Medivir AB (NASDAQ: MVIR) (STO: MVIR), a pharmaceutical company focused on developing innovative cancer therapies in areas of high unmet medical need, today announced that its selective cathepsin K inhibitor MIV-711 has received both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) for the treatment of Legg-Calvé-Perthes disease (LCPD).
The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals from birth to 18 years of age. Pediatric diseases recognized as “rare” affect fewer than 200,000 people in the United States. With an RPDD, MIV-711 qualifies for expedited review, and following marketing approval for LCPD, the company may be eligible for a Priority Review Voucher from the FDA. The voucher can be redeemed to receive priority review for a future marketing application or it can be sold to another company for their use.
– LCPD is a disease with significant impact on daily life and risk of long-term consequences for which there are no effective treatments. Children with LCPD often suffer from obesity and depression due to the forced immobility resulting from the disease. In more than half of children, the affected leg becomes shorter than the healthy leg, and in about 50% there is deformity of the hip joint, eventually leading to osteoarthritis. We are pleased that MIV-711 has received RPDD approval from the FDA and thus has the potential to become the first approved treatment option. We see a partnership as an attractive opportunity to advance the development of MIV-711 to ensure the speed of development,” said Jens Lindberg, CEO of Medivir.
To receive an RPDD, there must be supporting data indicating that the drug may be effective in the disease. MIV-711 has shown in an LCPD-specific animal model that it is capable of preventing hip head deformation and having positive effects on bone resorption biomarkers without negatively impacting normal bone formation. Similarly, clinical studies have shown that MIV-711 prevents bone resorption in patients with osteoarthritis, further supporting the potential clinical benefit in LCDP.
