Tegsedi (inotersen) to be made available on the NHS in England for people with hATTR amyloidosis

NICE issues positive recommendation for inotersen to treat adult patients in England with stage 1 or stage 2 polyneuropathy with hereditary transthyretin amyloidosis (hATTR)

LONDON, 16 April 2019 – Akcea Therapeutics UK Ltd., an affiliate of Akcea Therapeutics, Inc., announced today that the National Institute for Health and Care Excellence (NICE) has issued a positive Final Evaluation Document (FED) for Tegsedi™ (inotersen) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).[i] This decision will allow patients in England with this rare, inherited, severely debilitating and fatal disease to access the treatment on the NHS.

“This is a landmark day for people with hATTR amyloidosis who have had very limited options available to them to date,” commented Carlos Heras-Palou of the UK ATTR Amyloidosis Patients Association. “There is a critical need for innovative new therapies for people across the UK living with this debilitating disease. We hope inotersen will be available to patients in the UK very soon.”

hATTR amyloidosis is a severe, progressive, and life-threatening disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, the heart and intestinal tract.[ii] The progressive accumulation of TTR amyloid deposits in these organs often leads to intractable peripheral sensorimotor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations.2 hATTR amyloidosis causes significant morbidity and progressive decline in quality of life, severely impacting activities of daily living.2 The disease often progresses rapidly and can lead to premature death.2 The median survival is 4.7 years from diagnosis.[iii]

Inotersen is an antisense oligonucleotide (ASO) inhibitor of human transthyretin (TTR) production. It is the first and only subcutaneous RNA-targeting drug designed to reduce the production of human transthyretin (TTR) protein.[iv]

By publishing a FED, NICE has made the final recommendations on how inotersen should be used in the NHS. The NICE decision was based on clinical trial evidence that shows inotersen slows progression of the disease considerably, although its long-term benefits are uncertain. 1 The recommendation in the FED is expected to form the basis for NICE’s final Technology Appraisal Guidance (TAG), the final process step before the treatment is available on the NHS in England. Once the final guidance is published, the NHS mandate requires that inotersen is available for routine use within 90 days.

“We are delighted with this news that patients can access inotersen in England,” commented Dr. Richard A. Jones, SVP Head of Europe for Akcea Therapeutics. “hATTR amyloidosis is a debilitating disease that, to date, has had limited treatment options in the UK. We hope that other health technology assessment and reimbursement agencies across Europe will take NICE’s lead in making inotersen available as a treatment option for patients with this disease. This positive news further confirms Akcea’s commitment to advancing and making accessible transformative treatments for patients living with serious and rare diseases.”

The decision is based on evidence from the NEURO-TTR trial, a Phase 3 randomised placebo-controlled study evaluating inotersen compared to placebo. It demonstrated that patients taking inotersen had an improved course of neurological disease over the 15-month study period confirmed by the significant change from baseline compared to placebo in measures of neuropathy and quality of life as measured by the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) which were the two primary endpoints of the study.4,[v]Inotersen is associated with risk of thrombocytopenia and glomerulonephritis. Enhanced monitoring is required to support early detection and management of these identified safety risks.4,5

For important safety information for inotersen, including method of administration, special warnings, drug interactions and adverse drug reactions, please see the Summary of Product Characteristics (SmPC), available fromwww.medicines.org.uk/emc/product/10011/smpc.

 This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you may get. Reporting forms and information can be found at www.mhra.gov.uk/yellowcard. Alternatively, search for MHRA Yellow Card in the Google Play or Apple App Store, or contact the MHRA atyellowcard@mhra.gov.uk or 0800 731 6789 (10am to 2pm Monday to Friday only).

-ENDS-

ABOUT INOTERSEN

Inotersen is authorised in the EU for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).4 Inotersen, discovered and developed by Ionis Pharmaceuticals, is the world’s first and only subcutaneous RNA-targeting drug designed to reduce the production of human transthyretin (TTR) protein.4

The EMA marketing authorisation is based on data from the NEURO-TTR study that was a Phase 3 randomised (2:1), double-blind, placebo-controlled, 15-month, international study in 172 patients with hATTR amyloidosis with symptoms of polyneuropathy.4,5 In NEURO-TTR, inotersen demonstrated significant benefit compared to placebo in measures of neuropathy and quality of life as measured by the modified Neuropathy Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN) total score. Patients treated with inotersen experienced similar benefit regardless of baseline characteristics such as age, sex, race, region, Neuropathy Impairment Score (NIS), Val30Met mutation status, and disease stage.4,5

The marketing authorisation is also based on data from the NEURO-TTR Open Label Extension (OLE) that is an ongoing study for patients who completed the NEURO-TTR study, designed to evaluate the long-term efficacy and safety of inotersen.4,[vi]

ABOUT HEREDITARY TRANSTHYRETIN (hATTR) AMYLOIDOSIS

hATTR amyloidosis is a severe, progressive, and life-threatening disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, the heart and intestinal tract.2 The progressive accumulation of TTR amyloid deposits in these organs often leads to intractable peripheral sensorimotor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations.2 hATTR amyloidosis causes significant morbidity and progressive decline in quality of life, severely impacting activities of daily living.2 The disease often progresses rapidly and can lead to premature death.2 The median survival is 4.7 years following diagnosis.3

 

ABOUT AKCEA THERAPEUTICS

Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc., is a biopharmaceutical company focused on developing and commercialising drugs to treat patients with serious and rare diseases. Akcea is commercialising TegsediTM (inotersen) and advancing a mature pipeline of novel drugs, including Waylivra™ (volanesorsen), AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx, AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, all with the potential to treat multiple diseases. All six drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis’ proprietary antisense technology. Inotersen is approved in the U.S., E.U. and Canada. Volanesorsen is under regulatory review for the treatment of familial chylomicronemia syndrome, or FCS, and is currently in Phase 3 clinical development for the treatment of people with familial partial lipodystrophy, or FPL. Akcea is building the infrastructure to commercialise its drugs globally. Akcea is a global company headquartered in Boston, Massachusetts.

 

AKCEA’S FORWARD-LOOKING STATEMENT

This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. and the therapeutic and commercial potential of TegsediTM (inotersen). Any statement describing Akcea’s goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Akcea’s forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Akcea’s forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Akcea's programs are described in additional detail in Akcea's annual report on Form 10-K, and its most recent quarterly report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from the Company.

In this press release, unless the context requires otherwise, “Ionis”, “Akcea,” “Company,” “Companies,” “we,” “our,” and “us” refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.

Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutic® TegsediTM and Waylivra® are trademarks of Akcea Therapeutics, Inc.

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