BOSTON and CARLSBAD, Calif.,
Hereditary ATTR amyloidosis is an under-recognized, debilitating and progressive disease that is caused by the buildup of TTR proteins that misfold due to inherited mutations. hATTR is characterized by the deposition of amyloid fibrils throughout the body, including the nerves and can have a devastating impact on the patients’ quality of life (QoL). TEGSEDI is a once-weekly at-home subcutaneous injection that targets hATTR amyloidosis with polyneuropathy at its source by reducing the production of TTR protein.
“Patients with hereditary ATTR amyloidosis are progressively robbed of their ability to function, and ultimately, their lives. Even early symptoms - tingling, numbness or pain in the lower extremities – can significantly impair a person’s ability to work or engage in daily activities. If left untreated, the disease can progress rapidly,” said Dr. Thomas Brannagan, director of the Peripheral Neuropathy Center at
Of the 139 patients who completed the NEURO-TTR phase 3 study, 97% of patients elected to continue in the OLE study. Initiation of TEGSEDI, substantially reduced TTR protein levels 75% to 79% below baseline and sustained reductions throughout treatment. Patients who received TEGSEDI through the NEURO-TTR and OLE studies (104 weeks) continued to show benefit throughout, and patients who switched from placebo to TEGSEDI demonstrated an improvement in measures of neuropathy progression and QoL compared with a continued predicted worsening with placebo. Additionally, patients who initiated TEGSEDI as part of the OLE demonstrated a stabilization of neurologic disease progression by mNIS+7 and Norfolk QoL-DN within six months, while greater benefit in mNIS+7 (17.1 points) and Norfolk QoL-DN (11.9 points) was observed in patients treated earlier with TEGSEDI. No new safety signals were identified in the open-label extension study.
“For patients with hereditary ATTR amyloidosis a late diagnosis or delayed treatment can be devastating as the disease can quickly progress, leading to worsening symptoms, significant disability and a deteriorating quality of life,” said Sarah Boyce, president of Akcea Therapeutics. “TEGSEDI is an important drug for the hATTR community and these results further underscore our confidence in the mechanism of TEGSEDI targeting the disease at its source and powerfully knocking down TTR protein while improving a patient’s quality of life.”
"We believe the NEURO-TTR OLE data continue to demonstrate a favorable benefit-risk profile for TEGSEDI to treat patients with this devastating disease. Patients treated with TEGSEDI continue to experience improvements in measures of neuropathy and quality of life, regardless of when treatment is initiated but with greater effects tied to earlier treatment initiation. In the OLE study, over 40% of patients initiating treatment with TEGSEDI for the first-time experienced improvement in their quality of life and nearly 50% saw improvement in a measure of neurological disease progression," said Brett P. Monia, Ph.D., chief operating officer of
ABOUT TEGSEDI® (INOTERSEN)
TEGSEDI was approved by the
The approval is based on data from the NEURO-TTR study that was a Phase 3 randomized (2:1), double-blind, placebo-controlled, 15-month, international study in 172 patients with hATTR amyloidosis with symptoms of polyneuropathy. In NEURO-TTR, TEGSEDI demonstrated significant improvement 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 TEGSEDI experienced similar benefit regardless of subgroups such as age, sex, race, region, Neuropathy Impairment Score (NIS), Val30Met mutation status, and disease stage.
The approval 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 TEGSEDI.
For TEGSEDI’s full prescribing information, please visit www.TEGSEDI.com.
IMPORTANT SAFETY INFORMATION
TEGSEDI can cause serious side effects including:
Low platelet counts (thrombocytopenia): TEGSEDI may cause the number of platelets in your blood to be reduced. This is a common side effect of TEGSEDI. When your platelet count is too low, your body cannot form clots. You could have serious bleeding that could lead to death. Call your healthcare provider immediately if you have:
- Unusual bruising or a rash of tiny reddish-purple spots, often on the lower legs
- Bleeding from skin cuts that does not stop or oozes
- Bleeding from your gums or nose
- Blood in your urine or stools
- Bleeding into the whites of your eyes
- Sudden severe headaches or neck stiffness
- Vomiting or coughing up blood
- Abnormal or heavy periods (menstrual bleeding)
Kidney inflammation (glomerulonephritis): Your kidneys may stop working properly. Glomerulonephritis can lead to severe kidney damage and kidney failure that need dialysis. Call your healthcare provider immediately if you have:
- Puffiness or swelling in your face, feet, or hands
- New onset or worsening shortness of breath and coughing
- Blood in your urine or brown urine
- Foamy urine (proteinuria)
- Passed less urine than usual
Because of the risk of serious bleeding caused by low platelet counts and because of the risk of kidney problems, TEGSEDI is available only through a restricted program called the TEGSEDI Risk Evaluation and Mitigation Strategy (REMS) Program. Talk to your healthcare provider about how to enroll in the TEGSEDI REMS Program.
Do not use TEGSEDI if you have:
- A platelet count that is low
- Had kidney inflammation (glomerulonephritis) caused by TEGSEDI
- Had an allergic reaction to inotersen or any of the ingredients in TEGSEDI. See the end of the Medication Guide for a complete list of ingredients in TEGSEDI
Before you start TEGSEDI, tell your healthcare provider about all of your health issues, including if you:
- Have or had bleeding problems
- Have or had kidney problems
- Are pregnant or plan to become pregnant. It is not known if TEGSEDI can harm your unborn baby
- Are breastfeeding or plan to breastfeed. It is not known if TEGSEDI can pass into your breast milk or harm your baby. Talk with your healthcare provider about the best way to feed your baby while you are taking TEGSEDI
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Especially tell your healthcare provider if you take vitamin A or beta-carotene supplements, blood thinners (anticoagulants), or drugs that affect blood clotting.
Your healthcare provider will test your blood and urine to check your platelet counts and kidney and liver function before you start TEGSEDI. While you are receiving TEGSEDI, you will be monitored closely for symptoms, which includes checking your platelet counts every week (or more frequently as needed), kidney function every 2 weeks, and liver function every 4 months. If your healthcare provider has you stop taking TEGSEDI, you will need to continue to get your blood and urine tested for 8 more weeks after treatment.
TEGSEDI may cause serious side effects, including:
Stroke. TEGSEDI may cause a stroke. One person taking TEGSEDI had a stroke, which occurred within 2 days after the first dose. Get emergency help immediately if you have symptoms of stroke, including sudden numbness or weakness, especially on one side of the body; severe headache or neck pain; confusion; problems with vision, speech, or balance; droopy eyelids.
Inflammatory and immune system problems. Some people taking TEGSEDI had serious inflammatory and immune system problems. Symptoms of inflammatory and immune system problems included unexpected change in walking, weakness and spasms in legs, back pain, weight loss, headache, vomiting, and problems with speech.
Liver effects. TEGSEDI may cause liver problems. Your healthcare provider should do laboratory tests to check your liver before you start TEGSEDI and while you are using it. Tell your healthcare provider if you have symptoms that your liver may not be working right, which could include unexpected nausea and vomiting, stomach pain, being not hungry, yellowing of the skin, or having dark urine.
Allergic reactions. TEGSEDI may cause serious allergic reactions. These allergic reactions often occur within 2 hours after injecting TEGSEDI. Get emergency help immediately if you have any symptoms of a serious allergic reaction, including joint pain, chills, redness on palms of hands, muscle pain, chest pain, flushing, tremor or jerking movements, flu-like symptoms, high blood pressure, or difficulty swallowing.
Eye problems (low vitamin A levels). Treatment with TEGSEDI will lower the vitamin A levels in your blood. Your healthcare provider will tell you how much supplemental vitamin A to take every day; only take the amount they tell you to take. Call your healthcare provider if you get eye problems, such as having difficulty seeing at night or in low-lit areas (night blindness).
The most common side effects of TEGSEDI include injection site reactions (such as redness or pain at the injection site), nausea, headache, tiredness, low platelet counts (thrombocytopenia), and fever. These are not all of the possible side effects of TEGSEDI. Talk to your healthcare provider about any side affects you may be experiencing.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.
Please see Medication Guide and full Prescribing Information, including boxed WARNING.
ABOUT HEREDITARY TRANSTHYRETIN (hATTR) AMYLOIDOSIS
Hereditary ATTR 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. 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. Hereditary ATTR amyloidosis causes significant morbidity and progressive decline in quality of life, severely impacting activities of daily living. The disease often progresses rapidly and can lead to premature death. The median survival is 4.7 years following diagnosis. Additional information on Hereditary ATTR amyloidosis, including a full list of organizations supporting the hATTR amyloidosis community worldwide, is available at www.hattrchangethecourse.com or by visiting www.hATTRGuide.com.
ABOUT IONIS PHARMACEUTICALS, INC.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to treat a broad range of diseases including cardiovascular diseases, neurological diseases, infectious diseases, pulmonary diseases and cancer.
To learn more about Ionis follow us on twitter @ionispharma or visit www.ionispharma.com/.
ABOUT AKCEA THERAPEUTICS, INC.
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