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Akcea & Ionis Announce European Union Drug Approval for Ultra-Rare Disease

Akcea Therapeutics, Inc., an affiliate of Ionis Prescription drugs, Inc., and Ionis Prescription drugs, Inc. just lately introduced that WAYLIVRA has acquired conditional advertising authorization from the European Fee (EC) as an adjunct to food plan in adult patients with genetically confirmed FCS and at excessive danger for pancreatitis, in whom response to eating regimen and triglyceride-lowering remedy has been inadequate. This authorization follows the constructive opinion recommending approval offered by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). As a part of the conditional advertising authorization, Akcea and Ionis will conduct a non-interventional post-authorization safety research (PASS) based mostly on a Registry.

“WAYLIVRA is the only approved treatment for people with FCS and is a major milestone for the global FCS community. This also marks Akcea’s second drug approval in the last year. We are very grateful to all the patients and physicians around the world who participated in our clinical trials. Their insights about the challenges of this devastating disease, and their stories of how they have benefited from WAYLIVRA continue to motivate us,” stated Paula Soteropoulos, Chief Government Officer of Akcea Therapeutics. “We plan to launch WAYLIVRA in Germany this year followed by additional European countries in 2020. Our team is ready to deliver this treatment option to patients with FCS.”

FCS is an ultra-rare debilitating illness that can be life-altering. It’s brought on by impaired perform of the enzyme, lipoprotein lipase (LPL), which leads to vital danger and disease burden, including unpredictable and probably deadly acute pancreatitis as well as persistent problems resulting from permanent organ injury. It’s estimated that there are between 3,000 to 5,000 individuals dwelling with FCS worldwide, with approximately 1,000 individuals dwelling with FCS in Europe.

“WAYLIVRA is the only treatment available for patients with FCS, which makes this approval a landmark event for the global FCS community. Patients, their caretakers, and their families have been suffering without any therapeutic option. Now patients across Europe can access a medicine that may help address their severely elevated triglycerides, which can give them hope for better health. High triglycerides can lead to a multitude of severe and daily chronic symptoms, such as abdominal pain and increased risk of pancreatitis, which have a significant daily impact on people living with FCS,” added Jules Payne, Chief Government at HEART UK and Chair of FH Europe.

WAYLIVRA is an antisense oligonucleotide drug designed by Ionis and co-developed by Akcea and Ionis to scale back the production of ApoC-III, a protein that regulates plasma triglycerides. WAYLIVRA is a self-administered, subcutaneous injection in a single-use, prefilled syringe.

“The approval of WAYLIVRA is truly an important moment for people affected by FCS and for all of us in medicine who specialize in treating lipid disorders,” stated Dr. Eric Bruckert, Head of the Endocrinology and Prevention of Cardiovascular Disease department in Pitié-Salpêtrière Hospital in Paris, France. “Many clinicians are pleased to now have a treatment option available because of the significant burden of FCS on patients and their families.”

“WAYLIVRA is Ionis’ third medicine approved in just over two years. Our highly efficient and productive antisense technology has the potential to treat the untreatable by precisely targeting the root cause of disease. FCS is an example of a disease that our novel technology is ideally suited to address,” stated Brett P. Monia, Chief Working Officer at Ionis. “We are confident that our affiliate, Akcea, has built an expert team that is committed and ready to deliver WAYLIVRA to patients as soon as possible.”

The EC’s advertising authorization of WAYLIVRA is predicated on outcomes from the Part three APPROACH research and the continued APPROACH Open Label Extension research and is supported by outcomes from the Part three COMPASS research. Results from the APPROACH trial, the most important research ever carried out in sufferers with FCS, show that compared to placebo remedy with WAYLIVRA delivered clinically and statistically meaningful discount in triglycerides over the research period. An evaluation of sufferers with a history of recurrent pancreatitis occasions (≥ 2 events in the five years previous to Research Day 1) showed a big discount in pancreatitis attacks in WAYLIVRA-treated sufferers in comparison with placebo treated sufferers. The most typical hostile occasions within the APPROACH research have been injection website reactions and reductions in platelet ranges. Along with the open label extension research, there are also ongoing international Early Access Packages for WAYLIVRA.

WAYLIVRA is the only remedy indicated for individuals with familial chylomicronemia syndrome (FCS). Akcea is working to verify a path forward for WAYLIVRA within the U.S. and Canada. WAYLIVRA, a product of Ionis’ proprietary antisense know-how, is designed to scale back the production of ApoC-III, a protein that regulates plasma triglycerides and may affect different metabolic parameters.

The European Fee’s advertising authorization of WAYLIVRA is predicated on outcomes from the Part 3 APPROACH research and the continued APPROACH Open Label Extension (OLE) research and is supported by results from the Part 3 COMPASS research. Outcomes from the Part three APPROACH trial, the most important research ever carried out in sufferers with FCS, show that compared to placebo, remedy with WAYLIVRA decreased triglycerides 77% (-94% when in comparison with placebo). All sufferers within the trial maintained a low-fat eating regimen.

WAYLIVRA is related to danger of thrombocytopenia. Enhanced monitoring is required to help early detection and administration of thrombocytopenia. Probably the most incessantly noticed hostile reactions (greater than 10%) during remedy with WAYLIVRA have been occasions related to injection website reactions and discount in platelet ranges/thrombocytopenia.

The WAYLIVRA Early Entry Program (EAP) has been initiated in Europe, the U.S. and Canada and is at present enrolling eligible sufferers. Click on right here for more info on the WAYLIVRA EAP. For more info on WAYLIVRA, please visit www.WAYLIVRA.eu.

WAYLIVRA can also be presently in Part 3 medical improvement for the remedy of patients with familial partial lipodystrophy, or FPL. Akcea anticipates reporting top-line knowledge from this research in mid-2019.

FCS is an ultra-rare illness brought on by impaired perform of the enzyme lipoprotein lipase (LPL) and characterized by extreme hypertriglyceridemia (>880mg/dL or 10mmol/L) and a danger of unpredictable and probably fatal acute pancreatitis. Because of restricted LPL perform, individuals with FCS can’t breakdown chylomicrons, lipoprotein particles which might be 90% triglycerides. In addition to pancreatitis, FCS sufferers are liable to persistent problems as a result of everlasting organ injury, including persistent pancreatitis and pancreatogenic diabetes. They will expertise every day signs including stomach pain, generalized fatigue and impaired cognitions that have an effect on their means to work. Individuals with FCS additionally report major emotional and psychosocial effects together with nervousness, social withdrawal, melancholy and mind fog. Further info on FCS is accessible at www.fcsfocus.com, via the LPLD Alliance at www.lpldalliance.org and thru The FCS Basis at http://www.livingwithfcs.org. For a full listing of organizations supporting the FCS group worldwide, please click right here.

Because the chief in RNA-targeted drug discovery and improvement, Ionis has created an environment friendly, broadly applicable, drug discovery platform referred to as antisense know-how that can treat illnesses the place no other therapeutic approaches have confirmed efficient. Our drug discovery platform has served as a springboard for actionable promise and realized hope for sufferers with unmet needs. We created the first and only permitted remedy for youngsters and adults with spinal muscular atrophy as well as the world’s first RNA-targeted therapeutic authorised for the remedy of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all of the sufferers we have now but to succeed in with a pipeline of more than 40 novel medicines designed to deal with a broad range of illnesses together with cardiovascular illnesses, neurological illnesses, infectious illnesses, pulmonary illnesses and most cancers. For more info, visit www.ionispharma.com.

Akcea Therapeutics, Inc., an affiliate of Ionis Prescription drugs, Inc., is a biopharmaceutical firm targeted on creating and commercializing medicine to treat patients with critical and uncommon illnesses. Akcea is commercializing TEGSEDI (inotersen) and advancing a mature pipeline of novel medicine, including WAYLIVRA (volanesorsen), AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, with the potential to treat multiple illnesses. All six medicine have been discovered by and are being co-developed with Ionis, a pacesetter in antisense therapeutics, and are based mostly on Ionis’ proprietary antisense know-how. TEGSEDI is accredited in the US, EU, and Canada. WAYLIVRA is accredited in the E.U. and is at present in Part three medical improvement for the remedy of individuals with familial partial lipodystrophy, or FPL. Akcea is constructing the infrastructure to commercialize its medicine globally. Akcea is a worldwide company headquartered in Boston, MA. For more info, visit www.akceatx.com.