Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, announced it has submitted a new drug application to the FDA for volanesorsen, an investigational drug treatment for familial chylomicronemia syndrome.
“We are eager to provide these patients and their physicians with potentially the first effective tool to treat this devastating disease,” Paula Soteropoulos, president and CEO of Akcea, said in a press release. “We have successfully submitted marketing authorization applications in the U.S., the [European Union] and we are on track to submit our application for marketing authorization in Canada in September. We are also on track to launch volanesorsen globally in 2018 pending approval in the respective markets.”
Patients with familial chylomicronemia syndrome (FCS) are deficient in lipoprotein lipase, which makes them unable to effectively metabolize chylomicrons. This rare disorder is characterized by high triglyceride levels, symptoms like abdominal pain, and a risk for recurrent acute pancreatitis, which could be fatal, and no effective therapies are currently available.
“Volanesorsen is designed to reduce the production of ApoC-III, a protein produced in the liver that plays a central role in the regulation of plasma triglycerides and may also affect other metabolic parameters,” according to the press release.
“Because of drastically impaired function of lipoprotein lipase, patients with FCS have triglyceride levels that can reach 10 to 20 times that of healthy individuals,” Linda C. Hemphill, MD, of Massachusetts General Hospital and Harvard Medical School, said in the press release. “This frequently leads to recurrent episodes of acute pancreatitis, which can be fatal. Today, there is no therapy for FCS patients that can effectively reduce their triglycerides to levels that are even close to acceptable. I am encouraged that the significant reductions in triglyceride levels and reduced risk of pancreatitis in the APPROACH and COMPASS studies indicate that we may soon have an option for these patients.”
The companies are also evaluating volanesorsen for familial partial lipodystrophy (FPL), another rare genetic metabolic disorder characterized by the body’s inability to store fat in normal locations, leading to high triglyceride levels in the bloodstream, abnormal fat distribution, and metabolic abnormalities. Patients with FPL have an increased risk for acute pancreatitis, premature cardiomyopathy, atherosclerosis and liver disease.
The companies expect results from the ongoing phase 3 BROADEN study in 2019, and if these results are positive, they plan to file for marketing authorization for this additional indication.
The press release noted that the drug has received orphan drug designation from the U.S. and the EU for FCS, and from the EU for FPL.
Disclosures: Soteropoulos is employed by Akcea. Healio Gastroenterology and Liver Disease was unable to confirm Hemphill’s relevant financial disclosures.