Lexeo Therapeutics Advances LX2020 with FDA RMAT Designation
NEW YORK, August 5, 2026 Lexeo Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative...
Orphan Drug
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Can-Fite Launches Phase 2 Piclidenoson Trial for Lowe Syndrome
RAMAT GAN, Israel, Aug. 3, 2026 Can-Fite BioPharma Ltd. has launched its first clinical development program evaluating Piclidenoson for...
Opus Genetics Completes Enrollment in OPGx-LCA5 Phase 3 Trial
RESEARCH TRIANGLE PARK, N.C., Aug. 3, 2026 Opus Genetics, Inc. has completed patient enrollment in its registrational Phase 3...
Oculis Achieves Positive FDA Pre-IND Outcome for Privosegtor Program
ZUG, Switzerland, Aug. 3, 2026 Oculis announced that the U.S. Food and Drug Administration (FDA) has provided positive pre-Investigational...
Moleculin Reports Positive Interim MIRACLE Trial Data in Venetoclax-Failed AML Patients
HOUSTON, July 31, 2026 Moleculin Biotech, Inc. announced updated positive preliminary blinded interim results from Part A of its...
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Alexion, AstraZeneca Rare Disease Shares Phase 3 Ultomiris Trial Results
BOSTON, Mass., July 27, 2026 Alexion, AstraZeneca Rare Disease announced high-level results from its global Phase 3 clinical program...
Zevra Therapeutics to Seek EU Re-Examination for Arimoclomol After CHMP Opinion
BOSTON – July 24, 2026 Zevra Therapeutics, Inc. has announced that the European Medicines Agency’s (EMA) Committee for Medicinal...
Ipsen Announces Phase III BOLD Results for Bylvay in Biliary Atresia
PARIS, France, July 24, 2026 Ipsen announced that its Phase III BOLD clinical trial evaluating Bylvay® (odevixibat) in patients...
Nuvectis Pharma Announces China Approval of Ciprocopan for PNH
FORT LEE, N.J., July 23, 2026 Nuvectis Pharma, Inc. announced that ciprocopan (NXP100) has received marketing approval from China’s...
Xodus Medical Study Validates Hot Pink Pad Performance
PITTSBURGH, Pa., July 23, 2026 Xodus Medical has announced encouraging results from a new observational clinical study demonstrating that...
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