Universal reference book for medicines

Active substance: agalsidase alfa

Type: The drug for the treatment of hereditary enzymatic insufficiency

Manufacturer: Shire Human Genetic Therapies (USA) manufactured by Chesapeake Biological Laboratories (USA)
A typical clinical and pharmacological article
This information is a reference and it is not enough that the drug has been prescribed by a doctor ..

Fabry disease is a hereditary disease characterized by a deficiency of lysosomal alpha-galactosidase A, resulting in the accumulation of glycosphingolipids, predominantly globotriaosylceramide.

Agalsidase alfa (alpha-galactosidase A) catalyzes the hydrolysis of globotriososylceramide and other neutral glycosphingolipids with the alpha-galactic radical at the end in the cells of the body (including endothelial and parenchymal) to ceramide dihexonide and galactose.

The glycosylation profile is similar to that of the natural enzyme, which allows agsidase alpha to specifically bind to mannose-6-phosphate receptors on the surface of target cells.

Pharmacokinetic parameters do not differ significantly between men and women.

Distribution in the main tissues and organs is comparable in men and women.
V d - 17% of body weight.
About 10% of the administered dose is taken up by the liver.
Metabolised by peptide hydrolysis.

The average value of clearance in children (7-11 years), in adolescents (12-18 years) and adults - 4.2 ml / min / kg, 3.1 ml / min / kg and 2.3 ml / min / kg, respectively.

After 6 months of use the clearance is increased (in connection with the production of antibodies to agalsidase alpha, determined in low titre).

After a single dose of 0.2 mg / kg T 1/2 from the bloodstream in men and women - 91-125 min and 61-117 min, respectively.
In children (7-18 years), the drug administered at a dose of 0.2 mg / kg is excreted faster than in adults.

T 1/2 of the fabrics - more than 24 hours.

Renal elimination is negligible.

- Fabry's disease (deficiency of alpha-galactosidase A).

In / in the drip, under the supervision of a doctor who has experience in treating patients with Fabry's disease or other hereditary metabolic disorders.

Adults and children older than 7 years in a dose of 0.2 mg / kg for 40 minutes 1 time in 2 weeks.

If the kidney function is impaired (CC less than 60 ml / min), the kidney response to enzyme replacement therapy may be limited.

In patients with chronic renal failure, incl.
dialysis patients, or after a kidney transplant, dose adjustment is not required.

Dissolve the required volume in 100 ml of a 0.9% solution of sodium chloride.
When breeding, sterile conditions should be ensured. Agalcidase alpha does not contain preservatives or bacteriostatic substances. The solution is gently mixed without shaking.

Enter through the IV system (infusion pump) with a built-in filter (no later than 3 hours after dilution).

Criteria for the frequency of occurrence: very often (more than 1/10), often (more than 1/100 and less than 1/10), sometimes (more than 1/1000 and less than 1/100).

The most frequent undesirable effects were infusion reactions.
Infusion reactions of severe course are rare and manifested as fever, chills, tachycardia, nausea, vomiting, urticaria, angioedema. Usually such reactions develop in the first 2-4 months after the beginning of therapy with the drug. Over time, their frequency and severity decrease. Reactions associated with the administration of the drug and increased pain in children developed more often. The most frequent reactions in children were light-flow reactions associated with infusion (chills, fever, hot flashes, headache, nausea, dyspnea).

From the nervous system: very often - headache;
often - hypersomnia, dizziness, taste distortion, neuropathic pain, tremor, hypoesthesia, paresthesia; sometimes - parosmia.

From the senses: often - increased lacrimation, a decrease in the corneal reflex, tinnitus or its amplification.

From the CCC: very often - hot flushes;
often - tachycardia, increased blood pressure, palpitations.

On the part of the respiratory system: often - cough, hoarseness of the voice, sore throat, dyspnea, nasopharyngitis, pharyngitis, rhinorrhea, increased secretion in the oropharynx.

From the side of the digestive system: very often - nausea;
often - diarrhea, vomiting, pain (discomfort) in the abdomen.

From the skin: often - acne, erythema, rash, itching, reticulum live;
sometimes - angioedema, hives.

From the musculoskeletal system: often - a feeling of discomfort in the muscles and bones, myalgia, back pain, pain in the limbs, arthralgia, swelling of the joints;sometimes - a sense of heaviness.

Local reactions: often - a rash at the injection site.

Other: very often - chills, fever, pain and discomfort, weakness or weakness;
often - a feeling of heat or cold, asthenia, pain and a feeling of heaviness in the chest, flu-like syndrome, malaise, peripheral edema; sometimes - a decrease in tissue saturation with oxygen.

- lactation period;

- Children under 7 years.

With caution: pregnancy.

Use with caution in pregnancy.
Application during lactation (breastfeeding) is contraindicated.
In patients with hepatic impairment, efficacy and safety have not been studied.

In patients older than 65 years, efficacy and safety have not been studied.

A widespread kidney damage can limit their response to enzyme replacement therapy, possibly due to irreversible changes.
In such cases, impairment of renal function is an expected natural progression of the disease.

In patients over 65 years of age or with liver failure, efficacy and safety have not been studied.

Light and short-term reactions do not require drug therapy and cancellation of treatment.
To prevent the development of these reactions 1-24 hours prior to administration, administration of either intravenous or intravenous antihistamines or GCS is possible.

With the development of mild or moderate acute infusion reactions, it is necessary to immediately stop the injection of the drug, provide medical care, and, if possible, resume the introduction of the drug.

With the development of allergic reactions (including severe) it is necessary to immediately stop the drug and start symptomatic therapy.

On the background of therapy in patients, antibodies of IgG class to agsidase alpha can be produced.
In 24% of male patients 3-12 months after the start of treatment, a low IgG antibody titer was detected. After 12-54 months, 17% still showed antibodies, while 7% showed signs of developing immunological tolerance, which was confirmed by the disappearance of IgG antibodies over time. The remaining 76% of patients did not detect antibodies.
It should not be administered concomitantly with chloroquine, amiodarone or gentamicin (inhibit the intracellular activity of alpha-galactosidase).

Pharmaceutically incompatible with other medicines.

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