## Facts

- Baker Norton Pharmaceuticals, Inc. developed Paxene, a paclitaxel drug intended (among other uses) to treat AIDS-related Kaposi’s sarcoma, a rare disease.
- Bristol-Myers Squibb Co. developed Taxol, also containing paclitaxel, for the same orphan indication.
- Paxene and Taxol differed in manufacturing, excipients, impurity profiles, and stability/degradation characteristics, but shared the same active ingredient (paclitaxel).
- FDA approved Taxol for second-line treatment of Kaposi’s sarcoma on August 4, 1997, triggering seven-year orphan-drug exclusivity for that indication.
- FDA later advised Baker Norton that Paxene was approvable but would not receive final approval during Taxol’s exclusivity unless Paxene was not the “same drug” or was “clinically superior.”
- FDA treated Paxene as the “same drug” as Taxol under its orphan-drug regulations because both products contained the same active moiety, and FDA found Baker Norton had not shown clinical superiority.
- Baker Norton sued under the Administrative Procedure Act, challenging FDA’s “same drug” interpretation and the decision to withhold approval; the parties filed cross-motions for summary judgment, and BMS intervened to defend the exclusivity.

## Issues

1. Whether the Orphan Drug Act permits FDA to define “same drug” for exclusivity purposes primarily by reference to the same active moiety/active ingredient rather than finished-product characteristics.
2. Whether FDA’s “same drug” and “clinical superiority” regulations are a permissible construction of the Orphan Drug Act under Chevron.
3. Whether FDA acted arbitrarily or capriciously in concluding Paxene was not clinically superior to Taxol and in withholding final approval during Taxol’s exclusivity period.

## Decision

- The court granted summary judgment for FDA and the intervenor BMS and denied Baker Norton’s motion.
- The court held the Orphan Drug Act does not unambiguously define “same drug” at the product-formulation level, leaving a statutory gap for FDA to fill.
- The court upheld FDA’s regulation treating drugs with the same active moiety as the “same drug,” with approval during exclusivity allowed only upon a showing of clinical superiority.
- The court held FDA reasonably applied its framework in treating Paxene as the same drug as Taxol and requiring evidence-based clinical superiority, not merely manufacturing or excipient differences.
- The court found FDA’s decision was not arbitrary or capricious because FDA applied an established regulatory test, considered relevant factors, and explained the connection between the shared active moiety, the lack of clinical-superiority evidence, and the approval bar.

## Legal Principles

- When a statute does not directly resolve a technical definitional question delegated to an agency, courts apply Chevron and defer to a reasonable agency construction.
- FDA may define “same drug” under the Orphan Drug Act by reference to the same active moiety/active ingredient, even if the products differ in excipients, manufacturing processes, or impurity profiles.
- Orphan-drug exclusivity bars approval of a later product for the same indication if it is the “same drug,” unless the later product demonstrates “clinical superiority” (e.g., greater efficacy, greater safety, or a major contribution to patient care).
- Under APA review, an agency decision is upheld when it reflects reasoned decisionmaking, applies a stated regulatory standard, and is supported by an adequate explanation; disagreement with the agency’s policy choices or technical judgments is insufficient.

## Conclusion

The court sustained FDA’s active-moiety-based “same drug” definition for orphan-drug exclusivity and upheld FDA’s refusal to approve Paxene during Taxol’s exclusivity period because Baker Norton did not establish clinical superiority.


Baker Norton Pharms., Inc. v. Food & Drug Admin., 132 F. Supp. 2d 30 (D.D.C. 2001)