Irish specialty drug developer Inflazome has secured orphan drug designation for Inzomelid, a therapy that it is developing.
Inflazome specialises in seeking therapies for rare inflammatory conditions. Specifically, it has focused on a protein complex called the NLRP3 inflammasome, which regulates the body's immune response. If overactive, it is implicated in a wide range of serious medical conditions, including Alzheimer's and Parkinson's.
In this case, Inzomelid is being tested as a treatment for a group of three related inflammatory conditions called Cryopyrin-Associated Periodic Syndromes (Caps), which can affect the skin, joints, the central nervous system and the eyes.
First phase
In March, the Dublin-based company announced positive first phase clinical trial results for the drug. The trial also showed that it was safe and well tolerated in healthy subjects.
It is planning a Phase II trial later this year to develop the dose for Caps patients.
The company was founded by Trinity immunologist Luke O'Neill and Matt Cooper, a University of Queensland chemist who has worked extensively in the areas of inflammation and superbugs.
Orphan drug designation makes it easier for medicines to gain approval to get to market and also extends their patent life. It is granted only to drugs treating such a small patient population that they will never make a profit without government support.
