gamesindustryblog.com
gamesindustryblog.com June 26, 2017


FDA approves costly drug to fight Duchenne Muscular Dystrophy

12 February 2017, 12:55 | Elizabeth Houston

Until now, the drug was not sold in America because no company thought it was worth the effort. It has been available for years in other countries, and the family of the individuals with Duchenne Muscular Dystrophy. Those with the disease usually lose the ability to walk in their teens and have a life expectancy around 25 to 30 years.

Now, with an FDA-approved med on the market-the only one green-lighted to treat all DMD patients age 5 and older-the import option is off the table for patients, and Marathon is slapping an $89,000-per-year list price on the med. With the orphan designation, the company is getting seven years of exclusive rights to sell the drug in the U.S.

Since so few Americans are affected by the disease, only around 15,000, the drug was approved under an "orphan drug" status, which is a special status given to drugs that treat rare diseases or conditions.

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.

Leading stocks in today's market: Twenty-First Century Fox, Inc. (NASDAQ:FOXA)
The average broker rating assists investors by providing them with a general feel for analyst sentiment on company shares. Twenty-First Century Fox, Inc.'s (FOXA) witnessed a loss of -0.19% in recent trading period with closing price of $31.40.

Ghias also says that that after rebates and discounts, the price will drop to $54,000, and with insurance and financial programs most patients will pay "zero to low out-of-pocket expense, " according to the Washington Post. USA patients have been importing it since the 1990s after clinical studies showed it could reduce inflammation with less unwanted side effects than another steroid. In general, the muscle strength was maintained among those taking the drug until the end of the 52-week study.

The most common side effects include facial puffiness (cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, and central obesity.

"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 at the FDA.

Other less-common AEs include problems with endocrine function, increased susceptibility to infection, hypertension, the risk of gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decreased bone density, and vision problems, such as cataracts. Patients have been importing the drug since the 90s, but now they will have to pay 50 to 70 times what they used to pay if they want to get it in the U.S.



Other News

Trending Now

Senate passes bill to bring ride-sharing to upstate
James Seward, R-Milford, Otsego County - includes a lower tax than Cuomo's proposal, which would tax each ride 5.5 percent. The two ride-hailing companies are now prohibited from operating outside of the New York City area.

Dr Richard Beale Reveals Conditions Surrounding Jayalalithaa's Death
The press conference was initiated as rumours circulating claimed that Jayalalithaa could had been "poisoned to death". Trashing the theory that Jayalalithaa died on an earlier date, the doctors revealed, "Jayalalithaa was speaking well".

These Are The First Set Photos Of Alicia Vikander As Lara Croft
For now, check out some of the Tomb Raider reboot set photos below, which show Vikander in Laura Croft's signature outfit. The original Lara Croft: Tomb Raider film was released in 2001 with a sequel - The Cradle of Life - coming out in 2003.

Blizzard conditions confirmed in some areas as storm hammers MA
Forecasters predict New York City will see six inches or more, while Boston is due for as many as 18 inches. Many government offices also were shuttered with MA and CT ordering non-emergency workers to stay home.

UP polls: Akhilesh Yadav to address six rallies in Sitapur
Dharmendra Yadav represented the party at the all-party meeting called by the Speaker before the Budget session. Akhilesh today addressed six election rallies in Sitapur as part of his vigorous campaigning in the state.

Drop Ivanka's line? Nordstrom gets Twitter response from Trump
White House press secretary Sean Spicer echoed the president's anger with the high-end fashion store in a news conference today. Nordstrom said that it cuts about 10 percent of brands each year over performance issues.

U.S. judge orders Google to release foreign emails to aid FBI investigation
In July previous year , a court ruled in favour of Microsoft in a similar case. In the Microsoft case, the data requested was stored on Irish servers.

US Department of Justice formally appeals Trump travel ban ruling
A USA federal judge temporarily blocked President Donald Trump's travel ban on refugees and immigrants. Taking to social media on Saturday Mr Trump mocked Judge Robart, appointed by President George W.

Co Down woman wins case for late partner's pension
Today's ruling will raise further questions about the differentiation in the new rules between married and unmarried couples. Brewster's lawyers had argued that the requirement of the nomination form was disproportionate and in effect discriminatory.

Egypt vs Cameroon AFCON Final
The Lions were dealt a big blow when a number of senior players retired from the national team just days before the tournament. That's why the players who are on the bench keep motivation. " Cameroon is a very strong and organised team".