• News
  • Medicine

Duchenne Muscular Dystrophy Treatment Emflaza Approved by FDA

Duchenne Muscular Dystrophy Treatment Emflaza Approved by FDA

Simply put, it's because the drug has never been approved in the USA despite widespread availability to treat other inflammatory conditions like arthritis in dozens of other nations. And, like other pharma companies that scoop up "orphan drugs" - meds used for diseases that don't affect more than 200,000 people nationwide - Marathon says the high cost is justified because of the money it has poured into R&D.

According to The Wall Street Journal, the FDA-approved deflazacort would be offered by Marathon Pharmaceuticals for $89,000 or so a year, up to 70 times its cost outside the US.

The U.S. Food and Drug Administration gave approval Thursday to Marathon Pharmaceuticals to sell a drug by the brand name of Emflaza, previously sold under the generic name deflazacort, to treat Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness. It can be administered to patients 5-years-old and above. As the disease progresses, patients lose muscle strength, and usually end up confined to a wheelchair by the time they reach their early teens. It affects one in every 3600 male kids worldwide.

Emflaza is marketed by Marathon Pharmaceuticals, headquartered in Northbrook, Illinois.

The FDA also granted the "rare pediatric disease priority review voucher", that encourages the development of new drugs and treatment of rare pediatric diseases.




The drug wasn't previously sold in the US because there was no company that would think it was profitable enough to warrant the effort of getting FDA approval. But U.S. patients have been importing it from foreign countries since the 1990s after clinical trials showed its potential to reduce inflammation with fewer side effects than another steroid.

The new, American, list price for this drug is $89,000 a year.

Deflazacort is the usual treatment for DMD across the world, but it is not a cure.

Emflaza side effects are similar to other corticosteroids, such as puffiness of the face, weight gain, increased appetite, infection of the upper respiratory tract, fequent urination, stomach fat, cough, and unwanted hair growth. To compare, patients can snag the treatment for less than $1 through online pharmacies. In addition, patients who were given deflazacort lost the ability to walk later than those patients who were taking the placebo.

As well, some patients on the drug can develop certain problems with endocrine functions, elevated blood pressure, risks of gastrointestinal perforation, skin rashes, cataracts, and susceptibility to infections.

Leave Your Comment

Leave Your Comment


Latest News

  • Davis warns peers to fulfil 'patriotic duty' over Brexit bill

    Davis warns peers to fulfil 'patriotic duty' over Brexit bill

    Corbyn said: 'The government does not have a blank cheque to set up an offshore tax haven in Britain. The Bill was approved in the House of Commons with a majority of 494 against 122.
    Army Clears Way for Dakota Access Pipeline Construction to Resume This Week

    Army Clears Way for Dakota Access Pipeline Construction to Resume This Week

    Almost 80 protesters were arrested last Wednesday near the Dakota Access Pipeline in North Dakota, authorities said. Try as he might, Donald Trump will not be able to break the will of those standing up against this pipeline.

    Trump Acknowledges That The 'Replacement' Of Obamacare Will Span Years

    It took us five years to be in a position to remodel it to our tastes. Rob Casalou, chief executive of the nonprofit St.
  • Melania wins 'substantial' settlement from blogger over prostitution claims

    Melania wins 'substantial' settlement from blogger over prostitution claims

    Trump ever worked as an "escort" or in the 'sex business.' "The original article provided denials from Trump's spokesman". The suit says the Daily Mail article "impugned her fitness to perform her duties as First Lady of the United States ".

    Analyst's Proposition on The Gap, Inc. (GPS), Twilio Inc. (TWLO)

    The company presently has an average rating of "Buy" and a consensus target price of $39.75. (NASDAQ:CA) for the current quarter . In the current quarter , Twilio forecast revenue of $83 million at the midpoint of guidance, above expectations of $78.3 million.
    Weston schools on two-hour delay, freezing rain

    Weston schools on two-hour delay, freezing rain

    The state could see significant snow on Thursday , possibly up to a foot, the National Weather Service is advising. The winter storm watch will begin at midnight Wednesday and last through 6 p.m. on Thursday , the agency said.
  • Sears, Kmart dump Trump products from e-stores

    Sears, Kmart dump Trump products from e-stores

    Retailers like Bloomingdale's, Amazon (AMZN, Tech30), Lord & Taylor, Macy's (M) and Zappos all still carry Ivanka Trump products. Ivanka Trump's clothing and accessories line has taken a hit in recent months.
    The Crown Season 2 Will Also Feature American Royalty

    The Crown Season 2 Will Also Feature American Royalty

    Season two is now in production and will usher in the 1960s after Her Majesty's Armed Forces fight an illegal war in Egypt. As well as this, Matthew Goode has also been announced as playing Lord Snowdon, the first husband of Princess Margaret.

    Israel starts demolition of West Bank Jewish outpost

    Science and Technology Minister Ofir Akunis of Netanyahu's Likud party said the argument was over the right to the Land of Israel. That admonishment marked the first public disagreement between Trump and Netanyahu , who plans to visit the White House Feb. 15.
  • Verizon resurrects unlimited data, because T-Mobile and AT&T aren't f*cking around

    Verizon resurrects unlimited data, because T-Mobile and AT&T aren't f*cking around

    Open Signal ranked Sprint in the last place among the "Big Four" U.S. carriers in its latest State of Mobile Networks report . Verizon management has argued that T-Mobile and Sprint don't have the network capacity to sustain unlimited data offerings.
    Top 3 questions about Mesa woman's deportation

    Top 3 questions about Mesa woman's deportation

    Ahead of Garcia de Rayos' deportation, dozens of immigration activists Wednesday night blocked the gates of ICE's Phoenix office. It's likely that by Thursday morning, Garcia de Rayos had completed processing and was in Mexico - away from home and family.
    Final Fantasy 15 Multiplayer Mode Character Creation Tool Revealed

    Final Fantasy 15 Multiplayer Mode Character Creation Tool Revealed

    The new teaser clip gives a sneak peek at the some of the new content coming to Final Fantasy XV this spring and beyond. However, Square Enix has not yet given up on Nintendo yet, dedicating many Dragon Quest games to the console instead.
  • Sting, Timberlake, Legend and Miranda to perform at Oscars

    Sting, Timberlake, Legend and Miranda to perform at Oscars

    Although they were written by the film's composer Justin Hurwitz, Legend appears in the film as a central musical character. Justin Timberlake is slated to perform his commercially successful song "Can't Stop the Feeling! " from the movie Trolls.
    Saturday Night NBA Props: Warriors vs Thunder

    Saturday Night NBA Props: Warriors vs Thunder

    It was Adams' 11th double-double of the season and the seventh time he has scored 20 or more points for the Thunder this season. Bench strength The Sixers got 65 points from their bench and all five reserves finished with a plus-rating for the night.
    Chandler, Scott Earn USTFCCCA All-Academic Honors

    Chandler, Scott Earn USTFCCCA All-Academic Honors

    The Franklin, Massachusetts native earned All-East Region team and NE10 All-Conference second team for the second time this year. Overall, there were 431 teams that earned distinction from the USTFCCCA for their efforts both athletically and academically.
  • Don't Visit Parks During Snowstorm, City Says

    Don't Visit Parks During Snowstorm, City Says

    The storm may be trouble for commuters, but it's good news for ski fans, who've been disappointed with the until-now mild winter. With the arrival of another big storm on Thursday, San Francisco International Airport is plagued by delays and cancellations.
    Cheeses manufactured in TN being recalled over listeria fears

    Cheeses manufactured in TN being recalled over listeria fears

    - Wisconsin-based cheesemaker Sargento Foods Inc .is recalling a specialty cheese because of possible bacterial contamination. The Meijer Brand Colby Cheese and Colby Jack Cheese were sold in Meijer stores from November 10, 2016 to February 9, 2017.
    Arrest made in Karina Vetrano case, helped by DNA evidence, NYPD says

    Arrest made in Karina Vetrano case, helped by DNA evidence, NYPD says

    On Saturday, he was arrested by police at his home in East New York , not far from Spring Creek Park where Vetrano was murdered. Her father repeatedly tried to call her cell phone while she was out and after two hours grew anxious and called police.

Breaking News



Recommended

Confusion at Rangers after Warburton denies resigning

The Rangers board convened on Wednesday and agreed to accept the management team's resignations and the deal was agreed. The statement had explained events during a seemingly busy week, and stressed that "absolute commitment is essential".

Bali landslide leaves 12 people dead, homes wiped out

Dewa Made Indra, an agency official in Bali , said a new landslide in Subaya village on Friday killed one person. The national weather agency said heavy rain would continue in Bali on Saturday and Sunday.

Activision Says Call of Duty: Infinite Warfare 'Didn't Resonate' With Fans

Some are now wondering if Activision will go back to World War II or something, taking a hint from the success of Battlefield 1 . The Q4 2016 earnings call by Activision revealed the company made a staggering $3.6 billion from in-game sales in 2016.

Russian Federation halted Syrian army, rebel clash in northern Syria

MOSCOW, Russia (A.W.)-A Russian airstrike killed three Turkish soldiers in northern Syria on February 9. There was no immediate comment from Damascus on the reported clashes.

Elizabeth Warren Silenced: Senator Barred from Speaking for Rest of Sessions Debate

Mr McConnell's objection to Ms Warren's speech was put to a vote and senators voted 49-43 in his favour. The rule was enacted after a 1902 fistfight on the Senate floor . "I don't get to speak at all ".

Tesla Model S P100D Sets New and Insane Record

Once the cars accelerate beyond 70 miles per hour , super cars like the $1.5 million Ferrari LaFerrari overtake the Model S. But when it comes to accelerating off the line at a red traffic light, this Model S leaves the pack in the rear view.

Hideki Matsuyama Defends Title at Waste Management Phoenix Open

Ultimately, Matsuyama drained a birdie putt on the par-4 17th - his fourth playoff hole - to defend his title at TPC Scottsdale. Week-long attendance for the tournament this year was estimated at 655,434, by far the most for a golf tournament in history.

A Japanese Effort To Remove Hazardous Space Junk Has Failed

Earlier, Japanese agency reported that space junk collector is encountering some problem and has not been successfully deployed. It's a frustrating setback given the mounting risks posed by the almost two million bits of junk now swirling around our planet.

Afghanistan: Over 900 children killed in 2016 violence

The number of children killed in Afghanistan's conflict rose by 25% in 2016, according to the United Nations mission in Afghanistan.

Red-hot Lukaku scores four in 'crazy' Everton victory

Naturally the players will think, "Here we go again", in terms of our recent performances in the way we have conceded goals. Lukaku scored four goals as Everton defeated Bournemouth 6-3 at Goodison Park on Saturday in the Premier League.