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rare disease drugs

PharmaDeviceNews rare disease drugs
Representative image of a clinical researcher reviewing rare disease therapy data, as Beren Therapeutics faces an extended FDA decision timeline for adrabetadex in infantile-onset Niemann-Pick disease type C.
Pharma & Biotech

Why the FDA extended its adrabetadex review and what it means for Beren Therapeutics

Adrabetadex faces a longer FDA review in infantile-onset NPC. The delay could shape rare-disease drug standards in 2026.

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Representative image: Pediatric endocrine care is drawing renewed attention as Neurocrine Biosciences’ CRENESSITY two-year CAHtalyst Pediatric data show durable hormone control, reduced glucocorticoid exposure and potential long-term clinical benefits for children with classic congenital adrenal hyperplasia.
Pharma & Biotech

Neurocrine’s CAHtalyst pediatric data sharpens the case for CRENESSITY durability

Steroid reduction is the real test in pediatric CAH. Neurocrine’s CRENESSITY data now faces the durability, access and adoption question.

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Pharma & Biotech

Can NovelMed Therapeutics challenge established PNH drugs with its Bb-targeting antibody Ruxoprubart?

NovelMed Therapeutics advances Ruxoprubart for paroxysmal nocturnal hemoglobinuria with Phase II data and a new subcutaneous trial. Discover what comes next.

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Pharma & Biotech

Can Idorsia Pharmaceuticals Ltd’s lucerastat redefine Fabry disease treatment beyond enzyme replacement therapy?

Discover how Idorsia Pharmaceuticals Ltd’s FDA-aligned Phase 3 plan could make lucerastat the first oral therapy for all Fabry disease patients. Read the analysis.

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