Orphan disease exclusivity
There’s a first time for everything! And on August 8th, 2012, FDA decided, for the first time since the enactment of the Orphan Drug Act, to rescind a period of 7-year orphan drug exclusivity. The decision came in the form of a response granting in part and denying in part a citizen petition (Docket No. FDA-2011-P-0213) CSL Behring (“CSL”) submitted to FDA in March 2011 requesting that the Agency: (1) revoke the orphan drug designation and the period of orphan drug exclusivity FDA subsequently granted to Octapharma USA, Inc. (“Octapharma”) for the December 4, 2009 approval of WILATE (von Willebrand Factor/Coagulation Factor VIII Complex (Human)) under BLA No. 125251 “for the treatment of spontaneous or trauma-induced bleeding episodes in patients with severe von Willebrand disease (VWD) as well as patients with mild or moderate VWD in whom the use of desmopressin is known or suspected to be ineffective or contraindicated;” and (2) “refrain from making any orphan drug designations and approval decisions based on hypothetical claims of superiority.” FDA decided not to revoke orphan drug designation for WILATE, and also denied CSL’s request regarding “hypothetical claims of superiority.”
WILATE is the “same drug” as HUMATE-P (Antihemophilic Factor/von Willebrand Factor Complex (Human)) for orphan drug purposes. That is, FDA considers them to be “chemically the same drug” and for the same orphan indication. FDA approved HUMATE-P on April 1, 1999 for the same orphan disease as WILATE. Because WILATE is the “same drug” as HUMATE-P, the issue of “clinical superiority” comes into play – for obtaining orphan drug designation and for obtaining orphan drug exclusivity.
FDA’s orphan drug regulations at 21 C.F.R. § 316.20(a) state that “a sponsor of a drug that is otherwise the same drug as an already approved orphan drug may seek and obtain orphan-drug designation for the subsequent drug for the same rare disease or condition if it can present a plausible hypothesis that its drug may be clinically superior to the first drug.” FDA’s orphan drug regulations define a “clinically superior” drug as “a drug . . . shown to provide a significant therapeutic advantage over and above that provided by an approved orphan drug (that is otherwise the same drug)” in one of three ways: (1) greater effectiveness as assessed by effect on a clinically meaningful endpoint in adequate and well controlled trials; (2) greater safety in a substantial portion of the target population; or (3) demonstration that the drug makes a major contribution to patient care.
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