RARE Daily

FDA Approves Pyros’ Vigafyde Oral Solution

June 18, 2024

Rare Daily Staff

The U.S. Food and Drug Administration approved Pyros Pharmaceuticals Vigafyde oral solution as a monotherapy for the treatment of pediatric patients 1 month to 2 years of age with infantile spasms where the potential benefits outweigh the potential risk of vision loss.

Infantile spasms (IS) are a rare, severe form of epilepsy that typically begins in children less than one year old. Infantile spasms can appear as subtle but repetitive movements that can often be overlooked or misdiagnosed. IS can lead to long-term permanent issues such as continued seizures, other forms of epilepsy, autism spectrum disorder, and developmental issues.

Vigabatrin is a medication used in the treatment of infantile spasms and is designed to inhibit the enzyme GABA transaminase, consequently increasing gamma-aminobutyric acid (GABA) levels in the brain. This release is thought to enhance seizure control for patients by modulating neuronal excitability. Pyros said its impact may extend beyond seizure management, with emerging research indicating potential benefits in mitigating neurodevelopmental complications associated with certain epilepsy syndromes. The approval of Vigafyde marks the first new drug indicated for infantile spasms in 15 years.

Vigafyde is expected to be available in the second half of 2024. Pyros Total Care, the company’s personalized comprehensive support program, is available to assist families throughout the treatment journey.

The drug carries a boxed warning. Vigafyde can cause permanent vision loss. Because of this risk and because Vigafyde, when it is effective, provides an observable symptomatic benefit; patient response and continued need for treatment should be periodically assessed.

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