- Denali Therapeutics Inc. announced that the **U.S. Food and Drug Administration (FDA)** has extended its review timeline for the Biologics License Application (BLA) seeking accelerated approval of **tividenofusp alfa** for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.  **[1714899_0001714899-25-000180_exhibit991pressrelease1013.htm:0]** **[1714899_0001714899-25-000180_dnli-20251013.htm:1]** 
- The **Prescription Drug User Fee Act (PDUFA) target date** has been extended by **three months**, moving from **January 5, 2026, to April 5, 2026**.  **[1714899_0001714899-25-000180_exhibit991pressrelease1013.htm:0]** **[1714899_0001714899-25-000180_dnli-20251013.htm:1]** 
- This extension is a result of Denali's submission of updated clinical pharmacology information, which the FDA classified as a **Major Amendment**.  **[1714899_0001714899-25-000180_exhibit991pressrelease1013.htm:0]** **[1714899_0001714899-25-000180_dnli-20251013.htm:1]** 
- The company stated that the extension is **not related to efficacy, safety, or biomarkers**, and no additional data were requested by the FDA.  **[1714899_0001714899-25-000180_exhibit991pressrelease1013.htm:0]**

Denali Therapeutics Announces FDA Review Extension for Tividenofusp Alfa BLA

- The FDA has extended the review timeline for Denali Therapeutics' biologics license application for **tividenofusp alfa**, a treatment for Hunter syndrome, from **January 5, 2026, to April 5, 2026**.  **[1714899_02c99752a9b24a74ac5d291fab1fcd69]** 
- This **three-month delay** is due to the submission of **updated clinical pharmacology data** classified as a Major Amendment, but is **not related to concerns over the drug's efficacy, safety, or biomarkers**.  **[1714899_02c99752a9b24a74ac5d291fab1fcd69]** 
- Denali Therapeutics, with a market capitalization of approximately **$2.19 billion**, is preparing for the potential commercialization of tividenofusp alfa, which holds **Fast Track and Breakthrough Therapy designations**.  **[1714899_02c99752a9b24a74ac5d291fab1fcd69]** 
- The company currently reports **no revenue growth**, a **negative earnings per share (EPS) of -2.8**, and negative returns on equity and assets, but maintains strong liquidity with a **current and quick ratio of 10.27** and a **low debt-to-equity ratio of 0.05**.  **[1714899_02c99752a9b24a74ac5d291fab1fcd69]**

Denali Therapeutics' Hunter Syndrome Drug Review Extended by FDA

- Denali Therapeutics Inc. announced that the **U.S. Food and Drug Administration (FDA)** has **extended its review timeline** for the Biologics License Application (BLA) of **tividenofusp alfa** for the treatment of **mucopolysaccharidosis type II (MPS II)**, also known as Hunter syndrome.  **[1714899_6cb775fde706428798a13a5f0a6179c9_0]** 
- The **Prescription Drug User Fee Act (PDUFA) target date** has been extended from **January 5, 2026, to April 5, 2026**.  **[1714899_6cb775fde706428798a13a5f0a6179c9_0]** 
- This extension is a result of Denali's submission of **updated clinical pharmacology information** in response to an FDA request, which the FDA classified as a **Major Amendment**.  **[1714899_6cb775fde706428798a13a5f0a6179c9_0]** 
- The company stated that the extension is **not related to efficacy, safety, or biomarkers**, and they believe the updated information does not affect the clinical pharmacology or benefit-risk conclusions of the BLA.  **[1714899_6cb775fde706428798a13a5f0a6179c9_0]**

- Denali's lead program, **tividendifusp alfa pidufidate (TIVI)**, an enzyme replacement therapy for Hunter syndrome, has a **PDUFA date of January 5** and is expected to launch in the **U.S. in 2026**.  **[1714899_tx_382210_0]** **[1714899_tx_382210_1]**  The company anticipates **premium pricing** and **20% COGS** for TIVI.  **[1714899_tx_382210_3]** 
- The **COMPASS confirmatory trial** for TIVI is expected to complete Cohort A by the **end of 2025**, with a full readout anticipated **two years from the end of 2025**, which will support broader global approvals.  **[1714899_tx_382210_4]** 
- Denali is pursuing an **accelerated approval path** for its Sanfilippo (MPS IIIA) program, with a Phase 3 trial expected to begin soon, targeting an estimated **300-400 patients in the U.S.**.  **[1714899_tx_382210_4]** **[1714899_tx_382210_5]** 
- The company is expanding its Enzyme Transport Vehicle (ETV) platform to include a **Pompe program**, focusing on the larger **Late-Onset Pompe Disease (LLPD)** population, and aims to file **one to two Investigational New Drug (IND) applications per year** across its pipeline.  **[1714899_tx_382210_6]** **[1714899_tx_382210_7]** **[1714899_tx_382210_8]**

Denali Therapeutics (DNLI)