Details

What is Zolgensma (onasemnogene abeparvovec) for?

Zolgensma (onasemnogene abeparvovec) is an adeno-associated virus vector-based gene therapy indicated for the treatment of:

It is given as a one-time-only dose.

Zolgensma (onasemnogene abeparvovec) is available in vial form containing 2 × 10^13 vector genomes/mL solution for infusion. It is provided as a customized kit to meet dosing requirements for each patient and comes with vials of onasemnogene abeparvovec.

How does Zolgensma (onasemnogene abeparvovec) work?

Onasemnogene abeparvovec, the active ingredient in Zolgensma, works by targeting the underlying mechanism of SMA: a lack in the survival of motor neuron (SMN) protein. This protein is crucial for the function of nerves that control muscle movements. In SMA patients, nerve cells are unable to work properly, and over time this leads to muscle weakness.

In most cases of SMA, the SMN1 gene is mutated. The production of SMN protein is directed primarily by SMN1, and partly by SMN2. However, the protein produced by the SMN2 gene is not stable.

Onasemnogene abeparvovec is a vector that contains a functional copy of the SMN gene. When injected, it passes into the nerves from where it replaces the function of the missing or nonworking SMN1 gene and it will tell the motor neuron cells to start making SMN protein. The new gene does not become part of the child’s DNA.

Onasemnogene abeparvovec is an adeno-associated virus vector-based gene therapy. This type of virus does not make people sick.

Although Zolgensma (onasemnogene abeparvovec) keeps motor neuron cells functioning and keeping strength in the muscles, it is not a cure and cannot reverse the damage already caused by SMA before treatment.

Where has Zolgensma (onasemnogene abeparvovec) been approved?

Zolgensma (onasemnogene abeparvovec) was approved for the treatment of children aged 2 years and below with SMA by:

Zolgensma (onasemnogene abeparvovec) received Fast Track, Breakthrough Therapy, and Priority Review designations by the FDA. Additionally, it received Orphan Drug designation, which provides incentives to encourage the development of drugs for rare diseases.

The FDA also awarded the manufacturer a rare pediatric disease priority review voucher, under a program intended to encourage the development of new drugs and biological products for the prevention and treatment of certain rare pediatric diseases.

Please note that this medicine may have also been approved in other regions than the ones we’ve listed. If you have a question about its approval in a specific country feel free to contact our support team.

How is Zolgensma (onasemnogene abeparvovec) taken?

The standard dosage is:

The medicine is given via slow intravenous infusion (into a vein) over 60 minutes.

Before Zolgensma (onasemnogene abeparvovec) can be given, several lab tests need to be completed:

A course of an oral corticosteroid, to be determined by the treating doctor, should be started the day before infusion with Zolgensma (onasemnogene abeparvovec). On the day of infusion, the second dose of the oral corticosteroid should be given. This helps manage increased liver enzyme reactions to the medicine by the body’s immune system.

Warning: Viral respiratory infection before or after infusion of the medicine can lead to more serious complications. If you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever, contact the treating doctor immediately.

Complete information about Zolgensma (onasemnogene abeparvovec) dosage (modification) and administration can be found in the official prescribing information listed in our references section.

Note: Please consult with your treating doctor for personalised dosing.

Are there any known adverse reactions or side effects of Zolgensma (onasemnogene abeparvovec)?

Common adverse reactions

The most common adverse reactions (≥10% of patients) listed in the prescribing information include:

Use in a specific population

References

Clinical trials

The Food and Drug Administration (FDA), USA approval of Zolgensma (onasemnogene abeparvovec) for the treatment of spinal muscular atrophy (SMA) in children aged 2 years and younger was based on the data of two phase 3 clinical trials: START (NCT02122952) and STR1VE (NCT03306277).

The START study was performed to establish safety and find the appropriate dose of onasemnogene abeparvovec.

The STR1VE study involved 22 children with SMA who were treated after symptoms appeared (symptomatic). All patients received Zolgensma (onasemnogene abeparvovec) through an intravenous infusion over 60 minutes. There was no placebo group. Results were measured against the natural history of SMA.

Results

The primary efficacy outcomes were reaching developmental motor milestones (e.g., head control and the ability to sit without support for at least 30 seconds at the 18-months-of-age study visit) and event-free survival. An event is defined as death, the need for permanent ventilatory support (such as tracheostomy), or the need for respiratory support (not due to illness or surgery) for ≥16 hours a day for at least 14 days.

These outcomes are a co-primary outcome which were assessed in sequence: the measure of functional independent sitting was determined first and, only when this endpoint met statistical significance, was the outcome of event-free survival assessed.

Zolgensma (onasemnogene abeparvovec) significantly increased event-free survival.

A majority of children reached the motor milestone at 16 months of age:

There is an ongoing long-term follow-up (LTFU) study that monitors the safety of many patients from the START study. The results show the safety and efficacy of Zolgensma up to 5 years after treatment and 5 years of age or older. As of December 2019, 100% (10/10) of patients were alive and did not need permanent breathing support, and 100% (10/10) have maintained motor milestones achieved at the end of the START study.

Please refer to the summary of product characteristics below and in the resource section for comprehensive information about the safety and effectiveness of Zolgensma (onasemnogene abeparvovec) for the approved indication.

References

Price & Costs

Zolgensma (onasemnogene abeparvovec) treatment costs

The cost for a monthly or yearly treatment of Zolgensma (onasemnogene abeparvovec) depends on your prescription requirements which includes the dosage in mg and medicine type (Vial).

The price of the medicines you see on sale is the cost set by the manufacturer. In addition, shipping costs and a Named Patient support fee will apply.

Make an enquiry and we will help you calculate the total price to buy Zolgensma (onasemnogene abeparvovec) online and have it delivered where you live. From your side, we’ll simply need a prescription from your treating doctor. In some countries we might also need an import license. Our Patient Support team will guide you throughout the process.

Zolgensma (onasemnogene abeparvovec) price and additional costs

Zolgensma (onasemnogene abeparvovec) reimbursement

It might be possible for you to claim the cost of Zolgensma (onasemnogene abeparvovec) via your country's healthcare system. Some countries have a government fund providing financial assistance for citizens to access lifesaving medical treatment abroad. The best thing would be to check with your country's own health ministry. We also highly recommend checking out local patient blogs, communities and patient organisations to find out more about your options.

Medical insurance

Generally, we do not support medical insurance, but we do provide you with an invoice after you make the payment that you may send to your medical insurance company so they can reimburse you. In some limited circumstances, we do accept insurance. Contact us for more details. You can pay by bank transfer or credit card.

Crowdfunding

Crowdfunding might also be an option for you. It is a means of raising funds by using an online platform to group together many small donations from friends, family and others, in order to help you afford medical treatment. Here are some crowdfunding platforms where you can seek support: GoFundMe, GiveForward, FundRazr and Watsi.

Shipping

Room temperature shipping

For this medication, standard shipping applies. This means that a consistent room temperature of 15 to 25°C is maintained during transit.

Your order will be shipped to you by our partner couriers, such as DHL, UPS and specialised cold chain couriers. If you would like your order to be shipped by a specific courier, please inform us when you place the order.

Our standard delivery time varies from 5 to 10 working days.

The shipping cost depends on the medicine and the destination country. After making an enquiry, you will receive a detailed quote that includes the shipping costs.

You can also pick up your medicine from our partner pharmacy in Amsterdam or our partner pharmacy in Luxembourg after the order has been paid for.

Our Process

Four steps to access the medicines you need

How and where to access and buy Zolgensma: You can access Zolgensma from everyone.org if the drug has not been approved or is not available in your country.