Zolgensma, the revolutionary gene therapy drug used to treat spinal muscular atrophy (SMA), has been making headlines for its astronomical price tag. The U.S. Food and Drug Administration (FDA) in May 2019 approved Zolgensma to treat pediatric patients under 2 with SMA.
It includes those who are pre-symptomatic or who have already developed symptoms. It is currently the most expensive drug in the world. However, the high cost of Zolgensma has sparked controversy and raised questions about its accessibility and affordability.
Especially in countries like India, where healthcare costs are a significant barrier to treatment.
What is Zolgensma?
Zolgensma is a gene therapy used to treat spinal muscular atrophy (SMA), a rare and often fatal genetic disease that affects the muscles used for movement. Zolgensma is a one-time treatment that delivers a functional copy of the SMN1 gene.
It is mutated or missing in patients with SMA. The therapy uses a harmless adeno-associated virus (AAV) to deliver the functional gene to the patient’s cells.
Zolgensma has shown promising results in clinical trials, with many patients experiencing significant motor function and survival improvements. However, Zolgensma is still a relatively new and complex treatment, like all gene therapies.
Its long-term safety and effectiveness are still being studied. Discussing the potential risks and benefits of Zolgensma with a qualified healthcare professional before considering the treatment is vital.
For Which Diseases Zolgensma is Used?
Zolgensma is a gene therapy drug that is used to treat spinal muscular atrophy (SMA). SMA is a rare, genetic disorder that affects the nervous system. It is caused by a mutation in the SMN1 gene, which results in a deficiency of the survival motor neuron (SMN) protein. This protein is essential for the development and function of motor neurons, which control muscle movement.
Zolgensma is a one-time treatment that is administered by a single intravenous infusion. It works by delivering a working copy of the SMN1 gene into the patient’s cells. This allows the cells to produce more SMN protein, which can help to improve muscle function and slow the progression of the disease.
Zolgensma was approved by the U.S. Food and Drug Administration (FDA) in May 2019 for the treatment of SMA in children under the age of two. It is the most expensive drug in the world, costing $2.125 million per dose.
Zolgensma has been shown to be effective in slowing the progression of SMA and improving the quality of life for patients. In a clinical trial, children who received Zolgensma at an average age of 6.9 months were able to sit independently for the first time at an average age of 14.5 months. They were also able to reach for objects and crawl at an earlier age than children who did not receive the treatment.
Zolgensma is not without risks. The most common side effects are fever, headache, and muscle weakness. Serious side effects, such as inflammation of the brain or spinal cord, can occur in rare cases.
If you are considering Zolgensma for your child, it is important to talk to your doctor about the risks and benefits of the drug.
Benefits of Zolgensma
Zolgensma is a revolutionary gene therapy that has shown remarkable benefits in treating spinal muscular atrophy (SMA), a rare genetic disorder that can cause muscle weakness and respiratory failure.
Zolgensma is a one-time treatment that replaces the missing or defective SMN1 gene, which is responsible for producing a protein critical for the survival of motor neurons.
Here are some of the benefits of Zolgensma:
1. Improvement in motor function
One of the primary benefits of Zolgensma is the improvement in motor function that it can provide. Children with SMA typically experience progressive muscle weakness lead to difficulty with motor functions such as crawling, walking, and even breathing.
In clinical trials, Zolgensma has shown significantly improved motor function in infants and children with SMA. It allows them to achieve milestones such as sitting up, crawling, and walking. This improvement in motor function can impact a child’s quality of life and independence.
2. Increased survival
SMA is a life-threatening condition that can significantly reduce life expectancy. Infants with the most severe form of SMA typically do not survive beyond their first few years of life. However, studies have shown that treatment with Zolgensma can significantly increase survival rates in infants with SMA.
In clinical trials, infants who received Zolgensma had a much higher chance of survival than those who did not receive the therapy.
3. Reduced need for respiratory support
SMA can cause respiratory problems that require assistance with breathing. Infants with SMA may require mechanical ventilation or other respiratory support to help them breathe. However, Zolgensma has reduced the need for respiratory support in infants and children with SMA.
In clinical trials, many infants who received Zolgensma could breathe independently without mechanical ventilation.
4. Improved quality of life
SMA can have a significant impact on a child’s quality of life. Children with SMA may experience chronic pain, difficulty with movement, and reduced independence. However, treatment with Zolgensma can improve the overall quality of life for infants and children with SMA.
By improving motor function, reducing the need for respiratory support, and allowing children to participate in activities that they may not have been able to before, Zolgensma can significantly improve the overall quality of life for patients and their families.
5. Potential for lifelong benefits
One of the most significant benefits of Zolgensma is the potential for lifelong benefits. Zolgensma is a one-time treatment that replaces the missing or defective SMN1 gene with a functional copy of the gene.
It means that infants and children who receive the therapy may continue to experience improvements in motor function and quality of life for many years. Some studies have shown that the benefits of Zolgensma can last up to five years after treatment.
6. Cost-effectiveness
Despite the high cost of Zolgensma, several studies have shown that it is a cost-effective treatment for SMA. In a study published in the Journal of Managed Care & Specialty Pharmacy, researchers found that the cost of Zolgensma was comparable to the cost of other treatments for SMA when considering the therapy’s long-term benefits.
The study also found that Zolgensma was more cost-effective than other treatments regarding quality-adjusted life years (QALYs) gained.
7. Minimally invasive
Zolgensma is administered through a one-time intravenous infusion, making it a minimally invasive treatment option. Unlike other treatments for SMA, such as Spinraza, which require multiple injections for several years, Zolgensma only requires a single infusion. It can significantly reduce the burden on patients and their families.
Overall, the benefits of Zolgensma in treating SMA are significant and can potentially transform the lives of patients and their families. While the cost of the therapy can be a significant barrier to access, efforts are being made to make the treatment more affordable and accessible to those who need it most.
Zolgensma Injection Price in India
Zolgensma has not yet been approved for use in India, but that hasn’t stopped patients and families from seeking access to the drug. The high cost of Zolgensma has prompted some families to launch crowdfunding campaigns to raise the funds needed for treatment, while others have turned to international markets to obtain the drug. As of May 2023, Zolgensma’s price in India is around INR 16 crore.
It is important to note that the drug is administered as a single intravenous (IV) injection, which can significantly impact the overall cost of treatment. The Zolgensma injection price in India is around INR 16 crore, making it one of the most expensive drugs in the world.
This high cost is due to the complex and expensive manufacturing process of producing the therapy. While the Zolgensma injection price in India is undoubtedly high, it’s important to remember that the therapy has shown great success in treating SMA and can potentially transform the lives of patients and their families.
With continued efforts to make the treatment more affordable and accessible, we can hope that more patients in India and around the world can access this life-changing therapy.
Why is Zolgensma so Expensive?
Zolgensma, a gene therapy approved for treating spinal muscular atrophy (SMA), is one of the most expensive drugs on the market, with a price tag of over INR 16 crore. But why is Zolgensma so expensive?
The high cost of Zolgensma has generated controversy and debate, with many questioning why a single treatment can cost so much. Here are some of the reasons why Zolgensma is so expensive:
1. Research and Development Costs
Developing a new drug or therapy is an expensive and time-consuming process. Pharmaceutical companies invest millions of dollars in research and development to bring a new therapy to market. In the case of Zolgensma, the therapy was developed by AveXis, a biotech company that was acquired by Novartis in 2018 for $8.7 billion.
The high cost of developing Zolgensma, which included clinical trials, regulatory approval, and manufacturing, is a significant factor in the drug’s high price.
2. Small Market Size
SMA is a rare genetic disorder that affects approximately 1 in 10,000 live births. The small market size for Zolgensma means that the cost of developing and manufacturing the therapy must be spread across a relatively small number of patients.
It can drive up the cost of the therapy, as the manufacturer must recoup its costs from a limited patient population.
3. One-Time Treatment
Zolgensma is a one-time treatment that provides long-lasting benefits. While this is a significant benefit for patients and their families, it also means that the manufacturer can only sell the therapy once to each patient. It differs from traditional pharmaceuticals, which are often taken daily or weekly and provide ongoing revenue for the manufacturer.
4. Manufacturing Costs
Gene therapies like Zolgensma are complex, requiring specialised facilities and processes. The manufacturing process for Zolgensma involves a viral vector to deliver a functional copy of the missing or defective SMN1 gene to patients.
The viral vector must be carefully produced and purified to ensure it is safe and effective for patients. The cost of manufacturing Zolgensma is a significant factor in the drug’s high price.
5. Health Outcomes
Zolgensma provides significant health benefits to patients with SMA, including improved motor function, increased survival, and a reduced need for respiratory support. These benefits can significantly impact the quality of life for patients and their families.
The manufacturer of Zolgensma argues that the therapy is priced based on the value it provides to patients rather than its cost of production.
6. Cost-Effectiveness
Despite the high upfront cost of Zolgensma, some studies have shown that the therapy can be cost-effective in the long term. A study published in the Journal of Managed Care & Specialty Pharmacy found that Zolgensma was cost-effective compared to other treatments for SMA when considering the therapy’s long-term benefits.
The study also found that Zolgensma was more cost-effective than other treatments regarding quality-adjusted life years (QALYs) gained.
7. Insurance Coverage
The manufacturer of Zolgensma has also established patient assistance programs to help patients access the therapy, regardless of their ability to pay. While the high cost of Zolgensma may be a barrier to access for some patients, insurance coverage and patient assistance programs can help to mitigate this barrier.
Thus, the high cost of Zolgensma is due to several factors, including the high price of research and development, the small market size for SMA, the one-time nature of the therapy, and the complex manufacturing process.
Is Zolgensma Approved In India?
No, Zolgensma is not currently approved for use in India. It was approved by the US Food and Drug Administration (FDA) in May 2019, and it is currently available in the US, Europe, and other countries. However, it has not yet been approved by the Drug Controller General of India (DCGI).
The DCGI is currently reviewing Zolgensma for approval. The review process is expected to take several months. If Zolgensma is approved, it is likely to be available at a lower cost than it is currently in the US.
In the meantime, there are a few ways that people in India can access Zolgensma. One way is to participate in a compassionate use program. These programs allow people to receive experimental drugs that are not yet approved for public use. To qualify for a compassionate use program, patients must meet certain criteria, such as having a life-threatening condition and having no other treatment options.
Another way to access Zolgensma is to import it from another country. This can be a complicated process, as it requires obtaining government approval and paying for the drug. However, it may be the only option for some people who need Zolgensma.
Which Authority Can Approve Zolgensma in India?
The Drug Controller General of India (DCGI) is the regulatory body responsible for approving new drugs in India. The DCGI will consider a number of factors when making a decision on whether to approve Zolgensma, including the safety and efficacy of the drug, the cost of the drug, and the availability of other treatment options.
In addition to the DCGI, the Indian government may also consider other factors when making a decision on whether to approve Zolgensma. For example, the government may consider the economic impact of approving the drug, the potential impact on the healthcare system, and the public interest.
The approval process for Zolgensma in India is likely to be lengthy and complex. However, there is a possibility that the drug could be approved in the near future. If Zolgensma is approved, it will be a major breakthrough for patients with SMA in India.
Here are some additional details about the DCGI:
- It is a government agency under the Ministry of Health and Family Welfare.
- It is responsible for ensuring the safety, quality, and efficacy of drugs in India.
- It has the authority to approve or reject new drugs for sale in India.
Is Zolgensma Available In India?
Zolgensma is not currently available for purchase in India. It is a gene therapy drug that is used to treat spinal muscular atrophy (SMA), a genetic disorder that affects the nervous system. The drug is very expensive, costing around ₹16 crores (US$20 million) per dose. This makes it unaffordable for many people in India.There are a few ways that people in India can access Zolgensma. One way is to participate in a compassionate use program. These programs allow people to receive experimental drugs that are not yet approved for public use. To qualify for a compassionate use program, patients must meet certain criteria, such as having a life-threatening condition and having no other treatment options.
Another way to access Zolgensma is to import it from another country. This can be a complicated process, as it requires obtaining government approval and paying for the drug. However, it may be the only option for some people who need Zolgensma.
The Indian government is currently considering approving Zolgensma for public use. If the drug is approved, it will be available at a much lower cost than it is currently. This will make it more affordable for people in India who need it.
Zolgensma Success Rate
Despite its high cost, Zolgensma has shown promising results in clinical trials, with many patients experiencing significant improvements in motor function and survival. In one clinical trial, for example, infants with SMA who received Zolgensma had a 95% survival rate at 24 months, compared to a 31% survival rate for untreated infants.
The Zolgensma success rate has led to calls for the treatment to be made more affordable and accessible to patients who need it. Advocates argue that the high cost of the treatment is a barrier to access for many patients, especially in developing countries where healthcare costs are significantly lower.
Important Things to Know About Zolgensma
- It is a one-time treatment that is administered by injection.
- It is designed to replace a missing or defective gene that causes SMA.
- It has been shown to be effective in slowing the progression of SMA and improving the quality of life for patients.
- It is the most expensive drug in the world.
Why Choose Ketto For Raising Funds For Zolgensma?
Raising funds for Zolgensma treatment can be a challenging task, given the high cost of the treatment. However, there are several strategies that patients and their families can use to raise funds for the treatment, and Ketto can be an effective platform for doing so. Here are some tips on how to raise funds for Zolgensma using Ketto:
1. Start a crowdfunding campaign
Ketto is an online crowdfunding platform that allows patients and their families to create a campaign to raise funds for Zolgensma treatment. The campaign should include a compelling story that explains the patient’s condition and why Zolgensma is necessary, as well as information about the cost of the treatment and how the funds will be used.
2. Share the campaign on social media
Once the campaign is created, sharing it on social media is important to reach a wider audience. Share the campaign on Facebook, Twitter, and other social media platforms, and encourage friends and family to share it.
3. Reach out to local media
Local media outlets can be a great way to spread the word about the campaign and raise awareness about the patient’s condition. Contact local newspapers, TV stations, and radio stations, and ask if they would be willing to cover the story.
4. Host a fundraising event
Hosting a fundraising event, such as a charity walk or run, can be a great way to raise funds for Zolgensma treatment. Use the event to promote the crowdfunding campaign and encourage attendees to donate.
5. Utilise Ketto’s tools and resources
Ketto provides several tools and resources to help patients and their families raise funds for Zolgensma treatment. These include email templates, social media graphics, and fundraising tips and advice.
Ketto provides a platform for patients and their families to raise funds for Zolgensma treatment and other medical expenses related to SMA. By creating a campaign on Ketto, patients and their families can reach out to a wider audience and share their stories with others who may be willing to donate to their cause.
Ketto also provides a secure platform for donations, ensuring the funds raised are used for the intended purpose. The platform allows donors to make one-time donations or set up recurring donations. It provides various payment options to make it easy for people to donate from anywhere in the world.
Overall, Ketto can be a useful tool for patients and their families seeking to raise funds for Zolgensma treatment and other medical expenses related to SMA. It provides a platform for patients to share their stories, connect with a wider audience, and allows donors to contribute to a cause they believe in.
Conclusion
In conclusion, Zolgensma is a highly effective gene therapy with a high success rate in treating spinal muscular atrophy (SMA), a rare and often fatal genetic disease. However, the high cost of the treatment can be a significant barrier to access, especially in countries like India, where healthcare costs are generally lower.
However, several options are available for patients and their families to raise funds for Zolgensma treatment, including crowdfunding platforms like Ketto. By creating a compelling campaign and reaching a wider audience, patients and their families can increase their chances of raising the necessary funds to access this life-saving treatment.
Ketto is a popular crowdfunding platform that allows individuals, organisations, and nonprofits to raise funds for various causes. In addition to helping people raise funds, Ketto offers various resources, including fundraising tips, marketing support, and crowdfunding consulting services.
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