AstraZeneca attributes its Phase 3 HICKORY miss in hypophosphatasia to trial design, as Mereo seeks a path forward with the FDA and Mirum wins approval…
July 08, 2026
Vanda Pharmaceuticals Wins FDA Rare Pediatric Disease Status For Ultra-Rare Nerve Disorder Drug
Vanda Pharmaceuticals Inc. (Nasdaq: VNDA) has received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for VCA‑894A, an investigational antisense therapy aimed…
June 30, 2026
FDA Grants Monopar Therapeutics Rare Pediatric Disease Designation for Wilson Disease Treatment
Monopar Therapeutics Inc. (NASDAQ: MNPR), a clinical-stage biopharmaceutical company, secured Rare Pediatric Disease designation from the FDA for ALXN1840 in the treatment of Wilson disease…
Tempus AI (NASDAQ: TEM) just added another rare‑disease data asset to its oncology flywheel, and the stock inched higher on the news. Shares rose about…
June 04, 2026
FDA Signals a Renewed Posture Toward Rare Disease
A recent meeting between acting FDA Commissioner Kyle Diamantas and senior executives from fifteen rare disease nonprofits offers a useful read on where the agency…
February 26, 2026
Investing in Innovation during Rare Disease Day 2026
Rare Disease Day 2026 serves as a powerful reminder of the urgent, high-stakes innovation occurring across the biotechnology landscape. For companies like Larimar Therapeutics (NASDAQ:…
February 09, 2026
Ultragenyx’s Gene Therapy UX111 Shows Durable Efficacy in Sanfilippo Syndrome Studies
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) reported that its investigational gene therapy UX111 demonstrated sustained cognitive improvements and significant biomarker gains in long-term follow-up from two…