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Insmed Incorporated Message Board

  • fudfighter4 fudfighter4 Jan 8, 2013 2:27 PM Flag

    A 505(b)(2) Rapid Approval NDA for Arikace?

    For the benefit of the two people who are having difficulty in grasping the concept of the FDA using the Amikacin label as the basis for the Arikace label, here is some info from the Regprofessional site on the approval route I'm guessing will apply in this instance -

    "The 505(b)(2) New Drug Application – A Rapid Approval Route

    The 505(b)(2) application is one of three established types of new drug application (NDA), and it is a pathway to approval that can potentially save pharmaceutical sponsors both time and money. However, many sponsors are unsure how to evaluate the possible benefits of using this type of application.

    The 505(b)(2) regulatory pathway is defined in The Federal Food Drug and Cosmetics Act as an NDA containing investigations of safety and effectiveness that are being relied upon for approval and were not conducted by or for the applicant, and for which the applicant has not obtained a right of reference.

    These applications differ from the typical NDA (described under Section 505(b)(1) of the Act), in that they allow a sponsor to rely, at least in part, on the FDA's findings of safety and/or effectiveness for a previously approved drug (the "reference drug").

    Section 505(b)(2) was added to the Act in 1984 with the goal of avoiding unnecessary duplication of preclinical and certain human studies. However, the sponsor must still provide any additional preclinical or clinical data necessary to ensure that differences from the reference drug do not compromise safety and effectiveness."

    One assumes that a QIDP designation under the new legislation (signed into law in July last year) would overlay the pre-existing 505(b)(2) pathway.

    From the CATO site -



    ..... 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"

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    • It seems that at least ten users of this forum have found fault with what I've posted. Here's a summary of the likely approval route for Arikace as I see it. Perhaps one of you will take the opportunity to point out the areas where my reasoning is flawed?

      1. One of the aims of the legislation signed into law last July is to expedite the availability of therapies to control pathogens which have the potential to pose a serious threat to public health, such as "multi-drug resistant gram negative bacteria, including Acinetobacter, Klebsiella, Pseudomonas, and E. coli species". A sponsor of a drug candidate with the potential to control one of the named pathogens can now apply for said drug candidate to be designated as a Qualified Infectious Disease Product.

      2. The 1984 505(b)(2) regulatory pathway provides for a New Drug Application based partly upon the FDA's findings of safety and/or effectiveness for a previously approved drug. The following are examples of changes to approved drugs which would be appropriate to submit as 505(b)(2) applications: Changes in dosage form, strength, route of administration, formulation, dosing regimen, or indication.

      3. Amikacin injection is FDA-approved for the treatment of treatment of serious infections due to susceptible strains of Gram-negative bacteria, including Pseudomonas species, Escherichia coli, species of indole-positive and indole-negative Proteus, Providencia species, Klebsiella-Enterobacter-Serratia species, and Acinetobacter (Mima-Herellea) species.

      4. Arikace is basically amikacin with a new route of administration. The ongoing Arikace EU pivotal clinical trial is intended to demonstrate efficacy in controlling arguably the most inherently difficult to treat of the pathogens specifically targeted by the new legislation (Pseudomonas) in an environment where it is particularly difficult to deliver the antibiotic to the pathogen (the lungs of an individual with Cystic Fibrosis).

      5. Under 569B the FDA is allowed to 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.

      6. If Arikace is designated as a Qualified Infectious Disease Product (the FDA has already determined that amikacin is effective in controlling Acinetobacter, Klebsiella, Pseudomonas, and E. coli) it will qualify for priority review.

    • For newbies, QIPD and QDIP are synonymous for Qualified Infectious Disease Product. From the new legislation -

      "(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.


      ..... 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."

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