LUMIZYME

Lumizyme ® (alglucosidase alfa) is a medication that replaces a missing or deficient enzyme in people with Pompe disease. If you have Pompe disease, ask your healthcare provider if Lumizyme is an appropriate treatment for you.

How long can you live with Pompe disease without treatment? Without treatment, babies with classic infantile-onset Pompe disease typically don’t survive beyond the age of 2, and most children with the nonclassic type don’t live past early childhood.

Lumizyme is administered by intravenous infusion, and should be reconstituted, diluted, and administered by a health care professional. Lumizyme is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α‑glucosidase (GAA) deficiency).

Myozyme will remain available to existing patients treated with Myozyme through December 31, 2014. 3. Patients currently receiving Myozyme: product will not be available for re-order after December 31, 2014.

While there is no cure for Pompe disease, enzyme replacement therapy can reduce or slow the progression of the disease. This lifesaving therapy was developed at Duke following clinical trials that allowed children with Pompe disease to access the therapy before it was widely available.

Symptoms begin in the first months of life, with feeding problems, poor weight gain, trouble breathing, muscle weakness, enlarged heart, floppiness, and head lag. Many infants with Pompe disease also have enlarged tongues.

During the studies in patients with infantile-onset Pompe disease, the most common side effects with Myozyme (seen in more than 1 patient in 10) were tachycardia (rapid heartbeat), flushing (redness), cough, tachypnoea (rapid breathing), vomiting, urticaria (itchy rash), rash, pyrexia (fever) and decreased oxygen …

Symptoms at onset Almost all the patients retrospectively remember difficulties during physical education or athletic games during their childhood. These symptoms are associated with, or often preceded by, diffuse muscle pain, lumbar pain and muscle cramps at the lower limbs.

QuantityPer unitPrice1$1,025.05$1,025.05

Lumizyme ® (alglucosidase alfa) treatment is generally covered by the U.S. Medicare program, as long as it is considered medically necessary.

Hemgenix. Cost: $3.5 million per one-time dose. … Elevidys. Cost: $3.2 million per one-time dose. … Skysona. Cost: $3 million per one-time dose. … Zynteglo. Cost: $2.8 million per one-time dose. … Zolgensma. Cost: $2.1 million per one-time dose. … Myalept. Cost: $1.3 million annually. … Danyelza. … Zokinvy.

A year later, after the U.S. FDA approved Zynteglo, bluebird found a much more welcoming market and priced its gene therapy accordingly. The company landed on a price of $2.8 million per dose, making Zynteglo the most expensive drug in the U.S.

The REMS for Lumizyme (alglucosidase alfa) was originally approved on May 24, 2010, and the most recent REMS modification was approved on July 16, 2012.

QuantityPer unitPrice25 milliliters$240.13$6,003.18

QuantityPer unitPrice1$1,025.96$1,025.96

The recommended dosage of LUMIZYME is 20 mg/kg body weight administered every 2 weeks as an intravenous infusion. LUMIZYME does not contain any preservatives. Vials are single-use only.

The total volume of infusion is determined by the patient’s body weight and should be administered over approximately 4 hours. If severe infusion or allergic reactions occur, immediate discontinuation of the administration of Lumizyme should be considered, and appropriate medical treatment should be initiated.

Nexviazyme outperforms Lumizyme in analysis In either case, this ratio reflects that a person taking Nexviazyme is more than twice as likely to benefit than one on Lumizyme, with statistical significance. Nexviazyme was similarly favored when patients were divided by sex and baseline lung function.

Lumizyme (alglucosidase alfa) is an enzyme replacement therapy. It replaces an enzyme (protein) that helps the body break down a large sugar called glycogen. People with Pompe disease aren’t able to break down glycogen because they’re either missing that enzyme or the enzyme doesn’t work.

Lumizyme, Myozyme (alglucosidase alfa) dosing, indications, interactions, adverse effects, and more.

DateArticleMay 25, 2010Approval FDA Approves Lumizyme for Late-Onset Pompe DiseaseJan 21, 2010Genzyme’s Lumizyme BLA Receives Six-Month FDA ReviewDec 7, 2009Genzyme to Reopen Enrollment in Alglucosidase Alfa Temporary Access Program

Lumizyme is administered by intravenous infusion, and should be reconstituted, diluted, and administered by a health care professional. Lumizyme is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α‑glucosidase (GAA) deficiency).

M6P (mannose-6-phosphate) is part of the molecule that binds to certain receptors on the muscle cell and helps enzyme replacement therapy reach your muscle cells. NEXVIAZYME has ~15x more M6P than alglucosidase alfa (Lumizyme) and was made to improve uptake of the treatment into the muscle cells.

Enzyme replacement therapy can help improve muscle tone and reduce glycogen storage in individuals with Pompe disease. The following drugs have been approved: Alglucosidase alfa (Myozyme©) to treat infantile-onset Pompe disease. Lumizyme© to treat individuals of all ages with Pompe disease.

Myozyme will be reconstituted, diluted and should be protected from light before it is given to you or your child. It is given as a drip through a needle placed into a vein (intravenous infusion), usually in the arm. This takes approximately 4 hours.

NEW YORK, NY — August. 6, 2021 — The Muscular Dystrophy Association (MDA) today celebrates the decision by the US Food and Drug Administration (FDA) to grant accelerated marketing approval to avalglucosidase alfa Nexviazyme for the treatment of people 1 year of age and older living with late-onset Pompe disease.

What Is Pompe Disease? Pompe disease is a rare genetic condition that causes muscle weakness that gets worse over time. It can have a serious effect on many of the body’s systems. Pompe disease is also called acid maltase deficiency disease and glycogen storage disease type II.

The recommended dosage of NEXVIAZYME is either 20 mg or 40 mg for each kilogram of body weight—your healthcare provider will calculate the appropriate dosage for you. The infusion usually takes approximately 4-5 hours for those receiving 20 mg/kg and approximately 5-7 hours for those receiving 40 mg/kg.

❖ Lumizyme and Myozyme are proprietary names for alglucosidase alfa, a recombinant form of the enzyme acid alpha- glucosidase, which is required for glycogen cleavage. These enzyme preparations are indicated for use in patients with Pompe 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$. Consequently, the incremental cost-effectiveness ratio (ICER) was 45415$.

The FDA has approved a two-component therapy — Pombiliti (cipaglucosidase alfa-atga) with Opfolda (miglustat) — to treat adults living with late-onset Pompe disease.

The manufacture of Lumizyme ® (alglucosidase alfa) employs recombinant DNA technology to produce the human GAA enzyme using Chinese hamster ovary (CHO) cells. Production begins with genetic modification of host CHO cells to express the human GAA gene.