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.
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.
A new drug to treat the rare disease spinal muscular atrophy is set to become the most expensive in the world at up to $5 million – and experts worry it could set a costly precedent
THE PROMISE OF AN EXPENSIVE TREATMENT IS BETTER THAN NONE TO SMA PATIENTS
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.
‘#SPINRAZA GOALS’: BROTHER AND SISTER WITH SMA START CRAWLING
Born with spinal muscular atrophy, Ruby (left) and Landon (right ) Sun had both lost the ability to walk, crawl or, in Landon’s case, sit up – until they started gene therapy
Ruby and Landon Sun, now eight and five, were both born with SMA.
Just after her first birthday, when she was learning to walk across the living room, Ruby started falling, was diagnosed and spent the following five years in a wheelchair.
Landon started losing control of his legs around 10 months.
Both children got their first Spinraza injections in 2017.
Within weeks of their loading doses, Landon was sitting up, and Ruby was walking on her knees.
‘They’re very proud, we joke around the house, whenever they do something really impressive, or exciting, they’ll say “hashtag Spinraza goals!”‘ their mother, Danyelle, says.
There is still no cure.
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.
A NEW DRUG IS SAID TO BE FASTER – BUT IS IT BETTER?
Now, there may be a different shot on offer, in the form of a single injection of an altered virus in Zolgensma.
Novartis claims the drug will work more quickly and effectively by replacing the exact faulty gene (SMN1) involved in SMA.
But the use of viral vectors for gene therapy can be dangerous – and Novartis has acknowledged that at least one patient death in the small trials – one consisted of 12 patients, another of 15 – it has conducted has died.
‘When you have a very sick patient on a drug who dies, it’s not always easy to determine if it was the disease or the drug,’ says Dr Robert Klitzman, director of Columbia University’s bioethics program, who has written extensively on drug pricing.
‘It’s not clear to me that we have all the information we need to understand how safe and effective each of these drugs are in the short term or the long term.’
Yet Novartis is confident its gene therapy will get the FDA’s approval this month, and it’s already setting its sights on a high price tag.
PRICES ARE GETTING HIGHER WITH EACH NEW RARE DISEASE DRUG – AND THERE’S NO ONE TO STOP IT
Like Spinraza before it, part of the problem with Zolgensma’s price is that ‘much of the research that has eventually led to these drugs was [partially] funded by the government, but the NIH,’ says Dr Klitzman.
‘The drug companies say, “look, individual patients aren’t going to pay for the drugs, the government is”‘ – via Medicaid – ‘but the government isn’t allowed, by law to negotiate drug prices.
‘And these drug companies, which have done a lot of great things and given us a wonderful drug also have contributed enormous sums of money to both political parties and enabled them to pass legislation that has given [the companies] enormous economic power, so as we get new drugs for gene therapy, companies are seeing it as “the sky’s the limit.”‘
In 2018, Novartis made political donations in the US to the tune of $1.14 million, on the books.
Biogen gave over $2.34 in political contributions the same year.
Critics of Big Pharma suspect that those donations to politicians and lobbying bodies may help to ensure that no laws capping drug prices are passed.
‘Are the drug prices too high? Yes. How high should they be? It depends on how effective they are, and we need to see more data to know how high the prices should be,’ says Dr Klitzman.
IS THE FDA’S EXPECTED APPROVAL FOR ZOLGENSMA COMING TOO SOON?
He suspects that the FDA is similarly in the pocket of pharmaceutical companies, where the agency has better access to drug makers’ money.
‘These decisions, ethically, should be based on risk, benefit and relative value for people’s lives, not political viewpoints when they may be paid by PR companies that are pushing for approval,’ Dr Klitzman said.
‘In the FDA’s [likely] passing of both drugs – because I think this is precedent setting – with other disease treatments that are gene therapies – we should be looking at the bottom line: what patients are getting better and for how long.’
Instead, the SMA drugs may be setting a very different trend, in which approvals and prices run wild – and up.
‘These drugs are overpriced and this is becoming an increasing problem is that there’s no upper limit to how much drug companies decide to charge nor, is there a clear standard that is socially agreed upon,’ Dr Klitzman says.