The US approved medicine of Novartis for gene therapy, which is the world’s most expensive drug, priced at $2.125 million equivalent to approximately INR 14 crore but out -of pocket costs for patients will be based on insurance.
This most expensive drug can treat a rare disorder that destroys all of baby’s muscles controls and kill almost all of those with this disease within a couple of years.
This medicine sold by Swiss drugmaker Novartis, it is a gene therapy which treats a condition called spinal muscular atrophy.
This treatment targets the defective gene which weakens a child’s muscles that they become unable to move, swollen or breathe and eventually cause death.
It affects about 500 babies each year alone in the US.
The Food and Drug Administration approved this treatment called Zolgensma for all the children under the ages of 2. This is confirmed by a genetic test to any three types of disease. This therapy is only for one time and takes about an hour.
Novartis give some relieve and said it will let insurers make payments in installments of $425,000 per year for over 5 years.
The other medicine for the disease approved in the US is called Spinraza. It must be given every four months. Biogen, Spinraza’s maker, charges a price of $750,000 for the first year and after that $350,000 per year.
Clinical and Economic Review which is independent non-profit group rates the value of expensive of new medicine, calculated that the new gene therapy is justifiable at a cost of $1.2 million to $2.1 million because it dramatically transforms the lives of families affected by this disease.
The defective gene prevents the body from making enough of a protein which allows nerves controls movements to work normally. Eventually, nerves die without protein.
At least 90% of patients die at the age of 2 and if any still alive need a ventilator to breathe. Patients with less severe become disable and love up to several decades.
This therapy that also called Zolgensma works by supplying a healthy copy of the defective gene and allow nerves cells and let produce needed protein. Which prevent any deterioration in the nerves cells and let the baby grow normally.
Babies given Zolgensma after six months stop losing muscle controls but the drug can’t reverse the damage.
The first ever treated children with this therapy, the name is Evelyn Villarreal of eight weeks. Her family had lost their first child to spinal muscular atrophy at 15 months. After Evelyn was born, the test shows that she also had a disease. So her family enrolled her at Nationwide Children’s Hospital in Columbus, Ohio.
Now she is 4 ½ and now there is no muscle problem.
Its too early to know how long the benefit of the treatments will last but doctors hopes are rising that it could for last for a lifetime.
“It’s beginning to look that way,” Dr. Jerry Mendell, because a few children treated who are now 4 or 5 still have no symptoms.
The FDA said side effects include vomiting and potential liver damage. So patients must be monitored for the first few months after treatments.
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