Final UK approval:
PTC Therapeutics has announced that the National Institute for Health and Care Excellence has issued final guidance recommending Translarna (ataluren) for ambulatory patients aged five and over with nonsense mutation Duchenne muscular dystrophy in connection with a managed access agreement with NHS England.
A rare genetic disease, primarily affecting males, Duchenne muscular dystrophy is a progressive muscular disorder caused by the lack of functional dystrophin protein. Disease sufferers lose the ability to walk from as early as 10 years old, and may begin to experience to potentially fatal lung and heart complications in their late teens and early twenties.
While rejected by the FDA in the US, Translarna has been approved by the EMA. NICE had backed the drug in initial guidance, and this latest guidance will mean Duchenne patients can now access the first approved therapy to treat the underlying causes of the disease.
Paul Lenihan, CEO of Action Duchenne, says: “We are delighted that this positive recommendation from NICE has now been published. This is such an important day for the Duchenne community, which has been working hard to make this ground-breaking drug available to boys with nonsense mutation Duchenne muscular dystrophy.”
Janis Clayon, General Manager for UK and Ireland at PTC Therapeutics, comments: “Translarna is the first and only licensed treatment to address the underlying cause of Duchenne muscular dystrophy. I am very pleased that the completion of this important review process undertaken by NICE and NHS England now ensures that a much-needed therapy is available for the children and young adults with nonsense mutation DMD that meet the requirements for the MAA.”
Amazon was in the same boat for YEARS. Losses were huge. Things are SLIGHTLY different there now.
And "biotech" is a completely different animal.
So the FDA wouldn't consider fast track, etc. because there is already something available to treat this, regardless of how ineffective that existing treatment is? Sounds about par for the course with them.
Thanks for all of your tips, hopeful200!
I mistyped PCTI when I was researching it. Accidentally ran across PTCT and picked some up under $7. Sold some at $8.25 and it's currently at $8.09. I give you credit for that one too. I'd rather be lucky, as the saying goes.
Thanks again! I owe you a beverage or three.
I think I remember seeing $14 or just below. It's possible it hit $17 and I was drunk and don't remember it, but either way, I don't remember it. I've been watching or in KTCC for over 10 years.
Ads are targeted based on several factors (search history and whatever information you have freely given Facebook, Twitter, Instagram, Linked In, etc.). I see none.
And, naturally, etrade gives me:
Opening orders for this security cannot be accepted online at this time.
Pop quiz, hotshot. Do I pick up the phone? Is this 1989?
FVE as an entity is in great shape. Very healthy. Occupancy numbers are mediocre. Were this company handled as a REAL company, it would be very profitable and it's market cap would be at least 5x what is now. HOWEVER, FVE lives in Portney's world where things are "distorted". Showing a profit would be counterproductive to the agenda.
Hopefully some of the institutional owners see the discrepancies and take some action.
Interesting. I'm a little depressed that volume didn't move the price up, but I suppose I should be happy it didn't drop it more than a nickel. What will tomorrow bring?