Massachusetts, USA-based drugmaker Genzyme says that it has completed enrollment in a Phase II trial of Genz-112638, a novel oral therapy being developed for the treatment of Gaucher's disease.
The drug is a novel oral ceramide analog that is designed to inhibit the enzyme glucosylceramide synthase, the malfunctioning of which is responsible for the formation of excess glucocerebroside in the disease state.
The company also revealed that initial observations from the first five patients to have received treatment, indicate that the agent has a rapid action and meaningful impact on the disease, including bringing about reductions in liver and spleen volume. The firm added that it intends to meet with regulatory agencies in the next few weeks to discuss its developmental strategy.
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