Takeda Launches CINRYZE In India, The First C1-I NH For prophylaxis In hereditary Angioedema Patients

·         CINRYZE™ is plasma-derived C1-I NH approved for routine prevention (prophylaxis), short-term prevention or pre-procedure prevention, and acute attacks of HAE.

·         Among adult HAE patients, 51% miss at least one day of work (mean 3.3 days), 44% students miss at least one day of school (mean 1.9 days) and 59% miss at least one day of leisure activities (mean 2.7 days) as result of their most recent HAE attacks.

·         With strong clinical evidence, CINRYZE™ is proven to reduce the frequency and severity of attacks with fixed dosing regimen.

Takeda Biopharmaceuticals India Private Limited (formerly known as Baxalta Bioscience India Pvt. Ltd.), a global values-based, R&D-driven biopharmaceutical leader launched CINRYZE™ an innovative injectable prescription medicine for the treatment of hereditary angioedema (HAE) patients. With eight years of global clinical experience proving efficacy and safety, CINRYZE™ has the potential to mark a breakthrough in the episodic treatment, short and long-term prophylaxis for HAE. Moreover, CINRYZE™ is the pioneer C1 esterase inhibitor (C1-I NH) approved by the FDA & EMA for the symptomatic management of HAE and for preventing future angioedema attacks.

CINRYZE™ is indicated in India for:

a.       Routine prevention (prophylaxis) of angioedema attacks in adults, adolescents and children 6 years of age and above with HAE[3].

b.       Treatment of angioedema attacks and pre-procedure prevention of angioedema attacks in adults, adolescents and children 2 years of age and above with HAE.

Speaking at the launch, Serina Fischer, General Manager, Takeda Biopharmaceuticals India Private Limited said, “At Takeda, we are committed to bringing innovative treatment to fulfil the unmet medical needs across our core therapy areas. We are confident that the launch of Cinryze will bridge the gap in the treatment of HAE patients in India. The launch furthers our commitment towards rare diseases patients in India.”

Sony Paul, Franchise Head, Rare Diseases, Takeda Biopharmaceuticals India Private Limited added, “As per data, there are likely to be more than 30,000[4] undiagnosed patients at present in the country. These patients are suffering due to the lack of diagnosis and treatment. With the launch of Cinryze, our aim is to treat acute attacks of HAE and prevent future HAE attacks by prophylactic regime   improving the quality of life of HAE patients.”

Economic costs and impact on QoL with HAE 

• HAE is associated with impaired emotional processes (alexithymia) and increased stress among paediatric patients [9]

• Adults with HAE were more likely to exhibit depressive symptoms than adults in the general population [1] 

• Sick days due to HAE and the need to work reduced hours because of HAE were estimated to cost $6,045 per patient per year and accounted for 11.5% of all socioeconomic costs as estimated by a study conducted in the United States [10]

Hereditary Angioedema (HAE) is a rare genetic condition that causes swelling in different parts of the body like limbs, face, abdomen, and larynx [5,6]. HAE is caused by a mutation in the gene which produce protein called the C1 esterase inhibitor leading to its reduced level or compromised functioning. [6] Symptoms of HAE often present in childhood, and while attacks can occur at any age, early onset may predict a more severe disease course [6]. Attacks often occur in children without a clear trigger,[5] and may affect a child’s participation in school, activities, and sports, which can leave them feeling socially isolated [7,8]. It can be life-threatening in severe cases in which the swelling attacks can manifest in the larynx (voice box), or trachea (windpipe) [5,6].

The disease is usually identified by recurrent episodes of fluid accumulation outside the blood vessels, causing rapid swelling of body tissues. The symptoms of HAE could be like an allergic reaction; however, the potential outcome could be life-threatening in case of HAE. Due to the lack of awareness among healthcare professionals, HAE is highly underdiagnosed in the country.

Takeda Biopharmaceuticals India Private Limited (formerly known as Baxalta Bioscience India Private Limited) is a part of Takeda Pharmaceutical Company Limited's group of companies headquartered in Japan. The company focuses on immunology, genetic diseases, haematology, oncology and gastrointestinal portfolios in the country. As a global leader in pharmaceuticals, Takeda has been focusing on driving comprehensive research to support the development of effective and innovative treatments in the company’s chosen therapy areas.

HAE, hereditary angioedema; QoL, Quality of life; FDA, Food and Drug Administration; EMA, European Medicines Agency

References:

1.       Lumry WR, Castaldo AJ, Vernon MK, et al. The humanistic burden of hereditary angioedema: Impact on health-related quality of life, productivity, and depression. Allergy Asthma Proc. 2010;31:407-14.

2.       Cinryze®. USFDA PI. March 2022. Cinryze®. SmPC EMA. October 2020

3.       CINRYZE®. Indian PI. Feb 2022.

4.       Jindal AK, Bishnoi A, Dogra S. Hereditary Angioedema: Diagnostic Algorithm and Current Treatment Concepts. Indian Dermatology Online Journal. 2021;12(6): 796-804. https://doi.org/10.4103/idoj.idoj_398_21

5.       Cicardi M, Bork K, Caballero T, et al. Evidence-based recommendations for the therapeutic management of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an International Working Group. Allergy. 2012; 67(2):147-157.

6.       Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.

7.      Read N, Lim E, Tarzi MD, et al. Patient hereditary angioedema: a survey of UK service provision and patient experience. Clin Exp Immunol 2014; 178:473-88.

8.       Mendivil J, Murphy R, Cruz Mdl, et al. Clinical characteristics and burden of illness in patients with hereditary angioedema: findings from a multinational patient survey. Orphanet Journal of Rare Diseases. 2021 (16).

9.       Savarese L, Bora M, Falco RD, et al. Emotional processes and stress in children affected by hereditary angioedema with C1-inhibitor deficiency: a multicenter, prospective study Orphanet J Rare Dis. 2018;13:115;

10.    Castaldo AJ, Jervelund C, Corcoran D, et al. Assessing the cost and quality-of-life impact of on-demand–only medications for adults with hereditary angioedema. Allergy Asthma Proc. 2021;42:108-17.