On this rare disease day, we thought It was time to reflect on some of the European Commission’s (EC) success stories from the framework programme.
Ceratium have been very active in targeting rare diseases. Working with a range of partners on multiple projects utilising EC funding to help advance the development of many orphan drugs and medicinal products. Rare disease continues to be a priority area for EC. Research funding. There has been major investment of over €2.4 billion made in FP7 and Horizon 2020 to fund more than 400 research and innovation projects in the area of rare disease, new funding is becoming available from Horizon Europe.
TIGER is the latest project in Ceratium’s rare disease portfolio, the aim is to advance the development of an intravenous (IV) platform technology that uses proprietary lipid nanoparticles (LNP) for a new class of mRNA-therapeutics with the potential to revolutionise care for patients with hard-to-treat cancers. The project builds on the current revolution in mRNA-based technologies that offer unparalleled opportunities for developing new therapeutics targeting cancers with high unmet clinical needs. Almost 5% of global cancers are caused by HPV, our targets include the rare cancers anal, vaginal and vulvar, in addition to some more common HPV+ cancers. Virus-associated cancers such as HPV are ideal candidates for immunotherapy because of the consistent presence of foreign antigens in comparison to non-viral cancers. Although the TIGER project is not a dedicated rare disease programme, there are a number of rare disease cancers involved that will be targeted by the novel immunotherapies. This has the potential to benefit the rare cancer community.
The EC funding really contributed to the success of the project for VISION-DMD, and the funding in the ODAK project helped with the late stage pre-clinical and early clinical development study.
The VISION-DMD project, has supported the clinical development of the orphan drug Vamorolone as a new therapy to revolutionise care for all patients with Duchenne muscular dystrophy (DMD). Following the success of the clinical trials, Vamorolone is being used by patients under expanded access and compassionate use in 11 countries worldwide. A new drug application (NDA) is currently under review by the FDA and subject to approval santhera plans to launch Vamorolone in the US in Q4 -2023 for the treatment of DMD. In Europe, the MMA has been validated and is under review by the European Medicines Agency (EMA).
The ODAK project undertook the pharmaceutical development of an orphan drug for the rare infectious eye disease Acanthamoeba Keratitis (AK). Results from the project have led the ophthalmic company SIFI, to potentially develop the first medicinal product to be approved for AK and have already launched their early access programme across EU countries in June 2022. A licence agreement with AVANZANITE BIOSCIENCE for AKANTIOR ® to commercialise and distribute in 26 countries within the European Economic Area and Switzerland is now in place. SIFI expects a potential positive opinion of the EMA Committee for Medicinal Products for Human Use in 2023.
