Marathon Pharmaceuticals' Duchenne muscular dystrophy drug wins FDA approval

MDA Celebrates News of FDA Approval of Emflaza for Treatment of Duchenne Muscular Dystrophy

MDA Celebrates News of FDA Approval of Emflaza for Treatment of Duchenne Muscular Dystrophy

The FDA has cleared a second drug for Duchenne muscular dystrophy, giving the green light to Marathon Pharma's corticosteroid Emflaza for patients aged five or more with the disease. Following rebates and other discounts, the price is $54,000 per year, said Marathon Pharmaceuticals CFO Babar Ghias.

The practice has prompted congressional investigations and hearings into companies including Valeant Pharmaceuticals International Inc. and Turing Pharmaceuticals LLC, the firm formerly run by onetime hedge-fund manager Martin Shkreli. The bad news is that it comes with a high price of $89,000 a year.

A recently FDA-approved drug used in steroid treatments, which has been on the worldwide market for years, will be introduced in the United States at the inflated price of $89,000 a year. Dr. Billy Dunn of the Division of Neurology Products with the Center for Drug Evaluation and Research at the Food and Drug Administration said the FDA is hopeful that Emflaza will help a wide range of patients with DMD.

The vouchers, in theory, exist for positive reasons as regulators try to encourage investment by companies in the development of drugs to use against rare diseases that are afflicting children.

For the last two decades, patients have been buying deflazacort from other countries ever since clinical trials demonstrated its effectiveness for treating Duchenne muscular dystrophy. It has been available for years in other countries, and the family of the individuals with Duchenne Muscular Dystrophy.

100 whales refloated in NZ rescue bid, but many stranded again
It's unusual to see such a large number of pilot whales traveling together, DOC Community Ranger Kath Inwood tells Westcott. Hundreds of whales have died overnight on a New Zealand beach after a mass stranding thought to be the largest in decades.

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.

The drug wasn't sold in the USA mainly because no company thought it would be profitable enough to warrant the effort of seeking FDA approval.

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. An overall stability in average muscle strength was maintained through the end of study at week 52 in the deflazacort-treated subjects. This is the 9th time that the FDA grants the pediatric disease priority voucher.

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).

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.

Recommended News

  • Neymar overtakes Ronaldinho goal record at Barcelona

    Neymar overtakes Ronaldinho goal record at Barcelona

    Real Betis striker Ruben Castro hit the post early on in scoreless stalemate that increased the winless streaks of both teams. Manager Luis Enrique is facing a selection headache ahead of this weekend's clash with Deportivo Alaves in the league.
    Sprint Announces FIVE Lines of Unlimited Data, Talk and Text for $90/month

    Sprint Announces FIVE Lines of Unlimited Data, Talk and Text for $90/month

    After the promotional period is over, customers will pay the additional United States dollars 10 per month for first line. T-Mobile customer? Sprint has an excellent offer for you! Then, you can add up to three more people to the plan for free.
    Fed's Tarullo to resign, creating opening at central bank

    Fed's Tarullo to resign, creating opening at central bank

    The departure will give Trump, who has promised to deregulate banks, a third vacant seat at the Fed to fill. During his time at the Fed, he served as chairman of its committee on supervision and regulation.
  • Knicks Owner James Dolan Bans Charles Oakley From MSG

    Knicks Owner James Dolan Bans Charles Oakley From MSG

    Despite couples taking selfies in front and parents ushering happy kids inside, all is not well at Madison Square Garden. Mostly Jim Dolan is surrounded with sycophants and henchmen who help him carry out his petty grudges and person feuds.
    Massive Anti-American Protests Break Out in the Islamic Republic of Iran

    Massive Anti-American Protests Break Out in the Islamic Republic of Iran

    Trump recently imposed fresh sanctions on Iranian individuals and entities in reaction to a missile test on January 29. Some signs specifically thank the USA people which, in this case, means progressives protesting Trump's travel ban.
    Krauthammer: 'No Slam Dunk' That SCOTUS Will Reinstate Travel Ban

    Krauthammer: 'No Slam Dunk' That SCOTUS Will Reinstate Travel Ban

    As a result, foreign travelers from the seven banned Muslim-majority countries have been allowed to enter the U.S. - for now. Judges wanted to know if the executive branch believes that Trump could bar Muslims from entering the country if he wanted.
  • 'Orange-colored' gator in SC becoming hot talker on social media

    'Orange-colored' gator in SC becoming hot talker on social media

    A predatory beast with leathery orange skin is gallivanting through social media, sowing fear in its wake. Alligators, except those of the Gainesville, Florida variety, aren't usually a deep orange color .
    Large Hole in California Dam Spillway Continues to Grow

    Large Hole in California Dam Spillway Continues to Grow

    They expect that water flowing out of the dam will catch up with the torrent flowing in from recent storms by sometime Saturday. Flows were halted so that engineers could assess the damage and determine how much the spillway could handle going forward.
    'World's heaviest woman' at 78st to have life-saving weight loss surgery

    'World's heaviest woman' at 78st to have life-saving weight loss surgery

    A special bed had been made by local Egyptian artisans in accordance with the safety precautions laid down by the airlines. She has suffered paralysis in right leg and right arm which has left her completely immobile even in her bed.
  • NCAA Selection Committee Releases Mid-Season Top 16 Seedings

    NCAA Selection Committee Releases Mid-Season Top 16 Seedings

    Kansas was the selection committee's No. 2 overall seed, projected in the Midwest Region that ends up in Kansas City, Mo. Florida and Kentucky would be the SEC's only two top-four seeds, with SEC co-leader SC left on the outside looking in.
    Romanian Justice Minister resigns over controversial corruption reforms

    Romanian Justice Minister resigns over controversial corruption reforms

    The projects proposed have been under public debate, organized by the Ministry of Justice and are now in the parliamentary debate. The government survived a vote of no-confidence this week and says a public consultation will now take place on justice reform.
    Will Rosie O'Donnell Play Steve Bannon On 'SNL'?

    Will Rosie O'Donnell Play Steve Bannon On 'SNL'?

    That made some women mad, and sparked a push to draft Rosie O'Donnell to play trump's chief strategist Steve Bannon . On Feb. 6, a report in Politico claimed that Trump was angry that his press secretary was played by a woman.

We are pleased to provide this opportunity to share information, experiences and observations about what's in the news.
Some of the comments may be reprinted elsewhere in the site or in the newspaper.
Thank you for taking the time to offer your thoughts.