Biohaven commences phase 3 spinal muscular atrophy trial


Biohaven has begun enrolling patients for a phase 3 trial of recombinant protein taldefgrobep alfa on individuals with spinal muscular atrophy. 

The study will monitor patients as they take taldefgrobep alfa in conjunction with other SMA treatments. 

Patients can enroll regardless of disease classification or ambulatory status. 

The phase 3 trial expects to enroll about 180 patients with SMA who are already taking doses of common SMA medications. 

"This is an important milestone for the taldefgrobep program and for people living with SMA. There have been great strides in advancing therapeutics in this challenging disease. Yet, there remains a significant unmet need to address the residual weakness and functional impairments, such as difficulty walking, that are caused by the disease," said Irfan Qureshi, MD, senior vice president of neurology at Biohaven, in a July 11 statement. 

"We are thankful for the SMA community that has welcomed and worked with us to develop this patient-focused study."

Taldefgrobep alfa is a human protein that can work to lower free myostatin and has been previously studied in patients with other neuromuscular diseases.

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