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Zolgensma vs Spinraza
Introduction
For patients diagnosed with spinal muscular atrophy (SMA), a rare and often fatal genetic disorder that affects motor neurons leading to muscle wasting and respiratory failure, certain treatments can help manage symptoms and improve survival. Zolgensma and Spinraza are two such drugs prescribed for SMA. They each work on different aspects of the disease but both have significant effects in children with SMA. Zolgensma is a gene therapy drug, it works by delivering a fully functional copy of the human SMN gene into the target motor neuron cells thus increasing survival motor neuron protein levels in children's bodies. On the other hand, Spinraza is an antisense oligonucleotide that modifies splicing of the SMN2 pre-mRNA to include exon 7, which results in increased production of full-length Survival Motor Neuron (SMN) protein.
What is Zolgensma?
Onasemnogene abeparvovec-xioi (the generic name for Zolgensma) is a gene therapy that was approved by the FDA in 2019, marking a major advancement over traditional treatment options for spinal muscular atrophy (SMA). Zolgensma works by introducing a functional copy of the survival motor neuron (SMN1) gene to halt disease progression with just one intravenous administration. It is prescribed for pediatric patients less than two years of age.
On the other hand, Nusinersen (the generic name for Spinraza), which was first approved by the FDA in 2016, increases production from an existing but usually non-functional backup SMN gene through repeated injections into the central nervous system. Both treatments target the root genetic cause of SMA and have demonstrated significant benefits in clinical trials; however, their different administration methods and dosing schedules may influence side effects and patient preference.
What conditions is Zolgensma approved to treat?
Zolgensma and Spinraza are both approved for the treatment of spinal muscular atrophy (SMA):
- Zolgensma is a one-time gene therapy used primarily for children under 2 years old, including those who are pre-symptomatic at diagnosis.
- Spinraza is an ongoing treatment given throughout the patient's lifetime. It can be used in individuals with SMA of all ages, from infants to adults.
How does Zolgensma help with these illnesses?
Zolgensma and Spinraza are both groundbreaking treatments for spinal muscular atrophy (SMA), a genetic disorder that affects motor neurons in the spinal cord, causing muscle weakness and atrophy. Zolgensma works by replacing the missing or faulty SMN1 gene with a fully functional copy. This allows the body to produce adequate levels of survival motor neuron protein which is crucial for muscle control. Once administered, Zolgensma offers a one-time treatment option that can prevent further progression of SMA.
On the other hand, Spinraza operates differently as it modifies the alternative SMN2 gene to boost its production of functional survival motor neuron protein. Unlike Zolgensma's one-time administration approach, Spinraza requires ongoing regular injections into the cerebrospinal fluid.
Both medications aim to increase availability of functional survival motor neuron proteins - essential elements responsible for controlling muscle movement - in patients suffering from SMA thus helping manage their condition and improve their quality of life.
What is Spinraza?
Spinraza is a brand name for nusinersen, which is an antisense oligonucleotide. It works by binding to the pre-mRNA and influences splicing to increase production of full-length survival motor neuron (SMN) protein. This protein plays a vital role in maintaining motor neurons; insufficient levels of SMN protein, as found in individuals with Spinal muscular atrophy (SMA), results in loss of motor neurons and muscle weakness.
Spinraza was first approved by the FDA in 2016, becoming the first disease-modifying therapy available for SMA. Unlike gene therapies such as Zolgensma that are administered once, Spinraza requires ongoing treatment through injections into the spinal fluid every four months after initial loading doses.
Side effects may vary among patients but can include respiratory infections and complications related to lumbar puncture procedure. Because it sustains SMN protein production over time rather than providing a one-time replacement like Zolgensma, it could potentially be beneficial for those who have later-onset forms of SMA or are diagnosed at an older age.
What conditions is Spinraza approved to treat?
Spinraza is an approved therapy for the treatment of spinal muscular atrophy (SMA), a genetic disorder characterized by weakness and muscle wasting due to the loss of motor neurons controlling movement. This medication can be used in patients across all types of SMA, regardless of disease onset (infantile, juvenile or adult). It's also approved for use in:
- Presymptomatic newborns diagnosed with SMA
- Children and adults manifesting symptoms related to SMA
How does Spinraza help with these illnesses?
Spinraza, an antisense oligonucleotide, works by altering the splicing of SMN2, a gene that is nearly identical to SMN1. This alteration enables it to produce more functional survival motor neuron (SMN) protein in patients with spinal muscular atrophy (SMA), a condition characterized by loss of motor neurons and progressive muscle wasting. Spinraza achieves this through its unique mechanism which involves being directly delivered into the central nervous system via intrathecal injection. Unlike Zolgensma which is a one-time gene therapy treatment replacing the function of the missing or nonworking SMN1 gene completely, Spinraza offers continuous management for SMA and may be prescribed when there are specific considerations such as age or disease severity that favor ongoing treatments.
How effective are both Zolgensma and Spinraza?
Onasemnogene abeparvovec (Zolgensma) and nusinersen (Spinraza) are both FDA approved treatments for spinal muscular atrophy, a genetic disease characterized by the loss of motor neurons leading to muscle weakness. Zolgensma was approved in 2019 while Spinraza has been in use since its approval in 2016.
Zolgensma is a gene therapy that works by replacing the faulty or missing survival motor neuron 1 gene which causes SMA. It's administered as a one-time intravenous infusion and is currently approved for pediatric patients less than two years old. Evidence from clinical trials shows that Zolgensma can significantly improve motor function and survival rates among infants with SMA type 1.
Nusinersen (Spinraza), on the other hand, doesn't replace the faulty gene but instead modifies the way it's processed within cells. This drug requires repeated lumbar puncture administrations throughout life with loading doses initially followed by maintenance doses every four months thereafter. Several clinical trials have shown substantial improvement in motor milestones and slowed disease progression across various types of SMA when treated with Spinraza.
Both treatments exhibit promising safety profiles relative to their benefits; however, they come with different risks largely linked to their methods of administration: potential liver damage for Zolgensma versus possible complications associated with repeated spinal injections for Spinraza.
A recent review showed evidence suggesting greater efficacy from early initiation of treatment regardless of whether using either Zolgensma or Spinraza indicating an important role for newborn screening programs.
Further research including head-to-head comparisons will be vital in understanding how these therapies compare regarding long-term effectiveness, safety profile, quality-of-life improvements, and cost-effectiveness given their considerable price difference – about $2 million USD per single-dose treatment course for Zolgensma compared to ongoing costs estimated at around $750k USD annually over lifetime treatment span using Spinraza.
At what dose is Zolgensma typically prescribed?
Zolgensma is a gene therapy administered as a one-time intravenous infusion. The drug's dosage is determined by the patient's weight at the time of dosing and is usually administered only once in a lifetime. On the other hand, Spinraza (nusinersen) requires an initial loading dose that includes four 12 mg injections administered intrathecally over a period of about two months, followed by maintenance doses given every four months thereafter for life. Dosing adjustments are not necessary based on age or body weight, but response to treatment should be regularly assessed.
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At what dose is Spinraza typically prescribed?
Spinraza treatment is typically initiated with a loading dose. The initial phase involves four doses of 12 mg each, given by injection into the spinal canal, spaced 14 days apart for the first two doses, and then 28 days after the second and third dose. Following this initial phase, a maintenance dosage of one injection every four months is recommended to sustain improvement in motor function. It's important to note that if there's no noticeable change after several weeks post-treatment initiation, don't rush assumptions - treatments like Spinraza may take time before significant improvements are observed.
What are the most common side effects for Zolgensma?
Common side effects of Zolgensma and Spinraza differ significantly due to their distinct mechanisms of action. Potential side effects for Zolgensma include:
- Elevated liver enzymes, indicating potential liver damage
- Vomiting
- Decreased platelet levels
- Decreased total protein in the blood
For Spinraza, common side effects may include:
- Respiratory infections such as colds and lung infections
- Fever
- Headache
- Vomiting
- Back pain Remember that every patient's experience with a medication can be unique, so this list does not cover all possible side effects. Always consult your healthcare provider to discuss any potential risks or concerns.
Are there any potential serious side effects for Zolgensma?
Zolgensma and Spinraza are treatments for spinal muscular atrophy. While they both have potential side effects, those associated with Zolgensma include:
- Elevated liver enzymes or other signs of liver damage: symptoms may include yellowing of the skin or whites of the eyes (jaundice), dark urine, severe nausea or vomiting, pain on the upper right side of your stomach area
- Hypersensitivity reactions: hives, itchy rash over the whole body, wheezing and trouble breathing, rapid heartbeat, sweating
- Low platelet levels - leading to unusual bruising or bleeding
- Decreased motor function - inability to use muscles that were previously under control
On the other hand, common serious adverse events related to Spinraza treatment might involve:
- Respiratory infection: coughing up mucus (phlegm), shortness of breath during rest or activity.
- Heart rate disturbances: unusually slow heart rate which can be dangerous in severe cases.
- Kidney problems marked by lower back pain and difficulties urinating.
In either case if these symptoms occur you should seek immediate medical attention.
What are the most common side effects for Spinraza?
Spinraza, another treatment option for spinal muscular atrophy (SMA), may have potential side effects including:
- Headache and back pain
- Mild to moderate skin reactions, such as redness, rash or itching around the injection site
- Respiratory infections such as colds and lung infections
- Constipation
- Increased risk of bleeding complications due to low platelet count
- Kidney damage in severe cases
It's important to note that these are possible side effects. Every patient can react differently, so it's crucial to discuss any health concerns with your healthcare provider before starting new medication.
Are there any potential serious side effects for Spinraza?
While generally considered safe, Spinraza can lead to certain side effects that might require immediate medical attention. These include:
- Signs of an allergic reaction such as hives, itching, rash or difficulty breathing
- Symptoms of kidney damage including changes in the amount or color of urine
- Severe back pain indicating potential damage at the injection site
- Unusual bruising or bleeding which may be signs of low platelet counts
- Breathing difficulties and chest tightness due to respiratory infections
Always seek immediate medical care if you notice any unusual reactions after receiving a dose of Spinraza. It's vital to remember that all medications carry some risk and it’s crucial to weigh these against their potential benefits under your doctor's guidance.
Contraindications for Zolgensma and Spinraza?
Both Zolgensma and Spinraza, along with most other Spinal muscular atrophy (SMA) medications, may lead to complications in some people. If you notice any worsening of your condition or any new symptoms, please seek immediate medical help.
Zolgensma is gene therapy administered as a one-time intravenous infusion while Spinraza requires repeated injections into the spinal fluid. Neither Zolgensma nor Spinraza should be given if you have advanced liver disease or an abnormality in certain liver enzymes. Always inform your healthcare provider about all medications and health conditions prior to starting treatment; special tests might be required to check liver function before administering these therapies.
Spinraza has been shown to increase the risk of bleeding complications and kidney damage in some patients who have severe thrombocytopenia or coagulation disorders so caution must be exercised under those circumstances.
On the other hand, Zolgensma can cause acute serious liver injury. Liver enzymes could elevate within just two weeks following infusion with Zolgensma and reach more than 20 times the upper limit of normal levels.
Therefore it's important that doctors closely monitor patients for at least three months after administration of either medication by checking complete blood counts and conducting regular assessments for potential signs of an adverse event such as unexpected bruising or bleeding which may indicate a possible effect on platelet count or clotting factor deficiencies.
How much do Zolgensma and Spinraza cost?
For the brand name versions of these drugs:
- The price of Zolgensma is about $2.1 million for a one-time infusion.
- Spinraza costs around $750,000 for the first year and then approximately $375,000 per year after that.
Thus, if you calculate it over 10 years (for simplicity), Spinraza would cost approximately $3.75 million compared to Zolgensma's one-time fee. Therefore, in terms of long-term treatment costs, Zolgensma could be less expensive than continuing with yearly doses of Spinraza. However, please note that cost should not be the primary consideration in determining which of these drugs is right for treating spinal muscular atrophy (SMA). Both medications have different administration routes and potential side effects which should also be considered.
Currently there are no generic versions available for either drug due to their relatively recent FDA approval and patent protection status. It's important to discuss all options with your healthcare provider before making any decisions regarding treatment plans.
Popularity of Zolgensma and Spinraza
Zolgensma and Spinraza are two revolutionary treatments for spinal muscular atrophy (SMA), a rare genetic disorder marked by loss of motor neurons, muscle weakness and atrophy.
Spinraza was the first disease-modifying treatment approved for SMA in 2016. It is given via intrathecal injection several times per year for life. In contrast, Zolgensma is a gene replacement therapy that corrects the underlying genetic defect causing SMA with just one infusion. Approved by FDA in 2019, it represents an entirely new approach to treating inherited diseases.
While exact prescription numbers are difficult to find due to the rarity of SMA, both drugs have been game-changers in managing this previously untreatable condition. The choice between these two therapies often comes down to specific patient factors such as age at diagnosis, severity of symptoms, insurance coverage and logistics related to repeated injections versus single infusion.
Conclusion
Both Zolgensma (onasemnogene abeparvovec-xioi) and Spinraza (nusinersen) are breakthrough treatments for spinal muscular atrophy, a rare, inherited neuromuscular disease. Backed by extensive clinical trials and real-world experience, both have shown significant effectiveness over traditional supportive care.
Zolgensma is a gene therapy that works by delivering a functional copy of the SMN1 gene to motor neuron cells, helping them produce the SMN protein necessary for muscle function. It's administered as a one-time intravenous infusion.
Spinraza, on the other hand, works differently - it modifies the patient’s backup SMN2 gene to produce more functional SMN protein. Unlike Zolgensma, Spinraza requires ongoing administration via lumbar puncture every four months after an initial loading dose period.
Both therapies come with high costs due to their innovative nature and rarity of condition they treat; however there are assistance programs available for eligible patients. Both may cause side effects: Zolgensma can cause elevated liver enzymes while Spinraza has been associated with respiratory infection risks and potential kidney damage in rare cases. As these are serious conditions requiring specialist intervention anyway, any decision between these two treatments should be made in careful consultation with experienced neurologists who specialize in treating SMA.