Astellas Pharma Submits New Drug Application for Avacincaptad Pegol in Japan
Astellas Pharma Inc. has officially submitted a New Drug Application (NDA) to Japan's Ministry of Health, Labour and Welfare for the conditional approval of avacincaptad pegol, an innovative intravitreal solution. This synthetic aptamer is designed to inhibit the complement C5 protein, potentially serving as a groundbreaking treatment for patients suffering from geographic atrophy (GA) secondary to age-related macular degeneration (AMD). If greenlit, it will stand as the first and only treatment option for GA in Japan.
Geographic atrophy is the advanced form of AMD, leading to progressive and irreversible vision loss. Currently, there are no approved therapies outside of the U.S. or Australia for this condition. It's estimated that over five million people worldwide grapple with GA, with approximately two-thirds facing severe visual impairment or legal blindness without immediate intervention. The psychological and daily life impacts on these patients can be profound, highlighting the urgent need for effective treatment solutions.
Marci English, Astellas’ Vice President and Head of BioPharma and Ophthalmology Development, commented on the NDA submission, stating it brings hope to many patients in Japan who currently have no approved treatment for this debilitating disease. She emphasized Astellas' commitment to collaborating with regulatory entities to facilitate patient access to this critical new therapy.
The basis for this NDA rests on results from overseas clinical trials, particularly the GATHER1 and GATHER2 studies. These randomized, sham-controlled trials assessed the safety and effectiveness of monthly 2mg intravitreal injections of avacincaptad pegol in individuals diagnosed with GA secondary to AMD. The findings illustrated that patients undergoing treatment with avacincaptad experienced a significant reduction in GA lesion growth while maintaining a favorable safety profile. Long-term effectiveness was noted, as reductions in lesion growth were observed over a two-year follow-up period in both GATHER1 and GATHER2 studies.
Avacincaptad pegol, also marketed as IZERVAY in the United States, specifically targets and inhibits the overactive complement system, known to contribute significantly to the degeneration of retinal cells. With its mechanism of action directed at complement C5 activity, the drug seeks to slow down the progression of GA, providing much-needed relief to affected individuals.
Age-related macular degeneration remains one of the leading causes of severe vision loss among elderly populations, severely affecting everyday activities that rely on central vision. As the disease progresses, it leads to the thinning and atrophy of retina tissues, culminating in additional irreversible vision loss. Therefore, the significance of potential treatments like avacincaptad pegol cannot be overstated.
The GATHER2 clinical trial specifically involved 448 participants diagnosed with GA secondary to AMD. The outcomes suggested a noteworthy safety record and efficacy, as patients receiving avacincaptad had a more favorable response compared to the sham group over the course of the study.
Astellas is not only focused on treating GA but also dedicated to developing innovative solutions for various health conditions through its research and development initiatives. The company's mission revolves around delivering transformative therapies that respond to unmet medical needs across several domains including oncology, ophthalmology, urology, immunology, and women's health.
As Astellas awaits the outcome of the NDA submission, the potential for avacincaptad pegol to change the landscape of treatment options for geographic atrophy in Japan remains optimistic, underlining the company's commitment to enhancing the quality of life for patients afflicted by this challenging condition.
Geographic atrophy is the advanced form of AMD, leading to progressive and irreversible vision loss. Currently, there are no approved therapies outside of the U.S. or Australia for this condition. It's estimated that over five million people worldwide grapple with GA, with approximately two-thirds facing severe visual impairment or legal blindness without immediate intervention. The psychological and daily life impacts on these patients can be profound, highlighting the urgent need for effective treatment solutions.
Marci English, Astellas’ Vice President and Head of BioPharma and Ophthalmology Development, commented on the NDA submission, stating it brings hope to many patients in Japan who currently have no approved treatment for this debilitating disease. She emphasized Astellas' commitment to collaborating with regulatory entities to facilitate patient access to this critical new therapy.
The basis for this NDA rests on results from overseas clinical trials, particularly the GATHER1 and GATHER2 studies. These randomized, sham-controlled trials assessed the safety and effectiveness of monthly 2mg intravitreal injections of avacincaptad pegol in individuals diagnosed with GA secondary to AMD. The findings illustrated that patients undergoing treatment with avacincaptad experienced a significant reduction in GA lesion growth while maintaining a favorable safety profile. Long-term effectiveness was noted, as reductions in lesion growth were observed over a two-year follow-up period in both GATHER1 and GATHER2 studies.
Avacincaptad pegol, also marketed as IZERVAY in the United States, specifically targets and inhibits the overactive complement system, known to contribute significantly to the degeneration of retinal cells. With its mechanism of action directed at complement C5 activity, the drug seeks to slow down the progression of GA, providing much-needed relief to affected individuals.
Age-related macular degeneration remains one of the leading causes of severe vision loss among elderly populations, severely affecting everyday activities that rely on central vision. As the disease progresses, it leads to the thinning and atrophy of retina tissues, culminating in additional irreversible vision loss. Therefore, the significance of potential treatments like avacincaptad pegol cannot be overstated.
The GATHER2 clinical trial specifically involved 448 participants diagnosed with GA secondary to AMD. The outcomes suggested a noteworthy safety record and efficacy, as patients receiving avacincaptad had a more favorable response compared to the sham group over the course of the study.
Astellas is not only focused on treating GA but also dedicated to developing innovative solutions for various health conditions through its research and development initiatives. The company's mission revolves around delivering transformative therapies that respond to unmet medical needs across several domains including oncology, ophthalmology, urology, immunology, and women's health.
As Astellas awaits the outcome of the NDA submission, the potential for avacincaptad pegol to change the landscape of treatment options for geographic atrophy in Japan remains optimistic, underlining the company's commitment to enhancing the quality of life for patients afflicted by this challenging condition.
Topics Health）
【About Using Articles】
You can freely use the title and article content by linking to the page where the article is posted.
※ Images cannot be used.
【About Links】
Links are free to use.