Pharma Focus Europe

Biohaven's Taldefgrobep Alfa Granted EU Orphan Drug Designation for Spinal Muscular Atrophy

Tuesday, August 01, 2023

Biohaven Ltd. (NYSE: BHVN) has recently obtained orphan medicinal product designation from the European Commission (EC) for taldefgrobep alfa, a novel anti-myostatin adnectin aimed at treating spinal muscular atrophy (SMA). The orphan designation acknowledges taldefgrobep's potential to bring significant benefits to SMA patients and offers regulatory and commercial incentives, including reduced regulatory fees for protocol reviews and scientific advice, as well as ten years of additional market protection. Earlier, the FDA had granted Biohaven fast track and orphan drug designations for taldefgrobep in SMA treatment.

SMA is a rare and progressive motor neuron disease that leads to compromised muscle mass development and growth, causing muscle weakness, atrophy, reduced motor function, and impaired quality of life. Inhibiting myostatin, a naturally occurring protein that limits skeletal muscle growth, is seen as a promising approach for treating SMA.

Taldefgrobep is a novel therapy that could enhance muscle function in SMA by blocking myostatin, used in combination with existing disease-modifying treatments that preserve motor neurons. Its uniqueness lies in its dual mechanism of action – it reduces overall myostatin levels by binding to myostatin and also acts as a receptor antagonist, blocking myostatin signaling in skeletal muscles.

Dr. Irfan Qureshi, Chief Medical Officer at Biohaven, expressed his delight in receiving the orphan drug designation for taldefgrobep alfa for SMA treatment. He emphasized the potential of taldefgrobep alfa to improve the lives of SMA patients and their families.

Biohaven, known for its innovative trials addressing neurodegenerative diseases, is currently enrolling participants in a Phase 3 clinical trial of taldefgrobep in SMA, called the RESILIENT study (NCT05337553).

Taldefgrobep alfa, also known as BHV2000, is a modified adnectin designed to specifically bind to myostatin (GDF-8). It is a fully human anti-myostatin recombinant protein that lowers free myostatin levels and acts as an Activin 2b receptor antagonist when forming the myostatin-taldefgrobep complex. Adnectins belong to a proprietary protein therapeutic class based on human fibronectin, an extracellular protein abundant in human serum.

Spinal Muscular Atrophy (SMA) is a rare genetic neurodegenerative disorder characterized by the loss of motor neurons, muscle atrophy in limbs and trunk, progressive muscle weakness, and often fatal outcomes. SMA is caused by insufficient production of the SMN (survival of motor neuron) protein, encoded by the SMN1 and SMN2 genes. In the United States, approximately 1 in 11,000 births is affected by SMA, and about 1 in every 50 Americans is a genetic carrier.

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