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Elevidys (delandistrogene moxeparvovec) is a gene therapy treatment for Duchenne muscular dystrophy (DMD)

Category: Specialty Medicines

Description

What is Elevidys(delandistrogene moxeparvovec-rokl)

ELEVIDYS is a prescription gene therapy used to treat ambulatory and non-ambulatory people with Duchenne muscular dystrophy who are at least 4 years old and have a confirmed mutation in the dystrophin gene.

Use in non-ambulatory people is approved under accelerated approval, which allows for drugs to be approved based on a marker that is considered reasonably likely to predict a clinical benefit. Treatment with ELEVIDYS increased the marker, ELEVIDYS micro-dystrophin (also called “micro-dystrophin”). Verification of a clinical benefit may be needed for ELEVIDYS to continue to be approved for non-ambulatory people with Duchenne.

What is Elevidys used to treat?

Elevidys is a one-dose treatment. After a single infusion, the drug is thought to work long-term to treat DMD in certain people. How long does Elevidys take to work? Elevidys begins to work shortly after you receive the infusion.

How many doses of Elevidys?

For DMD, your child will receive only one dose of Elevidys. For children that weigh less than 70 kg, the usual dose of this drug is 1.33 x 10¹⁴ vg per kg of body weight. For children that weigh more than 70 kg, the usual dose of the drug is a fixed dose of 9.31 x 10¹⁵ vg.

Who can get Elevidys?

What are the side effects of ELEVIDYS?

Clinical trials have found delandistrogene moxeparvovec-rokl to be largely safe. In clinical trials, the most common side effects were vomiting, nausea, fever, low platelet counts, and abnormalities on liver function tests. Most of these side effects appear during the first two weeks after treatment.

How long does it take for yeast infection tablets to work?

Generally, the active ingredients in thrush tablets start working right away to combat yeast overgrowth. However, immediate relief from symptoms isn’t typical. Most individuals begin to notice an improvement in symptoms within one to three days after starting the treatment.

Who created ELEVIDYS?

Sarepta is responsible for global development and manufacturing for delandistrogene moxeparvovec-rokl, and distribution within the U.S. will commence immediately.

Is Elevidy effective?

While the large, randomized study of delandistrogene moxeparvovec-rokl failed to meet its statistical primary endpoint of improvement versus placebo in the NSAA, the FDA found the observations regarding the secondary endpoints and exploratory endpoints to be compelling and to indicate clinical benefit compared to placebo.

What is the most expensive pill in the world?

Lenmeldy (atidarsagene autotemcel) is the most expensive drug on the market with a steep price tag of $4.25m. The gene therapy is developed for children with metachromatic leukodystrophy (MLD), a potentially fatal disorder caused by a mutation in the arylsulfatase-A (ARSA) gene, which leads to the buildup of fats.

What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy is a serious but rare genetic condition that becomes worse over time, leading to weakness and wasting away of the body’s muscles. The cause of Duchenne muscular dystrophy is a defective gene that codes for the dystrophin protein, an important component of muscle cells.

The lack of dystrophin causes symptoms such as trouble walking and running, falling frequently, fatigue, learning disabilities/difficulties, heart issues due to the impact on the heart muscle, and breathing problems due to weakness of respiratory muscles involved in breathing.

Symptoms typically begin in childhood, often between 3 to 6 years old, and as the disease progresses, life-threatening heart and respiratory problems can occur. Disease severity and life expectancy do vary, but patients often succumb to the disease in their 20s or 30s because of heart and/or respiratory failure.

Warnings

Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following infusion. Patients are closely monitored during and for at least 3 hours after the end of infusion for signs and symptoms of infusion-related reactions, including tachycardia, tachypnea, lip swelling, difficulty breathing, nasal flaring, urticaria, flushing, lip pruritus, rash, cheilitis, vomiting, nausea, rigors, and pyrexia.

Acute serious liver injury has been observed when using delandistrogene moxeparvovec-rokl. Before using this medicine, a liver function test should be performed. Then, liver function (clinical exam, GGT, and total bilirubin) should be monitored weekly for the first three months following delandistrogene moxeparvovec-rokl infusion.

Immune-mediated myositis (an immune response affecting muscles). Patients should contact their healthcare provider immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including difficulty swallowing, difficulty breathing, or a weak voice, as these may be myositis symptoms.

Acute serious myocarditis (inflammation of the heart) and troponin-I elevations have been observed following Elevidys infusion in clinical trials. Patients should contact their healthcare provider immediately if they experience chest pain and/or shortness of breath.

Pre-existing Immunity against AAVrh74 In AAV-vector based gene therapies. Preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with this infusion, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for the presence of anti-AAVrh74 total binding antibodies before infusion. Elevidys should not be administered in patients with elevated anti-AAVrh74 total binding antibody titers (≥1:400)

Infections. While taking corticosteroids, an infection before or after the Elevidys infusion could lead to more serious complications. Contact a healthcare provider immediately if symptoms suggestive of infection are observed (e.g., coughing, wheezing, sneezing, runny nose, sore throat, or fever).