Trapped by orphan drug rules, FDA blocks generic Treanda for 3 extra years

Once again, the FDA’s own regulations designed to foster rare disease drug development have forced the agency to extend the monopoly on an old drug.

In what Bernstein analyst Ronny Gal called an “illogical decision” that will cost the public $3 billion, the FDA granted Eagle Pharmaceuticals’ blood cancer drug Treanda three more years of exclusivity, blocking generics until December 2022.

The decision stemmed from Eagle’s court victory granting orphan drug exclusivity—not to Treanda—but to its newer counterpart, Bendeka, which shares the same active ingredient.

How did such a bizarre situation happen? Basically, FDA’s own regulations happened.

Bendeka got seven years of orphan drug exclusivity by court order. But a quirk in the FDA’s own rules deem Bendeka and Treanda as essentially the “same drug” because the newer drug hasn’t proven superior in the clinic. Therefore, exclusivity for Bendeka applies to Treanda, too.

Treanda, a chemotherapy, was approved by the FDA in 2008 and was expected to face generics this November. Bendeka won approval in December 2015 and sought seven years of orphan-drug exclusivity under the Orphan Drug Act.

FDA originally denied Eagle’s request, saying that Bendeka was not clinically superior to Treanda and thus didn’t merit orphan drug exclusivity. But Eagle sued in federal district court and won, even though the FDA argued the ruling would grant “lengthy monopolies for trivially modified versions of existing orphan drugs that do not represent a meaningful benefit to patients.”

The FDA is trying to appeal that ruling by the U.S. District Court in Washington, D.C. But in the meantime, the decision protects Treanda as well.

“[T]he scope of Bendeka’s exclusivity extends to all applications containing the same active moiety as Bendeka, bendamustine,” the agency said.

Generic versions of Treanda are barred until Bendeka loses orphan exclusivity on Dec. 7, 2022, unless another drug manages to prove superior efficacy, the agency said.

Gal, in a note to clients on Monday, criticized the FDA for its decision.

“This is poor performance by FDA which shows that even in the Gottlieb era, the risk-averse bureaucracy can get lost in its own maze of regulations,” the Bernstein analyst wrote.

The FDA, in its own defense, said: “Agency regulations may sometimes draw bright lines that may be over- or under-inclusive, but are nevertheless appropriate exercises of agency authority, and provide greater certainty to the regulated industry.”

After considering comments from stakeholders, including generics makers aiming to copy Treanda, the agency said it decided not to diverge from its “same drug” approach in this case, where a court order and the FDA’s own regulation seems to collide.

Ironically enough, the “clinical superiority” element only became law last August as part of the FDA Reauthorization Act of 2017 (FDARA).

Because the FDA “expects that only a limited number of pre-FDARA drugs may be affected by this decision,” the agency figured “promulgating new regulations would not be an effective use of agency resources.”

However, the pre-FDARA problem reflected in the Treanda case isn’t the only loophole in the FDA’s orphan drug program that reform advocates would like to see closed.

Catalyst Pharma recently came under public scrutiny for the $375,000 price tag on its rare disease therapy Firdapse. The drug’s active ingredient had been given for free to patient for three decades under FDA’s compassionate use program.

Therefore, Sen. Bernie Sanders, I-Vt., had argued Firdapse is basically an “old drug.” But Catalyst still got orphan drug exclusivity and listed Firdapse at the controversial price tag.

https://www.fiercepharma.com/pharma/trapped-by-orphan-drug-program-fda-blocks-generic-cancer-drug-for-3-extra-years