FDA Approves Muscular Dystrophy Drug, Will Cost Patients $89000
A drug company may be facing some heat after doing something similar to Martin Shkreli, the hated man accused of jacking up the price of a life-saving drug just to make some more money.
While corticosteroids are the standard treatment commonly used globally to treat DMD, this is "the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use" in the US, the agency said.
The two congressmen, who have previously eyed Valeant, Turing, and Mylan, among other pharma firms, on Monday wrote a letter to Marathon Pharmaceuticals seeking answers about its newly approved $89,000 treatment for Duchenne muscular dystrophy.
Emplaza is a corticosteroid that reduces activity within the immune system and decreases inflammation.
Mortgage rates level off
The short term, popular 15 year fixed rate mortgages can be had for 3.710% today and an April of 3.894%. The loan package features an April variable of 3.708%, according to today's updated loan information.
Because it's been available outside the U.S.at such lower prices, many have said the Northbrook, Illinois-based Marathon's price is unjustifiably high. FDA approval should also increase access to a drug that may be too expensive to buy outright for some, even in its generic form.
Deflazacort has always been used off-label for DMD treatment, but until now hadn't been approved to treat the condition.
Deflazacort is used to treat Duchenne muscular dystrophy (DMD), a rare genetic disease that causes heart and respiratory conditions, usually in younger men.
Sanders and Cummings noted that the drug has been imported by patients in the US for "decades". The drug now costs $1,200 overseas.
Hull vs Liverpool Reds crash again as Tigers' resurgence continues
If we could play our best against Spurs then prove the rest of it later in the season - that would be the best things". Now we have to change it - that's a football thing.
A drug to treat muscular dystrophy will hit the USA market with a price tag of $89,000 a year despite being available for decades in Europe at a fraction of that cost. Sarepta's drug treats a subset of DMD patients representing about 13% of the total.
One hundred and ninety-six male patients, who ranged in age from 5 to 15 years old, had a mutation of the dystrophin gene, and had the onset of weakness before they were five years of age, participated in a clinical study of the drug deflazacort.
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. It was also awarded a rare pediatric disease priority review voucher that can be used for another drug. The drug isn't a cure, but it has been shown to improve muscle strength, the FDA said in a statement announcing the approval. Fundamentally, the voucher is a way to get a future drug reviewed faster by regulators. An overall stability in average muscle strength was maintained through the end of study at week 52 in the deflazacort-treated patients. Other side effects include mood swings, elevated blood pressure, infection and serious skinj rashes.
Fake transfer news! Hakan Calhanoglu denies expressing Chelsea interest
Calhanoglu had been in great form for Bayer until his suspension, scoring seven times and providing seven assists in all competitions.
Sargento, Meijer recall cheese due to possible Listeria contamination
Meijer is also recalling its Meijer brand colby and colby jack cheeses that are sold exclusively through its deli counters. The products were packaged at the Sargento plant in Plymouth, Wisconsin, and were distributed nationwide.
Russian airstrike kills 3 Turkish soldiers in Syria by accident
The Russian airstrike coincided with a two-day visit to Ankara by new Central Intelligence Agency chief Michael Pompeo. Turkish troops and allied forces have been advancing towards the town from the north, east and west.
China's kicks off 2017 with above-forecast trade numbers
Commodity imports, such as iron ore, crude oil and coal, continued to grow in January, featuring general price rises. China's trade performance rebounded strongly last month with an unexpected bounce in both imports and exports.
Trump Says Iran's Nuclear Deal Inconceivable, Disastrous For Israel
President Alexander Lukashenko has congratulated Iran's President Hassan Rouhani and the Iranian people on the national holiday. Some political observers believe Tehran held its collective tongues Friday largely out of fear of further inciting Trump.
Volunteers document race to save surviving whales after 400 stranded
Farewell Spit has been described as a whale trap , as its long coastline can be hard for whales to navigate away from. However, they were unable to save the lives of an estimated 335 whales whose carcasses now lie strewn on the beach.
Dam spillway in Oroville, Calif., closed after large hole in concrete discovered
State officials say a break in the weather will allow engineers to truly assess the damage and plan repairs. A hole, about 250 feet long and 45 feet deep, opened up in the lower part of the main spillway on Tuesday.
US, Chinese military planes come close at South China Sea
"The Department of Defense and US Pacific Command are always concerned about unsafe interactions with Chinese military forces". Tensions have eased since the election of President Rodrigo Duterte, who has sought to mend ties with China.
Falcons Replacing Defensive Coordinator Richard Smith
Greater Atlanta Christian's communications director, Margie Isef, said the school is already looking to next year and #RiseUp2018. Per multiple reports, quarterbacks coach Matt LaFleur will also leave the team to become offensive coordinator with the Rams.
Tennis player Genie Bouchard learned her lesson after betting against Tom Brady
New England wound up pulling off the greatest comeback in Super Bowl history, stunning Atlanta with a 34-28 victory in overtime. Bouchard confirmed Monday afternoon she'll make good on her wager with 20-year-old John Goehrke.
Manchester City make major Aguero decision, fans will be divided
In the long balls he's a fighter and against taller central defenders he can win the ball and he's only nineteen years old. We can use him as a third man and give an assist with David [Silva] and Kevin [De Bruyne], especially in the second half.