‘Scientific efficacy of gene therapy for SMA is certified, don’t prevent campaigns’

* Photo: Anadolu Agency (AA)

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After Health Minister Fahrettin Koca released a written statement and indicated that "there was no concrete data proving the scientific efficacy of the gene therapy" for children with SMA, lawyer Dilşad Enhoş has spoken to bianet and said, "What the Minister said was contradictory. The related drug is now in the category recognized by the state."

CLICK - 'SMA campaigns make the state look weak'

While several opposition MPs, artists, physicians and scientists have been campaigning on social media, demanding that the three quarters of Turkey's 2020 grand New Year lottery prize (75 million lira) transferred to the Wealth Fund be used for the treatment of children with SMA, Health Minister Koca released a statement on January 3 and said:

"We are clearly against our children being used as test subjects. For the time being, there has been no concrete data indicating that the method put forward as gene therapy scientifically works ... We will not let our children be used as test subjects with the pressure of pharma companies."

Briefly about SMA and the situation in Turkey

Spinal muscular atrophy, or SMA, is a neuromuscular disease that causes gradual wasting of muscles. The first type is the one that affects babies in the first months of their lives. There are two drugs that work.

Spinraza is a drug that needs to be administered every four months to keep the symptoms in check. Zolgensma, which is a gene therapy, is a one-off cure that was approved by the U.S. Food and Drug Administration (FDA) in May 2019 and costs over two-million dollars.

While Turkey has added the gene therapy to the "Foreign Medicine List" upon a court verdict, the drug is not covered by the Social Security Institution (SGK). Parents of children with SMA and several others have been campaigning so that the children can be treated before it is too late.

Against this background, lawyer Dilşad Enhoş has noted that Zolgensma, an alternative to the Spinraza drug prefered in Turkey and used all through children's lives, has been approved by both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the SMA patients of all ages and in all SMA types.

On the list of foreign medicines

Enhoş has further underlined that academic research is conducted to get these approvals and briefly added the following:

"At this point, the Minister made contradictory remarks. Because the related gene therapy that the SMA patients are trying to access has been added to the Foreign Medicine List upon a lawsuit. In other words, there is no such thing as being test subjects for the medicine.

"SMA gene therapy Zolgensma has been added to the relared foreign medicine list of the Pharmaceuticals and Medical Devices Administration of Turkey dated December 22, 2020.

'Its scientific efficacy is certified'

"The company of the drug is Novartis. No applications for license were made in our country. It had to be brought from abroad.

"Legally speaking, it has to be in the following way: The doctor has to prescribe this medicine. And the Ministry of Health makes the application to the Pharmaceuticals and Medical Devices Administration of Turkey. It is procured for a single patient. This approval has been given for baby Yiğit, which certifies the scientific efficacy of the medicine.

"On the other side, the approval given by the Pharmaceuticals and Medical Devices Administration is only about bringing the medicine to our country. The cost of the medicine is still not met by the Social Security Institution (SGK). And this cost is 2.6 million dollars (17.7 million lira)."

'At least, don't prevent the campaigns'

According to lawyer Dilşad Enhoş, nine babies were sent abroad for this treatment, adding that the fundraising campaigns are not allowed by the governor's offices. She has made the following recommendations:

"Families are in a really difficult situation, there are families that cannot pay this money. In the first stage, if you do not repay it through the SGK, you can at least try to reduce the price as the Ministry.

"And don't prevent the campaigns. There are official statements suggesting that this amount can be reduced by three times. Then, perhaps, not nine, but 27 babies can receive this treatment.

'400 babies can undergo gene therapy'

"There are around 1,300 babies with SMA in Turkey. Most of them do not meet the criteria for this drug. There are around 400 babies who meet the criteria. A baby needs to be younger than two and be under a certain weight so that she or he can receive this treatment.

"The campaigns of families are not allowed, either. This medicine must, of course, be afforded by the state as required by both the right to life and the principle of social state." (AÖ/SD)

LAWYER DİLŞAD ENHOŞ