Ionis Pharmaceuticals (IONS) and Akcea Therapeutics (AKCA), an affiliate of Ionis Pharmaceuticals, announced that the Advisory Committee of the U.S. Food and Drug Administration’s (FDA) Division of Metabolism and Endocrinology Products voted 12-8 in support of approving the firms’ product WAYLIVRATM (volanesorsen) for familial chylomicronemia syndrome (FCS).
The PDUFA date for the FDA review and ruling is August 30, 2018.
WAYLIVRA, potentially the first and only treatment for people living with this serious and potentially fatal disease.
The Advisory Committee decision is based on data from two Phase 3 clinical trials, APPROACH and COMPASS, as well as the ongoing APPROACH Open Label study for WAYLIVRA.
Results from the Phase 3 APPROACH trial demonstrate that patients with FCS treated with WAYLIVRA achieved a statistically significant mean reduction in triglycerides of 77% from baseline and decreased the risk of pancreatitis.
According to the firms’ press release, the most common adverse events were injection site reactions and platelet declines.
Brett P. Monia, the chief operating officer at Ionis said, “People with FCS have severely elevated triglycerides, which lead to multiple severe daily and chronic symptoms, such as abdominal pain and increased risk for pancreatitis, which can be fatal. WAYLIVRA is the first drug to demonstrate substantial triglyceride lowering in clinical trials in people with FCS. WAYLIVRA illustrates how our antisense technology can create targeted drugs for people living with severe diseases who currently have no available therapeutic options.”
WAYLIVRA emanates from Ionis’ proprietary antisense technology. It is under regulatory review in the U.S., EU, and Canada for familial chylomicronemia syndrome (FCS). The drug reduces the production of ApoC-III, a liver-produced protein that plays a central role in the regulation of plasma triglycerides and may also affect other metabolic pathways. WAYLIVRA is in Phase 3 trial for familial partial lipodystrophy (FPL).
The U.S. and EU regulatory agencies have granted Orphan Drug Designation to WAYLIVRA for the treatment of FCS. If approved, WAYLIVRA would be the first and only therapy indicated for people with FCS.
Chylomicronemia syndrome or FCS is an ultra-rare disease caused by malfunction of the enzyme lipoprotein lipase (LPL). The disease is characterized by severe elevation of triglycerides (>880mg/dL) and by the risk of unpredictable and potentially fatal acute pancreatitis. The limited LPL function makes people with FCS unable to breakdown chylomicrons, which are lipoprotein particles that are 90% triglycerides. In addition to pancreatitis, FCS patients can also experience daily abdominal pain, generalized fatigue, in addition to major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog.
Using its proprietary antisense technology, Ionis created a large pipeline with over 40 drugs in development. SPINRAZA® (nusinersen) has been approved in global markets for the treatment of spinal muscular atrophy (SMA). Another advanced product, TEGSEDITM (inotersen), is under regulatory review for marketing approval in the U.S., EU, and Canada for the treatment of patients with hereditary ATTR amyloidosis, or hATTR.
Akcea Therapeutics is an affiliate of Ionis Pharmaceuticals. The firm’s pipeline has six novel drugs, discovered and being co-developed with Ionis. The drugs are based on Ionis’ proprietary antisense technology. Akcea is building the infrastructure to commercialize its drugs globally. The global firm is headquartered in Cambridge, Massachusetts.
Antisense technology is alive and is producing products were diseases are caused by genetic anomalies that lead to the production of pathological proteins, or to the lack of the good ones. Treating gene-derived diseases cannot possibly exist with other than antisense, genetic engineering and gene editing techniques.
The good news from Akcea is good news for its mother Ionis too. The competition by other gene therapy and gene editing techniques will not demean the value of the antisense technology or the antisense products at any time in the near future. The novel gene therapy and gene editing approaches will become more challenging in the future when there will be more and more gene therapy products and gene editing created therapies in clinical trials competing with the antisense approach and the antisense numerous products in clinical trials.
As a result of the good news announced about the FDA committee’s voting in favor of Akcea and Ionis product WAYLIVRA for the treatment of FCA, Akcea stock ACKA and Ionis stock IONS rallied in premarket hours, AKCA was trading at $23.85 UP over $3.40 and IONS was trading at $46.49 UP $0.54.
This is long-term Good news for the patients and the companies that created and developed the product regardless of whether investors would take advantage to continue buying or taking profit by selling.
As a matter of fact, after its premarketing early gains, in today’s early marketing, AKCA is trading at $23.20 DOWN $059. Ions stock is trading at $46.62 UP $0.6.
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