Index of drugs

Long-lasting, enzymatic replacement therapy in patients with a confirmed diagnosis of Pompe disease.

1 vial contains 50 mg alglucosidase alfa.

Recombinant form of human acid alpha-glucosidase. Produced by recombinant DNA using Chinese hamster ovarian cell culture. Alglucosidase alfa is thought to restore lysosomal alpha glucosidase activity, leading to stabilization or rebuilding of cardiac and skeletal muscle function.

Life threatening hypersensitivity (anaphylactic reaction) to the active substance or to other components of the preparation after another failed attempt to administer the drug.

Because of the potential for serious anaphylactic reactions associated with the infusion, appropriate therapeutic preventive measures, including cardiopulmonary resuscitation equipment, should be available during administration. If severe hypersensitivity or anaphylactic reactions occur, immediate discontinuation of the product should be considered and appropriate treatment initiated. Observe the current medical standards applicable to the treatment of sudden anaphylactic reactions. Based on clinical trials, the risk of developing infusion-related reactions appears to be higher in patients diagnosed with an infant with an increase in antibody titres and a greater risk of infusion-related reactions observed in patients who were infected with infusion during the infusion. acute illness (eg pneumonia, sepsis). Caution should be exercised when administering the preparation to patients who have experienced an infusion-related reaction (in particular anaphylactic reaction). If mild and transient reactions occur, medical assistance and discontinuation of the infusion may not be necessary. Most infusion-related reactions have been successfully treated by reducing the rate of infusion by temporarily interrupting or administering oral antihistamines and / or antipyretics and / or corticosteroids prior to the start of the infusion. Infusion-related reactions may occur during administration, usually up to 2 hours after administration, and are more likely at higher infusion rates. In clinical trials, the majority of patients have usually developed IgG antibodies to human recombinant alpha-glucosidase during the first 3 months of treatment, hence seroconversion is expected in these patients. In patients with an early stage of the disease treated with a higher dose of the preparation (40 mg / kg), a tendency to the development of antibodies with larger titers was observed. It seems that the likelihood of an unfavorable course of the disease and the formation of stable, high antibody titres is higher in CRIM negative patients whose endogenous GAA protein was not detected by Western blot analysis than in patients with CRIM-positive test (in whom endogenous GAA protein was detected using the same analysis). However, high, sustained antibody titres also occur in some CRIM-positive patients. It is believed that the adverse outcomes of the disease and high, sustained antibody titers have a multifactorial etiology. IgG titres should be monitored regularly. The state of patients with advanced Pompe disease (risk of cardiac and respiratory disorders) and patients with IgE antibodies to the product should be closely monitored during administration. Some patients with known IgE antibodies were successfully re-administered by reducing the rate of infusion and reducing the starting dose, and further intake of the preparation was under strict control.

Do not administer the preparation during pregnancy unless clearly necessary (no data on the use of alglucosidase alfa). It is recommended to stop breast-feeding during treatment with the preparation.Alglucosidase alfa can penetrate into breast milk, no data on the effect on infants fed with mother's milk using the product.

An early form of Pompe disease. Very common: decrease in oxygen saturation; tachycardia; acceleration of breath, cough; vomiting; urticaria, rash; hot flushes; fever. Common: acceleration of the heart rhythm, increase in blood pressure, increase in body temperature; cyanosis; trembling; belching, nausea; erythema, maculopapular rash, macular rash, papular rash, pruritus; hypertension, pallor; irritability, chills; anxiety. In addition, the following have been reported: rales, oxygen saturation, bronchospasm, increased breathing frequency, and periocular tissue edema. Late form of Pompe disease. Common: increase in blood pressure; dizziness, numbness, headache; tightness in the throat; diarrhea, vomiting, nausea; urticaria, macular rash, pruritus, hyperhidrosis; muscle spasms, muscle tremors, muscle aches; hot flushes; fever, chest discomfort, peripheral edema, localized swelling, fatigue, and feeling hot; hypersensitivity. In addition, the following have been reported: angioneurotic edema, tightness in the throat, chest pain other than cardiac origin, supraventricular tachycardia, acceleration of the heart rhythm, hypertension, cooling of peripheral body parts, pain at the infusion site. In patients with early and late-onset Pompe disease, uncontrolled clinical trials have also been observed in agitation, hypotension, face swelling, excessive tearing, cyanosis, nervousness, wheezing, anaphylactic shock and / or cardiac arrest. In addition, bradycardia, laryngeal edema, pressure in the throat, and acute respiratory failure were observed after marketing.

Treatment with the product should be under the supervision of a physician experienced in the treatment of Pompe disease or other congenital metabolic or neuromuscular diseases. The recommended dose is 20 mg / kg, given once every 2 weeks as an intravenous infusion. The drug administered gradually. It is recommended to start the infusion at an initial rate of 1 mg / kg / h. every 30 min until reaching a maximum speed of 7 mg / kg / h, unless symptoms of an infusion-related reaction occur. There is no evidence that special instructions should be used for adolescents, adults and elderly patients. The safety and efficacy of the preparation in patients with renal or hepatic failure have not been evaluated.