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Upsher-Smith showcases new rare disease treatments at Child Neurology Society annual meeting

Jim Maahs, Head of Commercial at Upsher-Smith, highlighted the company's dedication to expanding rare disease treatments.

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MAPLE GROVE, Minnesota — Upsher-Smith Laboratories is presenting its expanding portfolio of products at the 2024 Child Neurology Society (CNS) Annual Meeting in San Diego, California, from November 11-14. The company's presence at the CNS meeting includes showcasing two of its newest rare disease treatments—Vigafyde (vigabatrin) Oral Solution and Torpenz (everolimus) Tablets—alongside its Promise of Support program designed to enhance patient and caregiver access to critical treatment resources.

Newly Introduced Treatments for Infantile Spasms and Tuberous Sclerosis Complex

Vigafyde, available since September 2024, is the first ready-to-use vigabatrin oral solution developed to treat infantile spasms in children between one month and two years of age. With this product, caregivers no longer need to reconstitute the medication, simplifying treatment administration. Upsher-Smith emphasizes Vigafyde as a new option in vigabatrin therapies, with the product offering a significant advantage by being pre-mixed and ready for use. Vigafyde is indicated solely when the benefits of treatment outweigh the risks of potential vision loss, a severe adverse effect associated with vigabatrin-based therapies. 

The recently launched Torpenz tablets, introduced in August 2024, offer a new treatment for patients with tuberous sclerosis complex (TSC). Torpenz is indicated for treating renal angiomyolipoma in adults with TSC who do not require immediate surgery and for managing subependymal giant cell astrocytoma (SEGA) in patients aged one year and older. The product is part of Upsher-Smith's effort to provide accessible, effective treatment options for the TSC community, and it comes with the company's Promise of Support program for enhanced patient support.

Commitment to Rare Disease Treatment through the Promise of Support Program

A pillar of Upsher-Smith's rare disease strategy is its Promise of Support Program, established nearly a decade ago to offer continuous assistance to patients, caregivers, and healthcare providers. The program's offerings range from assistance with prior authorizations to help with uninterrupted medication access. Additional resources include copay assistance, educational materials, and streamlined prescribing processes through collaboration with a specialty pharmacy partner. Upsher-Smith's team at the CNS meeting encourages healthcare providers to learn how the program can ease the administrative and financial challenges that rare disease patients often face.

Enhanced Safety Protocols with Vigabatrin REMS

As part of the regulatory framework for vigabatrin products like Vigafyde, Upsher-Smith implements the FDA-mandated Risk Evaluation and Mitigation Strategy (REMS). This program is designed to help patients and healthcare providers weigh the risk of vision loss, a known side effect of vigabatrin, through regular vision testing and informed counseling. This REMS enrollment process ensures patients are continually monitored, helping to manage risks while offering effective treatment options.

Jim Maahs, Head of Commercial at Upsher-Smith, highlighted the company's dedication to expanding rare disease treatments. "With Vigadrone (vigabatrin) for Oral Solution's initial launch in 2018, we have steadily increased our offerings for the rare disease community. We are excited to bring Vigafyde and Torpenz to CNS and engage clinicians about our innovative, patient-centered solutions."

To learn more about Upsher-Smith's products, the Promise of Support program, and important safety information for their rare disease treatments, visit Upsher-Smith's website.

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