Buy Zolgensma | 100% Best Sales
$1,800,000.00
Zolgensma is a one-time-only gene therapy treatment for children aged less than two years with spinal muscular atrophy (SMA
Description
What is Zolgensma
Zolgensma is a brand-name prescription drug. It’s FDA-approved to treat spinal muscular atrophy (SMA) caused by genetic changes in the SMN1 gene. For this purpose, Onasemnogene abeparvovec-xioi is given to children less than 2 years old.
SMA is a rare condition that damages nerve cells in the brain and spinal cord. This leads to muscle weakness and trouble with activities such as breathing, speaking, swallowing, and walking. For more information about this condition, see the “Zolgensma for spinal muscular atrophy” section below.
Onasemnogene abeparvovec-xioi is a one-time-only gene therapy treatment for children aged less than two years with spinal muscular atrophy (SMA) that costs $2.1 million for the single treatment. The reason Zolgensma price is so expensive is because that is the price Novartis has decided it is worth because it “dramatically transforms the lives of families affected by this devastating disease” and the claimed cost of bringing new drugs to market. But this price is not without controversy.
For a start, the early development of Onasemnogene abeparvovec-xioi was financed by the National Institutes of Health and several charities devoted to finding treatments for SMA, including many U.S. charities such as Sophia’s Cure, Cure SMA, Getty Owl Foundation, Fighting SMA, Jadon’s Hope Foundation, the Gwendolyn Strong Foundation, and Miracle for Madison. Many of these charities use donations by patient families and friends to subsidize research and clinical trials into new medicines for SMA.
The CEO of Novartis, Vas Narasimhan, argues that gene therapies represent a medical breakthrough in the way that they offer hope of a cure for deadly genetic diseases with a single dose. In some cases, the alternative is a multi-dose treatment with incremental improvements. For example, an alternative to Onasemnogene abeparvovec-xioi price is Spinraza that is taken four times a year for life. The list price is $750,000 for the first year and then $350,000 per year after that, so about $4 million a decade.
But how do they calculate the actual cost? Many companies use a value-based pricing model which calculates the cost based on how many years of life gained as well as taking into account the effectiveness of the drug. But this means putting a price on how much a life is worth, as well as ensuring the company makes a reasonable margin on their investment. Little is disclosed about the true cost of bringing a new drug to market, and Novartis didn’t develop Onasemnogene abeparvovec-xioi but acquired it through the $8.7 billion purchase of US firm AveXis.
Most health insurance systems do not allow for one-time treatments at such a price and a big price tag is also no guarantee that a drug will meet all expectations. Novartis will allow payments over five years, at $425,000 per year, and has said it will give partial rebates if the treatment doesn’t work.
How does Zolgensma work?
Onasemnogene abeparvovec-xioi treats the genetic root cause of SMA by replacing the missing or nonworking SMN1 gene with a new, working copy of a human SMN gene. It does this by using a vector, which is a “carrier” that can get the new, working SMN1 gene into the body. The vector in this case is a virus called AAV9 that has had its DNA removed and replaced with the SMN1 gene. This type of virus does not make you sick but can quickly travel through the body to the motor neuron cells and deliver the new gene.
Onasemnogene abeparvovec-xioi sits inside the nucleus of the motor neuron cell and tells the motor neuron cell to start making new SMN1 protein. Once the genes reach their destination, the vectors are broken down and excreted from the body and do not become part of the child’s DNA.
How effective is Onasemnogene abeparvovec-xioi
Results from an open-label, single-arm clinical trial that enrolled 21 children (range of ages 0.5 to 5.9 months) who all received 1.1 x 1014 VG/kg Onasemnogene abeparvovec-xioi price reported:
- 13 of the 19 patients continuing in the trial reached at least 14 months of age without permanent ventilation, at the March 2019 data cut off
- 10 of the 21 patients (47.6%) achieved the ability to sit without support for ≥ 30 seconds between 9.2 and 16.9 months of age (mean age was 12.1 months). This was significant because based on study entry criteria none of these patients would have been expected to attain the ability to sit without support, and only approximately 25% of these patients would be expected to survive (being alive without permanent ventilation) beyond 14 months of age
- 16 of the 19 patients had not required daily non-invasive ventilator use.
Drug details
Onasemnogene Abeparvoveccontains the active ingredient onasemnogene abeparvovec. This drug is a type of gene therapy. It works by replacing an abnormal SMN1 gene with a normal SMN1 gene. For more information, see the “How Zolgensma works” section below.
Onasemnogene Abeparvovec comes as a liquid suspension. It’s given as a one-time intravenous infusion (an injection into a vein over a period of time) by a healthcare provider. The drug comes in one strength: 2 x 1013 vector genomes per milliliter (mL).* This is the number of viral particles that are in each mL of the drug.
FDA approval
In 2019, the Food and Drug Administration (FDA) approved Zolgensma price to treat SMA in children less than 2 years old. Zolgensma price is the first gene therapy to receive FDA approval for the treatment of SMA.
Effectiveness
Onasemnogene Abeparvovec is given as a one-time dose. Clinical studies haven’t looked at how effective the drug may be if a child receives more than one dose.
Zolgensma para que sirve price also hasn’t been studied as a treatment for advanced SMA. “Advanced” means the disease has worsened. This may lead to conditions such as paralysis of the arms and legs or needing to use a ventilator to breathe.
For more information about the effectiveness of Zolgensma, see the “Zolgensma for spinal muscular atrophy” section below.
Zolgensma is only available as a brand-name medication. Zolgensma contains the active ingredient onasemnogene abeparvovec-xioi.* It’s a biologic drug that isn’t currently available in biosimilar form.
Biologic drugs are made from living cells, so they can’t be copied exactly. Non-biologic drugs are made from chemicals and can be copied exactly. A generic drug is an exact copy of the active drug in a brand-name medication.
Although not exact copies, biosimilars are considered as safe and effective as their parent biologic drugs. Like generics, biosimilars usually cost less than brand-name drugs.
* The reason “-xioi” appears at the end of the drug’s name is to show that the drug is distinct from similar medications that may be created in the future.
As with all medications, the cost of Zolgensma price can vary. To learn about the price of Zolgensma price in your area, talk with your doctor or pharmacist. What you’ll pay depends on your child’s insurance plan, your location, and the cost of the visit to the facility where your child gets their Zolgensma dose.
Before approving coverage for Zolgensma, the insurance company may require prior authorization. This means that your child’s doctor and insurance company need to communicate about the prescription before the company covers the drug. The company will review the prior authorization request and decide if the drug will be covered.
If you’re not sure whether you’ll need to get prior authorization for Zolgensma, contact your child’s insurance company.
Financial and insurance assistance
If you need financial support to pay for Zolgensma para que sirve, or if you need help understanding your child’s insurance coverage, help is available.
AveXis Inc., the manufacturer of Zolgensma, offers the OneGene Program. It allows you to work with coordinators who can tell you about financial assistance available for Zolgensma para que sirve. For more information and to find out if you’re eligible, call 855-441-GENE (855-441-4363) or visit the program’s website.
Generic or biosimilar version
Zolgensma isn’t available in a biosimilar form. Zolgensma is a biologic drug that’s made from living cells. Non-biologic drugs are made from chemicals and can be copied exactly. A generic drug is an exact copy of the active drug in a brand-name medication.
Biologic drugs can’t be copied exactly. Instead of generics, biologics have biosimilars. Biosimilars are considered as safe and effective as their parent biologic drugs. Like generics, biosimilars usually cost less than brand-name drugs.
Other drugs can treat spinal muscular atrophy (SMA), and some may be a better fit for your child than others. If you’re interested in finding an alternative to Zolgensma, talk with your child’s doctor. They can tell you about other medications that may work well to treat SMA.
Examples of other drugs that may be used to treat SMA include:
- risdiplam (Evrysdi)
- nusinersen (Spinraza)
The Food and Drug Administration (FDA) approves prescription drugs such as Zolgensma to treat certain conditions. Zolgensma may also be used off-label for other conditions. Off-label drug use means using a drug for a purpose other than what it’s been approved for by the FDA.
Zolgensma is FDA-approved to treat spinal muscular atrophy (SMA) caused by genetic changes in the SMN1 gene. For this purpose, Zolgensma is given to children less than 2 years old.
Zolgensma para que sirve has certain limitations of use. Specifically:
- Zolgensma is given as a one-time dose. Clinical studies haven’t looked at how effective the drug may be if a child receives more than one dose.
- Zolgensma hasn’t been studied as a treatment for advanced SMA. “Advanced” means the disease has worsened. This may lead to conditions such as paralysis of the arms and legs or needing to use a ventilator to breathe.
About spinal muscular atrophy
SMA is a rare condition that damages nerve cells in the brain and spinal cord. It’s caused by SMN1 genes that are missing or aren’t working properly. Over time, the damaged nerve cells become weaker and eventually stop working. This leads to muscle weakness and trouble with basic activities such as breathing, speaking, swallowing, and walking.
There are five types of SMA:
- SMA type 0 (the most severe form of SMA)
- SMA type 1 (the most common form of SMA)
- SMA type 2
- SMA type 3
- SMA type 4 (the least severe form of SMA, and it typically appears in adults)
Symptoms of several types of SMA appear very early in life, often before 18 months of age. These symptoms can include:
- floppy muscles
- inability to kick while lying on the back
- inability to sit
- problems eating, such as choking or trouble swallowing
- trouble breathing
- weak reflexes
Effectiveness for spinal muscular atrophy
Zolgensma precio has been found effective for treating SMA. Currently, Zolgensma is the only gene therapy available for the treatment of this condition.
For more information about how the drug performed in clinical studies, see Zolgensma’s prescribing information.
Zolgensma and children
Zolgensma precio is only FDA-approved to treat SMA in children less than 2 years old. It isn’t approved for use in people older than this.
Before your child receives their Zolgensma dose, their doctor will prescribe a corticosteroid (a drug that helps treat inflammation). Prednisolone is an example of a corticosteroid. Your child will start taking this drug a day before their Zolgensma dose. They’ll need to take the corticosteroid for at least 30 days.
Taking corticosteroids with Zolgensma treatment helps prevent high levels of liver enzymes, which can lead to serious liver damage.* The doctor will monitor your child’s liver function before their Zolgensma dose and for at least 3 months afterward. If your child shows any signs of liver problems, they’ll continue taking corticosteroids until their liver function improves.
Reviews
There are no reviews yet.