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What is Zolgensma and Why Zolgensma Drug is So Expensive?

As since humans are born there is always a development in their genes . As humans are believed to evolve from monkey , there is a continuous genetic change kept on going. Also, many diseases with genetic change of human being have also evolved. From which spinal muscular injury is a rare condition. It is a life threatening disease that can cause death of a person . It can happen both in children and adults.

Spinal muscular atrophy and the Clinical Research Trends for the Development of the Drug

It is the condition where there is a loss in motor nuerons and progressive muscle wasting. There is an uncontrollable muscle weakness which is marked by disability of the muscles. It is diagnosed in early childhood as the patient is not able to walk or speak. It may also appear in later stages of life. The other problems may include :-

-‌weakness of voluntary muscles

-‌weakness of arms, legs

-‌weakness of respiratory muscles

-‌poor head control

-‌difficulty in swallowing


-‌joint contracture

This is a life threatening disease if not taken care. So , there is a medicine names Zolgensma given for patients of spinal muscular atrophy.

What is Zolgensma?

Zolgensma is the most expensive medicine given for the patients of spinal muscular atrophy. It is the best medicine known for gene therapy. It comes under the brand name

Onasemnogeneabeparvovec. It is directly infused to the vein. The company provides a copy of SMN protein in form of Zolgensma medicine. One injection of Zolgensma is of 16 crore rupees

Why is Zolgensma drug is so expensive?

It is so expensive because of the rarity of the disease as among 10,000 children only 1-2 get these disease. Government has put up heavy taxes on the import of the medicine. The high cost of this drug is being put up by the company itself as it dramatically transforms the lives of families who are being affected.

novel gene therapy zolgensma drug

The spinal muscular atrophy happens due to the SMN1 gene that codes for survival motor nueron protien which eventually leads to motor nueron cell survival. Motor nueron cells are responsible to communicate with our muscles and tell them about the muscle action that has to be done. But if the SMN 1 gene is not present then the motor nueron will not work and eventually life the no muscle action would be there. This disease happens in children with deficiency of SMA.

Hence, Zolgensma is a drug that is made up of working copy of human gene to essentially replace the one that is missing or not working properly. It is placed in Adeno associated virus 9 (AAV 9) vector which simply acts to take the gene where it is needed. Therefore, it delivers the gene directly to motor nuerons of the body.

The protien that accumulate in the nuerons starts producing the SMN  functional protien. These protiens bring back the lost functions of the child and hence, expanding the life span of children.

Clinical research Zolgensma drug and Clinical Research Associates

The pre- clinical research includes In Vivo use of animal models and in vitro use no use of animal models. There are variety in AAV9 vectors that provides the drug to be safer in all aspects. The clinical trial associates carry out these trials to see if the drug is safe for the people or not.

AAV has been used to treat various other diseases such as:-

– cystic fibrosis

– parkinsonism disease

– caravan disease

– limb girdle muscular dystrophy

these are list of diseases that have been significantly cured by AAV.

Clinical Research and Pharmacovigilance:

So many Clinical Research has been Going on for the Novel Gene Protein Therapy.

What is the Novel Gene Therapy and the Clinical Trials for Novel Gene Therapy?

Novel Gene Therapy is for the treatment of SMA in childrens. The Therapy by inserting a healthy gene into the genome into the precise spot in the form of Zolgensma Injection.

The Clinical Trials has been occurring from so many years. So many Clinical Research Associates have been involving in the process of the Clinical Research. The Well Trained Clinical Research Associates with Clinical Research Certification has been involved.

Clinical Trials for Zolgensma Injection:

Novartis is playing a crucial role in the development of Zolgensma Injection.

1 st clinical trial – An in vivo model has been successfully delivered in to airway of rabbits which had continued to be naturally expressed indicating that it was a successful treatment for adult cystic fibrosis patient.

2nd clinical trial- an in vitro model where AAV vector was able to bring a vital gene to skeletal muscle and central nervous system which allows the body to produce various types of protiens

3rd clinical trial- the AAV vector was able to produce dopamine to a patient of parkinsonism disease.

then clinical trials for Zolgensma were done where SMN gene was put into the AAV vector and three clinical trials were made:-

START clinical trial-

where 15 patients were given  Zolgensma injection

12 with high dose

results showed that all 12 people were able to breath properly without any breathing support.

75% could sit without help

17% could stand and walk without help

3 with low dose

3 patients required permanent breathing support.

hence, the Zolgensma is the medicine which is given in the earlier stages of the disease that is till the age of 2 years can cure the disease effectively.

side effects

the patients treated with Zolgensma had – elevated liver enzymes

– also they had vomiting

– low platelets

– elevated levels of cardiac troponin-I

– Thrombotic microangiopathy

Important Safety Information

– patients receive corticosteroid after the infusion of Zolgensma so proper blood tests should be taken.

– if the skin becomes yellow or eyes becomes yellow contact the doctor as it is s sign of liver toxicity

– viral infections can become a major respiratory problem after the drug infusion.

– seek immediate help if there is decreased platelet count.

– make the patient to wear diapers as some amount of Zolgensma can appear in the patient’s stool.

So, if your child is suffering from the condition of spinal muscular atrophy and the age is below 2 years you need to contact the doctor immediately for help.

Clin Infotech who is a well known Clincal Reserach Organization hiring for the Clinical Research Associates with Clinical Research Training and Certification for the various process of Clinical Trails. Clin Infotech Offers Clinical research course in hyderabad. Because of the best placement record Clin Infotech considered as the best clinical research institute in Hyderabad.