Akcea Therapeutics Inc. (NASDAQ: AKCA) and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) were each crushed on Tuesday after each announced that it received a Complete Response Letter (CRL) from U.S. Food and Drug Administration (FDA).
Specifically, this letter comes from the FDA’s Division of Metabolism and Endocrinology Products in regards to the New Drug Application (NDA) for Waylivra (volanesorsen).
For some quick background: Waylivra is also under regulatory review in the European Union and Canada for the treatment of people with familial chylomicronemia syndrome (FCS).
FCS is an ultra-rare, devastating hereditary disease that causes unpredictable and potentially fatal acute pancreatitis, as well as chronic complications due to permanent organ damage, and has a severe impact on daily living.
Paula Soteropoulos, CEO of Akcea, commented:
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.
Shares of Akcea were last seen down about 26% at $24.48, with a consensus analyst price target of $39.00 and a 52-week trading range of $15.20 to $40.75.
Ionis shares were down 15% at $45.65. The consensus price target is $58.64 and the 52-week range is $39.07 to $65.51.