Biohaven's Taldefgrobep Alfa Receives EU Orphan Drug Designation for Spinal Muscular Atrophy
- Taldefgrobep alfa, in Phase 3 global clinical development for Spinal Muscular Atrophy, granted EU Orphan Drug Designation in addition to previously receiving Fast Track and Orphan Drug Designation in the US
SMA is a rare, progressively debilitating motor neuron disease in which development and growth of muscle mass are compromised, resulting in progressive weakness and muscle atrophy, reduced motor function, impaired quality of life and often death. Inhibition of myostatin, a naturally occurring protein that limits skeletal muscle growth, an important process in healthy muscular development, is a promising therapeutic strategy for SMA.
Taldefgrobep has the potential to be a novel therapy to enhance muscle function in SMA by blocking myostatin, when used in combination with currently available disease modifying therapies that help preserve motor neurons. Taldefgrobep's novelty in a field of myostatin inhibitors is based on its unique, dual mechanism of action. It binds to myostatin to lower overall myostatin levels and also functions as a receptor antagonist, thereby blocking myostatin signaling in skeletal muscles.
As a leader in innovative trials addressing neurodegenerative diseases,
About Taldefgrobep alfa
Taldefgrobep alfa (also known as BHV2000) is a modified adnectin designed to specifically bind to myostatin (GDF-8). Taldefgrobep is a fully human anti-myostatin recombinant protein that lowers free myostatin and acts as an Activin 2b receptor antagonist with the myostatin-taldefgrobep complex. Adnectins are an established proprietary protein therapeutic class based on human fibronectin, an extracellular protein that is naturally abundant in human serum.
About Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a rare genetic neurodegenerative disorder characterized by the loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk and progressive muscle weakness that is often fatal and typically diagnosed in young children. The underlying pathology of SMA is caused by insufficient production of the SMN (survival of motor neuron) protein, essential for the survival of motor neurons, and is encoded by two genes, SMN1 and SMN2. In the
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