World's Costliest $2.1 Million Gene Therapy By Novartis Approved By FDA
Vas Narasimhan, CEO of Novartis. PHOTO CREDIT: Bloomberg photo by Jason Alden.
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World’s Costliest $2.1 Million Gene Therapy By Novartis Approved By FDA

World’s Most Expensive Gene Therapy Drug By Novartis – Worth $2.1 million has been given a green signal by the FDA.

Zolgensma – is a one-time gene therapy treatment for spinal muscular atrophy – a disorder where there is weakness & wasting of muscles. It is amongst the leading cause of infant deaths affecting one in every 11,000 infants born. In earlier announcements, Novartis had already mentioned that could price tag the treatment somewhere in between $ 1millin to $5 million.

This gene therapy treatment for SMA will cost one time – $2.1 million or $425,000 per year for a duration of 5 years. Novartis said it is touch with insurers for the creation of 5-year agreements & other payment options for the ease of the patients based on
the success of the treatment.

Novartis initiatives will lay the floor for the development of more such one-time gene therapy treatments for rare diseases. The pricing factor is something which needs to be worked on. The government & Insurers need to collaboratively frame possible ways for people to pay conveniently for these high end million dollar treatments.

Vasant Narasimhan – Novartis CEO said that Zolgensma is a historic advancement in treatment for spinal muscular atrophy (SMA). Its a landmark one-time gene therapy treatment to date & the company’s goal is to make sure the therapy reaches to a large number of patients, he added.

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Drug Cost assessing body – ICER, Institute for Clinical and Economic Review had earlier stated that Zolgensma’s cost should cross the $1.5 million margin. Later, ICER released a further statement, which said after analysis of the clinical results and taking into account FDA’s Nod – Zolgensma’s price is well under the upper bound of ICER’s value-based price benchmark range.

With this historic approval by FDA, Zolgensma apart from becoming the World’s Costliest $2.1 Million Gene Therapy, also becomes the second gene therapy to be approved after Luxterna – Spark Therapeutics eye disease treatment.

Novartis Press Release

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