How do I get Zolgensma?
Zolgensma is given through an intravenous (IV) infusion that takes about an hour. It is a one-time treatment. Zolgensma is an SMN-enhancing therapy that works by replacing the function of the missing or nonworking SMN1 gene with a new, working copy of an SMN gene.
How much is Zolgensma treatment?
With a cost of $2.1 million for a one-dose treatment, Zolgensma is currently the most expensive drug in the United States. Zolgensma treats spinal muscular atrophy (SMA), a genetic disorder that causes muscle wasting and weakness.
Is Zolgensma curative?
Because Zolgensma is a “potentially curative one-off gene therapy” that can provide "exceptional benefit” to patients, NICE reviewers concluded it's worth the high cost in certain patients. For babies aged seven to 12 months, a “national multidisciplinary clinical team” should discuss using the medicine, NICE said.
Is Zolgensma cost effective?
A value-based price for Zolgensma was estimated at between $1.1 million to $1.9 million per treatment, given a cost-effectiveness threshold of $100,000 to $150,000 per quality-adjusted life year gained.
Why is Zolgensma not a cure?
However, zolgensma is not a cure: Timing is critical. Although the treatments can rapidly increase SMN protein production after administration, some irreversible damage may have already occurred in the nervous system. This may happen even before a baby is born.
How long does Zolgensma prolong life?
Zolgensma led to achievement of new milestones years after treatment – including sitting – with sustained durability in children now up to six years old and more than five years post-treatment.
Does insurance pay for Zolgensma?
Aetna rolls out in-network coverage for Zolgensma, other multimillion-dollar gene therapies. Aetna is launching a designated network to cover new gene therapies for rare diseases, the insurer announced Thursday.
How quickly does Zolgensma work?
How long does it take to work? Zolgensma starts working to treat SMA as soon as it's injected. In clinical studies of the drug, some children saw improvements in their SMA symptoms as early as 1 month after they received the drug.
Can SMA be cured completely?
It's not currently possible to cure spinal muscular atrophy (SMA), but research is ongoing to find new treatments. Treatment and support is available to manage the symptoms and help people with the condition have the best possible quality of life.
Is Zolgensma free in USA?
To date, our collective efforts in the US and globally have enabled approximately 170 babies and children around the world to receive Zolgensma free-of-charge. This is a significant percentage of the more than 700 patients in total who have received Zolgensma to date.
Is Zolgensma permanent?
Official answer. No, Zolgensma (onasemnogene abeparvovec-xioi) is not a cure for SMA (spinal muscular atrophy). In addition, it cannot reverse any damage to motor neurons already caused by SMA before treatment. Since damage is not reversible, early treatment with Zolgensma is very important.
Which countries cover Zolgensma?
Argentina | es.Brazil | pt.Canada | en.Canada | fr.Central America | es.Colombia | es.Mexico | es.United States | en.More items...•
How much does Zolgensma cost in the USA?
The current list price of Zolgensma is over $2 million, making it one of the most expensive drugs in both the U.S.A. and the world. But unlike most drugs, Zolgensma is a one-time treatment.
Which countries cover Zolgensma?
Argentina | es.Brazil | pt.Canada | en.Canada | fr.Central America | es.Colombia | es.Mexico | es.United States | en.More items...•
How quickly does Zolgensma work?
How long does it take to work? Zolgensma starts working to treat SMA as soon as it's injected. In clinical studies of the drug, some children saw improvements in their SMA symptoms as early as 1 month after they received the drug.
How fast does Zolgensma work?
Zolgensma is given as a one-time intravenous (IV) infusion that takes about one hour and is given at a treatment center. Zolgensma has been shown to: improve muscle strength. increase survival without the need for permanent breathing support.
What is Zolgensma for?
ZOLGENSMA ® (onasemnogene abeparvovec-xioi) is a gene therapy for pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) 1
What is the OneGene program?
The OneGene Program™, offered by Novartis Gene Therapies, is a comprehensive, individualized support program for families with pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) and their healthcare providers throughout the ZOLGENSMA treatment journey.
What to do if you have questions about Zolgensma?
If you have questions about ZOLGENSMA, talk to your doctor today.
What is zolgensma used for?
ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). It is given as a one-time infusion into a vein. Now that you and your child’s doctor have chosen ZOLGENSMA, there are a few steps that need to be taken to determine if your child qualifies for ZOLGENSMA.
How long does it take for Zolgensma to work?
On treatment day your child will be infused with ZOLGENSMA. The actual ZOLGENSMA infusion takes about 60 minutes. Your child will also receive a second dose of the oral corticosteroid. After infusion, your doctor and care team will talk to you about post-treatment follow-up and additional medical monitoring.
What test is done to confirm SMN1?
Your doctor will order a genetic test for your child to confirm the SMN1 gene is missing or nonworking. In addition, your doctor may also test your child’s SMN2 gene copy number. Your doctor will also need to perform additional lab tests—an AAV9 antibody test and baseline medical tests.
Can a child get Zolgensma right away?
If your child’s immune system has built up a certain level of anti-AAV9 antibodies, he or she may not qualify for ZOLGENSMA right away. If your child’s anti-AAV9 antibodies are too high, your doctor has the option to retest. Speak with your doctor about the AAV9 antibody test.
Does Zolgensma require a baseline?
Your doctor’s office will follow up on the status of your insurance coverage for ZOLGENSMA. If approved, and if not previously completed, your doctor will perform baseline medical tests before treatment with ZOLGENSMA .
Can you get a viral infection after Zolgensma?
Viral respiratory infection before or after ZOLGENSMA infusion can lead to more serious complications. Contact your doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
What to do after a child has been treated with Zolgensma?
After your child has been treated with ZOLGENSMA, it is important to understand how to continue managing SMA and other health needs. This guide outlines potential members of a complete healthcare team, types of supportive therapies your child’s doctor may recommend, and how a nutritionist can help ensure nutritional needs for growth and development are being met.
Does SMA continue after treatment?
The management and monitoring of SMA continues after treatment with ZOLGENSMA. This guide provides a timeline of tests your child will need following treatment, questions to ask your doctor, and the types of specialist that may be part of your child’s healthcare team.
What is the ZOLGENSMA gene?from zolgensmacopayassist.com
ZOLGENSMA is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Is Zolgensma valid for commercially insured patients?from zolgensmacopayassist.com
This offer is valid for eligible commercially insured patients and is good for use only with a ZOLGENSMA® (onasemnogene abeparvovec-xioi) prescription at the time the prescription is filled or after the product is administered to the patient.
What is zolgensma used for?
ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into a vein. ZOLGENSMA was not evaluated in patients with advanced SMA.
How long after Zolgensma can you use hand hygiene?
Temporarily, small amounts of ZOLGENSMA may be found in the patient’s stool. Use good hand hygiene when coming into direct contact with bodily waste for 1 month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.
How many people are affected by SMA?
SMA affects about 1 in every 11,000
Does Zolgensma change DNA?
ZOLGENSMA does not change or become a part of a child’s DNA. To help you understand how this is possible, let’s look at how ZOLGENSMA works to treat SMA. ZOLGENSMA targets the genetic root cause of SMA by replacing the function of a missing or nonworking gene, called the SMN1gene.
Does Zolgensma stop SMA?
ZOLGENSMA stops the progression of SMA with a one- time-only dose
Can Zolgensma cause liver damage?
ZOLGENSMA can cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA. Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.
