Alexion seeks approval from FDA for PNH drug ALXN1210
The submission uses a rare disease priority review voucher, which designates the BLA for an expedited eight-month review by the FDA instead of the standard 12-month review. Alexion
The US Food and Drug Administration (FDA) has granted rare paediatric disease designation to Vanda Pharmaceuticals’ VCA-894A, an investigational antisense oligonucleotide therapy for Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S).
The designation is for the treatment of patients with medium to high folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer who received at least one, but no more than
The trial showed a statistically significant and clinically meaningful improvement in progression-free survival (PFS) for patients treated with EPd compared with pomalidomide and dexamethasone (Pd) alone. ELOQUENT-3 is