WORLD'S MOST EXPENSIVE DRUG

 OPENSIVE MIND// THE KB

Invention:- Zolgensma was developed by AveXis, a Chicago-headquartered biotech that Novartis bought in 2018 for $8.7bn. It had been manufactured in Maryland.

Zolgensma (onasemnogene abeparvovec-xioi), marketed by Novartis Gene Therapies, is an FDA-approved therapy to treat SMA. It is a type of treatment referred to as gene therapy or gene replacement therapy. More than 20 private health-insurance plans and four Medicaid plans have set up coverage policies for Zolgensma since the FDA approval, according to Novartis.

How it works ?

As a gene therapy, ZOLGENSMA^® (onasemnogene abeparvovec-xioi) is designed to target the genetic root cause of spinal muscular atrophy (SMA) by replacing the function of the missing or nonworking SMN1 gene with a new, working copy of a human SMN gene. ZOLGENSMA does not change or become a part of the child's DNA.

Pathophysiology:- Survival motor neuron (SMN) proteins facilitate proper signaling between CNS motor neurons and muscles in the body § SMA is an autosomal recessive condition caused by deletions or mutations in the SMN1 gene, resulting in a shortage of SMN protein o SMN2 gene produces a small amount of functional length SMN protein, and an increase in copy number may decrease severity of disease but cannot fully compensate for the loss-of-function of SMN1.

 

Why is it so expensive?

The reason Zolgensma is so expensive is because that is the price Novartis has decided it is worth because it “dramatically transforms the lives of families affected by this devastating disease” and the claimed cost of bringing new drugs to market. But this price is not without controversy. While a genetic test for SMA costs just under $500, more than 12,500 women would have to be screened to prevent one case of SMA, which affects only about 1 in 10,000 newborns.

Result of application

Zolgensma® data shows rapid, significant, clinically meaningful benefit in SMA including prolonged event-free survival, motor milestone achievement and durability now up to 5 years post-dosing.

Dosage:- Monitor troponin-I before ZOLGENSMA infusion and on a regular basis for at least 3 months afterwards (weekly for the first month, and then monthly for the second and third months until troponin-I level returns to baseline). Now that Zolgensma is finally approved in Europe, though, Novartis has an opportunity to steal market share from Biogen's SMA drug Spinraza. That's because the European approval allows the Zolgensma to be used in children weighing up to 21 kilograms, which basically covers any child under the age of 5.

Comparison of Zolgensma and Spinraza:-

An indirect comparison of the two treatments indicates that Zolgensma may be more effective than Spinraza among infants with symptomatic SMA type 1 in terms of overall survival, independence from permanent assisted ventilation, motor function and motor milestones.

Availability:-

Novartis Gene Therapies' Zolgensma (onasemnogene abeparvovec) will be available for use in the UK's National Health Service (NHS) following recommendations by regulators in England and Wales, as well as Scotland. Zolgensma is deemed to be the most expensive drug in the world with a list price of £1.79m per dose.