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Health

FDA poised to approve $5 MILLION gene therapy to treat muscular atrophy

Monday 20/May/2019 - 08:50 PM
Sada El Balad
Edited by Ahmed Moamar
EFA sets date to begin new soccer season
A new gene therapy for the rare disease، spinal muscular atrophy، will be the most expensive in the world at up to $5 million، after it gets the anticipated Food and Drug Administration (FDA) approval this month، the Daily Mail said.

Novartis's drug، Zolgensma، is only the second drug in the world to treat the rare muscle wasting disease، spinal muscular atrophy (SMA) and its eye-watering price outstrips its predecessor، Spinraza، which costs $750،000 for the first dose.

Before the debut of Biogen's Spinraza، there was no cure or even treatment available for the debilitating and often deadly disease.

But there's a dark side to these advancements، especially in gene therapies.

With desperate families of pediatric patients and neither competition nor regulation to keep drug companies from hiking their prices، pharmaceutical companies like Biogen and Novartis can be wild with their price tags.

Currently the two are locked in a marketing battle - and the outcome could set precedent for the pricing of emerging drugs for rare diseases.
Between 10،000 and 25،000 American children and adults have SMA، a genetic disease that leaves them with deteriorating motor nerves.

Many with the disease cannot walk or stand. Some can barely move and eventually require a respirator to do the work of their lungs for them
But gene therapy has offered some hope for a better and longer life for people with the debilitating disease.

The first of these therapies، Spinraza، was greeted with fanfare from patients and their families، but left the jaws of ethicists، advocates and many health policy makers on the floor with its high list price.

Spinraza costs $750،000 combined for the first four loading doses - injected directly into the spinal fluid - followed by a maintenance doses given every four months for the rest of the patients life، running about $375،000 a year.

Once injected، the gene therapy provides SMA sufferers with something akin to a helper gene that produces a similar protein to the one they lack.

Some 7،500 people around the world have received Spinraza. It doesn't cure the disease، it doesn't work for everyone، and it doesn't work as well for some as others.

But it's a shot at improvement.

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