U.S. flag An official website of the United States government

On Oct. 1, 2024, the FDA began implementing a reorganization impacting many parts of the agency. We are in the process of updating FDA.gov content to reflect these changes.

U.S. Food and Drug Administration
  1. Home
  2. For Industry
  3. Medical products for rare diseases and conditions
  4. FDA’s Overview of Catalyst Pharms., Inc. v. Becerra
  1. Medical products for rare diseases and conditions

FDA’s Overview of Catalyst Pharms., Inc. v. Becerra

Update January 23, 2023: The FDA has carefully considered the complex legal and regulatory implications of the decision made by the U.S. Court of Appeals for the Eleventh Circuit in Catalyst Pharms., Inc. v. Becerra. In a Federal Register Notice, the agency explains that, at this time, while complying with the court’s order in Catalyst, FDA intends to continue to apply its regulations to matters beyond the scope of that order. These regulations tie the scope of orphan-drug exclusivity to the uses or indications for which the drug is approved. The agency believes its decision is in the best interest of public health, rare disease patients and rare disease product development. See the notice for more information. 

On September 30, 2021, the U.S. Court of Appeals for the 11th Circuit issued a decision in Catalyst Pharms., Inc. v. Becerra (Catalyst)—a decision that impacts drug companies (or sponsors) that research and market treatments for rare diseases and patients who need those drugs. The FDA expects that the statutory interpretation adopted in the Catalyst decision would affect the incentives for research and development of medical products for rare diseases. 

The Catalyst decision addressed the scope of orphan-drug exclusivity (ODE). Under the Catalyst decision, the first company to gain approval for any use for a drug that has been designated for a rare disease will, in most cases, have seven years of exclusivity for its drug for the entire rare disease. Specifically, the court held that under the statute, ODE 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.  

If, for example, the FDA grants orphan-drug designation to a drug for “treatment of cystic fibrosis,” and then approves that drug for “treatment of adult patients with a particular gene mutation with cystic fibrosis,” the approval of the application will block FDA from approving – for seven years – another company’s application for the same drug for any indication within cystic fibrosis, including for children and for patients without the particular mutation.

The FDA, by contrast, has interpreted the statute to mean that ODE blocks approval of the same drug for only the same approved use or indication. Under this interpretation, drug companies can continue to study and seek approval for other uses of the drug for that disease or condition, such as a pediatric indication for a product that had been approved for adults.

Key Impacts:

  • The Catalyst decision adversely resolves a statutory issue about the scope of ODE. The Catalyst decision interpreted the Orphan Drug Act to unambiguously tie ODE to the disease or condition for which the drug was designated, which would leave the FDA with no discretion to address the issue differently. In the coming months, the FDA will need to consider how the decision affects drugs with active terms of orphan drug exclusivity as well as currently marketed drugs, including generics. Going forward, the FDA expects that some drugs that are in late-stage development, or that have already been submitted for marketing application review, would be blocked from approval under the Catalyst decision’s interpretation of the Act. 
     
  • The Catalyst decision implicates approval of drugs for rare diseases. Because the FDA generally grants orphan-drug designation for the entire rare disease or condition—to incentivize sponsors early in drug development to study all persons with the rare disease or condition—and because FDA can only approve a drug for the specific indications for which there is adequate data in the marketing application, the designated disease or condition can represent a much broader population than the approved indication.  

    Sponsors could seek approval and exclusivity for their drugs by focusing on the smallest, easiest-to-study populations. Under the interpretation in the Catalyst decision, such an approval would result in exclusivity for their drug for the entire disease, even though the sponsors did not invest in studying and developing their drug for all individuals with the disease. 
     
  • The FDA expects that the Catalyst decision’s interpretation could adversely affect children—particularly the youngest pediatric populations—and other populations that are typically studied later in drug development. Because clinical studies in certain populations including children can be more challenging to conduct, sponsors often focus first on the populations who are easiest to study: adults with the fewest co-morbidities and complications. Except for certain oncology products, FDA cannot require pediatric studies of orphan-designated products. These barriers to development could mean that children (and other later-studied populations such as elderly individuals) would not have formulations, or dosing and safety information, appropriate for their needs for years after the product’s original approval.
     


Back to Top