FDA grants Medivir´s MIV-711 Rare Pediatric Disease Designation and Orphan Drug Designation for the treatment of Legg-Calvé-Perthes Disease
- MIV-711 granted Rare Pediatric Disease Designation and Orphan Drug Designation for Legg-Calvé-Perthes Disease.
- RPDD allows fast track review and potential Priority Review Voucher from the FDA.
- LCPD affects children aged 0-18 years with treatment options.
- MIV-711 aims to address unmet medical needs in LCPD patients.
- None.
Insights
The FDA's decision to award both Rare Pediatric Disease Designation and Orphan Drug Designation to Medivir's MIV-711 for the treatment of Legg-Calvé-Perthes Disease is a pivotal development, as it underscores the drug's potential to address a critical treatment gap in pediatric care. This twofold endorsement not only accelerates the review process but also enhances the drug's commercial viability through incentives like the Priority Review Voucher—a lucrative asset that may be sold or utilized to expedite FDA review for future drugs.
For shareholders and potential investors in Medivir, these designations might signal a fertile ground for growth given the commercial exclusivity period typically granted with Orphan Drug status. This could translate into reduced competition and a stronger market position if MIV-711 is approved. However, investors should also consider the clinical and regulatory hurdles that remain prior to the drug reaching the market, as well as the potential size of the pediatric segment afflicted by LCPD, to gauge the true commercial potential.
From a clinical perspective, LCPD's classification as a rare pediatric disease underlines the limited patient population, which often leads to challenges in drug development such as patient recruitment for trials. The success of MIV-711 could hinge on its ability to demonstrate significant efficacy and safety in this niche patient group. Given the debilitating nature of LCPD and the current lack of effective treatments, there could be a substantial impact on patient quality of life, should MIV-711 prove efficacious. This would represent a breakthrough in pediatric orthopedics.
Analyzing from an investment standpoint, the Priority Review Voucher that comes with the RPDD is an asset of considerable value, with previous vouchers being sold for figures upwards of
The FDA defines a rare pediatric disease as a serious or life-threatening disease in which the disease manifestations primarily affect individuals aged from birth to 18 years of age. Pediatric diseases recognized as "rare", affect fewer than 200,000 people in
- "LCPD is a disease, with a significant impact on daily life as well as risk for long-term sequalae, for which there are no available effective treatments. Children with LCPD are more likely to suffer from obesity and depression due to the forced immobility as a consequence of the disease. In more than half of the children, the affected leg becomes shorter than the healthy leg and about
To gain RPDD, there must be supportive data suggesting that the drug may be effective in the disease. MIV-711 has shown, in an LCPD-specific animal model, the ability to prevent femoral head deformity and positive impact on biomarker of bone degradation without negatively impacting normal bone formation. Similarly, clinical studies have shown that MIV-711 prevents bone degradation in patients with osteoarthritis, further supporting the potential clinical benefit in LCDP.
Cathepsin K is the main cysteine protease involved in bone resorption and cartilage degradation by osteoclasts, through the breakdown of key bone matrix proteins. Cathepsin K inhibition protects the damaged bone by reducing bone resorption and promoting bone formation, thereby addressing the key mechanisms causing pathological changes in LCPD, with potential to minimize long-term negative effects. Selective inhibition of cathepsin K has the potential to provide clinical benefit in diseases characterized by excessive bone resorption, including additional pediatric bone disorders.
For additional information, please contact:
Magnus Christensen, CFO, Medivir AB
Telephone: +46 8 5468 3100.
E-mail: magnus.christensen@medivir.com
About Legg-Calvé-Perthes Disease
Legg-Calvé-Perthes disease (LCPD) is a childhood hip disorder initiated by a disruption of blood flow to the head of the femur. Due to the lack of blood flow, the bone dies (osteonecrosis or avascular necrosis) and stops growing. Over time, healing occurs by new blood vessels infiltrating the dead bone and removing the necrotic bone which leads to a loss of bone mass and a weakening of the femoral head. The condition is most commonly found in children between the ages of 4 and 8, but it can occur in children between the ages of 2 and 15. It is estimated to affect about 10 in 100.000 children per year in the
About MIV-711
MIV-711 is a potent and selective inhibitor of cathepsin K, the principal protease involved in breaking down collagen in bone and cartilage. It has been shown to slow, stop or reverse the progressive degeneration of joints affected by osteoarthritis. By inhibiting cathepsin K and increased/excessive activity of osteoclasts, MIV-711 has the potential to prevent degradation of bone and cartilage and counteract the osteonecrosis induced by lack of blood flow, to prevent femoral head deformity and long-term negative effects as well as positively impact Quality of Life.
About Medivir
Medivir develops innovative drugs with a focus on cancer where the unmet medical needs are high. The drug candidates are directed toward indication areas where available therapies are limited or missing and there are great opportunities to offer significant improvements to patients. Medivir is focusing on the development of fostroxacitabine bralpamide (fostrox), a smart, targeted chemotherapy designed to selectively treat liver cancer cells and to minimize side effects. Collaborations and partnerships are important parts of Medivir's business model, and the drug development is conducted either by Medivir or in partnership. Medivir's share (ticker: MVIR) is listed on Nasdaq Stockholm's Small Cap list. www.medivir.com.
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SOURCE Medivir
FAQ
What designations did MIV-711 receive from the FDA?
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