Clinical Development Strategy
Zywie is currently working with the FDA to confirm the clinical and pre-clinical requirements to support approval of Ziva for the treatment of T3 GD. Based on this guidance, Zywie plans to finalize its proposed protocol for two Phase 2/3 placebo-controlled pivotal studies with an integrated natural history study. Zywie has been granted orphan drug designation by the US FDA for the use of Ziva in the treatment of GD.
source site Well-Defined Primary Endpoint FOR TYPE 3 GD
Zywie has completed a meta-analysis of T3 GD and has used these findings to design a clinical study that will provide sufficient data on the safety and effectiveness of Ziva to support a near-term decision on its approvability for marketing as a new treatment for T3 GD.
- To minimize the risk of delay, Zywie will proceed with two pivotal studies and plan to file for approval based on the results of the first pivotal study.
- Ziva is a potential treatment for an orphan disease with no approved treatment.
POTENTIAL OPPORTUNITIES BEYOND TYPE 3 GD
Zywie is also evaluating the potential to pursue other indications, including:
- Type I Gaucher Disease (a substantially larger market than Type 3 GD because there are about 20 times more patients with T1 than with T3 GD)
- Lewy Body Dementia (a subset of Parkinson’s Disease with significant neurological symptoms)
- Other subsets of patients with Parkinson’s Disease (which has a similar disease mechanism that is likely to respond to treatment with Ziva).
There’s a significant overlap between Gaucher and Parkinson’s. Please see Publications Section for a large number of both studies and assumptions.