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Posts tagged with "Regulatory Affairs"

  • Clinical Trials

    Stem Cells: The Center of “Right to Try”

    I wanted to point out an excellent editorial on the whole “Right to Try” issue at Nature. The authors note correctly that stem cells are the therapeutic area where these battles are being fought most often, since their regulatory status (particularly with any sort of autologous cell treatment) is sometimes unclear, the number of possibl… Read More
  • Clinical Trials

    Right To Try: Here We Go

    At what point should an experimental drug be made available for anyone to try it? The usual answer is “Unless you’re enrolled in a clinical trial, then not until it’s no longer an experimental drug”. There’s always compassionate use, but that’s a hard topic to deal with, and one that has a different answer for… Read More
  • Regulatory Affairs

    The Reasons for Failure at the FDA

    Here’s a good retrospective in JAMA, from the FDA, about what’s happened when the agency has rejected a new molecule entity (NME). The authors look over the data set from 2000-2012 to see what the most common reasons for trouble were, and what happened after that in each case. Overall, 302 new molecules were submitted… Read More
  • Regulatory Affairs

    The 2013 Crop of New Drugs

    Here’s Lisa Jarvis’s roundup of the 2013 drug approvals (PDF version). There’s also a list of notable late-stage clinical failures, and unfortunately, these tended to be potentially bigger drugs than the ones that got through last year. I have to wonder if the structures included along with the chart were chosen especially to high… Read More
  • Clinical Trials

    PTC’s Latest Ataluren Woes

    I wrote here about PTC Therapeutics and their drug candidate for Duchenne muscular dystrophy (ataluren, PTC124). Opinion has been divided, to put it mildly, about how it works and whether it works at all. Well, the saga continues. The company is having a rough time with that program these days, though. PTC applied to the… Read More
  • Cardiovascular Disease

    Merck Pulls One Out

    Merck’s vorapaxar, a thrombin antagonist that many had thought might never make it, has received a positive FDA advisory committee vote. I’m glad to see it – peripherally, I go way back with this compound (well, its ancestors), and I really had doubts that Merck could get things to fly. Anticoagulants are a very tricky… Read More
  • Business and Markets

    The 2013 Drug Approvals: Not So Great?

    So we finished the year with 27 new drugs approved. Compared to the 39 approvals the year before, is that a reversion to the mean, a sudden downturn, a meaningless fluctuation, or what? John Carroll seems to be in the “something to worry about” camp at FierceBiotech: The wave of new drug approvals that had… Read More
  • Regulatory Affairs

    Merry Christmas From the FDA

    I see that the FDA is in the holiday spirit – they’ve told Ariad that they can bring Iclusig back to the market in the US, subject to restrictions, and Amarin, who have been trying, quite unsuccessfully, to get approval for wide use of a fish-oil-pill, have at least gotten a stay of execution for… Read More
  • Regulatory Affairs

    23 And Me And the FDA

    As everyone will have heard the personal-genomics company 23 and Me was told by the FDA to immediately stop selling their product, a direct-to-consumer DNA sequence readout. Reaction to this has been all over the map. I’ll pick a couple of the viewpoints to give you the idea. From one direction, here’s Matthew Herper’s article… Read More
  • Clinical Trials

    Sarepta’s Approval Woes

    I briefly mentioned Sarepta and etiplirsen, their proposed therapy for Duchenne muscular dystrophy (DMD) in September. In that post, I made reference to the “delirious fun of investing in biotech”. Well, the company recently got some regulatory news that illustrates that point even more clearly. The FDA told Sarepta that it would not ge… Read More
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