Denali tividenofusp alfa gets FDA Breakthrough Therapy; BLA submission in early 2025

Denali Therapeutics Inc.

FDA granted Breakthrough Therapy Designation for tividenofusp alfa in Hunter syndrome (MPS II) on Jan 7, 2025.
Denali expects BLA submission under accelerated approval pathway in early 2025; U.S. launch targeted late 2025/early 2026.
Seeking FDA alignment on accelerated approval for DNL126 in Sanfilippo syndrome after CSF HS normalization in Phase 1/2.
Cash, equivalents, and marketable securities ~$1.28B as of Sep 30, 2024; cash runway extended into 2028.
Plan to advance 1–2 TV-enabled programs into clinic per year over next three years across ETV, OTV, ATV.

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