US. Regulators have approved the most expensive drug ever for a rare condition that destroys a child's muscle control and kills almost all patients with the most common type of disease within a few years.
The treatment costs $ 2.125 million. The expenses for patients vary depending on the insurance coverage.
The drug, distributed by the Swiss drug manufacturer Novartis, is a gene therapy that treats a hereditary disease called spinal column atrophy. The treatment targets a defective gene that weakens a child's muscles to such an extent that it can not move and ultimately can not swallow or breathe. Every year about 400 babies are born in the USA.
The Food and Drug Administration approved on Friday the treatment called Zolgensma for all children under 2 years of age who have been confirmed by a genetic test to have one of the three types of children with the disease. The therapy is a one-time infusion that lasts about an hour.
Novartis announced that insurers will make payments of $ 425,000 per annum over a five-year period and will provide partial discounts if treatment fails.
Another drug for the US-approved disease is a drug called Spinraza. Instead of a single treatment, it must be given every four months. Biogen, Spinrazas manufacturer, estimates a list price of $ 750,000 for the first year and $ 350,000 per year thereafter.
The independent charitable group Institute for Clinical and Economic Review, which evaluated the value of expensive new drugs, calculated that price. The cost of new gene therapy amounts to between $ 1.2 and $ 2.1 million, as it "lives the families affected by this devastating disease changed dramatically. "
ICER president dr. Steven D. Pearson called the price for the treatment. A positive result for the patients and the entire healthcare system.
The defective gene, which causes spinal muscular atrophy, prevents the body from producing enough of a protein to function normally with the nerves that control the movement, without the protein killing the nerves.
Bei At least 90% of patients, at the age of 2 years, die and all living patients need a respirator to breathe, children with less severe types are slower disabled and can live for up to a few decades.
Zolgensma provides a healthy copy of the defective gene, allowing nerve cells to begin to produce the required protein, stopping the deterioration of the nerve cells and allowing the baby to develop more normally.
In patients' tests, babies with the most severe form of the disease had Six months after birth Zolgensma suffers from limited muscle problems earliest ones did best.
Babies given Zolgensma after six months no longer lose muscle control, but the drug can not reverse damage already done.
Evelyn Villarreal was one of the first children to be treated at the age of eight weeks. Her family from Centerville, Virginia, lost their first child after 15 months with back muscle atrophy. Two years later, when Evelyn was born, a test showed she was also suffering from the disease, and the family enrolled her in the gene therapy study at the Nationwide Children's Hospital in Columbus, Ohio.
Evelyn is now 4½ years old and shows no other muscle problems than getting small issues up, said her mother, Elena Villarreal. She feeds herself for a long time, she draws and speaks well and will go to kindergarten in autumn.
"She is very active and goes to the playground a lot," said Elena Villarreal. "She goes and even jumps."
It is still too early to know how long the benefit of the treatment will last, but according to Drs. Jerry Mendell, a neurologist at Nationwide, is raising doctors' hopes that they could last a lifetime children. Mendell led one of the early patient studies and is Evelyn's doctor.
"It's starting to look that way," he said, because some children who are now 4 or 5 have no symptoms.
Early diagnosis is why Novartis has collaborated with states to obtain genetic testing for newborns that are required at birth. It is expected that most states will have this requirement until next year.
The FDA said the side effects included vomiting and potential liver damage, so patients need to be monitored for the first months after treatment.
Follow Linda A Johnson at https://twitter.com/LindaJ_onPharma .
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