AP&S helps a startup specialty pharma commercialize a novel generic formulation to treat infantile spasms.

Oct 25, 2024

OUTCOME: AP&S secured a US patent on the new formulation, Pyros was acquired, A better treatment is now available for the treatment of infantile spasms.

Infantile spasms are a severe pediatric epilepsy affecting ~1,200 U.S. infants annually, typically starting at 1–2 months and peaking at 4–8 months. Untreated or poorly treated, they can cause serious, ... irreversible developmental harm; early diagnosis and prompt therapy can enable normal development. Standard therapy, vigabatrin (VGB), is unstable once mixed with water. Caregivers must prepare each dose through a 15-step process, and the solution’s bitterness leads many infants to spit out medication, forcing guesswork on redosing. Underdosing risks seizures; overdosing risks retinal toxicity with irreversible visual field loss in ~34% of treated children. Pyros Pharmaceuticals set out to commercialize a ready-to-use, flavored VGB oral solution to simplify dosing. Because the formulation used known components, patentability—and therefore exclusivity—was challenging. AP&S reviewed a licensable PCT application covering oral formulations (including VGB) and concluded its claims were unpatentable over prior art, even after the owners proposed a narrower set. Upon becoming U.S. counsel on the licensed case, AP&S crafted claims that overcame prior art and secured U.S. Patent No. 12,016,857 specifically covering Pyros’s formulation. Upsher-Smith (Vigadrone®) opposed seven Pyros trademarks in the U.S. and Europe. Assessing Pyros’s strengthened IP and the clinical advantages of a ready-to-use product for infants, Upsher-Smith’s parent, Bora Pharmaceuticals, acquired Pyros rather than pursue uncertain, protracted oppositions. As CEO Michael Smith noted, the deal aligned with a mission to improve care for rare diseases. The resulting product, VIGAFYDE™, is the first and only ready-to-use vigabatrin oral solution—and the first new infantile spasms treatment approved in 15 years. A recent study showed both new and experienced caregivers delivered significantly more accurate, less variable doses with VIGAFYDE™ than with solutions prepared from VGB powder. Case study link: https://www.apslaw.com/case-studies/aps-guides-a-startup-specialty-pharmaceutical-company-in-the-commercialization-of-a-novel-formulation-of-a-generic-drug-for-treating-infantile-spasms/#_ftn1