Higher and Higher (Into the Fire): Jacobus Appeals Orphan Drug Case to SCOTUS

April 21, 2022By Sara W. Koblitz

Our last post on the Eleventh Circuit’s September 2021 decision in Catalyst v. Becerra got a lot of attention.  We’d like to think that this is because the scope of orphan drug exclusivity is as fascinating to everyone as it is to us, but if we’re honest with ourselves, it’s probably because it was an ode to Ms. Britney Jean Spears.  Well, Catalyst v. Becerra is back with Intervenor Jacobus Pharmaceuticals, Inc. submitting a Petition for Writ of Certiorari to the Supreme Court.  Aren’t we Lucky?  Ok, ok, we’ll stop.

In Catalyst v. Becerra, innovator-drug sponsor Catalyst sued FDA for approving the “same drug for the same disease or condition” as its orphan drug-designated product, Firdapse (amifampridine), during its seven-year orphan drug exclusivity period.  FDA approved Firdapse for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) in adult patients in November 2018 with orphan drug exclusivity and subsequently approved a second amifampridine product, Ruzurgi, in May 2019 for the treatment of pediatric patients with LEMS.  Catalyst sued FDA in June 2019 alleging that the plain language of the Orphan Drug Act precluded FDA’s approval of Ruzrugi in any LEMS patients until the expiration of the orphan drug exclusivity period covering Firdapse.  Jacobus intervened, and the case made its way to the Eleventh Circuit.  In September 2021, the Eleventh Circuit held that FDA’s interpretation of the Orphan Drug Act such that exclusivity was limited to the indication for which the drug was approved—rather than the disease or condition for which the drug was designated—is foreclosed by the plain language of the Orphan Drug Act.  Consequently, the Court held, FDA’s approval of Ruzurgi contravened the plain language of the Orphan Drug Act in violation of the Administrative Procedures Act and ordered FDA to rescind Ruzurgi approval.

On April 7, 2022, Jacobus appealed the Eleventh Circuit’s decision to the Supreme Court.  In urging the Court to review the Eleventh Circuit’s decision, Jacobus argues that the Eleventh Circuit’s extension of exclusivity from the specific indication for which the drug was approved to the entire disease or condition for which the drug was designated “eviscerated FDA’s long-standing practice and created a circuit split” with the Fourth and D.C. Circuits “over whether the [Orphan Drug Act] unambiguously forecloses FDA’s use-based approach.”  With some dramatic flair, Jacobus further states “If allowed to stand, the results of the Eleventh Circuit’s decision would be catastrophic.”  This is because the decision would lead to uncertainty for orphan designated drug sponsors while leaving patients with fewer options.

Jacobus makes two main arguments to convince the Supreme Court to hear its case.  First, Jacobus explains that the Eleventh Circuit’s decision creates a circuit split between the Fourth and the D.C. Circuit “both of which upheld FDA’s use-based approach.”  Citing Sigma-Tau Pharms., Inc. v. Schwetz, 288 F.3d 141 (4th Cir. 2002), Jacobus states that the Fourth Circuit determined that a use-based approach to exclusivity was consistent with the plain language of the Orphan Drug Act.  Orphan drug exclusivity, in other words, “protects uses, not drugs for any and all uses” and is “disease-specific, not drug-specific.”  Jacobus also cites to Spectrum Pharms., Inc. v. Burwell, 824 F.3d 1062 (D.C. Cir. 2016) to explain the circuit split, as the D.C. Circuit there deferred to the Agency’s interpretation of the Orphan Drug Act because the language does not “unambiguously foreclose FDA’s interpretation.”

Second, Jacobus points to the “catastrophic consequences” of the Eleventh Circuit’s refusal to defer to FDA’s interpretation of the scope of orphan exclusivity.  Jacobus states that the “end-result” of the decision “was to leave the pediatric population out in the cold because the drug had already been approved (only) for an adult population.”  (This is somewhat hyperbolic given that the Eleventh Circuit noted that only there are only “dozens” of pediatric LEMS patients.)  Jacobus also cites to the policy arguments that will be familiar to our Britney blog post fans: that narrow uses can now cut off competition for an entire disease or condition, and that this policy raises a significant threat of serial exclusivity.  But most of this section is dedicated to dismantling the Eleventh Circuit’s plain language discussion.  There, Jacobus argues that the term “same disease or condition” must be read in the context of the new drug approval provisions of the FDC Act.  Jacobus explains that the exclusivity provisions in the Orphan Drug Act (21 U.S.C. § 360cc(a)) states that FDA “may not approve another application under [new drug provisions at 21 U.S.C.] section 355.”  Because the new drug provisions (21 U.S.C. § 355) are approved for “uses” or “indications” rather than diseases or conditions, Jacobus argues that orphan drug exclusivity must be limited to the “use” approved in the application and not to the entire condition designated.

The Petition for Writ of Certiorari was filed by Intervenor Jacobus, so we have not yet seen how FDA will respond.  Given that FDA likely wants to preserve its existing interpretation of the scope of orphan drug exclusivity, we’d expect that a Government Petition would be firmly in favor of Certiorari.  The Agency’s and Catalyst’s briefs are due on May 11, 2022.  Nevertheless, we suspect that FDA, as it did the last time it was challenged on its interpretation of the Orphan Drug Act, may be working on a legislative fix for the issues at hand.