Denali Therapeutics Announces Initiation of Rolling Submission for Accelerated Approval of Tividenofusp Alfa for Hunter Syndrome
Denali Therapeutics, a biotechnology company based in South San Francisco, California, recently announced the initiation of a rolling submission of a Biologics License Application (BLA) for the accelerated approval of tividenofusp alfa for the treatment of Hunter syndrome (MPS II). The company revealed that the submission has been received by the Center for Drug Evaluation and Research (CDER) of the U.S. Food and Drug Administration (FDA).
Collaborative Engagement with the FDA
Denali Therapeutics has been maintaining regular, collaborative, and productive engagement with CDER. The company is aligned with CDER on the content of the BLA data package, including the use of cerebrospinal fluid heparan sulfate (CSF HS) as a surrogate endpoint to support an accelerated approval. This surrogate endpoint is a biomarker that is believed to correlate with clinical benefit in Hunter syndrome.
Timeline for Submission and Commercial Launch
Denali expects to complete the BLA submission in the first half of May 2025. The company continues to prepare for a potential commercial launch in the U.S. in late 2025 or early 2026.
Impact on Individuals with Hunter Syndrome
For individuals with Hunter syndrome, the potential approval of tividenofusp alfa as a treatment represents a significant step forward. Hunter syndrome is a genetic disorder caused by the deficiency of iduronate-2-sulfatase, leading to the accumulation of heparan sulfate and dermatan sulfate in various body tissues. This condition can cause a range of symptoms, including coarse facial features, skeletal abnormalities, and cognitive impairment.
Currently, there are no FDA-approved treatments for Hunter syndrome. Enzyme replacement therapies, such as Cerezyme and Elaprase, are available but have limitations, such as the need for frequent infusions and potential side effects.
If approved, tividenofusp alfa could offer a more convenient and potentially more effective treatment option for individuals with Hunter syndrome. The use of CSF HS as a surrogate endpoint in the clinical trials suggests that the drug could have a positive impact on the disease’s progression.
Global Implications
Beyond the U.S., the potential approval of tividenofusp alfa could have significant implications for individuals with Hunter syndrome worldwide. Denali Therapeutics has ongoing clinical trials in Europe, Australia, and other regions.
- In Europe, Denali Therapeutics has initiated a Marketing Authorization Application (MAA) for tividenofusp alfa. The European Medicines Agency (EMA) has granted PRIME (Priority Medicines) designation to the drug, which aims to expedite the regulatory review process.
- In Australia, Denali Therapeutics has submitted a Biologics Application to the Therapeutic Goods Administration (TGA) for accelerated approval. The TGA has granted Priority Review status to the application.
If approved in these regions, tividenofusp alfa could provide a new treatment option for individuals with Hunter syndrome, improving their quality of life and potentially slowing the progression of the disease.
Conclusion
Denali Therapeutics’ initiation of a rolling submission of a BLA for accelerated approval of tividenofusp alfa for the treatment of Hunter syndrome marks a significant milestone in the development of a potential new treatment for this debilitating condition. The collaborative engagement between Denali Therapeutics and the FDA, as well as the potential use of CSF HS as a surrogate endpoint, bodes well for the drug’s chances of approval. For individuals with Hunter syndrome, the potential approval of tividenofusp alfa could offer a more convenient and effective treatment option. The global implications of this development are also noteworthy, as Denali Therapeutics continues to pursue regulatory approvals in Europe and other regions.
As we await the FDA’s decision, the potential approval of tividenofusp alfa represents a beacon of hope for the Hunter syndrome community, offering the promise of improved quality of life and potentially slowing the progression of this debilitating condition.
