Following a positive opinion from the European Medicines Agency (EMA), the European Commission (EC) has granted orphan medicinal product designation to Inhibrx, Inc. for INBRX-109 for the treatment of chondrosarcoma.
“We are highly optimistic about the potential of INBRX-109 in chondrosarcoma to address a high unmet medical need,” said Inhibrx CEO, Mark Lappe.
“The positive opinion issued by the EMA is excellent news and acknowledges the potential of INBRX-109 as a treatment for patients throughout Europe who suffer from this debilitating, rare condition.”
The EC grants orphan designation to drugs and biologics intended for the safe and effective treatment, prevention, or diagnosis of a disease that is life-threatening or chronically debilitating impacting fewer than five in 10,000 patients in the European Union.
Orphan drug designation in the EU can provide certain benefits, including reduced regulatory fees, clinical protocol assistance and the potential for up to ten years of market exclusivity following regulatory approval.
Chondrosarcoma is an orphan bone cancer with approximately 2,800 new patients diagnosed annually in the U.S. and the EU. There are currently no therapeutics approved for the treatment of chondrosarcoma.
INBRX-109 is a precision-engineered, tetravalent death receptor 5 (DR5) agonist antibody designed to exploit the tumor-biased cell death induced by DR5 activation.
In 2021, the FDA granted Fast Track designation to INBRX-109 for the treatment of patients with unresectable or metastatic conventional chondrosarcoma and orphan-drug designation to INBRX-109 for chondrosarcoma in the U.S.
In June 2021, Inhibrx initiated a randomized, blinded, placebo-controlled, potential registration-enabling phase 2 trial of INBRX-109 in conventional chondrosarcoma, which is currently ongoing.
In November 2021, Inhibrx provided updated results from its ongoing phase 1 clinical trial evaluating the efficacy and safety of INBRX-109 in patients with conventional chondrosarcoma.
Preliminary disease control was observed in 16 of the 18 evaluable patients (89%) measured by RECISTv1.1, with two of the 18 achieving partial responses (11%). Based on preliminary results of the ongoing phase 1 trial at that time, the median progression-free survival (PFS) was 7.4 months, and the median overall survival had not been reached. Three patients had exceeded 61 weeks on treatment with INBRX-109, with 77 weeks being the longest duration of stable disease observed at that time.