Sunday, February 12, 2017



Last week, the Food and Drug Administration announced that a drug previously not officially available to patients in the United States had been approved. Deflazacort, a corticosteroid, has been shown to be useful and life-prolonging for patients with Duchenne muscular dystrophy, a rare and fatal disease. Its U.S. launch has been delayed, however, after lawmakers questioned the dramatic price hike that came with its debut here.
How much of a price increase? That depends on who you ask. The drug will be marketed under the name Emflaza, and its sticker price will be $89,000 for a year’s worth of pills. The CEO of Marathon Pharmaceuticals, the company marketing the drug, said that the typical cost per patient after discounts and insurance rebates would be $54,000 per year.
The Wall Street Journal reports that families currently pay an average of $1,200 to import deflazacort from countries where it’s available. The price varies: One mother reports paying $800 to $1,000, and other families pay up to $1,600.
Marathon Pharmaceuticals didn’t invent or even perform its own clinical trials on deflazacort, but bought the rights to data from clinical trials performed on Duchenne muscular dystrophy patients in the 1990s, using that to submit an application to the FDA. The clinical trial data showed that the drug helped patients keep more muscle strength, and patients taking it retained their ability to walk for longer.
The company can do this because of the Orphan Drug Act, which gives pharmaceutical companies exclusive patent rights for a few years after developing a new drug or gaining approval for a different use for an existing one. It’s an important program when used as intended, but this isn’t really its intended use.
There are only around 12,000 patients with the disease in this country, and the Washington Post reports that an estimated 7-9% of them even have access deflazacort. In theory, the cost to patients will be close to zero between insurance and discounts, but the high price to insurers is still frustrating.
Lawmakers have even taken up the cause: Sen. Bernie Sanders (VT) and Rep. Elijah Cummings (MD) sent Marathon a letter asking for “detailed information” about the drug’s development, including how much it actually cost Marathon to bring it to market.
Rep. Robert Aderholt of Alabama, the chairman of the committee that funds the FDA, also questioned the drug’s price hike.
“It makes me question whether the current construct of how FDA approves orphan drugs does more harm than good if companies have found a way to game the system,” Aderholt said in a statement yesterday.
Coincidentally, yesterday Marathon announced that it plans to delay the Emflaza launch to discuss the pricing scheme with caregivers and other stakeholders. It was originally supposed to go on the market last month.
As of February 12, 2017:

I have previously written about Duchenne muscular dystrophy, a rare and fatal, genetic muscle disease that affects children  (CLICK HERE FOR PREVIOUS POST OF SEPTEMBER 19,2016) and (CLICK HERE FOR PREVIOUS POSTS PRIOR TO SEPTEMBER 19, 2016)

Also check for further information- Cure Duchenne Organization website-(Click Here).

What Is Duchenne Muscular Dystrophy?

DMD is a degenerative neuromuscular disorder causing severe progressive muscle loss and even premature death. It is one of the nine types of muscular dystrophy and it is mainly caused by the absence of dystrophin, an important protein that helps keep the muscle cells intact.
Symptoms of this disease first appear during childhood, usually between ages 3 and 5. It primarily affects boys, but can also affect girls in rare cases.
Patients affected with DMD usually do not survive beyond their teen years, but thanks to advancement in medicine and cardiac care, the life expectancy of boys affected by the condition is increasing dramatically. Some of the patients have reached young adulthood, with some having families of their own and others living into their 40s and 50s.

                       UPDATED FEBRUARY 12, 2017

A first corticosteroid treatment for Duchenne muscular dystrophy (DMD) regardless of mutation, Emflaza  (Generic name is deflazacort), has been approved for use in patients ages 5 and older by U.S. Food and Drug Administration (FDA) as of February 9, 2017.
“We are in a new era in the treatment of Duchenne muscular dystrophy. For the first time, patients in the U.S. with Duchenne will have widespread access to an FDA approved medicine that is indicated for all genetic forms of the condition,” Timothy M. Cunniff, an executive vice president at Marathon Pharmaceuticals, the drug’s developer, said in a company press release.
Corticosteroids, including deflazacort, are widely used in many countries to slow the decline in muscle strength in boys with DMD. They work by reducing inflammation and suppressing the immune system. 
“This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy,” said Billy Dunn, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in an FDA agency press release. “We hope that this treatment option will benefit many patients with DMD.”
Emflaza will be available in tablet form and as an oral suspension. Its effectiveness in delaying DMD symptoms has been demonstrated in several clinical trials.
One Phase 3 study involved 196 DMD patients, ages 5 to 15, all with a documented mutation in the dystrophin gene and evidence of muscular weakness before 5 years old. The 12-week trial compared the effectiveness of two corticosteroids —  deflazacort (0.9 mg/kg/day) and prednisone (1.2 mg/kg/day) — against placebo.  

Results showed that both drugs significantly improved muscle strength in treated patients compared to placebo, but the lower dose of deflazacort caused fewer side effects, including weight gain, while demonstrating efficacy. Significant improvements in scores of muscle strength were also maintained for a one year, the study reported.
A two-year trial involving 29 DMD patients also showed a “numerical advantage” in average muscle strength among deflazacort treated boys compared to placebo, the FDA reported in its release. Treated patients also appeared to retain an ability to walk for a longer time than those in the placebo group. 
Common side effects associated with Emflaza included facial puffiness, weight gain, upper respiratory tract infections or coughing, and increased daytime urination.
“EMFLAZA is an important new drug with proven benefit in boys with Duchenne muscular dystrophy, increasing muscle strength and physical function,” Robert C. Griggs, MD, with the University of Rochester Medical Center and an early investigator of deflazacort, said in the Marathon release. “By undertaking the research needed to secure FDA approval … we now know more about the drug, its dosing and possible interactions. These are all advances in care for patients with Duchenne.”
The FDA previously designated Emflaza an Orphan Drug as a treatment for a rare disease with a high unmet need, and approved its use under priority review.
Marathon announced it plans further studies of Emflaza in DMD patients, including a trial examining the safety and effectiveness of different dose regimens in younger patients, and a safety and efficacy study in non-ambulatory patients.
The company will also continue to work on other DMD treatments it is developing, Cunniff said.
“We are pleased to learn that the FDA has approved EMFLAZA,” added Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy, the largest U.S. nonprofit group for Duchenne MD. “While steroids are considered standard of care for Duchenne patients, there has been no steroid specifically approved for Duchenne. The FDA approval … provides options for our families when making crucial decisions about care with their providers. We hope that this approval gives more families access to this important medication.”

Deflazacort has been available outside the United States for decades.
The big issue facing Marathon will be how to price the drug. Marathon did not invent deflazacort and patients have been able to import it inexpensively for personal use for years because there was not an approved U.S. treatment.
That will now change. Patients will no longer be allowed to import the drug now that an FDA-approved product is available. Northbrook, Illinois-based Marathon said the drug will carry a list price of $89,000 a year but that the company will provide a "robust" assistance program for patients who are not covered by insurance.

Yes $89,000 a year. That's the price tag although It's been available in other countries for years and patients in this country were allowed to import it. Now they'll be able to get it in the U.S. but there are mixed reactions that that news.
Gabe Griffin's family has been fighting for years to help Gabe live longer. He was diagnosed with Duchenne muscular dystrophy at the age of three. ABC33/40 has spent time with him in his backyard and at charity events. His father Scott Griffin says Gabe has been taking deflazacort for six years. When they tried to take him off the drug it didn't go well. "We did notice that he started to get weaker. We have stairs coming up our front door he was having trouble navigating those."
Griffin says they've been paying out of pocket $418 a year to have the drug shipped here from Europe. When he heard the FDA approved it in the U.S. "my initial thought was wow won't have to go through the headache of coming through customs and then you get to the price tag."
MDA Executive Director Terri Wilson says having the drug in the U.S. will make it more readily available for families. MDA says based on the patient assistant programs it's estimated the co-pay for the drug will be less than what families spent out of pocket to import it from other countries.
Griffin says he's skeptical because even with vouchers and assistant programs the drug still may cost a lot more. "I have a $3,000 deductible and so my insurance company doesn't pay anything until my $3,000 deductible is met so I went from paying $418 a year to essentially $3,000 a year."
The MDA says this is the second drug approved by the FDA in the past six months to treat patients with Duchenne muscular dystrophy and that is unprecedented. They are celebrating that fact.
The FDA approval of Emflaza marks a third major proof point of MDA’s research program in recent months. Exondys 51 in September 2016 became the first disease-modifying drug approved by the FDA to treat DMD. 

But Griffin says there are still a lot of questions about that $89,000 price tag like how much will insurance cover and how much will get passed onto the patient. 

He is waiting to see what happens.

What happens to those victims of this horrific disease who cannot afford the enormously expensive cost of this medication, do they not get access to this drug, do they continue to suffer in agony, do they stay alive or die because a pharmaceutical company cares more about profit than human lives?