In 2016, the world was bombarded with news coming from the FDA that a particular muscle condition found in babies had no cure. Families began to lose hope — as more and more babies die each year. But the FDA, after making such an announcement, forgets one thing; nothing is impossible with technology.
Today, the FDA shocked the world as it approves the most expensive medicine ever created in history, which can topple down the same rare condition that destroys a baby’s muscle control years ago. The said disease kills nearly all of those affected within a couple of years.
The medicine developed by the Swiss drugmaker, also known as Novartis, can be sold at a sum of 2.125 million dollars. However, out-of-pocket costs for patients will vary based on the type of insurance they have and its coverage.
The Food and Drug Administration on Friday approved the treatment called Zolgensma, which specializes for children under age two who are confirmed having any four types of the said disease through genetic testing. The therapy includes a one-time infusion, which takes about an hour long.
FDA explained that the medicine is a gene therapy that treats an inherited condition called spinal muscular atrophy. Medical Express indicated that the treatment focuses on a defective gene that weakens a child’s muscle, which resulted in immovability, and if uncured, leads to an inability to swallow or breathe.
So, why did Novartis took time and effort to produce a drug, knowing that it costs more than half of the price of a house?
Records show that spinal muscular atrophy in the most common type, which is also the most severe, at least 90 percent of patients die by age 2 and the lucky ones who are still alive need a ventilator to breathe. Children experiencing less–severe types become disabled eventually and can live up to a couple of decades. Medical Express also revealed that every year, about 400 babies in the U.S are born with this condition.
The defective gene, which causes spinal muscular atrophy, prevents the body from making enough protein that allows nerves which control movement to work typically. The specific nerves die off without the protein. While the number continues to increase since 2016 and alarm families, Novartis studied the said disease carefully. The company saw the need to create a drug that will provide a solution to the families affected by the disease.
After three years, it brought the world the first ever drug that can change the lives of millions of babies, while making a considerable profit.
How does Zolgensma work wonders?
The drug supplies an excellent copy of the faulty gene — which allows nerve cells to start producing the needed protein. This medicine will halt the deterioration of the nerve cells and will enable the baby to develop more normally. Inpatient testing, babies with the most severe form of the disease who took Zolgensma within six months of birth, had limited muscle problems, and others who were given the treatment also performed best.
Although many families, especially those who are affected with the disease, protested on the amount of money they would spend in just one infusion, Novartis promised that the company is creating a solution to cater the less privileged ones. It will let insurers make payments over five years, meaning, a sum of only 425,000 dollars will be paid per year. However, if the treatment doesn’t work; the company will give partial rebates.
Meanwhile, in the United States, the same medicine for the disease that was also approved is called Spinraza. But unlike Zolgensma, this drug must be given every four months. The maker is known as Biogen, which charges a price of 750,000 dollars for the first year and 350,000 dollars a year after. Just like the latter, it also resorted to installments to cater to families with low income.
The day after Zolgensma was approved; it created a spur of disbelief from the public. They questioned as to why this drug costs so much. FDA and independent non-profit groups are positive enough that these drugs will create miracles in the future.
Even the Institute for Clinical and Economic Review, which rates the value of expensive new medicines, calculated that the price of the new gene therapy is justifiable at the cost of 1.2 to 2.1 million dollars because it “dramatically transforms the lives of families affected by this devastating disease.”
The FDA, on a conference, did not indicate how long the benefit of the treatment lasts, but medical practitioners’ hope that it would last a lifetime. As with the side effects, the FDA said that patients would be experiencing vomiting and potential liver damage — so they must be monitored for the first few months after treatment.