News Related to Office of Orphan Products Development

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TuHURA gains FDA orphan drug designation for IFx-2.0 in cutaneous melanoma

May 17, 2026

TuHURA Biosciences said the FDA granted orphan drug designation to IFx-2.0 for stage IIB to stage IV cutaneous melanoma based on Phase 1 data. The company said its Phase 3 IFx-2.0 study in first-line Merkel cell carcinoma continues to enroll.

Insmed Reports Strong 2025 Results, FDA Grants Orphan Drug Status to Treprostinil Palmitil

Feb 28, 2026

Insmed reported total revenues of $606.4 million for 2025, with ARIKAYCE growing 19% annually. The FDA granted orphan drug designation to treprostinil palmitil for pulmonary arterial hypertension treatment in January 2026.