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Insmed Incorporated Message Board

  • fudfighter4 fudfighter4 Nov 24, 2012 11:00 PM Flag

    Two early opportunities for the FDA to show willing?

    This extract from the new legislation is the main reason for my expectation of an early marketing authorisation for Arikace -

    "As a result of these remarkable scientific and medical advances, the FDA should be encouraged to implement more broadly effective processes for the expedited development and review of innovative new medicines intended to address unmet medical needs for serious or life-threatening diseases or conditions, including those for rare diseases or conditions, using a broad range of surrogate or clinical endpoints and modern scientific tools earlier in the drug development cycle when appropriate. This may result in fewer, smaller, or shorter clinical trials for the intended patient population or targeted subpopulation without compromising or altering the high standards of the FDA for the approval of drugs."

    It seems to me the FDA is between a rock and a hard place here. On the one hand (doubtless primarily as a result of complaints from politicians in the pockets of big pharma) the new legislation puts pressure on the FDA to shorten the drug approval process. On the other hand, the FDA must not compromise or alter its high standards.

    Those high standards have traditionally stipulated that a drug is not approved until safety has been established via testing in animals for long periods.

    I'm guessing the FDA will be anxious not to be seen as being stuck in its ways. But the time necessary to establish the safety of drugs currently starting the development process will surely translate to a long delay before the FDA is able to demonstrate that it's listening to the people who approve its annual budget ($4,486,368,000 next year).

    I expect the FDA to be looking for "quick wins" here - and both Arikace and iPlex would appear to be prime candidates because of the safety data already available.

    In the case of iPlex -

    "The Secretary shall, at the request of the sponsor of a drug, expedite the development and review of such drug if the drug is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. (In this section, such a drug is referred to as a `breakthrough therapy'.)"

    Will the data from the ongoing study in Sweden satisfy the following criterion? -

    "preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints"

    I would suggest that if iPlex prevents the ROP developing in the first place, that will qualify as a "substantial improvement" over the retinal ablation laser therapy currently used to control it.

    Although I've been referring to the provisions in the new legislation for the expedited development and review of "breakthrough therapies" - as Bo has pointed out, Insmed may (also?) propose Arikace for designation as a "qualified infectious disease product". In that regard I can't help but wonder if Renu's comment in the presentation earlier this year that amikacin is currently used to treat multi-drug resistant tuberculosis was entirely innocent.

    "(d) Designation-

    ..... '(1) IN GENERAL- The manufacturer or sponsor of a drug may request the Secretary to designate a drug as a qualified infectious disease product at any time before the submission of an application under section 505(b) for such drug. The Secretary shall, not later than 60 days after the submission of such a request, determine whether the drug is a qualified infectious disease product.

    '(4) DESIGNATION PRIOR TO REGULATIONS-

    ..... The Secretary shall designate drugs as qualified infectious disease products under subsection (d) prior to the promulgation of regulations under this subsection, if such drugs meet the definition of a qualified infectious disease product described in subsection (g).

    '(g) Qualified Infectious Disease Product- The term 'qualified infectious disease product' means an antibacterial or antifungal drug for human use intended to treat serious or life-threatening infections, including those caused by--

    ..... '(1) an antibacterial or antifungal resistant pathogen, including novel or emerging infectious pathogens; or

    ..... '(2) qualifying pathogens listed by the Secretary under subsection (f).'.

    '(f) Qualifying Pathogen-

    ..... '(1) DEFINITION- In this section, the term 'qualifying pathogen' means a pathogen identified and listed by the Secretary under paragraph (2) that has the potential to pose a serious threat to public health, such as--

    .......... '(A) resistant gram positive pathogens, including methicillin-resistant Staphylococcus aureus, vancomycin-resistant Staphylococcus aureus, and vancomycin-resistant enterococcus;

    .......... '(B) multi-drug resistant gram negative bacteria, including Acinetobacter, Klebsiella, Pseudomonas, and E. coli species;

    .......... '(C) multi-drug resistant tuberculosis; and

    .......... '(D) Clostridium difficile."

    The following provision, imo, is of potential relevance to both Arikace and iPlex -

    'SEC. 569B. USE OF CLINICAL INVESTIGATION DATA FROM OUTSIDE THE UNITED STATES.

    ..... '(a) In General- In determining whether to approve, license, or clear a drug or device pursuant to an application submitted under this chapter, the Secretary shall accept data from clinical investigations conducted outside of the United States, including the European Union, if the applicant demonstrates that such data are adequate under applicable standards to support approval, licensure, or clearance of the drug or device in the United States.

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    • REPORT TO yAHOO!

    • More on the "qualified infectious disease product" designation, from the CATO site -

      "QDIP REGULATIONS

      Exclusivity:

      ..... NCE Exclusivity: 10 years (ANDAs for QDIPs that are NCEs cannot be submitted until 9 years after approval)
      ..... Clinical Investigation Exclusivity: 8 years
      ..... Orphan Drug Exclusivity: 12 years
      ..... Pediatric Exclusivity: Extends exclusivity by 6 months

      Regulatory Pathway:

      ..... Sponsor can request QDIP designation any time before NDA submission, and the FDA will confirm or deny QIDP designation within 60 days of the request submission
      ..... Sponsors can request that the FDA provide written recommendations for "non-clinical and clinical investigations" for QIDP drug approval
      ..... QIDP designated drugs qualify for priority review IF the NDA is submitted after 09 Jul 2012
      ..... Sponsors can also request fast-track status"

      The FDA has already granted QIDP designations. Durata Therapeutics announced on November 5 -

      "Dalbavancin is among the first anti-infective agents to receive QIDP designation through the new Generating Antibiotic Incentives Now (GAIN) statute, created by Congress to encourage the development of therapies for drug-resistant organisms known to cause serious or life-threatening infections."

      Btw - Arikace is indeed considered by the FDA to be a New Chemical Entity (although the active ingredient amikacin is already FDA-approved). The NCE exclusivity would supplement the patent protection, by preventing an application for FDA approval of a generic version for nine years after Arikace is approved.

    • even funnier is that you write that the "fda will be anxious to be seen as not being stuck in its ways."

      Really??? This is the funniest bit of tripe I have ever read. You don't have the hint of a clue about how the FDA, DOT, EPA, OSHA, HHS or any of the many alphabet soup agencies do business. Here is the REALITY...they do not consider "being stuck in its ways" ever. Never ever. Not even the hint of a thought of that nonsense you spew.

      • 2 Replies to satltsasw
      • If you are arguing that the current head of the FDA is so sure of her backing that she has no fear of calls for "a new broom to reorganise the FDA", here are a couple of snippets you should read -

        From September 2005:

        "WASHINGTON -- Embattled Food and Drug Administration Commissioner Lester Crawford abruptly resigned Friday, telling his staff that at age 67 it was time to step aside.

        President Bush designated the National Cancer Institute's director, Dr. Andrew von Eschenbach, to be the FDA's new acting commissioner.

        Crawford's resignation came just two months after the Senate, in a long-delayed move, elevated the longtime agency deputy and acting commissioner to the top job.

        His three-year tenure at FDA was marked by increasing criticism and a particularly rocky final 12 months. The painkiller Vioxx was pulled off the market for safety problems, FDA was embarrassed last fall when its British counterparts shut down a supplier of U.S. flu vaccine for tainted shots, and over the summer recalls of malfunctioning heart devices mounted.

        Finally last month, morale at the agency plummeted when Crawford indefinitely postponed nonprescription sales of emergency contraception over the objections of staff scientists who had declared the pill safe. FDA's women's health chief resigned in protest."

        From earlier this year:

        "The agency has drawn fire from some manufacturers as being too cautious in analyzing cancer and other drugs as well as medical devices."

        The head of any organisation which consumes such a large chunk of taxpayers' money will be only too aware of the old saying "He who pays the piper calls the tune". I'm still waiting for you, or ANYBODY, to point to a single likely barrier to an early US marketing authorisation for Arikace under the legislation enacted in July this year.

      • 1. Re your -

        [even funnier is that you write that the "fda will be anxious to be seen as not being stuck in its ways."

        Really??? This is the funniest bit of tripe I have ever read. You don't have the hint of a clue about how the FDA, DOT, EPA, OSHA, HHS or any of the many alphabet soup agencies do business. Here is the REALITY...they do not consider "being stuck in its ways" ever. Never ever. Not even the hint of a thought of that nonsense you spew.]

        From an article earlier this year -

        "The agency has drawn fire from some manufacturers as being too cautious in analyzing cancer and other drugs as well as medical devices.

        Some critics say the FDA is harming innovation and U.S. competitiveness with unnecessarily tough requirements. The medical device industry has argued vocally that the FDA is driving companies to Europe where developers see a quicker path to the market. Consumer advocates and others, meanwhile, argue that the FDA may sacrifice safety for speed and should demand more data from companies."

        You must be very naive to be able to read the following extract from the new legislation -

        "the FDA should be encouraged to implement more broadly effective processes for the expedited development and review of innovative new medicines"

        - and miss the clear implication in the manner in which that was worded. Why not "the FDA should be allowed to ....."? There is no implication here that the FDA has been asking for those powers - the implication is that encouragement was NEEDED.

        Individuals with responsibility for managing organisations funded primarily by the taxpayer don't tend to keep their jobs for long if they leave themselves open to the accusation of not being on the ball. Margaret Hamburg will most certainly be alive to the possibility that in a year's time her critics will be saying that nothing has changed, despite the "encouragement".

        2. Your claim to have already offered a valid argument in support of your belief that the Arikace program will cannot benefit from the new legislation was lame. All you've offered us is a flawed argument that the FDA will not be looking for quick wins. You've offered no reason for believing that Arikace will not be designated either as a "breakthrough therapy" or as a "qualified infectious disease product".

        Unless you do so, your argument that the share price cannot hit $500 next year is fundamentally flawed.

        You say -

        ""the regulatory path between here and commercial sales is longer than a year"

        - but this is only true if one assumes there will not be a development under the new legislation.

        Without such a development, the earliest revenue the Company is scheduled to earn from Arikace is that anticipated following its launch in Europe. Top-line data from the EU CF study is not due until around the middle of 2013. One would expect a further delay for the full data analysis, and preparation of the application for an EU marketing authorisation. The average time for the EU approval process is currently around a year.

        Do the maths. I don't think Insmed's decision around the middle of 2012 to bring on board the expertise of people like Altomari and Lewis was in preparation for an EU launch of Arikace anticipated late 2014. I think that decision is far more likely to have been prompted by the development that there was almost unanimous support for the proposed legislation (subsequently signed into law in July).

    • haha...you said that the regulation puts the FDA between and rock and a hard place. Who do you think writes the regulations???? They come from within just about 100% of the time. The FDA is NEVER between a rock and a hard place. And just how often have you dealt with the FDA or their regulations? Oh...never you say.

      • 1 Reply to satltsasw
      • The following is an extract from an article earlier this year -

        "The agency has drawn fire from some manufacturers as being too cautious in analyzing cancer and other drugs as well as medical devices."

        If you are arguing that Margaret Hamburg is so sure of her position that she has no fear of suggestions that a "new broom" is needed to reorganise the FDA ..... I'm still waiting for you to offer an argument against an early US marketing authorisation for Arikace which others here could take seriously.

    • FRAUD.

 
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