What is the most expensive medicine in the world so far?
And what is its price?
And what disease does it treat?
And what company makes it?
The answer is the drug "Zolgensma", manufactured by the company "Novartis", and works to treat a genetic disease called "spinal muscular atrophy SMA".
This rare genetic disease affects one in 10,000 children worldwide, and when untreated - in its most severe form - it leads to death in 90% of cases before the age of two years.
And the manufacturer, Novartis, got the green light last Tuesday to start a second production of the drug Zolgensma, and promised to facilitate access to this drug by negotiating discount terms with countries.
The start of a second production site for the drug Zolgensma
In a report published by the French newspaper "Luton" (letemps), writer Ram Etwarea said that Novartis had confirmed the news of the start of a second production site for Zolgensma;
The company told the newspaper that it would "strengthen the 'Extended Access' program aimed at introducing Zolgensma."
The author explained that the drug was approved for production in the United States in 2019, and the following year it was approved in the European Union and Switzerland, and it has already saved the lives of 18,000 children in research programs and clinical trials.
Subsequently, 100 doses were given free of charge in 2020, and more were given in 2021 to patients who would otherwise not have been able to pay for treatment, given that the treatment requires only one dose for the patient.
The price in Switzerland is confidential
The author shows that the drug Zolgensma is the most expensive treatment in the world.
According to a report in "Forbes" (Forbes), the price of a single dose is 2.1 million dollars.
The writer points out that Novartis is preparing from now on to increase its production capacity.
In fact, the US Food and Drug Administration gave the green light last Tuesday to its industrial site in Durham County, North Carolina, and it will also manufacture products for clinical trials of other gene therapies.
The company said, in its statement issued last Tuesday, that the “Innovation Center” is an innovation campus that already benefits from cooperation with the US government, while North Carolina Governor Roy Cooper said that “this operating license puts our state at the forefront of advanced gene therapies and we look forward to continuing our cooperation.” with Novartis.
Accordingly, the Swiss multinational will have a second production site in the United States for the treatment of genetic diseases, the first site in Libertyville, Illinois.
According to the author, production of the County Durham site will begin within 6 months, while Novartis stated that "complex and expensive gene therapies to produce are a promising part of medicine to treat serious diseases that have hitherto been considered incurable."
The writer reveals that the drug Zolgensma achieved $ 1.3 billion last year, noting that the CEO of Novartis, Vasant Narasimhan, said during 2020 that "the potential approval of this treatment in Europe represents an opportunity to earn billions of dollars."
Novartis defended the price of the drug by highlighting the $8.7 billion that was paid to purchase the rights to manufacture the drug until 2028 from the American laboratory "Avexis" that developed the drug.
Increasing demand for real estate
The author points out that the "Extended Access" program was launched in 2019, knowing that this program does not call for free access to the drug.
Despite the 2020-2021 COVID-19 pandemic, supply constraints, and other regulatory and logistical obstacles, Novartis has been able to deliver Zolgensma to more than 26 countries.
In practice, the company is negotiating with countries about purchase discounts, according to the company's statement, which confirmed that the contracts are based on its purchasing capabilities, financial ability, and annual or deferred payment terms.
What is spinal muscular atrophy?
Spinal muscular atrophy is a genetic genetic disease that makes muscles weak, causes the patient to have movement problems, and the disease worsens with time, and there is no current treatment for the disease, but there are treatments that help the patient deal with symptoms and improve his life, according to the National Health Service in Wales.
The disease does not affect the child's intelligence or ability to learn.
In most cases of spinal muscular atrophy, a child is born with the disease if both parents carry the gene that causes the disease.
Usually, the parents do not have the disease, but they carry a copy of the gene, meaning that the gene is recessive, and in order for the child to develop the disease, he must carry two copies of it: one from the father and the second from the mother.
If the parents are carriers of the disease gene, there is a 25% chance that the child will be born with it, a 50% chance that the child will carry the gene only, without the disease, and a 25% chance that the child will not carry the gene at all.
Symptoms of spinal muscular atrophy include:
Weakness or looseness of the legs and arms.
Movement problems such as walking or sitting.
Muscle twitching.
Bone and joint problems.
Swallowing difficulties.
breathing difficulties;
Zulgensma has a single dose of $2.1 million (Shutterstock)
Types of spinal muscular atrophy
There are several types of the disease that begin at different ages, as follows:
first type
It affects young infants, and symptoms appear during the first 6 months of life, and they have severe weakness in the arms and legs, and difficulties in movement, eating, breathing and swallowing, and the child cannot raise his head or sit.
Usually people with this type die in the early years, as a result of breathing problems.
second type
In this type, symptoms appear on affected children at the age of 7 months and 18 months, and are less severe than the first type, and include the following:
The child is able to sit without assistance, but not be able to stand or walk.
Weakness in arms and legs.
Tremor in fingers and hands.
Later, joint problems such as curvature of the spine (scoliosis) develop.
Weak breathing muscles and difficulty coughing, which can put them at risk of respiratory infections.
This type may shorten life expectancy, but most affected children live to adulthood and can live long lives.
third type
Symptoms usually appear after 18 months of age, but sometimes they may not appear until late childhood or early adulthood.
Symptoms of this type include:
The patient is able to stand and walk without assistance, but he may find it difficult to walk or get up from a sitting position.
Balance problems or difficulty running.
Slight tremors in the fingers.
Walking may become more difficult over time, and the patient may lose the ability to walk as he grows.
This third type usually does not affect life expectancy.
Fourth type
It affects adults and usually begins in early adulthood, and its symptoms include:
Weakness in the hands and feet.
difficulty walking
Muscle tremors and tremors.
This fourth type slowly worsens over time, but it does not cause breathing or swallowing problems, and does not affect life expectancy.