Denali climbs past Avlayah expectations with $3.6M in revenue
After launching in April, Hunter syndrome drug Avlayah surprised analysts with a strong first quarter, as patients rush to get access to Denali Therapeutics’ new option.
biospace · 4d ago
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Pfizer CFO to step down; Denali sells a regulatory fast-pass
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biopharma_dive · 1mo ago

brand

Avlayah

generic

tividenofusp alfa

MOA

12.1 Mechanism of Action Hunter syndrome is an inherited X-linked recessive lysosomal storage disease caused by a deficiency of iduronate-2-sulfatase (IDS), a lysosomal enzyme, that degrades heparan sulfate (HS) and dermatan sulfate (DS), the two primary glycosominoglycans (GAGs) in the lysosome. Insufficiency or absence of IDS leads to accumulation of GAGs, including HS and DS, and subsequent lysosome dysfunction in multiple organs and tissues, including the central nervous system (CNS). Tividenofusp alfa-eknm provides an exogenous source of IDS. The fragment, crystallizable (Fc) component of tividenofusp alfa-eknm binds to the apical domain of the transferrin receptor (TfR) and delivers IDS to peripheral tissues and to the CNS through receptor-mediated transcytosis across the blood-brain barrier. Tividenofusp alfa-eknm is internalized via binding to the mannose-6-phosphate receptor on the cell surface and transported into lysosomes where it is thought to exert enzymatic activity and reduce accumulated GAGs. In addition, since TfR is ubiquitously expressed, it is expected that the interaction of tividenofusp alfa-eknm and TfR will contribute to its uptake into cells in the brain and peripheral tissues.

indication

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome )

phase

marketed

Data sources

openFDA · ClinicalTrials.gov

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tividenofusp alfa

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by $DNLI Denali Therapeutics Inc.

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AVLAYAH (TIVIDENOFUSP ALFA-EKNM) — FDA approval

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Denali climbs past Avlayah expectations with $3.6M in revenue
After launching in April, Hunter syndrome drug Avlayah surprised analysts with a strong first quarter, as patients rush to get access to Denali Therapeutics’ new option.
biospace · 4d ago
Biogen validated the Alzheimer’s tau theory. CEO Viehbacher still isn’t betting the store
With so many unknowns, Biogen is carefully eyeing its next-gen Alzheimer’s treatment diranersen as a high-risk, high-reward project with the potential for a major payout around 2030.
biospace · 16d ago
Pfizer CFO to step down; Denali sells a regulatory fast-pass
A search is underway for a successor to Dave Denton, who took a job outside the pharmaceutical industry. Elsewhere, a new kind of Duchenne gene therapy got venture backing and a GSK deal paid divid…
biopharma_dive · 1mo ago

brand

Avlayah

generic

tividenofusp alfa

MOA

indication

Mucopolysaccharidosis II (MPS II) (Hunter Syndrome )

phase

marketed

Data sources

openFDA · ClinicalTrials.gov

last refreshed 3m ago

Found something off?

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tividenofusp alfa

🐶 often sniffed alongside

all catalysts(1)

Trials studying tividenofusp alfa

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tividenofusp alfa

🐶 often sniffed alongside

all catalysts(1)

Trials studying tividenofusp alfa

News(3)

Report a data issue

comments(0)