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July 12, 2022

Biohaven enrolls first patient in drug trial for spinal muscular atrophy


New Haven-based Biohaven Pharmaceutical Holding Co. has started enrolling patients in a Phase 3 clinical trial of its candidate to help treat spinal muscular atrophy.

The condition is a rare, often fatal genetic neurodegenerative disorder characterized by muscle atrophy. Patients with the disorder don’t adequately produce a protein which is essential for the survival of motor neurons, according to Biohaven.

The clinical trial will evaluate the safety and efficacy of Taldefgrobep alfa in combating the disorder when combined with other approved treatments. 

According to Biohaven, Taldefgrobep alfa has the potential to enhance muscle mass and strength in people with the disorder as it targets myostatin, a natural protein that limits skeletal muscle growth.

Biohaven plans to enroll approximately 180 patients in the placebo-controlled, double-blind global trial.

Dr. Irfan Qureshi, senior vice president, neurology at Biohaven, called the enrollment an important milestone for people living with the disorder.

"There have been great strides in advancing therapeutics in this challenging disease,” Qureshi said. “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.”

Dr. Han Phan, director of clinical research, rare disease research, said there continues to be “a high unmet need for safe and effective supportive treatments” for the disorder.

"Many patients still experience significant weakness and reduced levels of functioning,” Phan said, in an announcement. “Additional research is needed to help improve the quality of life for people living with (spinal muscular atrophy.)”

Contact Michelle Tuccitto Sullo at

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