ELELYSO

Elelyso is a medication that treats Type 1 Gaucher disease in adults and children (2 to 17 years old) who have at least one of the following symptoms: enlarged spleen or liver, low red blood cell count, or bone abnormalities. It contains taliglucerase alfa rpc, which is an Enzyme Replacement Therapy.

On May 1, 2012, the U.S. Food and Drug Administration approved ELELYSO® (taliglucerase alfa), an enzyme replacement therapy for Gaucher disease, as Protalix’s first product manufactured by ProCellEx.

Protalix, an Israeli biotherapeutics company, manufactures ELELYSO® (taliglucerase alfa) for injection.

ELELYSO is a recombinant analog of human lysosomal glucocerebrosidase that functions as an enzyme replacement therapy. It works by catalyzing the hydrolysis of glucocerebroside into glucose and ceramide, thereby reducing the buildup of glucocerebroside in the body.

Colchicine, also known as Colcrys or Mitigare, is a medication prescribed for the treatment and prevention of gout, a form of arthritis that causes severe pain. Nitroglycerin, morphine, phenazopyridine, phenobarbital, potassium chloride, and sodium fluoride are other medications that may be used for various medical purposes.

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Amylyx’s ALS therapy, AMX0035, has received FDA approval for the treatment of amyotrophic lateral sclerosis (ALS). Despite the underwhelming data, the FDA has shown regulatory flexibility and granted full approval for this severe neurological disorder that leads to paralysis and death.

The FDA grants approval for new human drugs and biological products, requiring companies to provide evidence of the product’s safety, effectiveness, and compliance with federal quality standards.

In February 2022, Pacritinib was granted its initial approval in the United States. This approval allows for the treatment of adults who have intermediate- or high-risk primary or secondary myelofibrosis (specifically post-polycythemia vera or post-essential thrombocythemia) and have a platelet count below 50 × 109/L.

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On June 4, 2021, Tembexa (brincidofovir) was approved by the U.S. Food and Drug Administration for the treatment of smallpox.

If individuals with disabilities are experiencing difficulties accessing the PDF files listed below, they can contact (301) 796-3634 for assistance. The approval date for these files is 8/27/2009.

CABENUVA is a medication that combines cabotegravir and rilpivirine. Rilpivirine is already approved in the U.S. as a 25 mg tablet that is taken once a day to treat HIV-1. It is used in combination with other antiretroviral agents in patients who have not received antiretroviral treatment before and are at least 12 years old and weigh at least 35 kg. These patients should also have a viral load of no more than 100,000 HIV RNA copies per milliliter.

Elelyso is administered intravenously through a drip, which should be infused over a minimum of 60 minutes. This infusion can take place at a hospital, clinic, or even at your home, depending on your doctor’s discretion.

If you are undergoing IV therapy for hydration or to alleviate a hangover, you can anticipate seeing results within a timeframe of 45 minutes to four hours following the treatment. However, if your goal is to enhance your energy levels, the effects of IV infusion therapy may become noticeable within a span of four days to two weeks after the treatment.

The average duration of IV therapy ranges from 15 to 90 minutes. Generally, it takes around 30 minutes, but the actual time may vary based on factors such as age, weight, and the specific type of IV chosen.

When undergoing outpatient infusion treatment or therapy, it is important to anticipate certain factors. The duration of the infusion process can vary, but it typically lasts for about an hour. However, the specific length of time required for this therapy depends on factors such as the type of medication being administered and the specific illness or infection being treated.

Causes: Mutations in the GBA gene result in Gaucher disease. The GBA gene is responsible for producing the beta-glucocerebrosidase enzyme, which is essential for breaking down glucocerebroside into glucose and ceramide.

Date: May 1, 2012
Article: FDA Approves New Orphan Drug Elelyso to Treat a Form of Gaucher Disease
Approval: FDA
Date: Dec 15, 2011
FDA Extends Taliglucerase Alfa PDUFA Date to May 1, 2012

Gaucher disease type 1 is the most prevalent form of the disease in western countries, affecting approximately 95 percent of patients. Common symptoms include enlarged spleen and liver, bone issues, and fatigue, while brain development remains unaffected. It is important to note that Gaucher disease type 1 is treatable.

Taliglucerase alfa, also known as Elelyso, is a biopharmaceutical drug created by Protalix and Pfizer.

VPRIV is given as a 60-minute infusion every other week, with a healthcare professional overseeing the process. While the infusion usually lasts up to 60 minutes, it may be necessary for you to remain for additional monitoring after the treatment.

Cerezyme infusions can take anywhere from 1 to 2 hours or longer to complete and can be administered in different settings, including a doctor’s office, a treatment center, or in some instances, at home.

Gaucher disease is a rare inherited genetic disorder characterized by a deficiency of an enzyme responsible for breaking down lipids. This leads to the accumulation of lipids in organs like the spleen and liver.

Gaucher disease occurs when there is a deficiency of the enzyme glucocerebrosidase (GCase), which is responsible for breaking down a fatty substance called glucocerebroside in the body. This leads to the accumulation of unprocessed glucocerebroside in macrophages, which are normal scavenger cells.

The subtype of Gaucher disease greatly impacts a person’s prognosis. Type 2, or the infantile form, often results in premature death, with most affected children passing away before the age of 5. However, adults with Type 1 Gaucher disease can typically expect a normal lifespan with the aid of enzyme replacement therapy.

Adam Rose, famous for his acting roles on “Veronica Mars” and “L.A.’s Finest,” was born with Gaucher disease (pronounced go-SHAY). This rare condition affects approximately 1 in every 40,000 live births.

Gaucher disease, an inherited genetic disorder, manifests in various symptoms such as bone pain, anemia, enlarged organs, abdominal swelling, and bruising and bleeding issues. The disease is categorized into three types, with certain types potentially resulting in severe brain damage and mortality.

Some of the main signs and symptoms of this condition include an enlarged liver and spleen, a low red blood cell count leading to anemia, easy bruising due to a decrease in blood platelets, bone abnormalities causing pain and fractures, and joint conditions like arthritis.

Although there is no cure for Gaucher disease, types 1 and 3 can be managed with medication and enzyme replacement therapy, which are typically successful. However, types 2 and 3 do not have effective treatments for the resulting brain damage.

To maintain strong bones, it is important for both children and adults with Gaucher disease to consume a nutritious diet that includes foods high in vitamin D and calcium. Regular exercise is also beneficial. Additionally, taking supplements such as vitamin D, calcium, or other recommended by your doctor may help alleviate Gaucher symptoms, especially if your diet is lacking in these nutrients.

Gaucher Disease: Clinical Phenotypes and Variants

Gaucher Disease is characterized by several major clinical phenotypes, including Type 1, Type 2, and Type 3. Type 1 is the most common form and is associated with non-neuronopathic symptoms. Type 2 is an acute infantile form, while Type 3 is a subacute juvenile form.

In addition to these major phenotypes, there are also variant forms of Gaucher Disease. These include the perinatal-lethal form, which is characterized by severe symptoms that are present at birth and often lead to early death. The cardiovascular form is another variant, which is associated with an increased risk of heart disease and other cardiovascular complications.

Signs and symptoms of Gaucher disease can manifest at any age, and it is not uncommon for individuals to remain undiagnosed until adulthood. Early onset osteoporosis may serve as one of several indicators, although it is worth noting that many healthcare professionals are not familiar with Gaucher disease.

Enzyme replacement therapy and substrate reduction therapy are the current treatments for Gaucher disease. These treatments have shown the ability to reverse many of the non-neurological symptoms associated with the disease. However, it is important to note that these therapies require continuous administration and are associated with high costs.