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The EC Broadens Takhzyro’s Label to Encompass for Hereditary Angioedema Prevention

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Takeda’s Takhzyro (lanadelumab) has received expanded approval from the European Commission, allowing its utilization in pediatric patients aged two years and older for the routine prevention of recurrent hereditary angioedema (HAE) attacks. This label extension builds upon the previous clearance granted for Takhzyro’s use in patients aged 12 years and older. Takhzyro, delivered via subcutaneous therapy every 15 days, has demonstrated noteworthy efficacy in curtailing the frequency of HAE attacks.

Hereditary angioedema is an uncommon genetic disorder characterized by recurrent episodes of tissue swelling, resulting from the accumulation of fluid outside blood vessels. It leads to the obstruction of normal blood and lymphatic fluid flow, causing rapid tissue swelling in various parts of the body. According to the US National Organization for Rare Disorders (NORD), approximately one in 50,000 to 150,000 individuals are affected by HAE. Notably, Takhzyro stands as the sole approved prophylactic treatment for preventing HAE attacks in pediatric patients aged two to 12 years.

Takhzyro operates as a monoclonal antibody that selectively binds to plasma kallikrein, a key factor in both classical and alternative complement pathways. By reducing its plasma concentration, the drug effectively prevents angioedema attacks. In line with GlobalData analysis, Takhzyro is anticipated to generate global sales of $1.85 billion by 2029. GlobalData is the parent company of Pharmaceutical Technology.

Hereditary Angioedema

Impressive Results from Phase III Trial in Hereditary Angioedema Treatments

The approval in Europe was founded on results from an open-label Phase III clinical trial (NCT04070326) involving 21 participants aged two to 12 years with HAE. The trial demonstrated that Takhzyro decreased the rate of HAE attacks by an impressive 94.8% compared to baseline, with a reduction from 1.84 attacks per month to just 0.08 attacks during treatment, as disclosed in a press release on June 30, 2022. Over 52 weeks, 76.2% of participants remained attack-free, with an average of 99.5% attack-free days. Importantly, there were no reported deaths or serious treatment-emergent adverse events (TEAEs) throughout the clinical trial.

The landscape of prophylactic treatments for HAE is continually evolving, with other drugs in development, including Astria Therapeutics’ STAR-0215. This monoclonal antibody plasma kallikrein inhibitor is currently undergoing Phase Ib/II trials (NCT05695248) and has received fast-track designation from the US FDA.

 

Resource: Pharmaceutical Technology, November 20, 2023

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