OneGene ProgramTM to support patients and families along their treatment journey with Zolgensma AveXis patient support program, called OneGene Program TM, is a comprehensive, individualized support program that provides a dedicated, personalized support team focused on the needs of each family throughout the Zolgensma treatment journey.
Full Answer
How is thrombocytopenia treated after Zolgensma infusion?
Administer a systemic corticosteroid to all patients before and after ZOLGENSMA infusion. Continue to monitor liver function for at least 3 months after infusion. Transient decreases in platelet counts, some of which met the criteria for thrombocytopenia, were typically observed within the first two weeks after ZOLGENSMA infusion.
What services does the onegene program offer?
Translation services available upon request. The OneGene Program is a team of highly trained and dedicated people who help ensure that every family has the support they need during their treatment journey and beyond.
Does Zolgensma increase troponin I?
Elevated Troponin-I Transient increases in cardiac troponin-I levels (up to 0. 176 mcg/L) were observed following Zolgensma infusion in clinical trials. The clinical importance of these findings is not known. However, cardiac toxicity was observed in animal studies.
Is Zolgensma inpatient or outpatient?
Zolgensma will primarily be administered in the hospital outpatient setting. Unlike other gene therapy products currently on the market, Zolgensma will not be restricted to certain hospital sites.
How much does Zolgensma treatment cost?
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 a one-time treatment?
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 many patients are treated with Zolgensma?
About Zolgensma Zolgensma is now approved in more than 40 countries and more than 1,800 patients have been treated with Zolgensma globally across clinical trials, managed access programs, and in the commercial setting.
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.
Does Medicare cover Zolgensma?
Medicare does not have an NCD for Zolgensma (onasemnogene abeparvovec-xioi).
What is life expectancy after Zolgensma?
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.
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.
Is Zolgensma a permanent cure?
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.
What is the life expectancy of someone with SMA?
The life expectancy of patients with spinal muscular atrophy (SMA) type I is generally considered to be less than 2 years.
What happens after Zolgensma?
Infections before or after ZOLGENSMA infusion can lead to more serious complications. Contact the patient's doctor immediately if you see any signs of a possible infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
How often is Zolgensma given?
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.
Does Zolgensma completely cure SMA?
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.
How long will Zolgensma work?
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.
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 much does SMA treatment cost?
In the USA, nusinersen, the first therapy for spinal muscular atrophy approved in 2016, costs $750 000 in the first year and $375 000 every following year for a patient's lifetime compared with onasemnogene abeparvovec, which costs $2.1 million for a one-off injection in five $425 000 instalments.
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 is Zolgensma used for?
ZOLGENSMA ® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA).
What is SMA Atlas?
SMA Atlas is where you will find an abundance of helpful resources about living with SMA, tips from experienced caregivers on the importance of building a complete healthcare team, and much more.
What is Zolgensma gene therapy?
Zolgensma (onasemnogene abeparvovec-xioi) is a proprietary gene therapy approved by the US Food and Drug Administration for the treatment of pediatric patients less than 2years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 ( SMN1) gene. Zolgensma is designed to address the genetic root cause ...
What is Zolgensma one time treatment?
One-time treatment with Zolgensma (onasemnogene abeparvovec-xioi) is designed to replace lifetime of chronic therapy for all pediatric patients with SMA
What is Avexis working on?
AveXis is working closely with payers to create 5-year outcomes-based agreements and novel pay-over-time options; Time is Neurons program to support rapid SMA treatment post-diagnosis
What is Zolgensma used for?
Zolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patient less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 ( SMN1) gene. Limitation of Use:
How many payers are in advanced discussions with Avexis?
Reflecting the pioneering nature of these programs, more than 15 payers are in advanced discussions of terms with AveXis, with some having already agreed, in principle, to terms.
How many patients were in the Str1ve trial?
The ongoing clinical trial, STR1VE, enrolled 21 patients (10 male and 11 female) with infantile-onset SMA. Before treatment with Zolgensma, none of the 21 patients required non-invasive ventilator (NIV) support, and all patients could exclusively feed orally (i.e., no need for non-oral nutrition). The mean CHOP-INTEND score at baseline was 31.0 (range 18 to 47). All the patients received 1.1 × 10 [1] [4] vg/kg of Zolgensma. The mean age of the 21 patients at the time of treatment was 3.9 months (range 0.5 to 5.9 months).
Does Avexis have a pay over time option?
AveXis has partnered with Accredo ® to offer a pay-over-time option of up to 5 years to help ease possible short-term budget constraints, especially for states, small payers and self-insured employers. In addition, CuraScript SD ® has been selected as the sole specialty distributor given its rare disease experience, including gene and cell therapies.
Indication
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.
Limitations of Use
The safety and effectiveness of repeat administration or the use in patients with advanced SMA (e.g., complete paralysis of limbs, permanent ventilator dependence) has not been evaluated with ZOLGENSMA.
Important Safety Information
BOXED WARNING: Acute Serious Liver Injury and Acute Liver Failure Acute serious liver injury, acute liver failure, and elevated aminotransferases can occur with ZOLGENSMA. Patients with preexisting liver impairment may be at higher risk.
WARNINGS AND PRECAUTIONS
Thrombocytopenia Transient decreases in platelet counts, some of which met the criteria for thrombocytopenia, were typically observed within the first two weeks after ZOLGENSMA infusion. Monitor platelet counts before ZOLGENSMA infusion and on a regular basis for at least 3 months afterwards.
ADVERSE REACTIONS
The most commonly observed adverse reactions (incidence ≥5%) in clinical studies were elevated aminotransferases and vomiting.
What is the ZOLGENSMA gene?
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?
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.
