Vaderis Therapeutics' VAD044 Receives FDA Fast Track Designation for Treating Hereditary Hemorrhagic Telangiectasia

Vaderis Therapeutics AG, a clinical-stage biotechnology company focusing on treatments for rare diseases associated with vascular malformations, today announced that the US FDA has designated the VAD044 as a Fast Track product for the treatment of Hereditary hemorrhagic telangiectasia (HHT). The FDA’s Fast Track process is designed to expedite the development and review of drugs that treat serious conditions and address unmet medical needs.

Publish Date: 18-11-2024 Source: Vaderis Therapeutics AG

Hereditary hemorrhagic telangiectasia (HHT), also known as Osler-Weber-Rendu disease, is a rare genetic disorder that causes abnormal blood vessel formation, leading to the development of arteriovenous malformations (AVMs). These AVMs are abnormal connections between arteries and veins that can form in various organs, including the lungs, liver, and brain. This can result in serious complications such as brain abscesses, stroke, and heart failure. Individuals with HHT often experience frequent and severe nosebleeds, visible blood vessels on the skin and mucous membranes, and gastrointestinal bleeding, which can lead to iron-deficiency anemia. HHT is inherited in an autosomal dominant pattern, with mutations in the ENG (endoglin) and ACVRL1 (ALK1) genes being the primary genetic causes. While there are currently no specific FDA-approved drugs for the treatment of HHT, treatment strategies focus on managing symptoms and complications, such as frequent nosebleeds and pulmonary AVMs.

However, the current Hereditary Hemorrhagic Telangiectasia (HHT) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies VAD044 has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Hereditary Hemorrhagic Telangiectasia (HHT) treatment. The key companies in the advanced development stage are Vaderis Therapeutics.

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Hereditary Hemorrhagic Telangiectasia (HHT) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2023 to 2033.

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