Internal Development (Skyclarys)
2023
Why
The organization internally developed Skyclarys for the treatment of Friedreich's ataxia, securing exclusive rights to the first FDA-approved therapy for this devastating rare neurological disorder.
Impact
The internal development provided the organization with a proven rare neurology platform and a deep pipeline of early-stage assets in Friedreich's ataxia, positioning the company at the forefront of the next-generation rare neurology market.
Outcome
Skyclarys was approved by the FDA in 2023, representing a major breakthrough in rare neurology and a potential blockbuster asset for the organization.