Takeda biopharmaceuticals launches new injectable drug for rare genetic disorder in India

Hereditary Angioedema (HAE) is a rare genetic condition that causes swelling in different parts of the body like limbs, face, abdomen, and larynx.

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Takeda Biopharmaceuticals on Wednesday announced that it has launched an innovative injectable prescription drug for the treatment of hereditary angioedema (HAE) patients.

The company claims that the drug, CINRYZE, has the potential to mark a breakthrough in the episodic treatment of short and long-term prophylaxis for HAE.

Hereditary Angioedema (HAE) is a rare genetic disorder characterised by recurrent episodes of severe swelling (angioedema). The swelling usually occurs on the limbs, face, intestinal tract, and airway.

With eight years of global clinical experience proving efficacy and safety, CINRYZE is the pioneer C1 esterase inhibitor (C1-I NH) approved by the USFDA and EMA for the symptomatic management of HAE and for preventing future angioedema attacks.

“CINRYZE is the first plasma-derived C1-INH approved for prophylaxis in hereditary angioedema patients in India. Along with prophylaxis, it has also been approved for pre-procedure prevention and acute attacks of HAE. Cinryze is approved for the treatment of angioedema attacks in children above 2 years of age, adolescents and adults,” Dr. Sandeep Arora, Head of Medical Affairs and Patient Services, Takeda Biopharmaceuticals India Private Limited told Financial Express.com.

Currently, in India, there are no other C1-INH treatments available for prophylaxis or for the treatment of patients in 2-6 years age group, Dr. Arora said.

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The drug is indicated for routine prevention (prophylaxis) of angioedema attacks in adults, adolescents, and children 6 years of age and above with HAE. Moreover, the treatment of angioedema attacks and pre-procedure prevention of angioedema attacks in adults, adolescents, and children 2 years of age and above with HAE.

“At Takeda, we are committed to bringing innovative treatment to fulfill 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,” Serina Fischer, General Manager, Takeda Biopharmaceuticals India Private Limited said in a statement.

According to Sony Paul, Franchise Head, Rare Diseases, Takeda Biopharmaceuticals India Private Limited, these patients are suffering due to the lack of diagnosis and treatment.

“The disease is grossly under-recognized in India. As per our estimate, there are more than 30,000 undiagnosed patients which is a significant burden. With the launch of CINRYZE, we want to make treatment accessible to patients who are living with HAE,” Paul told Financial Express.com.

Hereditary Angioedema (HAE) is a rare genetic condition that causes swelling in different parts of the body like limbs, face, abdomen, and larynx.

“The two most common variants of HAE, type I and type II, are due to either deficiency or dysfunction of C1-INH, respectively. In clinical studies, the intravenous administration of CINRYZE resulted in a significant increase in systemic levels of C1 inhibitor within 1 hour after administration and temporarily restores the natural regulation of the contact, complement, and fibrinolytic systems thereby controlling the swelling or the propensity to swell,” Dr. Arora told Financial Express.com.

Moreover, symptoms of HAE often present in childhood, and while attacks can occur at any age, early onset may predict a more severe disease course.

According to experts, attacks often occur in children without a clear trigger and may affect a child’s participation in school, activities, and sports, which can leave them feeling socially isolated.

Moreover, it can be life-threatening in severe cases in which the swelling attacks can manifest in the larynx (voice box), or trachea (windpipe). Due to the lack of awareness among healthcare professionals, HAE is highly underdiagnosed in the country.

On the price of the drug and its affordability status as compared to other market players, Paul said: “We are launching CINRYZE at competitive pricing for Indian patients. We follow a fair pricing strategy and are launching Cinryze in line with the strategy.”

The company (formerly known as Baxalta Bioscience India Private Limited) focuses on immunology, genetic diseases, haematology, oncology, and gastrointestinal portfolios in the country. 

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This article was first uploaded on December fourteen, twenty twenty-two, at one minutes past eleven in the morning.
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