Ionis Pharmaceuticals
Ionis Pharmaceuticals works on 19 rare diseases tracked on Trial Friend, including Acromegaly, Alexander Disease, Amyotrophic Lateral Sclerosis and 16 more, with 14 active clinical trials (7 recruiting) and 3 FDA-approved rare disease drugs.
Ionis Pharmaceuticals is a leader in antisense oligonucleotide (ASO) therapies targeting RNA for rare genetic, neurological, and cardiovascular diseases. Approved products include Tryngolza (olezarsen) for familial chylomicronemia syndrome (2024), Dawnzera (donidalorsen) for hereditary angioedema (2025), and Zanvastro (zilganersen), approved in September 2026 as the first and only treatment for Alexander disease. Through its partnership with Biogen, Ionis developed tofersen (Qalsody) for SOD1-ALS and Spinraza for spinal muscular atrophy. The pipeline includes ION582 for Angelman syndrome (Phase 3) and sefaxersen, partnered with Roche, for IgA nephropathy, which met its primary proteinuria endpoint at a prespecified interim analysis of the Phase 3 IMAgINATION study in September 2026.
Ionis Pharmaceuticals Drug Pipeline
Ionis Pharmaceuticals has 14 active clinical trials across 4 development stages, with 7 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Ionis Pharmaceuticals Clinical Trials (14)
Active and recruiting clinical trials sponsored by Ionis Pharmaceuticals, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
Ionis Pharmaceuticals FDA-Approved Drugs (3)
Medications developed or marketed by Ionis Pharmaceuticals that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| DONIDALORSEN | DAWNZERA subcutaneous | Aug 21, 2025 | |
| OLEZARSEN SODIUM | TRYNGOLZA subcutaneous | Dec 19, 2024 | |
| ZILGANERSEN | ZANVASTRO | Sep 3, 2026 |
Ionis Pharmaceuticals Trial Locations
Ionis Pharmaceuticals clinical trials are running at 234 sites in 27 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (19)
Diseases targeted by Ionis Pharmaceuticals's clinical trial and drug development programs
Acromegaly is a rare hormonal disorder caused by excessive growth hormone production, typically from a pituitary adenoma, resulting in abnormal growth of hands, feet, and facial features. It also caus...
Alexander disease is a rare inherited leukodystrophy caused by mutations in the GFAP gene affecting astrocyte function. The condition leads to progressive neurological deterioration with abnormal prot...
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord. This causes progressive weakness and paralysis of voluntary muscles whil...
Angelman Syndrome is a rare neurological disorder caused by loss of function of the UBE3A gene on the maternal chromosome 15. People with this condition typically develop normal until 6-12 months of a...
ATTR amyloidosis is a rare progressive disease where abnormal transthyretin protein accumulates as amyloid deposits in the heart, nerves, and other organs. It can be inherited (hereditary ATTR) or dev...
Cystic fibrosis is an autosomal recessive genetic disorder affecting the CFTR protein, which normally regulates chloride transport. Defective CFTR causes thick, sticky secretions in the lungs and dige...
Patient Resources
Organizations and resources related to Ionis Pharmaceuticals's rare disease focus areas
Frequently Asked Questions About Ionis Pharmaceuticals
Common questions about Ionis Pharmaceuticals's rare disease programs, clinical trials, and treatments.