The FDA approved a new drug for the treatment of spinal muscular dystrophy, and the treatment cost was $265,438 +0.25 million. Novartis, a well-known pharmaceutical factory in the United States, recently obtained the approval of the US Food and Drug Administration (FDA) to market a gene therapy Zolgen *** a for the treatment of spinal muscular dystrophy (SMA), which is suitable for children under 2 years old. This new drug is the first one-off treatment for spinal muscular atrophy in the world, which can be completed with only one injection.
The price of this new drug for rare diseases is NT$ 67 million (US$ 265,438+US$ 2,500), which once again sets a new record of the most expensive drug in the world, breaking the previous record of Luxturna, an ophthalmic gene therapy drug, which is also gene therapy. The price of this drug is NT$ 265,438+00,900 (US$ 850,000).
Spinal muscular dystrophy is a rare genetic disease called spinal muscular dystrophy (SMA), which is caused by gene mutation of motor neurons. The gene exists in protein of motor neuron, and this function protein is very important for motor neuron, which controls the muscle movement of the whole body. If there is no functional protein, there will be muscle weakness, problems such as looking up, swallowing and breathing disorders. Most sick children will die before the age of 2 because of respiratory failure.
Spinal muscular atrophy is a hereditary disease and the second most serious autosomal recessive disease in children. In Taiwan Province Province, the carrying rate of SMA is about 2-4%, and about 1 person is the carrier for every 30 to 50 people. If both husband and wife are SMA carriers, then every fetus, whether male or female, has a 1/4 chance of becoming an SMA patient. The neonatal morbidity is about1/6000-110000. If there are190,000 newborns in Taiwan Province Province every year, there will be more than 10 neonatal cases in one year.
In the past, other treatments cost NT$ 6.5438+27.5 million (US$ 4 million). Before Zolgen *** a was developed and marketed, the FDA approved a drug Spinraza for the treatment of spinal muscular atrophy in September, 2065.438+06, which was the first drug approved for this disease. This is an antisense oligonucleotide (ASO) injected into the spinal cord. The total cost of chronic treatment for 10 is about NT$ 1 275,000 (US$ 4 million). Patients must receive/kloc-0 injections every four months, and they need lifelong injections, otherwise they will fail. Zolgen *** a, on the other hand, introduces new DNA into human body to correct the wrong gene, and adopts intravenous injection, which only needs one injection, without frequent spinal cord puncture injection as in the past.
However, this new drug also has side effects. Taiwan Province Province has not approved the import, and Zolgen *** a is not completely without side effects. The side effects are increased liver enzymes and vomiting caused by acute liver injury. The FDA mentioned in the approval requirements that patients need to be warned of possible side effects in the label.
At present, the US Food and Drug Administration in Taiwan Province Province has not approved the import of this new drug, but many large teaching center hospitals in China have begun to support the promotion of genetic screening for spinal muscular atrophy, so as to avoid bringing this expensive and rare disease to the next generation due to heredity.