A Cheap Muscular Dystrophy Drug Will Cost US Patients $89000

In a Thursday press release, the Food and Drug Administration (FDA) granted approval for a drug by Marathon Pharmaceuticals, a United States company focused exclusively on the development of new treatments for rare diseases.

The efficacy of Emflaza was demonstrated in a clinical study (n=196) of male patients aged 5-15 years at the start of the trial with documented mutation of the dystrophin gene and onset of weakness before age 5.

At week 12, those on deflazacort had improvements in a clinical assessment of muscle strength across a number of muscles compared with those on placebo. In Canada and other countries around the world, deflazacort has been used for years to treat DMD and the pills or oral suspensions range from about $1,000 to $2,000 a year for patients. An overall stability in average muscle strength was maintained through the end of study at week 52 in the deflazacort-treated subjects.

Common side effects include facial puffiness (Cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency (pollakiuria), unwanted hair growth (hirsutism), and excessive fat around the stomach (central obesity).

Less common side effects include problems with endocrine function, increased susceptibility to infection, elevation in blood pressure, risk for gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decrease in the density of the bones, and vision problems.

Patients who get immunosuppressive doses of corticosteroids should not be given live or live attenuated vaccines, the FDA warned.

FDA officials today announced the approval of Emflaza (deflazacort) tablets and oral suspension for the treatment of Duchenne muscular dystrophy (DMD). Unlike etelirsen, the drug will be approved for all DMD patients age 5 years and older.

"We now have a treatment option for kids and adults with Duchenne, which is a major advance for the community", said MDA chief medical and scientific officer Valerie Cwik. Individuals with this condition lose little by little their ability to perform activities on their own, and they usually require the use of a wheelchair before their 20s.

A voucher known as a priority review was given to the company, which essentially is equal to a ticket that can be used to get a drug in the future reviewed faster by regulators, or can be sold for hundreds of millions of dollars to another pharmaceutical company.

Related news:

Hot News

isis-killer-beheading-video-story-top Lawmaker introduces bill to repeal HB2
Feb 12, 2017 - 00:50
Brockman, who is bisexual, is one of only two openly LGBT lawmakers in the General Assembly. But the bill might not get a hearing in the Republican-dominated legislature.

Activision Blizzard (ATVI) is Reiterated by Stifel to "Buy"
Feb 11, 2017 - 00:49
The share price of $134.14 is at a distance of 38.55 percent from its 52-week low and down -1 percent versus its peak. KeyCorp. (NYSE:KEY) jumped 71.47 percent over the past one year, while it declined -0.99 percent year to date (YTD).

Google confirms merging YouTube & Play Music teams for 'best possible product'
Feb 11, 2017 - 00:48
Youtube Go v 0.26.67 comes with an install size of 6.77 MB, and app size of 8.5 MB and runs on Android Jellybean 4.1 and above. Previously available in a very limited private testing phase, YouTube Go is now in open beta.

Arizona woman deported to Mexico despite complying with immigration officials
Feb 11, 2017 - 00:47
Then the administration of President Barack Obama changed its priorities on immigration and she was allowed to stay, they said . It's likely that by Thursday morning, Garcia de Rayos had completed processing and was in Mexico - away from home and family.

isis-killer-beheading-video-story-top Facebook now lets you check the weather forecast on mobile and desktop
Feb 11, 2017 - 00:47
Though given the sometimes capricious nature of the Facebook feed, you might not always find the information when you need it. Fortunately, it's somewhat easy to go directly to that weather information, that is if you have Facebook on your smartphone.

Russian Runner Banned, Loses London Gold For Doping
Feb 11, 2017 - 00:46
The IAAF said it would no comment on the case as Savinova still has 45 days to decide whether to make an appeal. She has not raced since 2013 after being suspended following an investigation.

isis-killer-beheading-video-story-top Flights at South Florida airports disrupted by Northeast snowstorm
Feb 10, 2017 - 02:18
Photos taken across the iconic U.S. city show wrapped-up workers shovelling snow in Times Square as snowflakes fall around them. Winds are expected to reach 30 miles per hour with up to 45 miles per hour gusts as temperatures drop into the 20s overnight.

Steve Popper: Waiting for a real answer from Phil Jackson
Feb 10, 2017 - 02:07
The true answer may be revealed before Feb 23 when the trade deadline ends, according to the National Basketball Association . "[Jackson's] underestimating 'Melo's willingness to stay", a source familiar with Anthony's thinking told The Vertical .

Buffalo Wild Wings 4Q net earnings drop 38.2%
Feb 09, 2017 - 00:44
Zacks provides the average brokerage recommendation (ABR) for thousands of stocks for institutions and retail investors alike. Laurion Capital Management LP bought a new stake in Buffalo Wild Wings during the third quarter worth about $211,000.

Free Syrian Army surrounds al-Bab town in Islamic State fight
Feb 09, 2017 - 00:42
Erdogan also complained that the U.S.in the past showed little support for the campaign against the Islamic State. The Turkish Armed Forces also hit 254 Daesh targets in northern Syria, said the General Staff statement.

isis-killer-beheading-video-story-top 26% of teen e-cigarette users "dripping" e-liquid
Feb 09, 2017 - 00:40
In general, the process of vaping relies on the e-cigarette's wick (passed through a coil), and tank. Krishan-Sarin a psychiatry researcher at Yale.

Utah now ranks 14th nationally in solar industry jobs
Feb 09, 2017 - 00:39
Employers polled in the census expect another 10 percent increase this year, for a total of 286,335 solar workers. In Texas, there are 9,396 solar jobs and more than 260,000 solar jobs in the nation.

Tomb Raider: First Photos of Alicia Vikander as Lara Croft Released
Feb 08, 2017 - 00:43
And she certainly looks the part of Lara, too, sporting a tight tank top, combat boots, and her dark hair pulled back. Variety reports that Daniel Wu ( Into the Badlands ) has been cast in the Tomb Raider reboot alongside Vikander.

No conspiracy, mystery in Jaya's death: Doctors
Feb 07, 2017 - 01:13
He also described as "ridiculous" the idea of exhuming her body for a re-postmortem as suggested by the Madras High Court. This was for nominating AIADMK candidates for elections to two seats and bypoll to one segment previous year .

Here are games getting significant boost with PS4 Pro's new firmware
Feb 07, 2017 - 01:13
Users are also notified that when "Boost Mode" is enabled, they might encounter "unexpected behaviors" during gameplays. Like a turbo button from a '90s PC, the feature is said to improve performance on games that saw unstable frame-rates.