«Zolgensma» the most expensive drug in the world at 8 million dirhams

A consultant demands insurance companies cover treatment for genetic diseases

  • Spinal muscular atrophy is a disease caused by a genetic defect that weakens a person's muscles.

    From the source

  • Fatima Al-Bastaki: "The price is exaggerated, especially since the company producing the drug did not develop it."

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Dr. Fatima Al-Bastaki, a consultant pediatric and clinical geneticist at Latifa Hospital in Dubai, called for the need for insurance companies to cover the cost of treating rare genetic diseases, so that those affected can obtain the appropriate treatment, explaining that the medicine for treating spinal muscular atrophy disease is the most expensive worldwide Its price is eight million dirhams, and it is authorized to treat children under the age of two, and it is a single intravenous injection that takes about an hour.

And issued the genetic drug «Zolgensma» for the treatment of spinal muscular atrophy disease list of the most expensive drugs in the world, amid questions about the reasons behind this amount, and what it provides to the patient without other drugs.

Al-Bastaki considered that the price is exaggerated, especially since "Novartis", the company that produces the drug, did not develop the drug, but rather obtained it after purchasing the American company, AveXis.

She emphasized that "spinal muscular atrophy" is a fatal disease resulting from a genetic defect that weakens a person's muscles, so that he becomes unable to move, and may ultimately impair the ability to swallow or breathe, and children who are born with the disease will die before the age of two if they do not receive treatment. The disease does not affect the child's intelligence, or his ability to learn, and the incidence of SMA is 10 per 100,000 people globally.

Dr. Al-Bastaki said that “Zolgensma” is a gene therapy that deals with the root cause of the disease, that is, the defective gene. Muscle movement and function, also improves survival ».

This treatment depends on administering the healthy gene during the virus vectors for muscle atrophy, by injecting the sick child intravenously once, so that it improves the response of the child's motor neurons, and the dose of treatment is determined according to the weight of the patient.

Al-Bastaki explained that the main causes of "muscular spinal atrophy" are the result of an abnormal or missing gene known as "the motor neuron 1 gene" (SMN1), which is responsible for producing a protein essential for motor neurons.

She continued: “Individuals with SMN have insufficient levels of the SMN protein, which leads to the loss of motor neurons in the spinal cord, and then to the weakness and wasting of skeletal muscles, and this weakness is often more severe in the trunk, leg and arm muscles than the muscles of the hands. And feet ».

She added: “Genetic reasons are also behind the emergence of this disease, especially if one of the parents is a carrier of the mutated gene, as the probability of inheriting the disease reaches 25% in every pregnancy, and the occurrence of this disease can be avoided through genetic examination before marriage, and through diagnosis. "Pre-implantation genetic material through IVF technology, and this is if there is a history of disease in the family."

Novartis, the "drug producer", announced that it is working with health insurance companies to allow payments to be divided over five years (ie 425 thousand dollars annually), and pledged to provide compensation unless the treatment succeeds. However, it remains unclear how these measures will be activated in Every country.

She explained, to "Emirates Today", that the reason behind the increase in the price of the drug to become the most expensive in the world is that the treatment of children with muscular spinal atrophy is a great burden in terms of cost to health systems, for example in Europe, the estimated cumulative health care cost per child ranges between 2.5 And four million euros, during the first 10 years alone, including rising caregiving requirements, limitations in career choices and advancement, and a social and emotional burden on families.

And she continued: "Gene therapies have the ability to reduce the long-term financial burden on patients, families and health-care systems, by relying on one treatment instead of lifelong treatments."

Novartis confirmed that “Zolgensma” is unique because it is a gene therapy, which means that we take an active copy of the gene and insert it into the cells of the child with spinal muscular atrophy, to replace what the child suffers from a deficiency in this regard, thus stopping the progression of the disease And the healthy gene is delivered through a carefully designed virus, which allows us to treat safely and effectively, but this advanced technology requires a large investment.

And she continued: "Some of the latest data showed that the drug works well even in children with more advanced disease in the first place, and 800 patients have been treated with this drug around the world."

List of alternative treatments

The US Food and Drug Administration approved the drug nucinercin, as the first drug approved for treating children and adults with spinal muscular atrophy, and it is given by injection into the fluid surrounding the spinal cord, and the benefit is better documented in infants and children, and its cost ranges between 625 thousand dollars to 750 thousand dollars per year The first, then $ 375,000 annually, and for life.

The administration also finally approved the drug Evresdi, a drink to be given orally to patients from the age of two months and adults, with an estimated cost of $ 340,000 annually.

Emirates Today provides treatment for Munira

Last week, Emirates Today succeeded in securing the price of a Zolgensma treatment injection for the Syrian child Munira, who suffers from spinal muscular atrophy, which caused her to be completely immobile, in addition to her rapid breathing and an accelerated heartbeat.

A group of institutions and semi-governmental and private entities in the state provided the full amount, to give this girl hope for a normal life, after her family was desperate for the ability to provide the required eight million dirhams.

Emirates Islamic Bank provided four million dirhams, Dubai Islamic Bank provided two million dirhams, the Dubai International Financial Center provided one million, and Wasl Properties provided one million dirhams, to complete the amount needed to treat the child (Munira).

• "Novartis": "The drug provides a treatment that saves millions of dollars, which are spent on alternative methods."

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