Hereditary Angioedema (HAE) is a rare genetic disease that results in recurrent episodes of swelling of the skin and mucous membranes, affecting approximately 1 in 50,000 people worldwide. Currently, there is no cure for HAE, and the treatments available only manage the symptoms. However, researchers are developing new drugs to improve the management of HAE. In this article, we will discuss the latest developments in Hereditary Angioedema Pipeline Drugs Market.
Hereditary angioedema (HAE) is a rare genetic disorder that causes episodes of swelling in various parts of the body. This swelling can be life-threatening if it occurs in the throat, leading to difficulty breathing. Although there is no cure for HAE, various drugs are in development that may help manage symptoms and prevent attacks. In this article, we will take a closer look at the latest developments in hereditary angioedema pipeline drugs.
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What is Hereditary Angioedema?
Hereditary angioedema (HAE) is an inherited condition that affects approximately 1 in 10,000 to 1 in 50,000 people worldwide. HAE is caused by mutations in the genes that regulate the production of a protein called C1 inhibitor. This protein helps to control a system in the body called the complement system, which plays a role in inflammation and immune responses.
In people with HAE, the levels or function of C1 inhibitor are reduced, leading to the uncontrolled activation of the complement system and the release of bradykinin, a peptide that causes blood vessels to dilate and become leaky. This results in episodes of swelling in various parts of the body, including the face, hands, feet, abdomen, and genitals.
Current Treatments for Hereditary Angioedema
There is no cure for hereditary angioedema, but various treatments are available to manage symptoms and prevent attacks. These treatments include:
- C1 inhibitor replacement therapy: This involves infusing the missing or defective C1 inhibitor protein into the bloodstream to help control the complement system and prevent swelling.
- Bradykinin receptor antagonists: These drugs block the effects of bradykinin and can help reduce swelling during an attack.
- Kallikrein inhibitors: These drugs target an enzyme called kallikrein, which plays a role in the production of bradykinin.
- Plasma-derived therapies: These therapies involve infusing plasma from healthy donors to help replace missing or defective proteins in people with HAE.
Latest Developments in Hereditary Angioedema Pipeline Drugs
Several drugs are currently in development for the treatment of hereditary angioedema. Here are some of the latest developments:
Berotralstat (BCX7353)
Berotralstat (BCX7353) is an oral small molecule drug that works by inhibiting kallikrein, an enzyme that plays a role in the production of bradykinin. In clinical trials, berotralstat has been shown to reduce the frequency of HAE attacks and improve quality of life in people with HAE. Berotralstat was approved by the FDA in December 2020 under the brand name Orladeyo.
Lanadelumab (DX-2930)
Lanadelumab (DX-2930) is a monoclonal antibody that targets plasma kallikrein, an enzyme that plays a role in the production of bradykinin. In clinical trials, lanadelumab has been shown to reduce the frequency of HAE attacks in people with HAE. Lanadelumab was approved by the FDA in August 2018 under the brand name Takhzyro.
TAKHZYRO® (lanadelumab-flyo)
TAKHZYRO® (lanadelumab-flyo) is a monoclonal antibody that targets plasma kallikrein, an enzyme that plays a role in the production of bradykinin. In clinical trials, TAKHZYRO has been shown to reduce the frequency of HAE attacks in people with HAE.
