Highlights
- Radiopharm has hit another milestone, securing Rare Pediatric Disease (RPD) Designation from the US FDA.
- The designation is for the company’s DUNP19 technology, being studied to treat osteosarcoma.
- With this designation, the companies get access to a priority review voucher (PRV), which they can use for accelerated approvals or other actions.
- Recently, DUNP19 also secured the ODD for DUNP19 in osteosarcoma treatment.
Shares of Radiopharm Theranostics (ASX:RAD) edged up by 12% on 16 September 2022 as the company secured a new grant from the US Food & Drug Administration (FDA).
The ASX-listed radiopharmaceutical firm has updated on the US FDA grant of a Rare Pediatric Disease (RPD) Designation to its DUNP19 technology. The company is studying this technology as a treatment for osteosarcoma.
LRCC15 expression is produced by cancer cells and the surrounding tumour microenvironment cells. However, it is not produced by healthy normal tissues. Further, LRRC15 production is very high in tumours that are aggressive and resistant to treatment.
How is Radiopharm going to benefit from RPD designation?
The RPD program is intended to progress the development of drugs with the potential to treat serious, rare pediatric diseases.
- Using RPD, businesses can get a priority review voucher (PRV) from the FDA when a marketing authorisation is approved.
- A PRV may be used by the company to speed up clearance, or it may be given or sold to other businesses for use in a similar way.
- Price of two recent examples of PRV sales ranged from US$105 million to US$110 million.
What is Osteosarcoma?
Osteosarcoma is a kind of bone cancer. It majorly affects adolescents, children, and young adults. At present, surgery and chemotherapy are the only available treatments for the disease. An aggressive form of osteosarcoma is an ideal candidate for proof-of-concept testing of DUNP19 as it has one of the highest expressions of LRRC15.
Orphan Drug Designation (ODD)
Recently, DUNP19 also secured the ODD for the treatment of osteosarcoma. The ODD will enable the company to get access to user fee exemptions, tax credits, potential seven-year market exclusivity after approval, and other benefits.