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DRUGS

Novartis now has the most expensive drug ever after getting US approval

Swiss pharmaceutical company Novartis on Friday announced it had received US regulatory approval for a gene therapy that treats a rare childhood disorder and has a price tag of $2.1 million, making it the most expensive drug in history.

Novartis now has the most expensive drug ever after getting US approval
The Basel headquarters of Swiss pharmaceutical giant Novartis. Photo: SEBASTIEN BOZON / AFP
The company said Zolgensma was a one-time treatment for spinal muscular atrophy, a disease that affects about 1 in 10,000 births and which results in death or the need for permanent ventilation by the age of two in 90 percent of cases. 
 
But the announcement comes as the administration of US President Donald Trump has vowed to tackle soaring drug costs.
 
Novartis defended the pricing by saying that gene therapy was a transformative new type of treatment and was 50 percent cheaper than current treatments.
 
“Zolgensma is a historic advance for the treatment of SMA and a landmark one-time gene therapy,” Novartis CEO Vas Narasimhan said in a statement, which added that the company was working with government and insurers to accelerate coverage.
 
The total cost will be $2.125 million payable over five years at $425,000 per year.
 
Zolgensma works by providing a functional copy of the defective gene responsible for SMA to halt the disease's progression via a one-time intravenous infusion.
 
The US Food and Drug Administration said the drug's safety had been tested in an ongoing clinical trial and a completed clinical trial involving 36 patients between the ages of two weeks and eight months.
 
Most of the evidence of its effectiveness was based on the results of the ongoing trial, which found that “patients treated with Zolgensma… demonstrated significant improvement in their ability to reach developmental motor milestones” including head control and the ability to sit without support.
 
Jerry Mendell, a doctor involved in the trial at Nationwide Children's Hospital in Columbus, Ohio, added that the “level of efficacy, delivered as a single, one-time therapy, is truly remarkable and provides a level of unprecedented hope for families.”
 
The most common side effects of Zolgensma are elevated liver enzymes and vomiting, the FDA said.
 
Gene and cell therapies leverage the biology to reverse diseases ranging from congenital blindness to pediatric leukaemia.

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NOVARTIS

Switzerland’s Novartis to help make Pfizer-BioNTech Covid vaccine

Swiss pharmaceuticals giant Novartis said Friday it had signed an initial agreement to help produce the Pfizer-BioNTech vaccine against Covid-19, as countries scramble to boost supplies.

Switzerland's Novartis to help make Pfizer-BioNTech Covid vaccine
Novartis will help manufacture Pfizer vaccine. Photo by AFP.

The rare act of cooperation — in an industry usually marked by cut-throat competition — comes after French pharma group Sanofi announced earlier this week that it would also team up with rivals Pfizer and BioNTech to help produce 125 million doses of their jab.

The two-dose vaccine, which is based on mRNA technology, has been shown to be around 95 percent effective and has been approved for use by the World Health Organization and in some 50 countries.

But it is in limited supply as nations around the world race to immunise their populations against the coronavirus, which has killed nearly 2.2 million people in just over a year.

Novartis said in a statement that it would use its sterilised manufacturing facilities at its site in Stein, Switzerland to help produce the Pfizer-BioNTech jabs.

Under the agreement, the company said it would “take bulk mRNA active ingredient from BioNTech and fill this into vials under aseptic conditions for shipment back to BioNTech for their distribution to healthcare system customers around the world”.

Once a final agreement is reached, Novartis said it expected to begin production in the second quarter of the year, with initial shipment of finished product expected in the third quarter.

Steffen Lang, Head of Novartis Technical Operations, stressed that the company was “committed to leverage our manufacturing capabilities to help support the supply of COVID-19 vaccines and therapeutics around the world”.

“We expect this to be the first of a number of such agreements,” he said in the statement.

Novartis said it was already in “advanced discussions” with a number of other companies about with other production tasks, including of mRNA, therapeutic protein and raw material production for Covid vaccines and therapeutics. 

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