MEdPACTO

Eligible for a rare pediatric disease priority review voucher that is worth $100M

On September 15th, MedPacto, a genome-based drug discovery and clinical-stage biotechnology company, announced that it received a Rare Pediatric Disease Designation from the U.S. FDA for Vactosertib as a monotherapy for the treatment of relapsed/refractory metastatic osteosarcoma.

Followed by having received Orphan Drug Designation from the FDA last year, this Rare Pediatric Designation will further expedite the development process for osteosarcoma therapy.

A rare pediatric disease is serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect patients from birth to 18 years.

Under this program, a sponsor who receives approval for a drug or biologic for a "rare pediatric disease" may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product. Priority review vouchers may be sold or transferred.

Priority review vouchers (PRVs) have been trading at around $100 million lately. Back in July, The Swiss pharma Novartis bought an FDA priority review voucher from Mallinckrodt for $100M. It reportedly used two PRVs in 2020 for multiple sclerosis drug Kesimpta and anti-inflammation therapy Cosentyx’s expansion into axial spondyloarthritis.

In a preclinical trial, Vactosertib significantly reduced osteosarcoma cancer cell growth and pulmonary transfer, improving the survival rate of a mouse by 100 percent and also restraining lung metastasis by 100 percent.

“Although Osteosarcoma is the leading cause of deaths due to cancer in a pediatric population, there is no standard treatment.”, “Vactosertib will offer a promising orally available therapeutics for patients with very few options are currently available.”, stated MedPacto representative.

MedPacto received IND approval for a Phase1/2 clinical trial in August.