Bonn A two-year-old girl from Remscheid received the world's most expensive medication at Bonn University Hospital. At the age of seven months, she was diagnosed with spinal muscular atrophy (SMA), a very rare disease. At the end of May, 2021, she received an injection that could save her life.
Ajla still says "Nana" instead of "Mama," but her mother Suada Cerimovic is thrilled nonetheless. Until recently, the two-year-old from Remscheid hardly spoke a word, could neither stand nor walk or even breathe on her own. At the age of seven months, she was diagnosed with spinal muscular atrophy (SMA), a very rare disease that, if left untreated, usually leads to death within a few years. But today, Ajla is happily babbling away and even manages to sit in her high chair for half an hour, Cerimovic says. For her, this is a sign that the therapy the family has fought for is already having an effect. At the end of May, Ajla received an infusion of Zolgensma, the most expensive drug in the world, at the University Hospital in Bonn. The cost: two million euros.
But it was a long road for the family until then. When Suada Cerimovic and her husband Rasim learned of their daughter's diagnosis, their world collapsed. At the time, they were told that the average life expectancy for those with SMA was around two years. And the chance of a cure was minimal. At least Ajla got an injection every four months with the drug Spinraza (into the spinal cord), the second therapy approved in Germany, but it is a lifelong treatment.
Meanwhile, Cerimovic heard about the possibility of beating the disease, perhaps forever, with a single dose of Zolgensma. "But doctors initially advised us against it," the 37-year-old says. But reports of the first child to benefit from the drug in Germany encouraged the family not to give up. Until 2017, there was no effective treatment for spinal muscular atrophy at all. Then the drug Spinraza from the U.S. company Biogen came on the market, it has to be injected into the spinal fluid every four months. This also involves high costs, up to 500,000 euros per year. Two years later, Zolgensma was approved in the USA, and a year later in Europe.
It is a gene therapy; a functional variant of the SMN1 gene is introduced into the body's cells. This is intended to repair the hereditary genetic defect caused by SMA. In this disease, impulses from the brain can no longer be transmitted to muscles in the body; untreated children usually die of respiratory failure within two years.
Zolgensma can only be administered once - the earlier, the better are the chances of success. Studies show that treated children survive longer and develop better motor skills. Novartis, the manufacturer, justifies the high price with the extremely lengthy and costly manufacturing process, as well as the one-time-only treatment.
When it comes to covering the costs, many health insurance companies initially react dismissively. Attorney Johannes Kaiser from Olpe has specialized in such cases. Most health insurance companies point out that there are no long-term studies, he says. "But since the drug has only been around since 2019, of course no results can be available yet," Kaiser says. Ajla's parents contacted Kaiser because their health insurance company resisted covering the treatment.
While looking for help, she came across the lawyer online, says Suada Cerimovic. The fight for the costs to be covered lasted longer than a year until Kaiser was able to reach an agreement with the insurance company. It was a particularly rocky road for the family, says the lawyer. It was important to receive the medication before the child's second birthday, and Ajla was just past that.
Cerimovic is all the happier that her daughter's health has improved after only a short time. "I hope that her example will encourage other parents who are affected," she says. Ajla is being cared for in a Remscheid nursing home, and her mother is with her every day. There is not enough space in their apartment to adequately care for the child, and the family also has three sons aged 15, 13 and eight. They are getting a bit of a raw deal at the moment, as father Rasim is the sole breadwinner, out working to support the family. Colleagues, friends and relatives have also helped the family financially. Suada Cerimovic hopes that she and her husband will eventually be able to afford a larger apartment where Ajla can live with them. For she firmly believes that this will be the case. "Ajla has already accomplished so much," she says. "I believe in this little girl.”