Taldefgrobep alfa scores orphan drug designation in Europe
A Phase 3 trial is testing to see how therapy affects motor function after a year
The European Commission has granted orphan drug status to taldefgrobep alfa as a potential treatment for spinal muscular atrophy (SMA).
The designation is given to therapies that may improve care for rare diseases. It confers certain incentives, including a reduction in some regulatory fees and a guarantee of 10 years of market exclusivity if the therapy is approved. Taldefgrobep is being developed by Biohaven Pharmaceuticals, which acquired the rights to it last year.
“We are delighted that the European Commission granted orphan drug designation for taldefgrobep alfa for the treatment of SMA,” Irfan Qureshi, MD, chief medical officer at Biohaven, said in a company press release.
Taldefgrobep is designed to block the activity of the protein myostatin, which normally helps limit the growth of muscles so they don’t get too large. By blocking myostatin, the therapy, which is injected under the skin, or subcutaneously, should promote muscle growth and strength in people with SMA. It was given orphan drug and fast track designations by the U.S. Food and Drug Administration.
Taldefgrobep’s effects on muscle weakness
Biohaven is developing taldefgrobep to be used with available disease-modifying therapies, which boost levels of the SMN protein, the defect of which causes SMA. These therapies can slow or stop the progression of the disease, but can’t reverse muscle atrophy and damage that’s already occurred, so many patients still have substantial muscle weakness even with treatment.
“Children and adults living with SMA experience significant muscle weakness and functional impairments affecting their quality of daily life, and a substantial unmet medical need persists,” Qureshi said. “We are excited about the potential for taldefgrobep alfa to improve the lives of patients and families affected by SMA.”
Biohaven is sponsoring a Phase 3 trial called RESILIENT (NCT05337553), which is testing taldefgrobep against a placebo with the main goal of evaluating how it affects scores on a standardized measure of motor function after about a year.
The RESILIENT study is open to patients ages 4-21 who have a confirmed diagnosis of SMA and are on an approved disease-modifying treatment. The study is recruiting participants at more than 50 sites in the U.S. and Europe.
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