BridgeBio Pharma received FDA Fast Track Designation for Investigational Therapy for the Treatment of Limb-girdle Muscular Dystrophy Type 2i (LGMD2i)
BBIO, a company whose mission was to develop and develop innovative medicines for people with genetic diseases and cancers with clear genetic drivers, today announced the United States Food and Drug Administration approved Fast Track designation for the investigation of BBP The FDA grants development programs Fast Track designation to support the development and expedite its review process for drugs that are investigated to treat serious conditions and fill unmet medical needs. The FDA uses this program to provide patients with access to new medicines as soon as possible. This is the fifth Fast Track designation that BridgeBio has received this year for an investigational therapy.
In addition to its 14 programs, the company's LGMD2i investigational therapy is one of the Company'' most extensive and highly specialized medical treatment centers. These programs are administered by the medical center or commercially located patients with
BridgeBio's first program is the now-approved drugs for Molybdenum Cofactor Deficiency (MoCD) Type A and previously-treated locally advanced or metastatic cholangiocarcinoma ( The second wave of programs include the company's four major near-term catalysts for its product candidates for the treatment of transthyretin (TTR), achondroplasia, congenital adrenal hyperplasie
LGMD2i is one of BridgeBio's leading programs in development, which includes several programs already in the cancer and mendelian space in clinics.
LGMD2i is a inherited recessive muscular dystrophy caused by mutagenesis of fukutin-related protein (FKRP). Approximately 7000 patients have potentially treatable mutations FKRP is a critical enzyme that combines sexy molecule with DG, aka alpha-dystroglycan. A robust cushioning system is provided by fully glycosylated DG proteins due to the defective FKRP enzyme function. The muscle cells of patients who are affected by LGMD2i lack robust resuscitation system Several medical conditions often result in severe and severe hemodynamic problems. In general, the underlying condition of the body's epoch (molar and thoracic) dysfunction (like girdle) and the
"Currently, there are no approved treatment options for people born with liar-girdle muscular dystrophy type 2i. People with this disease may lose their ability to perform routine daily activities, and ultimately lose the ability of walking, need ventilatory support or face the risk of heart failure, said Douglas Sproule, M.D., a medical We are grateful that the FDA granted our program Fast Track designation because of the potential of our investigational therapy to treat this very serious condition. We hope that designation will allow us to address this unmet medical need by allowing us, to offer our medicine to patients faster.
BBP-418 is being investigated as a treatment for LGMD2i. The investigational therapy is designed to overcome the enzymatic limitation of the defective FKRP enzyme by supplementing endogenous sugar molecules to glycosylate DG and to improve muscle cell integrities, which results in Clinical trials are ongoing to determine the safety and efficacy of BBP-418.
The FDA approved Orphan Drug Designation for the treatment of LGMD2i and the European Medicines Agency approved for LGDM. BridgeBio is currently advancing its Phase 2 clinical trial in subjects with a genetically confirmed diagnosis of LGMD2i. If the development program is successful, BBP-418 may be the first approved therapy for the treatment of patients with LGMD2i.
About Limb-girdle Muscular Dystrophy Type 2i Type 3ii-type 3o3u4 lbgynthnal systorophy During childhood, LGMD2i is a monogamous autosomal recessive disease with relapse of function in the -DG gene (FKRP) gene mutation. The majority of people develop Cardiomyopathy is progressive with an annual loss of 0.4% of left ventricular ejection fraction (LVEF). Patients with heterozygous genotypes have an early childhood onset with an more severe clinical course, rapid mobility loss by 20 years, more frequent cardiac involvement (25%), and respiratory failure by 30 years.
About BridgeBio Pharma, Inc. BridgeBio Pharma Inc. is a biopharmaceutical company founded in 1985 to discover, create, test and deliver transformative medicines to treat patients with cancer and genetic diseases with clear genetic drivers. The company's commercial organization is focused on delivering the company two first approved therapies. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information, visit the site for more details. bridgebio.com is a bridge bio.de service. Follow us on LinkedIn and Twitter.
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