Rare Disease Trial Updates: March 2026

In March 2026, Trial Friend tracked 5 rare disease clinical trial updates across 5 conditions, including 5 FDA-related actions and 0 trial status changes, alongside enrollment updates and new trial site openings sourced from ClinicalTrials.gov and FDA announcements.

This is a permanent monthly archive. For the latest updates, see the live current events feed.

Major Updates: FDA Actions, Status Changes & Results

FDA ApprovalMar 26

FDA Approves First Gene Therapy for Severe Leukocyte Adhesion Deficiency Type I

The U.S. Food and Drug Administration today approved Kresladi (marnetegragene autotemcel), the first gene therapy for the treatment of severe Leukocyte Adhesion Deficiency Type I (LAD-I).

FDA DesignationPolycystic Kidney DiseaseMar 26

FDA grants orphan drug designation to tamibarotene for Polycystic Kidney Disease

The FDA granted orphan drug designation to tamibarotene, developed by Rege Nephro Co., Ltd., on 2026-03-26 for the treatment of autosomal dominant polycystic kidney disease. Orphan designation is an incentive for rare disease drug development, not an approval: it gives the sponsor tax credits, fee waivers, and 7 years of market exclusivity if the drug is eventually approved. It often signals that a company is committing to a full development program.

FDA ApprovalHunter SyndromeMar 25

FDA Approves Drug to Treat Neurologic Manifestations of Hunter Syndrome

The U.S. Food and Drug Administration approved Avlayah (tividenofusp alfa-eknm) to treat certain individuals with Hunter syndrome (Mucopolysaccharidosis type II or MPS II).

FDA DesignationIdiopathic Pulmonary FibrosisMar 23

FDA grants orphan drug designation to tadalafil for Idiopathic Pulmonary Fibrosis

The FDA granted orphan drug designation to tadalafil, developed by Shenzhen Hanhui Pharmaceutical Technology Co., Ltd, on 2026-03-23 for the treatment of idiopathic pulmonary fibrosis. Orphan designation is an incentive for rare disease drug development, not an approval: it gives the sponsor tax credits, fee waivers, and 7 years of market exclusivity if the drug is eventually approved. It often signals that a company is committing to a full development program.

FDA DesignationAmyotrophic Lateral SclerosisMar 2

FDA grants orphan drug designation to meloxicam for Amyotrophic Lateral Sclerosis

The FDA granted orphan drug designation to meloxicam, developed by AniMusCure Inc., on 2026-03-02 for the treatment of amyotrophic lateral sclerosis. Orphan designation is an incentive for rare disease drug development, not an approval: it gives the sponsor tax credits, fee waivers, and 7 years of market exclusivity if the drug is eventually approved. It often signals that a company is committing to a full development program.