Gene theraphy for Spinal Muscular Atrophy approved by FDA
A new drug to treat spinal muscular atrophy (SMA) approved by the Food and Drug Administration (FDA) on Friday will come with a price tag of $2.1 million, making it the most expensive medicine in the world.
The drug, called Zolgensma, treats SMA, a genetic disease that causes debilitating muscle weakness and paralysis, and is a leading cause of infant mortality.
The high price tag is the latest illustration of how innovative drugs, while holding immense promise for improving the lives of the patients who need them, also pose a financial burden on the health care system through their cost.
The maker of the drug, Novartis, argued on Friday that the cost is still less than what it costs to treat people with SMA, which the company said was $4.1 million over 10 years.
"Zolgensma is a historic advance for the treatment of SMA and a landmark one-time gene therapy,” said Vas Narasimhan, Novartis’s CEO. “Our goal is to ensure broad patient access to this transformational medicine and to share value with the healthcare system.”
A top FDA official said the drug provides the opportunity for improved care for people with SMA.
“Children with SMA experience difficulty performing essential functions of life. Most children with this disease do not survive past early childhood due to respiratory failure,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival.”
The drug will treat children under the age of two.
https://thehill.com/policy/healthcare/445451-fda-approves-worlds-most-expensive-drug-at-%242.1M