Tag Archives: orphan drug

Sanofi’s Rilzabrutinib Receives Breakthrough and Orphan Designations for Warm Autoimmune Hemolytic Anemia

(IN BRIEF) Sanofi has received Breakthrough Therapy designation from the U.S. FDA and orphan drug designation from Japan’s Ministry of Health, Labour and Welfare for rilzabrutinib (Wayrilz) in warm autoimmune hemolytic anemia, based on data from the ongoing LUMINA 2 … Read the full press release

Basilea Launches FORWARD‑IM Phase 3 Trial of Fosmanogepix in Invasive Mold Infections

(IN BRIEF) Basilea Pharmaceutica has initiated FORWARD‑IM, a global Phase 3 trial evaluating fosmanogepix in adults with invasive mold infections. Building on positive Phase 1 and Phase 2 results—and following the FAST‑IC Phase 3 candidemia trial—this open‑label study will randomize about 160 patients to … Read the full press release

BARDA Invests Additional USD 39 Million to Accelerate Phase 3 Trials of Basilea’s Fosmanogepix and BAL2062

(IN BRIEF) Basilea Pharmaceutica has secured an extra USD 39 million from BARDA under its Other Transaction Agreement, bringing potential non-dilutive funding to USD 268 million over 12 years. This new tranche will finance the ongoing phase 3 trial of … Read the full press release