=Akcea Therapeutics (AKCA) and Ionis (IONS) : FDA rejects rare disease drug

The FDA rejected a drug developed by Massachusetts-based biotech Akcea Therapeutics Inc. in partnership with California-based Ionis Pharmaceuticals that's intended to treat a rare lipid disorder, halting hopes for a treatment for the currently unserved, debilitating disease.


Akcea Therapeutics and Ionis (IONS) receive Complete Response Letter for WAYLIVRA from FDA  

• Akcea Tx (AKCA) an affiliate of Ionis Pharma (IONS) received a Complete Response Letter (CRL) from the Division of Metabolism and Endocrinology Products of the U.S. FDA regarding the New Drug Application (NDA) for WAYLIVRA (volanesorsen).
• "We are extremely disappointed with the FDA's decision. FCS is an ultra-rare and debilitating disease. Our disappointment extends to the patient and physician community who currently do not have a treatment available to them. We continue to feel strongly that WAYLIVRA demonstrates a favorable benefit/risk profile in people with FCS as was reflected in the positive outcome from our Advisory Committee hearing in May. We will continue to work with the FDA to confirm the path forward,"