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Niemann-Pick disease type C

PharmaDeviceNews Niemann-Pick disease type C
IntraBio’s AQNEURSA has become the first FDA-approved treatment for ataxia in patients with ataxia-telangiectasia after significant neurological improvement in Phase 3 testing. Representative image.
Features & Analysis

AQNEURSA is the first ataxia-telangiectasia treatment. Can a 12-week improvement change a lifelong disease?

IntraBio’s AQNEURSA has become the first approved treatment for ataxia in people with ataxia-telangiectasia after a randomized Phase 3 trial showed a significant improvement in neurological function. The approval ends decades without a specific therapy for the disorder’s ataxia, but it does not resolve whether levacetylleucine can slow long-term neurodegeneration or alter the immune, pulmonary

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IntraBio’s pivotal Phase III CACNA1A trial is enrolling patients as levacetylleucine moves into a key clinical test, with recruitment expected to be completed in October 2026. Representative image.
Pharma & Biotech

What IntraBio’s IB1001-304 Phase III trial could reveal about CACNA1A treatment

IntraBio’s Phase III CACNA1A trial is enrolling, with October recruitment completion targeted as levacetylleucine enters a pivotal test.

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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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Pharma & Biotech

Why Beren Therapeutics’ adrabetadex NDA could redefine approval standards in ultra-rare pediatric disease

FDA acceptance of adrabetadex puts disease modification for infantile-onset NPC under review. See what this decision could change for rare disease approvals.

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