World's most expensive drug: Approved drug for muscle disease
The Swiss pharmaceutical company Novartis has received approval in the United States for gene therapy to treat a rare muscle disorder. The single dose of the most expensive drug in the world is said to cost over two million US dollars.
Single dose costs more than two million US dollars
The Swiss pharmaceutical company Novartis received approval from the US Food and Drug Administration (FDA) on Friday for gene therapy for the treatment of a rare muscle disease. The price of the drug immediately sparked controversy over the rising cost of drugs and their payment. Because: A single dose of the preparation should cost $ 2.125 million.
For the treatment of a genetic muscle disease
AveXis, whose parent company is Novartis, markets gene therapy called Zolgensma, as reported by various media, including the news channel “CNN”.
The FDA approved the use of the drug in children under two years of age to treat the inherited disease of spinal muscular atrophy (SMA).
The hereditary disease leads to progressive atrophy of the muscles and to premature death or lifelong disability.
"In Germany there are around 5,000 people affected at all ages and around 2 million Germans are carriers for SMA," explains the German Muscle Foundation on its website.
"Worldwide, the numbers are 375,000 people affected and 150 million people are carriers," it continues.
Controversy over the rising cost of drugs
The Novartis remedy is intended to help those affected.
"The potential of gene therapy products that change the lives of patients who may be at risk of fatal or worse, or even death, gives hope for the future," said FDA Commissioner Dr. Ned Sharpless in a message.
However, the price of the drug immediately sparked controversy about the rising cost of drugs and their payment.
"It's a symbol of our broken system that is forcing us to pay the price that drug companies charge for life-saving new drugs," said David Mitchell, founder of the Patients For Affordable Drugs advocacy group, according to CNN.
Novartis officials said the price had achieved the targeted cost efficiency. The newly approved treatment would cost about half of what another treatment for the disease, Spinraza, could cost over ten years.
The company expects the drug to be approved in Europe and Japan later this year.
Therapy currently in use
Spinal muscular atrophy (SMA) is currently being treated with Spinraza from the US biotechnology company Biogen.
"The drug must be administered intrathecally, i.e. by a lumbar puncture (nerve water withdrawal in the same amount as the drug is supplied). This has to be repeated regularly, ”explains the German Society for Muscle Diseases (DGM) on its website.
"The treatment regime provides for an application at intervals of 14 days, then 30 and 60 days and then every four months," it continues.
According to the Reuters news agency, treatment costs $ 750,000 a year in the first year of treatment and subsequently $ 375,000 a year.
Novartis said the most recent price range for the drug, now approved in the United States, was $ 1.5 to $ 5 million. (ad)