3 for 3 on Orphan Drug Requests
In hockey they call it a “hat trick” and in horse racing they call it the “trifecta”. At Advaxis, we call it “mission accomplished”.
Late last week, we received notice from the U.S. FDA Office of Orphan Products Development that our lead immunotherapy drug candidate, ADXS-HPV, has been granted orphan drug designation for the treatment of Stage II-IV invasive cervical cancer.
The benefits of orphan status are considerable and for each of our multiple orphan designations we qualify for various financial benefits and incentives, including:
- clinical protocol assistance with the FDA
- tax credits for the cost of clinical research
- waiver of PDUFA (prescription drug user fee act) filing fees (potentially millions)
- annual grant funding to defray the cost of clinical testing
- seven year period of exclusive marketing after an orphan drug is approved
This is our third orphan designation granted in about 6 months, adding to the previously granted indications for HPV-associated head & neck and anal cancers.
It’s important to keep in mind that the FDA grants orphan designation to drugs/products to promote the development of products intended to treat rare diseases affecting fewer than 200,000 individuals in the United States. Based on the relatively few women that develop invasive cervical cancer in the U.S., we believed that ADXS-HPV would qualify for orphan status in this indication. However, the FDA initially denied our request for orphan drug status in cervical cancer on the basis that the total number of women who have ever had cervical cancer exceeded the statutory maximum of 200,000.
Our regulatory team went back to the drawing board and provided an additional analysis that supported ADXS-HPV for the treatment of a medically relevant subset of patients – patients with Stage II-IV invasive cervical cancer – a subset that did qualify for orphan status. The new request resulted in a hard-earned and very valuable orphan status for Stage II-IV invasive cervical cancer. Our determination to continue to work with the FDA through this process has allowed us access to the benefits of orphan status that may be helpful in making our drug candidate available to women with cervical cancer.
We intend to pursue the financial and regulatory advantages available under orphan designation to make a difference in HPV-associated cancers worldwide and to bring value to our supportive stockholders.
We will continue to identify other orphan drug indications for our proprietary immunotherapies. Why stop at three when there is the potential for more?
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Gregory T. Mayes, Chief Operating Officer
Forward Looking Statements
This blog may contain forward-looking statements, including, but not limited to: statements as to the anticipated timing of clinical studies and other business developments, statements as to the development of new constructs, expectations as to the adequacy of our cash balances to support our operations for specified periods of time and as to the nature and level of cash expenditures, expectations as to market opportunities, our ability to take advantage of those opportunities, and the risk factors set forth from time to time in Advaxis' SEC filings, including but not limited to its report on Form 10-K for the fiscal year ended October 31, 2012, which is available at www.sec.gov. The Company undertakes no obligation to revise these forward-looking statements which may be made to reflect the events or circumstances after the date hereof or to reflect the occurrence of unanticipated events, except as required by law. You are cautioned not to place undue reliance on any forward-looking statements.