Rivulet Media Inc (fka RIVU)

[Lewisinhk]

Your inquiry is answered in CFR 21 Part 316.


§316.24 Deficiency letters and granting orphan-drug designation.

(a) FDA will send a deficiency letter to the sponsor if the request for orphan-drug designation lacks information required under §§316.20 and 316.21, or contains inaccurate or incomplete information. FDA may consider a designation request voluntarily withdrawn if the sponsor fails to respond to the deficiency letter within 1 year of issuance of the deficiency letter, unless within that same timeframe the sponsor requests in writing an extension of time to respond. This request must include the reason(s) for the requested extension and the length of time of the requested extension. FDA will grant all reasonable requests for an extension. In the event FDA denies a request for an extension of time, FDA may consider the designation request voluntarily withdrawn. In the event FDA considers a designation request voluntarily withdrawn, FDA will so notify the sponsor in writing.

Also of interest, Dr. Koos can submit orphan application at any phase of the development before a marketing application.

§316.23 Timing of requests for orphan-drug designation; designation of already approved drugs.


Even if the application is denied, Dr. Koos has the opportunity to file an amendment.


§316.26 Amendment to orphan-drug designation.


(b) FDA will grant the amendment if it finds that the initial designation request was made in good faith and that the amendment is intended to conform the orphan-drug designation to the results of unanticipated research findings, to unforeseen developments in the treatment or diagnosis of the disease or condition, or to changes based on FDA recommendations, and that, as of the date of the submission of the amendment request, the amendment would not result in exceeding the prevalence or cost recovery thresholds in §316.21(a)(1) or (a)(2) upon which the drug was originally designated.


http://www.ecfr.gov/cgi-bin/text-idx?c=ecfr&SID=238362420de6b80c19b2a9c44b73a843&rgn=div6&view=text&node=21:5.0.1.1.6.3&idno=21#se21.5.316_123