Feb 14 2017
In an interview, Marathon CFO Babar Ghias noted the company will likely receive much less in net revenue than the list price, after providing discounts to government insurers and financial support to patients who can not afford the medication.
Corticosteroids are a common treatment option for DMD around the world but this is the first time that the FDA has approved any drug in the class to treat the condition and also the first time that Emflaza was approved for any kind of use. Marathon said the research and development costs necessary to make that happen justify the high price-tag of the drug.
Emplaza is a corticosteroid that reduces activity within the immune system and decreases inflammation. The drug, deflazacort, can help to treat patients ages 5 years and above that suffers from the disastrous and deadly disease Duchenne muscular dystrophy (DMD).
DMD is the most well-known kind of muscular dystrophy that caused by an absence of dystrophin, a protein that keeps muscle cells in place. The top 100 orphan drugs in the US cost an average of $111,820 a year per patient in 2014, according to a report by Evaluate, a market research firm.
Marathon Pharma's chiefe financial officer Babar Ghias told reporters that the net price of the drug will be $54,000 after some rebates and discounts.
Sen. Bernie Sanders (D-VT) and Rep. Elijah Cummings (D-MD) on Monday wrote to Illinois-based Marathon Pharmaceuticals CEO Jeffrey Aronin, calling the company's plan to charge $89,000 annually for its recently approved treatment "outrageous".
"Marathon's apparent abuse of government-granted exclusivity periods and incentives to sell what should be a widely available drug for $89,000 a year is unconscionable", Sanders and Cummings wrote in the letter.
Sanders and Cummings accused the company of taking advantage of the FDA's orphan drug program, which will give it seven years of exclusive sales rights on the treatment.
Clinical studies of the drug deflazacort showed improvement in muscle strength compare to those who took a placebo. An overall stability in average muscle strength was maintained through the end of study at week 52 in the deflazacort-treated patients. The agency granted fast track designation and priority review to the application.
The sponsor is receiving a rare pediatric disease priority review voucher under a program meant to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. Other side effects include elevated blood pressure, mood changes, infection, and serious skin rashes.
Feb 14 2017
Cindy Crawford Responds To Amal Clooney's Baby News In The Best Way
He hit the jackpot". "I was so happy for him, and I was like, 'How far along is she?' And he goes, 'Eight weeks'". She announced: " Beyoncé is not the only superstar expecting twins".
Feb 12 2017
USA's pull-out from START-3 to trigger massive arms race
President Trump had another embarrassing phone call with a foreign leader - this one with potentially unsafe consequences. It is unclear whether Trump's national security adviser, Mike Flynn , briefed the president before he spoke with Putin.
Feb 12 2017
Volunteers in New Zealand work to stop fresh whale stranding
Some of the surviving whales had been re-floated at high tide this morning but many quickly became stranded once the tide ebbed. Sometimes the whales are old and sick, injured, or make navigational errors particularly along gentle sloping beaches.
Feb 12 2017
Ford to invest $1 bln in autonomous vehicle tech firm Argo AI
Experts have long warned that legacy automakers such as Ford risk being disrupted by tech companies like Google. The Argo AI team will focus totally on developing Level 4 autonomy without driver input.
Feb 11 2017
Federal Judges Refuse To Reinstate Trump's Travel Ban
Three federal judges heard oral arguments Tuesday to determine whether the travel ban would be reinstated. The President was angry that his emergency appeal to overturn Judge James Robart's ruling was denied .
Feb 10 2017
Family Vows to Fight for Arizona Mom Deported Under Trump's Order
As protestors surrounded the auto and held signs urging ICE not to breakup families, they shouted "liberation, not deportation". Immigrant advocates say her detention reflects the severity of the Trump administration's crackdown on illegal immigration.
Feb 09 2017
Panneerselvam revolts, says he was forced out as Tamil Nadu CM
He (OPS) is a traitor, somebody has asked him to speak like this, ' she said of OPS before whisking her MLAs away. Mr Panneerselvam also said he was willing to withdraw his resignation "if people wanted".
Feb 08 2017
January 2017 new auto registrations up 2.9% YOY
New auto sales reached a 12-year high in January, according to an industry trade association. In terms of market spread, private purchases were up by 5% and represented 44% of all sales.
Feb 07 2017
Sergio Aguero casts Man City future into serious doubt after Swansea snub
Meant to be a backup for Aguero, along with fellow backup Kelechi Iheanacho, Jesus has quickly gotten used to English soccer. Aguero is City's top scorer this season with 18, 11 more than Raheem Sterling , who is next on the list.
Feb 07 2017
Afghan civilian casualties hit record high in 2016
Of those casualty numbers, 3,512 were children - 923 dead and 2,589 injured, marking a 24% increase on the previous recorded figures.
Feb 13 2017End goal of Turkey in Syria is Raqqa: Tayyip Erdogan
Feb 12 2017Iran reacts to new United States administration at Islamic Revolution anniversary marches
Feb 12 2017World's heaviest woman weighing 500 kg lands in Mumbai for surgery
Feb 12 2017Lake Oroville overflows into emergency spillway
Feb 12 2017NCAA Tournament bracket revealed early on CBS: Top 16 seed expert predictions
Feb 10 2017Moscow 'Unconditionally' Condemns Deadly Attack on Red Cross in Afghanistan
Feb 10 2017Freddie Mac: Mortgage Rates In Holding Pattern Due To Economic Uncertainty
Feb 09 20171-2-Switch will include 28 minigames
Feb 07 2017Suspect In Queens Jogger's Violent Rape And Murder Arrested
Feb 06 2017After party, Sasikala to take full control of government
Feb 13 2017Listeria Recall: Meijer Brand Colby Cheese and Colby Jack Cheese
Feb 13 2017South Korea says North Korea test fires missile into sea
Feb 13 2017Steinmeier Becomes German President
Feb 11 2017Past Quarters Earnings Report for Cliffs Natural Resources Inc. (NYSE:CLF)
Feb 11 2017China, US Warplanes Nearly Collide Over Disputed South China Sea
Feb 10 2017Cognizant (CTSH) Q4 Earnings & Revenues Increase Y/Y February 08, 2017
Feb 10 2017Sarfraz Ahmed to lead Pakistan ODI team
Feb 09 2017Falcons Super Bowl appearance causes hilarious tardy excuses
Feb 09 2017Canada's population now at 35 million, growth in Waterloo Region and Guelph
Feb 09 2017Microsoft will soon bring UWP games to your Xbox One
Feb 08 2017Brexit French PM, Juncker say deal must be worse than membership
Feb 07 2017Hekmatyar removed from designated terrorists list