On October 19, 2017 Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company focused on oncology, reported that the European Medicines Agency (EMA) has granted Orphan Drug Designation for G100, Immune Design’s investigational intratumoral therapy, for the treatment of follicular non-Hodgkin’s lymphoma (Press release, Immune Design, OCT 19, 2017, View Source [SID1234521040]).

The EMA orphan drug designation is assigned to products targeting the treatment of rare diseases, which are defined as having a prevalence of not more than 5 in 10,000 people in the European Union (EU). This designation provides the sponsor with certain benefits, including protocol assistance, reduced fees for regulatory activities and up to 10 years of market exclusivity in the EU upon marketing approval for the designated indication.

G100 has also been granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of follicular non-Hodgkin’s lymphoma.

G100 is a product candidate from Immune Design’s GLAAS discovery platform. It contains a potent synthetic small molecule toll-like receptor-4 (TLR-4) agonist, Glucopyranosyl Lipid A (GLA), and is the lead product candidate in Immune Design’s Antigen Agnostic approach. G100 activates innate and adaptive immunity in the tumor microenvironment to generate an immune response against the tumor’s preexisting diverse set of antigens. A growing set of clinical and preclinical data have demonstrated the ability of G100 to activate tumor-infiltrating lymphocytes, macrophages and dendritic cells, and promote antigen-presentation and the recruitment of T cells to the tumor. The induction of local and systemic immune responses has been shown in preclinical studies to result in local and abscopal (shrinking of tumors outside the scope of the localized treatment) tumor control. Currently, G100 is being evaluated as both a monotherapy (with local radiation) and in combination with Merck’s anti-PD-1 agent, pembrolizumab, pursuant to a clinical collaboration with Merck, in a randomized Phase 1/2 clinical trial in patients with follicular non-Hodgkin’s lymphoma.