The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has approved ACH-4471 for orphan status. According to Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN), the oral inhibitor of complement factor D can now be used as a treatment for C3 glomerulopathy (C3G). The biopharmaceutical company has been working towards enhancing the awareness of C3G, which is a rare renal disorder and apparently it has no approved therapies.
Records have it that close to 4,000 people in Europe are suffering from C3G, which consists of dense deposit disease (C3G) and C3 glomerulonephritis (C3GN). However Chief Executive Officer of Achillion, Milind Deshpande says that the issuance of the positive orphan status to the treatment will go a long way in building the awareness.
The mechanism of action by EMA is a novel approach in treating C3G
C3G is a devastating illness, which affects the kidney. However, together with his team, Dr. Deshpande says, “The mechanism of action for our factor D inhibitors……………………we believe we are targeting the root cause of disease, which experts attribute to overactivation of the alternative pathway.”
There is also hope that ACH-4471 will help in reducing the unnecessary formation of C3 fragments in the blood. On the other hand, orphan drug designation by the EC is expected to open and develop a market for therapies, which will help in treating life-threatening or chronically debilitating conditions. Companies seeking protocol assistance from the EMA will also get an opportunity once the orphan drug designation has been approved.
Achillion is rapidly advancing its efforts to bring potentially life-saving medicines to patients
Achillion seems to be moving swiftly towards safeguarding the lives of millions of patients living with rare diseases. Alongside C3G, the company will be launching a new patient support initiative program and websites, which will engage patients and caregivers with resources and educational materials. Deshpande confirms that they are also proud of being able to support a global natural history study of C3G to track its course.
The company has proven beyond doubts that it is committed to innovative research its aim being addressing unmet medical requirements of patients living with C3G.