MedPacto's Bexmarilimab Monotherapy for Osteosarcoma Designated as 'Rare Pediatric Disease Drug' by US FDA

[Asia Economy Reporter Lee Gwan-joo] MedPacto announced on the 15th that its single-agent therapy with Bactosertib for osteosarcoma patients has been designated as a 'Rare Pediatric Disease Drug (RPDD)' by the U.S. Food and Drug Administration (FDA).

Following the orphan drug designation (ODD) received from the FDA last year, the recent RPDD designation is expected to accelerate the development of Bactosertib as a treatment for osteosarcoma.

The FDA grants RPDD designation for serious and life-threatening diseases that primarily affect children under 18 years old and fewer than 200,000 people. Upon receiving RPDD designation, applicants can apply for a Priority Review Voucher (PRV) under the pediatric rare disease treatment drug approval exemption. The PRV allows the FDA to complete the approval review within six months, which can be beneficial in the marketing approval process of other products and can be sold or transferred to other companies.

A MedPacto official stated, “Osteosarcoma, a representative pediatric cancer, currently lacks adequate treatments, so following the FDA clinical trial application, there have been many requests for clinical participation from both patients and domestic hospitals. With the FDA orphan drug designation followed by the pediatric rare disease treatment drug designation, we expect Bactosertib to establish itself as an effective treatment option for osteosarcoma patients in the future.”

Osteosarcoma is a malignant tumor arising from bone or bone-related cartilage and other osseous tissues, and is one of the most common pediatric cancers with a high incidence rate among children and adolescents.

In preclinical studies on osteosarcoma, Bactosertib inhibited the growth of osteosarcoma cancer cells and lung metastasis, improving the survival rate of mice by 100%. It also completely suppressed lung metastasis of osteosarcoma cancer cells.

This content was produced with the assistance of AI translation services.