The most expensive drug in the world will be reimbursed from September

The September list of reimbursed drugs will include the most expensive SMA drug – Zolgensma. It will be intended for children up to 6 months of age, said the Minister of Health, Adam Niedzielski.

1. From September, the most expensive SMA drug will be reimbursed, announced the Minister of Health, Adam Niedzielski
2. Zolgensma is one of the most expensive drugs in the world. One dose costs over $ 2 million
3. About XNUMX people suffer from spinal muscular atrophy (SMA) in Poland
4. More current information can be found on the Onet homepage

The SMA drug will be reimbursed

At a press conference in Warsaw, the head of the Ministry of Health said that the source of financing for the most innovative drugs in Poland is the Medical Fund.

He announced that the most expensive SMA drug, Zolgensma, will be on the list of reimbursed drugs in September.

– The reimbursement on the September list will include the most expensive, in terms of a single admission, drug against SMA. I am talking about the drug Zolgensma, which will be intended for children up to 6 months of age. It will, of course, be administered as a result of the screening program, said Minister Niedzielski.

He noted that genetic tests to diagnose SMA are currently underway, and that they are complemented by therapy, which was “a bit more difficult to take”. – Now we are introducing the possibility of this most expensive drug, i.e. Zolgensma – emphasized the head of the Ministry of Health.

Zolgensma – the most expensive medicine in the world

Onasemnogen abeparvovec, the tradename Zolgensma, is a gene therapy medicinal product that expresses the human survival motor neuron (SMN) protein.

It is a non-replicating recombinant vector based on adeno-associated virus serotype 9 [AAV9] based vector containing human SMN gene cDNA controlled by a hybrid cytomegalovirus enhancer / chicken beta actin promoter promoter.

Zolgensma works by increasing the level of the SMN protein in the motor neurons, a deficiency of which lies at the root of spinal muscular atrophy. With more of this protein, motor neuron degeneration stops and the disease stops progressing. If the patient’s muscles have not yet progressed to atrophy, the combination with appropriate physiotherapy and multidisciplinary medical care may bring the patient a significant improvement.

Zolgensma is one of the most expensive drugs in the world. The retail price set by the manufacturer is $ 2,125 million per package (1 dose).

In May 2020, the drug was conditionally approved for use in the European Union for the treatment of patients with spinal muscular atrophy who either have clinical symptoms of the first form of SMA or have no more than three copies of the gene SMN2 regardless of the form of SMA. Until now, it has not been reimbursed by the National Health Fund.

SMA – what is this disease?

Spinal Muscular Atrophy (SMA) is a genetically determined group of neuromuscular diseases that share the common feature of the loss of spinal cord motor neurons (which transmit impulses from the brain to the muscles), leading to muscle weakness and wasting.

The disease affects people of all ages, but in over 70 percent. In cases, the first symptoms of SMA appear in infancy or early childhood. Until the introduction of modern methods of respiratory care and pharmacological treatment, SMA was the most common genetic cause of death in children under the age of two.

There are four types of SMA:

1. SMA1 (acute form, Werdnig-Hoffmann disease) – the first symptoms of the disease most often appear in the second or third month of life. Attention is drawn to the lack of progress in the child’s motor development, quiet crying and easy fatigue when sucking and swallowing, which is the result of significant weakening of the intercostal muscles. The child never achieves the ability to sit up on its own. The prognosis for acute disease is poor. Most children die before the age of 2.
2. SMA2 (intermediate form) – The baby develops normally at first but never becomes able to walk independently. There is trembling of the fingers and with time significant weakness. There are joint contractures and curvatures of the spine. Survival in SMA2 patients depends on the level of care, but is usually shorter than in the general population.
3. SMA3 (mild form, Kugelberg-Welander disease) – the first symptoms usually appear before the age of 3. The child has trouble walking and climbing stairs. He falls over, stands up, leaning on objects, does not run. The course of the disease may also be slower and unnoticeable for a long time. As the disease progresses, there may be pseudo-hypertrophy of the calves, trembling of the fingers, and muscle twitching. Survival time is similar to that of the general population.
4. SMA 4 (adults) – the disease begins around 20-30 years of age. discrete, slowly progressing symptoms: trembling fingers, difficulty running, climbing stairs. Over time, weakness spreads to the distal (distal) muscles. The survival time is unchanged.

It is estimated that there are approximately 1000 SMA patients in Poland. About 45–55 babies are born each year and will develop spinal muscular atrophy at some point in their lives; 30–40 of them will have severe SMA.