When the Food and Drug Administration (FDA) accepted Zolgensma, a lifesaving medication to treat spinal muscular atrophy, parents of younger kids with the rare and deadly illness rejoiced.
However, that decline was rapidly strengthened by the price sticker: the most expensive ever for a single dose of a drug – $2.1 million.
“I was pretty shocked,” stated Sarah Stanger of Monroe, Ohio. “You know, as a teacher, we don’t have $2.1 million, and I don’t know anybody who does.”
Stanger’s son, the Duke, was diagnosed with the condition as an infant. When Zolgensma was permitted in May, doctors mentioned that the drugs were the best choice for Duke. But the family’s insurance company disagreed to pay for it.
Without the drug, Duke’s future is blank. In infants with SMA, nerve cells within the brain stem, and spinal cord that control the muscles needed for walking, speaking, breathing, and swallowing are destroyed because a critical protein is missing. Because the disease progresses, muscles weaken, and atrophy and patients lose their skill to roam, eat or even breathe, in line with the National Institute of Neurological Disorders and Stroke.
“Once these neurons die, there’s no reviving them,” Stanger told NBC News. “With no treatment, most youngsters will pass away by age 2.”
When the drug was licensed, Novartis, the maker of Zolgensma, said that it anticipated insurance companies would cover the cost of the treatment. Novartis additionally stated that the high value of the drug was warranted, and the one-time treatment was half the cost of a decade of therapy with a current SMA drug.