Disease gougers: New Big Pharma scandal as FDA approves drug for terminal muscular dystrophy – but manufacturer hikes the price from $1,000 to $89,000 a year

Emflaza, a steroid, is one of the only drugs known to slow the effects of DMD
Duchenne’s muscular dystrophy is a terminal disease that mainly affects boys
Previously, families could import the drug to the US for around $1,000 a year
Last week it was approved by the FDA for US use – but the manufacturer Marathon Pharmaceuticals hiked the price to $89,000 a year
Now Marathon has paused the launch amid outrage from families and lawmakers

A pharmaceutical company has been forced to ‘pause’ the launch of a crucial muscular dystrophy drug amid outrage over its $88,000 price-hike.

Emflaza, a steroid, is one of the only drugs believed to slow the effects of Duchenne’s muscular dystrophy (DMD), a terminal disease that disproportionately affects boys.

Until now sufferers in the US were forced to import the drug for around $1,000 since it did not have FDA approval.

Last week, after years of protests from families, the FDA gave the green light to Marathon Pharmaceuticals LLC to make and sell a US version.

However, within hours the families’ joy was tinged with anger as Marathon announced the drug would cost $89,000 a year.

On Monday, Marathon declared it was ‘pausing’ the launch of Emflaza following outrage from consumers and lawmakers.

DMD is a terminal disease that gradually robs sufferers (mainly boys) of muscular control. The FDA has approved a steroid that could slow the effects – but it is $89,000 a year (file image)

WHAT IS DMD? WHAT IS THE NEW DRUG?

Duchenne’s muscular dystrophy is a rare disease.

It is the most common type of muscular dystrophy.

DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact.

The first symptoms are usually seen between three and five years of age and worsen over time.

Life expectancy is usually in the 20s, though more and more sufferers are living into their early 30s.

The disease often occurs in people without a known family history of the condition and primarily affects boys, but in rare cases it can affect girls.

DMD occurs in about one of every 3,600 male infants worldwide.

The disease causes loss of movement, weakness, and eventually death.

THE DRUG

The FDA has approved Marathon’s drug Emflaza, which is generically known as deflazacort.

The effectiveness of deflazacort was shown in a clinical study of 196 male patients who were five to 15 years old at the beginning of the trial.

Each of the participants had documented mutation of the dystrophin gene, and onset of weakness before the age of five.

At week 12, patients taking deflazacort had improvements in a clinical assessment of muscle strength across a number of muscles compared to those taking a placebo.

Most of those taking deflazacort maintained stability in their muscle strength through to the end of study at week 52.

In another trial with 29 male patients that lasted 104 weeks, deflazacort helped the majority of patients maintain muscle strength.

Patients on deflazacort also appeared to lose the ability to walk later than those treated with placebo.

In a statement posted on the patient advocacy website Cure Duchenne, Marathon’s chief executive, Jeffrey Aronin, said the company back-pedaled amid ‘concerns about how the pricing and reimbursement details will affect individual patients and caregivers.’

The company said it would maintain its ‘expanded access program’ under which 800 patients currently receive the drug for nothing. It said patients currently importing the drug from overseas would continue to be able to do so.

The furore is the latest in a series related to U.S. drug prices, including Mylan NV’s emergency EpiPen allergy treatment, that have drawn the ire of patients and lawmakers of all political stripes.

Marathon’s drug, Emflaza, known generically as deflazacort, is a steroid, one of a class of drugs commonly used to treat Duchenne’s that patients could import for personal use because it was not available in the United States. In theory, last week’s announcement that it won U.S. approval closed off that option.

Earlier on Monday independent Senator Bernie Sanders of Vermont and Democratic congressman Elijah Cummings of Maryland, called on privately held Marathon to justify the price of the drug by documenting the cost involved in bringing it to market.

Marathon, the lawmakers wrote in a letter to Aronin, did not after all develop the product, which has been around for decades.

The company acquired rights to two clinical trials that had already been completed. And it conducted further toxicology and other tests to bring it through the U.S. regulatory process.

Marathon argues that the studies it conducted had value in determining proper dosing, potential side effects and drug-to-drug interactions, allowing physicians to prescribe ‘with confidence.’

In a separate statement, Marathon said it appreciated the opportunity to discuss ‘the value of an FDA approval of a medicine’ with Sanders and Cummings ‘and futhering our shared goal that every patient who needs this drug receives it.’

U.S. President Donald Trump has said he wants to lower drug prices and increase competition though he has not spelled out how.

The Food and Drug Administration approved Emflaza under its ‘orphan’ drug program that provides incentives to drugmakers to develop treatments for diseases with small patient populations.

The incentives include seven years of market exclusivity. Duchenne’s, a devastating muscle-wasting disease, affects some 15,000 patients in the United States, mostly young boys.

‘Marathon will have a monopoly on deflazacort for years to come, preventing less expensive generic competitors from entering the market, despite the fact that this drug is already available in generic form in other countries,’ Sanders and Cummings wrote.