News Kite’s CAR-T therapy most valuable pipeline orphan drug Drug tops list compiled by Evaluate Pharma
Views & Analysis Orphan drugs will fuel pharma's future - and a pricing pushb... Evaluate predicts sales could approach $8bn if approved.
News Teva files orphan blockbuster hope in tardive dyskinesia SD-809 will be vital for Teva if approved.
News Pharma must slash rare disease drug prices - patient organis... More rare disease drugs must also be approved, says EURORDIS.
News NICE rejects second Alexion ultra-rare disease drug NHS will not fund Kanuma for rare disease LAL-D.
News Marathon halts launch of Duchenne drug after price outcry Company says it will listen to concerns of patient advocates
News Merck rare tumour drug Ezmekly backed for NHS use Merck's drug for tumours associated with rare disease neurofibromatosis type 1, Ezmekly, has been cleared for NHS use in children as young as two.
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