Double thumbs up for Medivir from the FDA
Medivir’s drug candidate MIV-711 has received both Rare Pediatric Disease Designation and Orphan Drug Designation for the treatment of the rare childhood disease Legg-Calvé-Perthes disease.
– LCPD is a disease with a high risk of future complications such as osteoarthritis. MIV-711 has the potential to be the first drug to have a positive impact on disease progression, says Jens Lindberg, CEO of Medivir.
Medivir’s main focus is to position the drug candidate fostrox as the first liver-targeted, orally administered drug for the treatment of primary liver cancer, hepatocellular carcinoma (HCC).
The company has recently taken important steps forward with the candidate through a so-called Type C meeting with the US Food and Drug Administration (FDA). Read more in BioStock’s interview CEO Jens Lindberg here.
Partnership focus for MIV-711
To date, Medivir has out-licensed four clinical projects, birinapant, Xerclear, USP-1/TNG348 and MIV-701, as well as the preclinical projects USP-7 and MBLI/MET-X. The company is also actively seeking partners for two additional clinical projects: Remetinostat and MIV-711.
Positive response from the FDA
Recently, MIV-711 was granted both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) by the FDA, for the treatment of the rare childhood disease Legg-Calvé-Perthes disease (LCPD).
LCPD is a painful disease that affects about 1 in 1000 children between the ages of 3 and 12. It is characterized by an inflammation and loosening of the femoral head due to insufficient blood circulation to the hip joint. This leads to limited mobility, which often requires treatment to relieve pain and prevent future complications.
In more than half of the children, the affected leg becomes shorter than the healthy leg and about 50 per cent develop a deformed hip joint where ultimately osteoarthritis develops.
Regulatory benefits of RPDD and ODD
RPDD can be obtained from the FDA for rare pediatric (pediatric diseases) that are serious or life-threatening where there is supporting data to suggest that the drug may be effective in treating the disease. ODD, or Orphan Drug Designation, can be awarded to drug candidates that target diseases that affect fewer than 200,000 people in the United States.
By virtue of its obtained RPDD, Medivir is eligible to receive Fast Track Review and upon marketing approval in LCPD, the company may be eligible for a Priority Review Voucher from the FDA. This voucher can be redeemed to receive priority review of a subsequent marketing application for another product, or sold to another company.
ODD provides similar benefits, including market advantages, expanded exclusivity, and tax incentives, facilitating investment and promoting the development of therapies for rare pediatric diseases.
Comments from the CEO
BioStock reached out to Medivir’s CEO Jens Lindberg for a comment on the decisions from the FDA and how the company plans to take advantage of the regulatory benefits that RPDD and ODD bring.
Jens, what is the basis for you receiving Pediatric Disease Designation?
– In order for RPDD to be obtained, there must be supporting data to suggest that the drug can be effective against the disease. MIV-711 has demonstrated the ability to prevent deformity of the femoral head in an LCPD-specific animal model and has had a positive impact on the biomarker of bone degradation without adversely affecting normal bone formation. The fact that MIV-711 has also been shown to be able to prevent bone breakdown in patients with osteoarthritis is further support for the potential clinical benefit of LCDP.
MIV-711 is a so-called selective cathepsin K inhibitor. Can you tell us more about this and what significance this can play in a disease like LCPD?
– Cathepsin K is the major cysteine protease involved in bone resorption and cartilage breakdown by osteoclasts, through the breakdown of central bone matrix proteins. Cathepsin K inhibition protects the injured bone by reducing bone resorption and promoting bone formation, thereby addressing the key mechanisms that cause pathological changes in LCPD, with the potential to minimize negative long-term effects. Selective inhibition of cathepsin K has the potential to provide clinical benefit in diseases characterized by excessive bone resorption, which includes additional pediatric bone diseases.
– MIV-711 has shown positive effects on both bone and cartilage in joints in osteoarthritis patients after only six months of treatment. A completed phase II study showed that one year of treatment with MIV-711 continued treatment effect on bone and cartilage. In addition, a subgroup analysis of our phase II study with MIV-711 in osteoarthritis has shown significantly reduced osteoarthritis-related pain.
To wrap this up, you are not conducting the clinical development of MIV-711 on your own. What are your strategies for entering into licensing or collaboration agreements for continued development?
– Our ambition is to establish a partnership with a company that has greater experience in developing drugs for rare paediatric diseases. We have chosen to wait for the FDA’s decision on RPDD, partly because it has value for a potential partner, but perhaps even more so because the FDA’s approval of RPDD for MIV-711 is a validation of its medical potential in LCPD. At the same time, we have also ensured that there is an active substance of MIV-711 to initiate clinical development work. This means that we are now starting our outreach to identify the licensing partner that best takes over the baton and drives clinical development forward.
The content of BioStock’s news and analyses is independent but the work of BioStock is to a certain degree financed by life science companies. The above article concerns a company from which BioStock has received financing.
