Zolgensma – Price In India, Availability, Approval & More

In this article, we will delve into the intricacies surrounding the Zolgensma price, a groundbreaking gene therapy developed for spinal muscular atrophy (SMA) treatment, a rare & often debilitating genetic disorder. Zolgensma has gained significant attention for its remarkable efficacy in treating SMA and its high price tag, which has sparked debates and discussions within the medical community, among policymakers, and among patients and their families. Join us as we explore the factors that contribute to the pricing of Zolgensma, the challenges it poses for accessibility and affordability, and the ongoing efforts to make this life-changing treatment available to those in need. 

Zolgensma is a revolutionary gene therapy drug developed to treat Spinal Muscular Atrophy (SMA) in children under the age of two. It has been widely praised for its potential to provide a long-term solution to SMA and offer new hope to families affected by the condition. Not just that, Zolgensma is one of the world’s most expensive medications costing ₹ 16 crore in India. This comprehensive guide will provide an overview of Zolgensma, including why is Zolgensma’s cost in India and worldwide so high. For what disease is Zolgensma used?

Additionally, we will explore the potential side effects of the drug, as well as its efficacy and how it compares to other treatments for SMA. Finally, we will discuss the new GST guidelines for Zolgensma in India, preventive measures for its use, and how to cover the cost of Zolgensma for SMA treatment. And many such questions may rack up your minds with doubts about Zolgensma’s price, use, history, effectiveness, and availability in India.

Whether you’re seeking a better understanding of the financial aspects surrounding innovative therapies or are interested in the broader implications for healthcare systems, our blog aims to offer valuable insights into the complex issue of Zolgensma’s price.

Read More: Impactguru hospital finder

After reading this guide, you will get the answer to all your questions more effectively as we will try to cover everything about Zolgensma in detail.

Let’s get started with a few important questions related to Zolgensma:

Why Is Zolgensma Injection’s Price In India And Around The World So High?

Zolgensma drug by Novartis is one of three medications in the world recommended for SMA, with Genentech’s Evrysdi and Biogen’s Spinraza. However, Zolgensma has proved to be the most effective as it stops the progression of SMA, which is the need of the hour for Zolgensma patients in India. Like its effectiveness, the ing zolgensma price in India is also high, i.e., ₹16 crores, which makes it costlier than Spinraza and Evrysdi (an oral drug) costs around 5 crores approximately and ₹ 22- ₹72 lakhs each year.
The Zolgensma cost in India, which is priced at ₹ 16 crores, is quite expensive for most of the Indian population.

Is It Possible For Anyone To Get A Zolgensma Injection In India?

I’d like to give a gist before answering the question in detail at the end. Yes, it is possible to get Zolgensma injections in India free of cost without paying back anything in return. How? With online fundraising, many small lives got a second chance at life. You might wonder if it’s a hoax. No, it isn’t. You can read articles about how online crowdfunding platforms like ImpactGuru helped SMA patients raise money to import Zolgensma injections in India, priced ₹ 16 crores from the US.  

Does Zolgensma Justify Its High Price In India And Worldwide?

There is a big debate around Zolgensma’s price and whether it should come down to saving the lives of poor people. We will try to present our viewpoints on this topic- Why and how it’s important to bring down the zolgensma price in India, which costs ₹ 16 crores and will help you bring clarity on Zogensma’s cost in India. 

Starting with a piece of important information, Zolgensma- World’s most expensive drug, is not available in India. So any Zolgensma patients in India who have been prescribed Zolgensma drugs for SMA treatment need to import them from the US. One of the reasons that the Zolgensma price in India is so high is that there are a smaller number of SMA patients or low incidence of SMA disease, i.e., it affects only 1 in 3200 babies each year in India, and one in 10,000 live births around the world. 

And families who want to import Zolgensma in India need to write a letter to Novartis company, US, that they require Zolgensma as recommended by a medical practitioner for SMA treatment. Importing the drug is another reason why Zolgensma’s injection price in India is quite high. However, a pharmaceutical expert states that there is a significant difference in the Zolgensma price in India and the US. Zolgensma’s cost in India is only 4th or 5th of its price in the US. And the reason is the tariff imposed by firms in the USA when purchasing from them which makes the Zolgensma price in India higher. 

Now, How Can Zolgensma’s Price In India Be Brought Down?

The only way to reduce the Zolgensma cost in India is when a pharmaceutical company in India licenses and introduces Zolgensma, depending on the purchasing power of Indian consumers. And negotiations are held by the Union government on reducing Zolgensma’s cost in India by licensing it under an Indian Pharmaceutical company. 

But, there is one major issue in doing this- first, a drug can only be introduced in India only after it is available for people in the county who innovated the Zolgensma. Secondly, a market like India should have a sizable market opportunity that demands a minimum volume of Zolgensma drugs, not just the demand for 1000 tablets or 100 vials. 

Finally, it leaves only a few options to subsidize Zolgensma cost in India now, which is only possible when the Indian government directly approaches Novartis and negotiates the onasemnogene abeparvovec Zolgensma price in India. As the pricing of any drug depends on the country, it can only be licensed by DGCA (Drugs Controller General of India) for the Indian population.

Here, Novartis spokesperson said that Zolgensma India is priced 50% low compared to its set benchmarks, comprising the 10-year cost of the current standard to treat SMA disease. They also state that a one-time dose of Zolgensma is more cost-effective than other medications that can treat SMA for years, or the patient needs to be on life-long medication therapy. 

Is Zolgensma Available In India?

Zolgensma is neither manufactured in India nor available in India. However, a person who requires Zolgensma drug in India for SMA treatment needs to order it from the US after taking government approval and the doctor’s recommendation. 

There is another way through which children with SMA type 1 can get a free dose of the Zolgensma injections in India, priced ₹ 16 crores. Through gMAP (Global Managed Access Program), only a selected number of patients under 2 years of age get a one-dose free. It is only available in countries like India with no regulatory approval of Zolgensma.  

Now you know you can get Zolgensma imported from the US, but are you wondering how to cover the Zolgensma cost in India, priced at 16 crores? Don’t worry in the end; you will get your answer and one effective financial plan to get the Zolgensma drug at zero cost. 

Is Zolgensma Approved In India?

Firstly, we will try to understand who approved Zolgensma drug for SMA treatment of children less than 2 years of age worldwide- 

• Food and Drug Administration (FDA)
• Health Canada
• European Medicines Agency (EMA)

FDA gave Zolgensma various designations like Breakthrough therapy, Fast track, and Priority Review. However, Zolgensma is neither approved nor manufactured in India, so SMA patients need to import Zolgensma drug at a high price of 16 crores from the US through a doctor’s recommendation and government approvals following a process.  

The only drug Evrysdi manufactured by Roche is approved in India for SMA treatment. It is cheaper than the Zolgensma cost in India. 

Now, we will cover more information about Zolgensma, giving you more clarity on this topic.

What Are The Factors Influencing The Zolgensma Price?

The price of Zolgensma is influenced by several factors, some of which include:

1. Development Costs: The price of researching, developing, and obtaining regulatory approval for a gene therapy like Zolgensma is substantial. Pharmaceutical companies typically factor in these expenses when pricing their drugs.

2. Manufacturing Costs: Producing gene therapies can be complex and expensive. The cost of manufacturing Zolgensma, which involves the creation of a viral vector to deliver the therapeutic gene to patients, contributes to its price.

3. Clinical Trials: Conducting clinical trials to demonstrate the safety and efficacy of Zolgensma also adds to the overall cost. These trials are often extensive and require significant resources.

4. Regulatory and Compliance Costs: Companies must adhere to strict regulatory requirements when developing and marketing gene therapies. The costs of meeting these requirements are factored into the Zolgensma’s price.

5. Limited Patient Population: SMA is a rare disease, and Zolgensma is a targeted therapy for a specific genetic subtype. The limited number of potential patients can affect pricing since the costs need to be recouped from a smaller patient base.

6. Monopoly Pricing: In some cases, when a drug is the first or only treatment available for a particular condition, the manufacturer may have more pricing power due to limited competition.

7. Health Insurance Coverage: The availability of health insurance coverage for Zolgensma can influence its price. Manufacturers may price the drug higher if they anticipate that insurers will cover it.

8. Orphan Drug Status: Zolgensma has orphan drug status in some regions. This designation can provide certain benefits, such as extended market exclusivity, which can affect pricing.

9. Market Dynamics: Competition from other treatments, both existing and those in development, can influence pricing decisions. If there are alternative treatments available, the pricing strategy may be adjusted accordingly.

10. Negotiations with Payers: Negotiations with government health agencies, private insurers, and other payers can impact the final price patients pay for Zolgensma. Payers may seek discounts or rebates, affecting the drug’s net price.

It’s essential to note that the price of Zolgensma has been a subject of debate and controversy due to its high cost. Health policymakers and advocacy groups often discuss the balance between the need to recoup development costs and ensuring affordable access for patients, especially for rare diseases. Prices can also vary from one country to another due to differences in healthcare systems and negotiation processes.

What Is Zolgensma?

Zolgensma, also called onasemnogene abeparvovec-xioi in scientific terms, is a gene therapy prescribed to treat children younger than 2 years with SMA (Spinal Muscular Atrophy). Zolgensma is approved by the Food and Drug Administration (FDA) as a treatment for all types of SMA in children having mutations in the SMN1 (Survival Motor Neuron) gene. Zolgensma received the first approval from FDA in 2019, making it the second treatment for SMA after Spinraza (nusinersen) and the first gene therapy for SMA treatment. However, Zolgensma started with the AVXS-101 by AveXis and was later purchased by Novartis Pharmaceuticals Corporation.

Zolgensma is usually given as an intravenous (IV)infusion that lasts for an hour. It involves a one-time dose for SMA treatment. Zolgensma replaces the nonfunctional SMN1 gene with a healthy, new copy of the SMN gene. AAV9 virus takes the replacement gene into the body and infects the body cells with a new DNA. The most common symptoms or side effects reported in patients during clinical trials were vomiting and liver enzymes, including the risk of acute liver failure and severe liver injury. 

There is a higher chance of success if infants receive Zolgensma at the earliest after the diagnosis, as timing plays a vital role in any medical treatment. 

If you took a backstep because of financial difficulty in affording Zolgensma costs in India, don’t worry, ImpactGuru is there to support you financially…

History Of Zolgensma- From Clinical Trials To Approval From The FDA

• Treatment of symptomatic SMA patients

START study- It is one of the first studies conducted for Zolgensma.

Participants	In this study, 15 patients with SMA Type 1 were taken who showed symptoms of SMA, had two copies of the SMN2 gene and were 8  months younger when diagnosed with SMA.
Goal	To determine the exact dose of SMA and establish safety measures.

STR1VE Study

After the START study, this study took place for Zolgensma. 

Participants	In this study, 22 patients with SMA Type 1 were taken who showed symptoms of SMA, had two copies of the SMN2 backup gene, and were 6  months younger when diagnosed with SMA.
Goal	To determine its efficacy and establish safety measures.

START study (Ongoing trial)

In this study, SMA patients were asked to participate in long-term follow-up. They took 13 out of 15 patients from the START study for safety examination on a long-term basis. 

• Treatment of pre-symptomatic SMA patients

SPR1NT Study

In this study, asymptomatic patients were taken to examine Zolgensma’s effectiveness.

Participants	In this study, 29 patients with 2 or 3 copies of the SMN2 backup gene who were younger than 6 weeks took part in the trial.
Goal	To determine its efficacy and establish safety measures for future results.

The final approval of Zolgensma from the FDA was determined based on the data from the completed Phase 1 START trial and ongoing Phase 3 STR1VE trial. The results from all these data were outstanding, including high survival rates and fast improvement in motor function in just one month of dosing. It also includes the ability to sit without support in untreated SMA patients due to the Zolgensma drug.  

It was all about Zolgensma and how it started. Still, in the following sections, we will try to understand Zolgensma’s cost in India, its availability, and how to deal with high Zolgensma costs in India. 

For Which Rare Disease Is Zolgensma Used As Medication Therapy?

Zolgensma is used to prevent health complications and manage SMA (Spinal Muscular Atrophy) symptoms contributing to a better quality of life in SMA patients. Spinal Muscular Atrophy (SMA) is a genetic disorder affecting the nerve cells that manage muscle movement, also called motor neurons. They are situated in the spinal cord. 

In SMA patients, motor neurons in the spinal cord cannot reply to nerve signals which causes the muscles to weaken and damage. Over time. It leads to atrophy due to muscle inactivity. However, there isn’t a complete cure for SMA, but different approaches are recommended to reduce its harmful effect to some extent. It includes

• Medication like Zolgensma and Spinraza
• Ventilation assistance
• Support devices and wheelchairs
• Occupational therapy, and rehabilitation.

Types of SMA (Spinal Muscular Atrophy)

	SMA Type 1 ( infantile-onset or Werdnig-Hoffmann disease)	SMA Type 2 ( intermediate SMA)	SMA Type 3 ( Kugelberg-Welander disease)	SMA Type 4 (adult-onset SMA)
Age of Onset	From birth to six months	7 to 18 months	18 months to 15 years	18 to 50 years
Symptoms	Puling (Crying weakly)Trouble sucking, swallowing, breathing, weakness of muscles	The problem of ScoliosisArms, lower torso, and legs muscle weaknessRespiratory problems	Lack of strength in the arm, shoulder, hip, and leg musclesInfirm respiratory muscles	Twitching, tremors, and moderate muscle weakness
Lifespan	Lifespan is only upto 2 years of age	Live up to young adulthood	Unaffected	Normal

How Does A One-time Dose Of Zolgensma Stop The Progression Of SMA?

A one-time dose of Zolgensma targets the root cause of SMA in patients. Zolgensma majorly includes a vector and a gene. When Zolgensma is given to a person, a working, new SMN gene is kept inside the vector and acts like a vehicle that brings the SMN gene to motor neuron cells all over the body. Zolgensma’s one-time dose replaces the work of the SMN1 gene to produce SMN protein.

With adequate protein, the dying motor neuron cells may function and survive. Even motor movement in children will function normally; that seems impossible without Zolgensma. The results may differ among children based on factors such as how far SMA symptoms have spread before taking the Zolgensma drug. 

What Are The Benefits Of The Zolgensma Drug?

One of the studies shows that Zolgensma is effective as it limits the use of artificial ventilation in SMA patients. Out of 22 babies who took the Zolgensma drug, 20 were breathing properly without a ventilator after 14 months and were alive. But, in the normal case, only a quarter of untreated children can survive without requiring a ventilator.

It was also observed that babies who took Zolgensma could sit without support for around 30 seconds. Out of 22 babies who were given Zolgensma drug can sit unaided even after 18  months, which is impossible to achieve in the case of untreated babies with a rare disease like SMA.

How Effective Is Zolgensma In Treating SMA?

One of the results from an open-label clinical trial where 21 children ranging from the age group of 0.5 to 5.9 months were given 1.1 x 1014 VG/kg Zolgensma and reported some remarkable results given below:

• 16 out of 19 SMA patients didn’t require a non-invasive ventilator for daily use.
• 13 out of the 19 SMA patients were able to reach a minimum of 14 months of age without using compulsory ventilation by March 2019 data reports.
• 10 out of the 21 patients, i.e., 47.6%, were able to sit without any support for less than 30 seconds of age group 9.2 and 16.9 months with an average age of 12.1 months. 

Why It’s Zolgensma Given To Children?

Zolgensma is also called onasemnogene abeparvovec in scientific terms. It’s gene therapy that includes human genetic substances. It is prescribed to cure babies of age less than 9 months having a rare disease called SMA (Spinal Muscular Atrophy). In SMA, an abnormal gene known as ‘Survival Motor Neuron’ (SMN) produces important protein; if it is lacking in the human body, it can lead to the death of motor neurons, nerves controlling body muscles. 

Here, the Zolgensma drug protects viable muscle neurons but can’ revive dead motor neurons in the human body. Zolgensma drug sends a new, working SMN copy that helps the body to produce SMN protein. The gene is transferred into body cells where it’s required with the help of a modified virus, and it doesn’t cause human disease.

How Is Zolgensma Taken?

The standard dosage, a one-time dose, is recommended based on the child’s weight. Usually, dosage may range from 3.3 x 10^14 vector genomes (vg)/kg body weight to 2.31 x 10^15 vg/kg. This medication takes around 60 minutes and is given through slow intravenous infection. However, before taking the Zolgensma drug, the following tests are recommended given below:

• An adeno-associated virus 9 (AAV9) antibody test
• Blood tests
• SMA diagnosis to confirm its existence in the body
• Before the infusion, the first dose of an oral corticosteroid is administered based on the doctor’s recommendation, and on infusion day, the second dose is given to the SMA patient.  The oral corticosteroid course helps control liver enzyme reactions by the immune system to the medicine. 

When is Zolgensma given to the patient?

Zolgensma is given to your child using prednisolone through the mouth a day before the drug is administered. However, the dose of corticosteroid is determined on the basis of your child’s weight, and it is generally recommended 1 mg per kg body weight every day. 

What Are The Side Effects Of The Zolgensma Drug On Patients?

All types of medicines, be it Zolgensma, can have side effects. But, if your child experiences any side effects mentioned below because of the Zolgensma drug, it is recommended to take medical support. There are two types of side effects- 

Less Severe side effects- 

• Vomiting
• Fever

More Severe side effects- 

• Skin yellowing or whites of the eyes, vomiting, or lesser alertness.
• Bleeding or bruising for extended periods that usually don’t last for long when your child gets hurt.
• Problems while breathing, swollen legs, tummy area or arms, and blue or pale grey skin color.
• Decrease in the amount of urine released, getting bruised easily, and seizures. 

What Precautions Should Be Taken Before And After A Dose Of Zolgensma?

• There are chances of serious complications if a patient is infected before or after the Zolgensma drug is given. You should visit your doctor if you find any symptoms of infections like coughing, sore throat, fever, runny nose, or wheezing.
• When Zolgensma is infused into the body, it can lead to decreased platelet counts. When there is unusual bruising or bleeding, it is important to seek medical support as a precautionary measure.
• One week after the Zolgensma drug is given to a person, Thrombotic microangiopathy (TMA) cases are reported in Zolgensma patients in India and around the world. It is vital to take medical assistance when a patient experiences signs of TMA, such as seizures, less urine release from the body, or unusual bruising or bleeding. 

Is Zolgensma Available As Biosimilar Or Generic?

Presently, Zolgensma is unavailable in a biosimilar version and only as a brand-name drug. Biosimilars are a type of drug similar to gene therapy or biologic drug. And Zolgensma is a gene therapy, and it is usually termed a biologic medication. Biologics are produced from living cells, which makes it difficult to make the exact copy of this drug. On the other hand, generic drugs are made from chemicals similar to the active drug in a brand-name drug. Biosimilars are more cost-effective than brand-name drugs and safe as their parent drug. 

How To Overcome The Challenges And Barriers To Accessing Zolgensma For Patients In India?

Accessing Zolgensma, a gene therapy treatment for spinal muscular atrophy (SMA), can indeed present challenges and barriers for patients in India. However, various measures can be taken to overcome these obstacles:

1. Understanding the SMA Treatment: Begin by gaining a comprehensive understanding of Zolgensma, including its benefits, potential side effects, and eligibility criteria. It’s essential to know if the patient meets the specific requirements for receiving the treatment.

2. Medical Consultation: Seek advice from a qualified healthcare professional or specialist experienced in SMA and genetic therapies. They can evaluate the patient’s condition, provide accurate information about the treatment, and guide you through the process.

3. Identify Treatment Centers: Research and identify hospitals or treatment centers in India that offer Zolgensma therapy. Connect with their pediatric neurology or genetic medicine departments to gather information about their experience with the treatment, costs involved, and any waiting lists.

4. Insurance Coverage: Check if the patient’s health insurance policy covers gene therapy treatments like Zolgensma. Contact the insurance provider to understand the terms and conditions, required documentation, and reimbursement procedures. If insurance coverage is not available, explore alternative financial assistance options.

5. Financial Assistance: The high cost of Zolgensma—INR 16 crores—limits its access to most patients in India. Look for financial assistance programs provided by the pharmaceutical company manufacturing Zolgensma or charitable organizations that support patients with rare diseases. These programs may offer financial aid or guidance on fundraising initiatives.

6. Advocacy and Support: Connect with patient advocacy groups and support networks for SMA in India. They can provide valuable insights, resources, and guidance on accessing Zolgensma, including navigating bureaucratic processes, connecting with relevant authorities, and sharing experiences with other families who have gone through similar journeys.

7. Seek Government Support: Engage with local government representatives, health departments, or regulatory bodies to advocate for improved access to gene therapies like Zolgensma. Collaborate with patient advocacy groups to raise awareness about SMA and the importance of making these treatments accessible to those in need.

8. Clinical Trials and Research: Stay informed about ongoing clinical trials and research studies related to SMA and gene therapies. Participation in these trials can provide alternative options for accessing potentially life-changing treatments.

Remember, navigating the process of accessing Zolgensma in India may require persistence, patience, and collaboration with healthcare professionals, patient advocacy groups, and supportive networks.

What’s The New Rule Of GST Exemption On The Zolgensma Drug In India?

As per Union Finance Minister Nirmala Sitharaman, the GST council has given complete exemption on Zolgensma and won’t be any GST taxes on Zolgensma, costing around ₹ 16 crore to import from the US. It’s a relief for SMA patients who need Zolgensma and has to pay tax on drugs imported from the US, including its introductory price. 

If you want to know about Zolensma’s cost in India and how to deal with its high cost, you can try online crowdfunding.

One Last Question, Can I Get Financial Help To Pay For The High Price Of Zolgensma In India?

We know how difficult it can be as a parent to see your child suffering from minor to serious medical emergencies like SMA and financial crises make the whole situation more daunting. Anyone looking for much-needed financial support to cover the Zolgensma price in India can try online crowdfunding at ImpactGuru. Many people in India opted for online crowdfunding to raise funds to overcome the financial difficulty caused due to high Zolgensma cost in India. 

With online fundraising on ImpactGuru, all you need to do is tell your fundraiser story in a compelling yet honest way to get support for your cause from people worldwide. ImpactGuru is one of the effective yet powerful ways to reach people outside your close circle, like friends and family, and raise funds to cover the price of Zolgensma in India. ImpactGuru is one of the trusted leaders in online crowdfunding in India. ImpactGuru charges a 0 % platform fee to create or maintain your fundraising campaign, which will help you keep the maximum amount of money to cover the Zolgensma cost in India. 

Valuable tips to raise funds successfully to deal with Zolgensma price in India

• It is vital to include images and videos of your small child in your fundraising campaign to attract more contributions.
• Try to be transparent when communicating where and how funds raised will be used to cover the Zolgensma cost in India.
• Regularly post updates about the ongoing fundraiser that shows what your child’s progress is till now. This will help donor retention as they may feel it’s worth donating to this cause.
• Try to use a compelling title to encourage more people to read and share with other people.
• Present a real and unique story to people that help them build trust in your fundraising campaign and, at last, donate to help you cover the onasemnogene abeparvovec Zolgensma price in India.  

We are here to help you deal with the price of Zolgensma in India. Along with these fundraising tips, we also provide resources like 24*7 customer support, fundraising ideas, and expert assistance to make your journey flawless and easy. And, if you are eagerly waiting to get Zolgensma drug for your child but lack funds to deal with the price of Zolgensma in India, you can use ImpactGuru as a fundraising platform and help your child get a healthy and better future with Zolgensma injection.