Company: IPHYF
Filing Date: 2025-04-30
Form Type: 20-F
Source: 0001598599-25-000042
Chunk: 149

Company: Innate Pharma SA
Filing Date: 2025-04-30
Form: 20-F
Item: Item 4
Chunk 149
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 of the drug. The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition, a designation which carries with it the following incentives: the possibility of obtaining research grants from the American government for clinical trials; tax credits for a portion of research costs; a possible exemption from user fees; and the potential for a seven-year period of exclusivity if a marketing authorization is granted.

Regarding orphan drug exclusivity, if a product with orphan drug designation receives the first FDA approval for the disease or condition for which it has such designation or for a select indication or use within the rare disease or condition for which it was designated, the product will generally receive orphan drug exclusivity. Orphan drug exclusivity means that the FDA may not approve another sponsor’s marketing application for the same drug for the same use or indication for seven years, except in certain limited circumstances. Orphan drug exclusivity does not block the approval of a different product for the same rare disease or condition, nor does it block the approval of the same product for different indications. If a product designated as an orphan drug ultimately receives marketing approval for an indication broader than what was designated in its orphan drug application, it may not be entitled to exclusivity.

Orphan exclusivity will not bar approval of another product under certain circumstances, including if a subsequent product that is the same biologic for the same use or indication is shown to be clinically superior to the approved product on the basis of greater effectiveness than the approved drug, greater safety in a substantial portion of the target populations, or demonstration of a major contribution to patient care. Additionally, FDA may approve another application for the same biologic for the same use or condition notwithstanding the applicability of orphan drug exclusivity, if the company with orphan drug exclusivity is not able to assure a sufficient quantity of the drug.

In the European Union, equivalent legislation has been adopted to promote treatments for rare diseases (Regulation 141/2000/EC of December 16, 1999, as amended by Regulation 847/2000/EC of April 27, 2000). A medicinal product may be designated as orphan if: (a) it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting not more than five in 10,000 persons in the European Union when the application is made, or (b) it is intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition in