You have to cough when you breathe. Sitting independently is hardly possible. It is uncertain whether they will ever be able to walk. Anyone who sees young children with spinal muscular atrophy wants to help immediately. Around 80 children in Germany are affected by the rare hereditary disease every year. Without therapy, most die at the age of two or three years, some sooner. Your muscles get weaker. Since a complex interplay of the muscles is also necessary for breathing, they suffocate.
Since 2019 some of the children have been given a one-time injection. It costs 2.3 million euros and is the most expensive drug in the world. Zolgensma is the name of the drug from the Swiss pharmaceutical company Novartis. For the parents of sick children, the remedy in which a defective gene is exchanged for a functioning one is either a miracle cure or a last hope. The Federal Joint Committee (G-BA), the body that decides which examinations and therapies are reimbursed, announced last week that “no additional benefit compared to the comparator therapy could be determined” for Zolgensma.
In individual cases, health insurers will still cover the costs. In general, however, an additional benefit must be proven for sales over 50 million euros. Drugs for rare diseases with lower sales do not have to provide evidence. A comparative therapy against the rare muscle disease has been on the market since 2017. The remedy is called Spinraza, has to be injected into the spinal canal four times a year in an unpleasant procedure and costs 100,000 euros per single dose. After six years, so the argument from Novartis, the two million injection would be cheaper. If the effect lasts that long.
For the G-BA, the study situation has so far not been sufficient to attest Zolgensma an additional benefit. There are delicate discussions when it comes to how expensive a therapy can be to save a life and how many years can be gained with it. Josef Hecken, the chairman of the G-BA, emphasizes that the “decision on Zolgensma will most likely only be provisional”. It is planned to consult again about the expensive means “by summer 2027 at the latest”, then “statements on the long-term additional benefit” would be possible. For the parents, the hope of an effective therapy remains. Little Michael from near Ludwigsburg, for whose treatment a donation campaign was initiated in 2019 with great media sympathy, turned three in August. Two years ago he got the million dollar injection, recent photos show him trying to walk on a frame.
It is to be hoped that the therapy works. Hope can carry far. Hollywood filmed in 1993 the fight of parents against the fate of their son, who suffered from the nervous disease adrenoleucodystrophy (ALD). They did not subside until a therapy for the deadly ailment was developed. Unfortunately, “Lorenzo’s Oil” did not live up to expectations in the long run. The young man, whose suffering in the cinema had touched the audience, died in 2008.