CEREZYME

Cerezyme® (imiglucerase) for injection is indicated for treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions: anemia (low red blood cell count) thrombocytopenia (low blood platelet count) bone disease.

The average annual cost of ERT is ranged between 40,941.1$ and 69,176.5$ per patient. The lifetime cost of ERT, which starting from symptomatic, stage is3,477,710.8$ against 87,885.4$ without ERT; a difference of 3,389,825$. Consequently, the incremental cost-effectiveness ratio (ICER) was 45415$.

Gaucher (go-SHAY) disease is the result of a buildup of certain fatty substances in certain organs, particularly your spleen and liver. This causes these organs to enlarge and can affect their function. The fatty substances also can build up in bone tissue, weakening the bone and increasing the risk of fractures.

Lumizyme ® (alglucosidase alfa) is an enzyme replacement therapy produced by recombinant DNA technology. It provides an exogenous source of acid α‑glucosidase (GAA), an essential lysosomal enzyme that is deficient or absent in patients with Pompe disease, leading to intralysosomal glycogen accumulation.

The recommended dosage of Cerezyme based upon disease severity ranges from 2.5 units per kilogram 3 times a week to 60 units per kilogram once every 2 weeks. Your infusion time may vary based on your body weight and how you respond to treatment.

There are no known contraindications to the use of Cerezyme. Treatment with Cerezyme should be carefully re-evaluated if there is significant clinical evidence of hypersensitivity to the product. Therapy with Cerezyme should be directed by physicians knowledgeable in the management of patients with Gaucher disease.

Cerezyme does not contain preservatives.Reconstitute each 400-unit vial of Cerezyme by slowly injecting 10.2 mL of Sterile Water for Injection, USP, down the inside wall of each vial.Roll and tilt the vial to allow the powder to dissolve completely. … Withdraw up to 10 mL per vial.

Since approval by the FDA in 1994 and EMEA in 1997, enzyme replacement therapy with Cerezyme (imiglucerase for injection) has been the standard of care for the treatment of Gaucher disease.

Cerezyme (imiglucerase) is indicated for use as long-term enzyme replacement therapy in patients with a confirmed diagnosis of non-neuronopathic (Type 1) or chronic neuronopathic (Type 3) Gaucher disease who exhibit clinically significant non-neurological manifestations of the disease.

Mechanism Of Action The mannose sugars on imiglucerase mediate binding to and internalization by cells including macrophages. Cerezyme catalyzes the hydrolysis of glucocerebroside to glucose and ceramide.

The cost for Cerezyme intravenous powder for injection 400 units is around $1,817 for a supply of 1 powder for injection, depending on the pharmacy you visit. Quoted prices are for cash-paying customers and are not valid with insurance plans.

Outlook (Prognosis) How well a person does depends on their subtype of the disease. The infantile form of Gaucher disease (Type 2) may lead to early death. Most affected children die before age 5. Adults with the type 1 form of Gaucher disease can expect normal life expectancy with enzyme replacement therapy.

While Cerezyme is an enzyme replacement therapy (ERT) providing an analog of beta-glucocerebrosidase — defective in these people — Cerdelga is a substrate reduction therapy (SRT) that lessens the amount of the fat molecule glucosylceramide.

Cerezyme® (imiglucerase) for injection is indicated for treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions: anemia (low red blood cell count) thrombocytopenia (low blood platelet count) bone disease.

Enlarged spleen.Enlarged liver.Eye movement disorders.Yellow spots in the eyes.Not having enough healthy red blood cells (anemia)Extreme tiredness (fatigue)Bruising.Lung problems.

How Cerezyme works. Gaucher disease is a rare genetic disorder caused by mutations in the GBA gene, which encodes for an enzyme called beta-glucocerebrosidase. This enzyme is responsible for breaking down glucocerebroside (a component of cell membranes) into glucose (a simple sugar) and ceramide (a simple fat molecule) …

VPRIV (velaglucerase alfa) is a member of the lysosomal enzymes drug class and is commonly used for Gaucher Disease. The cost for VPRIV intravenous powder for injection 400 units is around $1,548 for a supply of 1 powder for injection, depending on the pharmacy you visit.

QuantityPer unitPrice1$1,025.96$1,025.96

Disease Overview Gaucher disease is a rare, inherited metabolic disorder in which deficiency of the enzyme glucocerebrosidase results in the accumulation of harmful quantities of certain fats (lipids), specifically the glycolipid glucocerebroside, throughout the body especially within the bone marrow, spleen and liver.

Cerdelga Prices, Coupons and Patient Assistance Programs. Cerdelga (eliglustat) is a member of the miscellaneous metabolic agents drug class and is commonly used for Gaucher Disease. The cost for Cerdelga oral capsule 84 mg is around $8,316 for a supply of 14 capsules, depending on the pharmacy you visit.

Generic Name:ImigluceraseTrade Name:CerezymeMarketing Approval Date:05/23/1994Approved Labeled Indication:Enzyme replacement therapy in patients with type I Gaucher’s disease.Exclusivity End Date:05/23/2001

Each vial of Cerezyme is for single use only. The powder for concentrate for solution for infusion has to be reconstituted with water for injections, diluted with 0.9% sodium chloride intravenous solution and then administered by intravenous infusion.

Cerezyme is administered by intravenous infusion over 1-2 hours. Dosage should be individualized to each patient.

Method of administration After reconstitution with water for injection and dilution with 0.9% Sodium Chloride intravenous solution the preparation is administered by intravenous infusion over 1 to 2 hours. For instruction on reconstitution and dilution of Cerezyme before administration, see section 6.6.

Dilute the Cerezyme solution promptly with 0.9% Sodium Chloride Injection, USP, to a final volume of 100 to 200 mL.

For 200 U vials, reconstitute each vial with 5.1 ml water for injections; the reconstituted volume is 5.3 ml. For 400 U vials, reconstitute each vial with 10.2 ml water for injections; the reconstituted volume is 10.6 ml. 5. Inject the water gently into a vial of Cerezyme.

Cerezyme is an analog of the human enzyme β-glucocerebrosidase (also called acid β-glucosidase). Cerezyme reduces the accumulation of GL-1 by cleaving the substrate into glucose and ceramide. References: Cerezyme [prescribing information].

Cerezyme is a biologic therapy that is indicated to treat: pediatric and adult patients with a confirmed diagnosis of Type 1 Gaucher disease that results in one or more of the following conditions: anemia. thrombocytopenia.

Mechanism Of Action The mannose sugars on imiglucerase mediate binding to and internalization by cells including macrophages. Cerezyme catalyzes the hydrolysis of glucocerebroside to glucose and ceramide.

Intended to treat Gaucher’s disease, a rare genetic disorder, Cerezyme was by no means the first orphan drug. What made it notable was the price. In the U.S., Cerezyme was priced at ~$200,000 per year for an average patient.

Enzyme replacement therapy (ERT) involves intravenous (IV) infusions to correct the underlying enzyme deficiency that causes symptoms of Gaucher disease (pronounced go-SHAY). In particular, a common use of enzyme replacement therapy is for lysosomal storage disease treatment.

Gaucher disease is passed down from parents to children (is inherited). It is caused by a problem with the GBA gene. It is an autosomal recessive disorder. This means that each parent must pass along an abnormal GBA gene for their child to get Gaucher.

2.4 Important Administration Instructions Swallow capsules whole, preferably with water, and do not crush, dissolve, or open the capsules. CERDELGA can be taken with or without food. Avoid the consumption of grapefruit or grapefruit juice (strong CYP3A inhibitors) with CERDELGA [see Drug Interactions (7.1)].