On orphan drug exclusivity, FDA decides to go its own way after court loss

The FDA on Monday offered its latest perspective on how it will interpret the laws governing the seven years of orphan drug exclusivity granted to new rare disease drugs, following a controversial court loss that left many rare disease drugmakers in limbo.

The agency effectively decided to return to its old ways in making orphan exclusivity determinations, following the Office of Orphan Products Development’s decision to stop making these determinations since late 2021 due to the court loss.

The case involved two rare disease drug developers, Catalyst and Jacobus, with a US appeals court overturning a prior FDA win, saying that the agency never should’ve approved Jacobus’ cheaper rare disease drug because Catalyst’s previously approved drug with a narrower indication and the same active ingredient had orphan drug exclusivity, barring such an approval.

But that court loss and subsequent silence from OOPD has meant that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on orphan exclusivity in the Orange Book.

FDA now says that it intends to continue to apply its regulations and tie the scope of orphan drug exclusivity to the uses or indications for which a drug is approved, which runs counter to what the court said should be the case with Catalyst, where orphan exclusivity could block any other competitors beyond the scope of the approved population.

But FDA has made its stance on that court decision well-known since, saying in December that it would have “far-reaching implications” and “blocks approval of another company’s application for the same drug for the entire disease or condition for which the drug is granted orphan-drug designation, regardless of whether the drug was approved only for a narrower use or indication.”

Consistent with the court’s decision, the agency said that it set aside the approval of Jacobus’ drug in that instance, but the agency also said it wouldn’t begin to apply exclusivity as the court said should be the case in Catalyst, saying it

continues to believe that the statutory text does not unambiguously require that orphan-drug exclusivity extend to the entire disease or condition for which a drug received orphan-drug designation if the drug is only approved for some uses within that disease or condition. Further, FDA believes that its statutory interpretation embodied in its regulations best advances the Orphan Drug Act’s purposes, appropriately balancing the need to incentivize the development of drugs for rare diseases and conditions with the need to provide patient access to orphan drugs.

FDA also said it thinks its interpretation of the law appropriately incentivizes companies to reach for newer patient populations too.

“These regulations incentivize sponsors to continue to develop a drug for use in all persons affected by a rare disease or condition,” the agency wrote in today’s Federal Register. “Thus, FDA believes that continued adherence to its validly promulgated regulations will best serve the public health by facilitating patient access to orphan drugs, especially for difficult-to-study patients such as young children.”

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