FDA Approves VIGAFYDE™ as First Ready-to-Use Vigabatrin Oral Solution

Pyros Pharmaceuticals, Inc., based in Parsippany, N.J., has announced the U.S. Food and Drug Administration (FDA) approval of VIGAFYDE™, a ready-to-use vigabatrin oral solution. This solution is the first new drug approved in 15 years specifically for the treatment of infantile spasms (IS) in pediatric patients aged 1 month to 2 years. The primary consideration for its use is that the potential benefits must outweigh the risk of vision loss.

Infantile spasms represent a rare but severe form of epilepsy that significantly challenges both patients and their families. Pyros Pharmaceuticals aims to provide a reliable and affordable treatment option to ensure that families dealing with an IS diagnosis can swiftly access this critical therapy. The approval of VIGAFYDE™ is a significant milestone, highlighting the company's dedication to supporting families navigating this complex condition.

Michael Smith, co-founder and Chief Executive Officer of Pyros, emphasized the importance of this approval, stating that it demonstrates the company's ongoing commitment to aiding families affected by infantile spasms. Edwin Urrutia, co-founder and Chief Operating Officer at Pyros, added that the approval is a significant development for the infantile spasms community, offering a renewed sense of hope and energy. Urrutia believes that this will further enhance efforts in disease education, care pathway development, and increased investment in IS research.

VIGAFYDE™ is projected to be available in the latter half of 2024. Additionally, Pyros Pharmaceuticals offers Pyros Total Care™, a personalized support program designed to assist families throughout the treatment process. This program provides comprehensive support including personal assistance and financial resources for caregivers whose children are starting or continuing therapy with VIGAFYDE™.

The support provided by Pyros Total Care™ includes a dedicated team comprising a nurse educator, reimbursement support staff, and a clinical pharmacist. This team is committed to ensuring that families have access to the resources and information they need to manage their child’s treatment effectively.

Infantile spasms typically begin in children under one year old and can often be misdiagnosed due to their subtle, repetitive movements. If left untreated, IS can lead to long-term issues such as ongoing seizures, different forms of epilepsy, autism spectrum disorder, and developmental challenges. Studies have demonstrated that successful treatment of IS can improve long-term outcomes for affected children.

Vigabatrin, the active ingredient in VIGAFYDE™, works by inhibiting the enzyme GABA transaminase, thereby increasing levels of gamma-aminobutyric acid (GABA) in the brain. This mechanism helps enhance seizure control in patients by modulating neuronal excitability. Beyond seizure management, emerging research suggests that vigabatrin may also help mitigate neurodevelopmental complications associated with certain epilepsy syndromes.

The FDA has required a Risk Evaluation and Mitigation Strategy (REMS) for all vigabatrin products, including VIGAFYDE™, to ensure informed risk-benefit decisions and proper use throughout the treatment process. Healthcare providers must be certified in the Vigabatrin REMS to prescribe VIGAFYDE™, and patients must be enrolled to receive it. This certification and enrollment process ensures ongoing monitoring and support for both prescribers and patients.

The approval of VIGAFYDE™ marks a significant advancement in the treatment of infantile spasms, offering a new avenue for managing this challenging condition. Pyros Pharmaceuticals remains dedicated to improving the lives of children affected by rare diseases through innovative treatments and comprehensive support services.