Tag Archive for: Orphan Drug Designation

Wednesday, the FDA denied Aldeyra Therapeutics’ application for ADX-2191 (methotrexate injection, USP), which the Massachusetts biotech was proposing to treat the rare eye cancer primary vitreoretinal lymphoma.

Novartis reported Thursday that a Phase III trial of iptacopan met its primary endpoint in paroxysmal nocturnal hemoglobinuria (PNH) – the company’s second win in this indication in as many months.

Mereo BioPharma will cut 40% of its headcount and significantly reduce expenses to support the advancements of its lead programs through the clinic, the company announced Tuesday. 

Inhibrx Inc. announced that the European Commission (“EC”), based on a positive opinion issued by the European Medicines Agency (“EMA”), has granted orphan medicinal product designation to INBRX-109 for the treatment of chondrosarcoma.

United Therapeutics announced on Tuesday that the company received U.S. Food and Drug Administration approval for its therapeutic Tyvaso DPI. Tyvaso DPI marks the first approval of a dry powder inhaler for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD).