Dubai – The Ministry of Health and Prevention in the United Arab Emirates (UAE) has approved the use of Lanadelumab Subcutaneous Injection for patients suffering from Hereditary Angioedema (HAE), a rare genetic disorder.
Lanadelumab Subcutaneous Injection received fast-track approval in the UAE as the first monoclonal antibody (mAb) treatment for HAE. The Injection is the first of its kind to be registered in the Middle East, Africa, India, Turkey, and the Commonwealth of Independent States (CIS), for the routine prevention of recurrent HAE attacks in patients aged 12 years and older. In line with the UAE health authorities’ vision to provide a comprehensive and excellent standard of care to the public and under the guidance of His Excellency Dr. Amin Hussain Al Amiri, Assistant Undersecretary of Public Health Policy and Licensing, this rare disease treatment is now registered and available, in record time.
HAE is a disorder characterized by recurrent episodes of severe swelling (angioedema). Episodes involving the intestinal tract cause severe abdominal pain, nausea, and vomiting, and swelling in the airway can restrict breathing and may be life-threatening. Symptoms typically begin in childhood and worsen during puberty. On average, untreated individuals have an attack every one to two weeks, and most episodes last for about three to four days2.
H.E. Dr. Maryam Mohamed Fatma Matar MD, Ph.D. Founder and Chairperson UAE Genetic Diseases Association (UAE GDA), said, “Timely diagnosis is the first step in the management of rare diseases and disorders. Specialized technology capabilities and tools are required to ensure proper genetic screening, with the ability to isolate and characterize specific mutations that can advise the management and subsequent treatment. Due to the acute need for individualized patient treatment for such disorders, there must be a greater investment in research to deliver accurate local data around prevalence. At the patient level, through their support groups, the UAE GDA is working with donor organizations to ensure wider accessibility of quality treatments.”
HAE is estimated to affect one in 50,000 people with no notable differences between men and women. The genetic cause of HAE in Middle Eastern Arab patients has not yet been determined.
Suleiman Al Hammadi, Professor of Pediatric Allergy and Clinical Immunology at UAE University and Consultant Pediatric Allergy and Clinical Immunology at Al Ain and Tawam Hospitals, added “Swelling of various parts of the body due to symptoms presented by HAE can progressively affect quality of life. Replacement enzymes have previously been intravenously injected two to three times a week in a hospital environment. With Lanadelumab, it is the first time that patients can administer a treatment subcutaneously at home. Safety margins of monoclonal antibody treatments are also higher. Patient groups will assist in alleviating the burden of the disease through awareness efforts.”
In a 26-week clinical study, which included 125 people with HAE, patients taking subcutaneous Lanadelumab 300 mg every two weeks had 83% fewer moderate to severe attacks, and 87% fewer attacks that needed on-demand treatment. In a post hoc analysis of the 16 weeks from Day 70 to Day 182, 77% of patients treated with Lanadelumab 300 mg every two weeks were attack-free compared to placebo Lanadelumab was generally well tolerated, with few cases of hypersensitivity.
“We are keen to support patients with the latest innovations to manage rare diseases, and the fast-tracked market authorization of the Injection by the Ministry is a testament to a national vision to support people with the best care. Our global team of researchers and scientists, together with our partners, harness cutting-edge science to push the boundaries of what is possible to bring transformative, life-changing therapies to patients worldwide. Our significant investment in research to resolve rare diseases such as HAE, which is under-recognized, under-diagnosed, and under-treated, seeks to create a better future for everyone,” Rodrigo Rodriguez, General Manager at Takeda Pharmaceuticals in the Middle East.
To promote education around HAE, an awareness website for patients, caregivers, and healthcare professionals is available at www.knowHAE.me in both English and Arabic with information on symptoms, diagnosis, and treatment options for HAE.
About Takeda Pharmaceutical Company Limited
Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people’s lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries. For more information, visit https://www.takeda.com.
 US National library of Medicine: https://ghr.nlm.nih.gov/condition/hereditary-angioedema. Last accessed May 2020
© Press Release 2020