Jazz Pharmaceuticals

BioMarin, Jazz, Otsuka CEOs Join NORD Summit Rare Disease Panel

Washington, D.C. – October 06, 2026 -- CNBC's Becky Quick will moderate a rare disease leadership dialogue featuring the CEOs of BioMarin, Jazz Pharmaceuticals and Otsuka North America Pharmaceutical alongside NORD CEO Pam Gavin, the National Organization for Rare Disorders announced.

Panel convenes three biopharma chiefs at NORD's Breakthrough Summit on October 26

The session, titled "Next Decade of Rare Disease: A CEO Leadership Dialogue," takes place at 11 a.m. ET on Monday, October 26, in Washington, D.C., during the three-day NORD Rare Diseases & Orphan Products Breakthrough Summit running October 25–27. Participants include Alexander Hardy, President and CEO of BioMarin; Renee Gala, President and CEO of Jazz Pharmaceuticals; Tarek Rabah, President and CEO of Otsuka North America Pharmaceutical; and NORD CEO Pam Gavin.

NORD Honors Six Firms for First-of-Kind Rare Disease Therapy Approvals

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.

Jazz's Ziihera Shows Significant Survival Edge Over Trastuzumab in GEA Trial

Dublin – September 03, 2026 -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that Ziihera (zanidatamab-hrii) combined with chemotherapy produced a statistically significant and clinically meaningful overall survival (OS) benefit versus trastuzumab plus chemotherapy in a second interim analysis of the Phase 3 HERIZON-GEA-01 trial in first-line HER2-positive gastroesophageal adenocarcinoma (GEA).

OS hazard ratio for Ziihera plus chemotherapy improved versus the first interim analysis

The company said the survival advantage strengthened compared with earlier data, reinforcing Ziihera's position following its FDA approval on August 25, 2026. No new safety signals emerged relative to previously reported HERIZON-GEA-01 data or the known profiles of the individual agents.