Norwell, Mass. – September 24, 2026 -- The National Organization for Rare Disorders (NORD) has named six companies and nonprofits as 2026 Industry Innovator honorees for delivering first-of-their-kind FDA-approved therapies to patients with rare diseases that affect a fraction of the more than 30 million Americans living with such conditions.
Six FDA approvals mark milestones across ultra-rare indications
Mighty Therapeutics was recognized for FORZINITY, the first therapy for Barth syndrome, a rare mitochondrial disease, shown to improve muscle strength and quality of life. UCB earned recognition for KYGEVVI, the first and only approved treatment for thymidine kinase 2 deficiency in patients whose symptoms began at or before age 12. Fondazione Telethon was honored for Waskyra, a gene therapy for Wiskott-Aldrich syndrome that stands as the first FDA-approved gene therapy for a primary immunodeficiency disorder and the first developed by a nonprofit.
Omeros was cited for YARTEMLEA, the first approved treatment for hematopoietic stem cell transplant-associated thrombotic microangiopathy, a frequently fatal transplant complication. Jazz Pharmaceuticals was recognized for MODEYSO, the first approved therapy for recurrent H3K27M-mutant diffuse midline glioma, an aggressive pediatric brain tumor. Verastem Oncology received recognition for the AVMAPKI FAKZYNJA co-pack, the first treatment specifically approved for KRAS-mutated recurrent low-grade serous ovarian cancer following prior therapy.
NORD CEO cites 95% treatment gap across more than 10,000 rare diseases
"With approximately 95% of more than 10,000 known rare diseases still lacking an approved treatment, advancing scientific progress is critical," said Pamela K. Gavin, NORD Chief Executive Officer. NORD CEO Pamela Gavin is presenting the Industry Innovation Awards to each recipient company at their U.S. headquarters.
Advocacy and research honors extend beyond industry recipients
The Foundation for Prader-Willi Research received the Abbey S. Meyers Leadership Award for its Global PWS Registry on NORD's IAMRARE platform, which contributed to an FDA-approved treatment. Stephanie E. Haridopolos received the Policy Changemaker Award for facilitating HHS's addition of Duchenne muscular dystrophy and metachromatic leukodystrophy to the Recommended Newborn Screening Panel. Mark Skinner received the Lifetime Achievement Award for decades of advocacy on bleeding disorders through the World Federation of Hemophilia and NORD's Board of Directors.
Community Champion, Policy Changemaker, Youth Leadership, Lifetime Achievement, and Abbey S. Meyers Leadership Awardees will be recognized in person at the NORD Breakthrough Summit on Oct. 26-27 in Washington, D.C.