Two Drugs in the Treatment of SMA Included in the Treatment Guidelines

Two Drugs in the Treatment of SMA Included in the Treatment Guide
Two Drugs in the Treatment of SMA Included in the Treatment Guidelines

SMA Science Board, Minister of Health Dr. They met at Bilkent Campus under the chairmanship of Fahrettin Koca. After the meeting, a written statement was made by Minister Koca. Minister Koca, in a written statement after the SMA Scientific Committee meeting, stated that the results of SMA scans, the treatment processes of the patients whose treatment continues, and the latest developments in treatment methods were discussed at the meeting.

Reminding that SMA is a hereditary, progressive, chronic, neurological disease, Koca said, “Until 2016, we were losing nearly 1 percent of babies with Type-90 form of the disease due to this disease, which had no known cure in the world, before they reached the age of 2. After the drug with the active ingredient 'nusinersen' started to be applied around the world in 2016, an opportunity arose to keep these babies alive. Immediately after this development, nusinersen treatment was started to be given free of charge to all our patients in our country, which can set an example in many countries around the world. Currently, 1024 of our patients receive this treatment free of charge.

Underlining that over time, scientific data show the necessity of applying this treatment as early as possible for it to be more effective, Koca pointed out that SMA newborn screenings were started across Turkey in May 2022 in this context.

Minister Koca said, “In this context, 753 thousand 350 babies have been screened for SMA so far. We delivered their treatment as soon as possible to those who were determined by their physicians that these babies needed medication. Our survival rate in the first 6 months was 100 percent in our babies who were diagnosed in the newborn screening program and whose loading dose of nusinersen treatment was completed.

“Our country is one of the few countries that can do the premarital screening program”

Stating that the SMA Scientific Committee has also meticulously followed the developments in the last 5 years regarding the prevention of the disease, and within this framework, the premarital screening program, which very few countries in the world can do, has been implemented. includes couples. Genetic counseling and selective pregnancy application that will enable them to have healthy babies are provided free of charge to couples who are found to be carriers of SMA in this screening. Our country is one of the few countries in the world that can do this," he said.

The final stage has been reached for the licensing of the SMA drug in solution form

Minister Koca emphasized that in addition to delivering the first drug treatment to patients and following their data, scientific developments regarding other drugs developed for use in SMA are also closely monitored by the Scientific Committee.

“In this context, together with nusinersen, all data regarding the drug with the active ingredient 'risdiplam' and the drugs named Zolgensma are also monitored. All three of these therapies are gene-based therapies. No superiority was demonstrated for any of the three treatments.

Of these, the legal procedures required for the drug with risdiplam active ingredient to enter our country have been completed by the relevant company and an application has been made. The scientific data regarding this drug, which will also allow our patients to be administered orally in the form of a solution, have been evaluated and the final stage has been reached for the licensing of the relevant drug. In today's meeting held by our scientific committee, all the details of our health system, from how the medicine is prepared and how it is delivered to our patients, were meticulously evaluated. We will have finalized the license process for this drug in the coming days. It was decided to offer this drug as an option for patients whose efficacy is known, nursinersen treatment is difficult to apply. As the country data increases, the application principles will be reviewed by the Scientific Committee.”

Thus, Koca emphasized that two drugs will now be included in the treatment guide for SMA patients.

“It is known that Zolgensma treatment does not benefit symptomatic patients”

Health Minister Koca underlined that all scientific data and developments regarding the evaluations made at the SMA Scientific Committee regarding the treatment called Zolgensma, also known as “gene therapy”, are closely followed.

Emphasizing that the main thing for the Ministry is the evaluation of the Scientific Committees and the protection of patients from the harms of global actors, Koca said, “In this sense, there have been serious reservations about both the scientific data and the applications on the patients. Inconsistencies were seen in the results of the experiment, which was the basis for the registration of the drug in the United States, and the scientific publication containing these data had to be removed from a very prestigious scientific journal.

Koca continued:

“At this point, we would like it to be known that every SMA baby born in our country receives a treatment whose effectiveness is known, and that we, as the Ministry of Health, do not approve any of the SMA aid campaigns. It is known that Zolgensma treatment does not benefit symptomatic patients. However, there are campaigns even for these patients who show symptoms, are connected to the device and will not benefit from the treatment. In fact, we have a large number of babies who were taken abroad with such campaigns, received Zolgensma treatment without our approval, and applied to our Ministry to continue the nusinersen treatment when they did not benefit.

Despite all this picture, in order not to ignore the possibility that it may provide an additional benefit to our patients, although they do not have a legal application for the entry of the drug to our country, the relevant company officials were interviewed and these data were re-submitted to our Scientific Committee by providing the scientific evidence. In the evaluation made by our Scientific Committee, it was seen that there is still no comparative scientific study showing the superiority of treatment with Zolgensma over other treatments.

“Our SMA Scientific Committee will make the evaluation as soon as possible”

Pointing out that the European Medicines Agency has recently restricted children older than 12 months due to the side effects in Zolgensma treatment, Koca made the following statements:

“This situation is closely followed by our Scientific Committee. However, there are studies published in the last 0 months showing that the drug Zolgensma has similar efficacy to other drugs on SMA Type-6 babies who do not have symptoms from 1-5 weeks newborn screening. It was thought that it would be appropriate to make a new evaluation by requesting real-life data from the relevant company for a new examination. Our SMA Scientific Committee will make this evaluation as soon as possible.”

Nursinersen, one of the three drugs used in the world in the treatment of SMA, has been licensed in our country, has applied for a risdiplam license and has come to the final stage. Zolgensma, on the other hand, did not take any initiative for licensing. Licensing is extremely important for the traceable and safe application of the treatment.”

“We will not allow the hope of our families to be used for commercial purposes”

Minister Koca stated that they aim to maintain the health service and care based on realistic expectations for SMA patients and their families at the highest level by following standard care rules, and made the following assessment:

“We would especially like to point out that we have not allowed the abuse of hope until now, and we will not allow it in the future. We will not allow the hope of our families to be misused for commercial purposes. We have previously declared that we will not allow our children to be used as subjects. We want it to be known that we maintain this position. However, we are ready to provide the necessary convenience for any treatment whose effectiveness has been proven with scientific evidence.”

Günceleme: 01/02/2023 12:12

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