.4 mg (+0.5 mg excess filling), sodium dihydrogen phosphate monohydrate 29.7 mg (+0.5 mg excess filling).

50 mg - glass bottles with a capacity of 20 ml (1) - packs of cardboard.

50 mg - bottles of glass with a capacity of 20 ml (10) - packs of cardboard.

50 mg - bottles of glass with a capacity of 20 ml (25) - packs of cardboard.


Description of the drug approved by the manufacturer for the printed edition of 2014.


Alglucosidase alpha is a recombinant form of human acidic a-glucosidase and is produced by recombinant DNA technology using the Chinese hamster ovary cell culture (CHO line).

Pompe disease is a rare, progressive metabolic myopathy leading to death, characterized by a deficiency of lysosomal hydrolase, acid alglucosidase alpha (rchcag).

It was found that Mayozyme replenishes the activity of lysosomal rchag, which leads to the stabilization or restoration of cardiac and skeletal muscle function (including the respiratory muscles), but due to the size of the molecule and the blood-brain barrier, penetration into the CNS appears unlikely.

The results of a clinical study of patients under 6 months of age with infantile Pompe disease showed prolonged survival of patients receiving Mayoim at a dose of 20 mg / kg or 40 mg / kg.
compared with the historical sample of patients who were not treated.
In patients receiving Mayoim, there was a greater survival and improvement of echocardiographic indices of cardiomyopathy, which was measured by a decrease in the mass of the left ventricle (LVH).

Analysis of efficacy between the groups receiving Mayozyme at a dose of 20 mg / kg or 40 mg / kg showed no significant differences, with respect to survival, survival without invasive artificial ventilation, survival without any artificial ventilation, reducing LVH, normalizing growth parameters and achieving stages of development of motor activity.
Based on these results, a dose of 20 mg / kg is recommended. 11 The vast majority of patients with infantile Pompe disease who received Mayoin treatment showed an improvement in heart function, stabilization or improvement in growth parameters, with greater variability in the effectiveness of treatment for motor and respiratory function.
In patients with infantile form of Pompe disease with established development of motor function, it was noted for its better safety and lower glycogen content in the quadriceps muscle at baseline.
It is worth noting that a large proportion of patients with better motor development results demonstrate stability or improvement in the parameters of growth (weight), while the vast majority of patients, regardless of the results of motor development or baseline indicators, observe the elimination of cardiomyopathy based on changes in LMW. In general, the evidence suggests that early diagnosis and early treatment can be critical to achieving better results in patients with Pompe disease from infancy.
Clinical studies of patients with late onset of Pompe disease also showed improvement in motor and respiratory function stabilization, but evidence of the effectiveness of Mayoise in this group of patients is still limited.


Clinical studies of patients with infantile Pompe disease demonstrated that the pharmacokinetic properties of the Mayozyme preparation are proportional to the dose and do not change with time.
After the first and sixth infusion of the Mayozyme preparation, the mean C max ranges from 178.2 to 263.7 Ојg / ml in the 20 mg / kg and 40 mg / kg groups, respectively, and the mean area under the plasma concentration-time curve (AUC) was in the range from 977.5 to 1.872.5 Ојg * h / ml. The mean plasma clearance (CL) was 21.4 ml / h / kg, and the average equilibrium volume (V ss ) was 66.2 ml / kg for both groups with small individual variability in the 15% and 11% groups, respectively . The mean T 1/2 was 2.75 hours for both groups.
There was no difference in pharmacokinetic characteristics of alglucosidase alpha in patients with late onset Pompe disease compared to patients who developed the disease in infancy.


- for long-term enzyme replacement therapy (FZT) in patients with a confirmed diagnosis of Pompe disease (insufficiency of CAG), both adults and children of all ages.

For patients with late onset of Pompe disease evidence of efficacy is limited.


Treatment with Mayozyme should be performed under the supervision of a doctor who has experience working with patients suffering from Pompe disease or other hereditary metabolic or neuromuscular diseases.

The recommended dosage regimen of alglucosidase alpha: 20 mg / kg body weight once every 2 weeks as an intravenous infusion.

Infusion should be performed with a gradual increase in the rate of administration of the drug, starting at 1 mg / kg / h and gradually increasing the dose by 2 mg / kg / h every 30 minutes, in the absence of infusion-related reactions, until a maximum rate of 7 mg / kg / h .
Special recommendations for the preparation Mayoim for each age group (children, adolescents, adults or elderly patients) are not available.
Evaluation of the safety and efficacy of Mayozyme in patients with renal or hepatic insufficiency was not carried out, and accordingly there is no recommendation for a special dosage regimen for such patients.

The patient's response to treatment should be assessed on a regular basis, based on a detailed analysis of all clinical manifestations of the disease.

Instructions for reconstitution and dilution of the drug

Before intravenous administration, Mayozyme should be reconstituted with water for injection, then diluted with 0.9% solution of sodium chloride for injection in accordance with the rules of good practice, especially with regard to asepsis.

Due to the protein nature of the preparation, particles in the form of white filaments and translucent fibers can form in the reconstituted and infusion solutions.Therefore, when administering the drug, an infusion apparatus with a built-in filter with a pore diameter of 0.2 microns should be used to bind a low molecular weight protein that removes visible particles and does not cause significant protein loss or activity.

In accordance with the individual dosage regimen for each patient, the required number of Mayozeim vial bottles is removed from the refrigerator and held at room temperature for approximately 30 minutes.
Since Mayoim does not contain preservatives, each vial of the drug is intended only for single use.

Each vial of Mayozheim 50 mg is reconstituted with 10.3 ml of water for injection.
Water for injection should be added slowly, drop by drop on the wall of the vial with the drug, gently tilting and turning the bottle, but neither turning and shaking, avoiding water directly on the lyophilizate. The reconstituted volume is 10.5 ml with an active substance content of 5 mg / ml and is a clear, colorless or slightly yellow solution that can contain protein particles in the form of thin white filaments or clear fibers. Do not use the drug in the event that during inspection, foreign particles are detected, in addition to those described above, or the color of the solution is changed. The pH of the reconstituted solution is about 6.2. After reconstitution, it is recommended that the contents of the vials be immediately diluted.

After reconstitution as described above, 1 ml of the reconstituted solution of the Mayozyme preparation in the vial contains 5 mg of alglycosidase alpha.
The reconstituted volume allows to accurately select 10.0 ml (equivalent to 50 mg of alglucosidase alpha) from each vial. Further, this volume should be diluted as follows: slowly recover the reconstituted solution from each vial to obtain a volume corresponding to the dose for the patient. The recommended final concentration of aglycosidase alpha in infusion tanks is from 0.5 mg / ml to 4 mg / ml. Remove air from the infusion container. An equivalent volume of sodium chloride solution for injection of 0.9% is recovered, which will be replaced by the reconstituted Mayozyme preparation. Slowly injected reconstituted Mayozyme directly into the solution of sodium chloride for injection 0.9%. Carefully turn over the infusion container or knead the infusion bag to mix the diluted solution. Avoid shaking or vigorous shaking of the infusion container.
Ready-made infusion solution should be administered as soon as possible after preparation.

Any unused product or waste should be disposed of in accordance with local requirements.


The table below shows the adverse drug reactions (NLR) in organ systems recorded in at least 2 patients in different clinical trials, divided according to the onset of Pompe disease and the frequency of observation as: very often (> 1/10) and often > 1/100 to <1/10), and also in order of decreasing severity of manifestations in the group with the same frequency.
Due to the small population of patients, NLR, developed in 2 patients, are classified as frequent.
In each group of reaction frequency, adverse reactions are presented in order of decreasing significance.

Abnormalities in organ systems Frequency Undesirable drug reactions

Infantile form of Pompe disease Pompe disease with late onset

On the part of the immune system is often hypersensitivity

From the side of the cardiovascular system very often Tachycardia, tides

often Cyanosis, hypertension, pallor of the Tides

* * Cardiac arrest, bradycardia, hypotension, vascular spasm

From the side of the nervous system often Tremor Dizziness, paresthesia, headache *

Mental disorders often Agitation

** Anxiety

From the sense organs ** Conjunctivitis

On the part of the respiratory system very often Rapid breathing, coughing

often Sore throat feeling

** Breath stop, apnea, respiratory distress syndrome, bronchospasm, wheezing, swelling of the pharynx, dyspnea, stridor

From the side of the digestive system very often Vomiting

often Desires for vomiting, nausea Diarrhea, vomiting, nausea *

* * Abdominal pain

Co of the skin very often Hives, rash

often Erythema, patchy-papular rash, spotted rash, papular rash, itching Hives, papular rash, itching, hyperhidrosis

** Periorbital edema, marbling of the skin, increased lacrimation, hyperhidrosis

From the musculoskeletal system often Muscle spasms, muscle twitching, myalgia

** Arthralgia

From the urinary system ** Nephrotic syndrome, proteinuria

Other disorders and complications together with administration very often Reduced oxygen saturation, pyrexia

often Increased heart rate, increased blood pressure, increased body temperature, irritability, chills Increased blood pressure, pyrexia, chest discomfort, peripheral edema, local edema, fatigue, sensation of fever

** Pain in the chest, swelling of the face, coldness of the limbs, soreness in the infusion site, reactions at the infusion site

Phenomena were registered more often in the placebo group

** NLRs, whose frequency estimates are not possible.
received additionally in nostmarketing and uncontrolled clinical trials, extended access programs. A small number of patients (<1%) during clinical trials and during commercial use of the drug suffered anaphylactic shock and / or cardiac arrest during the infusion of Mayozyme, which required resuscitation. Reactions usually developed shortly after the onset of infusion, mainly as a combination of edema, respiratory, cardiovascular, and / or skin symptoms. In several patients with moderate, severe or recurrent MI. including one patient who underwent an anaphylactic reaction, a positive result was observed for the presence of specific IgE antibodies to the Mayozyme preparation.

- hypersensitivity to any of the components of the drug.

With caution: with the repeated administration of Mayozyme to patients who developed unwanted drug reactions (NLP) during infusion or within 2 hours after (infusion reactions (IR)), especially anaphylactic.


Mayoim should not be used during pregnancy, if there is no absolute indication.
The potential risk to humans is unknown. Studies on the use of alglucosidase alpha in pregnant women have not been conducted, but studies in animals have demonstrated reproductive toxicity.
There are no clinical data on the effect of alglucosidase alpha on frogility.
Alglucosidase alpha may be excreted in breast milk. When applying the preparation, Mayozeim is recommended to stop breastfeeding, since there is no evidence of the effect of alglucosidase alpha through breast milk on newborns.

Precautions for use

Because of the possible development of serious, life-threatening, anaphylactic reactions associated with infusion with the administration of Mayozyme, it is necessary to have the readiness to immediately provide appropriate medical assistance in accordance with applicable standards, including resuscitation.
In the case of a severe anaphylactic reaction, the infusion of Mayozyme should be stopped immediately and appropriate treatment started.
Caution should be exercised when re-administering the Mayozyme drug to patients who developed NLR during infusion or within 2 hours after (IR).
Development of RI is more likely with a higher rate of infusion. With light and short-term side effects of special medical care or cessation of infusions may not be required, in most cases it is sufficient to reduce the rate or temporary discontinuation of infusions or the use of oral forms of antihistamines and / or antipyretics and / or corticosteroids before the infusion administration of the preparation Mayoheim.
Patients with Pompe disease in severe stages with impaired cardiac and respiratory function who have a higher risk of serious complications of MI should be closely monitored during the administration of the Mayozyme preparation.

In a clinical study, it was noted that approximately half of the patients receiving Mayoise in infancy developed MI, sometimes severe with a tendency to display more symptoms in patients receiving a higher dose (40 mg / kg).
Patients who develop high antibody titers in their infancy are more at risk for more frequent MI, as well as patients with acute illness (eg, pneumonia, sepsis). Prior to the administration of Mayozyme, the clinical condition of the patient should be carefully examined and all cases of both IR, and delayed and, possibly, immunological reactions should be monitored and recorded carefully.
In clinical studies, as a rule, during the 3 months of treatment, most patients developed IgG antibodies to the CRCH.
Thus, seroconversion is expected in most patients receiving Mayozyme. There was a tendency to produce higher antibody titers in patients receiving a higher dose (40 mg / kg). Apparently, there is no correlation between the onset of TS and the time of antibody formation. In vitro tests, a limited number of IgG-positive patients have a positive result with respect to inhibitory effects. In view of the rarity of this condition and the limited experience at the moment, the effect of antibody formation on the safety and efficacy of the drug has not been fully established. The likelihood of an unfavorable outcome and the formation of high and stable antibody titers is higher among PRIM negative patients who did not have an endogenous protein CRCH in Western blotting (PRIM is a cross-reactive immunological material;) compared with PRIM-positive patients. However, high and persistent antibody titers are also found in some PRIM-positive patients. It is believed that the lack of clinical effect and the development of high and stable antibody titers are determined by many factors. Antibody titers should be regularly monitored.
Patients with hypersensitivity reactions to rchKAG shows testing for IgE antibodies and other mediators of anaphylaxis, due to an increased risk of MI with the reappointment Mayozaym drug. Such patients should be monitored carefully, as there is a successful experience of their conduct during the reappointment Mayozaym drug in a lower starting dose and at a low infusion rate. Severe skin reaction to rchKAG, including ulceration and necrosis may immuno-conditioned. Several patients experienced nephrotic syndrome with high titers of antibodies IgG (> 102,400) and immune complexes in kidney biopsy flowing after termination LFT. Thus in patients with high titers of IgG antibodies need to periodically urine.
During the reception rchKAG patients should be observed at systemic immune-mediated symptoms of the skin and other organs. If this happens, the treatment should start, stop taking rchKAG. Before re-appointment is necessary to evaluate the risk / benefit ratio, as a result of immune-mediated reactions, taking into consideration the existing successful experience reappointment Mayozaym drug to patients under close medical supervision.
If any of these instructions side effects are compounded, or if you notice any other side effects not mentioned in the instructions, tell your doctor.
Note, if necessary features of the action of the drug at the first reception or its cancellation
No features of the medicinal product with its first reception or cancellation has been established. If you have questions about the first dose Mayozaym or its cancellation, consult your physician.
Description, if necessary, the doctor's actions (assistant), a patient at the admission of receiving one or more doses of the drug
If you missed receiving one or more doses of the drug Mayozaym or have any questions, consult your physician.
Impact on the ability to drive vehicles and manage mechanisms

Studies on the effect of the PA's ability to drive and use machinery has not been. On the day of vnutivennoy infusion should be particularly careful because of possible dizziness.

Cases of overdose have been identified. In clinical studies used dose to 40 mg / kg body weight.

Studies on the interaction of alpha alglyukozidazy with other drugs was conducted. Since alglyukozidaza alpha is recombinant human protein, drug-drug interactions by cytochrome P450 are unlikely.
In the absence of drug compatibility studies Mayozaym, can not be mixed with other medicines. You should inform your doctor about all medications you take.Mayozaym can be used with other drugs only if recommended by your doctor.

On prescription.


At a temperature of 2 В° C to 8 В° C.
Keep out of the reach of children. Shelf life - 2 years.
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