What rare diseases does Ionis Pharmaceuticals focus on?

Ionis Pharmaceuticals has active programs across 20 rare diseases: Acromegaly, Alexander Disease, Amyotrophic Lateral Sclerosis, Angelman Syndrome, ATTR Amyloidosis (Transthyretin Amyloidosis), Cystic Fibrosis, Dravet Syndrome, Familial Chylomicronemia Syndrome, Geographic Atrophy, Hereditary Angioedema, Homozygous Familial Hypercholesterolemia, Huntington Disease, IgA Nephropathy, Multiple Myeloma, Multiple System Atrophy, Myotonic Dystrophy, Polycythemia Vera, Spinal Muscular Atrophy, Spinocerebellar Ataxia, Thalassemia. These span several therapeutic areas. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Does Ionis Pharmaceuticals have a rare disease division?

Ionis Pharmaceuticals is a diversified pharmaceutical company headquartered in Carlsbad, United States, founded in 1989. While it operates across multiple therapeutic areas, it maintains a significant rare disease portfolio with 20 active rare disease programs including Acromegaly, Alexander Disease, Amyotrophic Lateral Sclerosis. Many of its rare disease treatments hold orphan drug designations from the FDA.

How can I find Ionis Pharmaceuticals clinical trials for rare diseases?

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Ionis Pharmaceuticals, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.

How do I enroll in an Ionis Pharmaceuticals clinical trial?

To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.

What FDA-approved rare disease drugs does Ionis Pharmaceuticals have?

Ionis Pharmaceuticals has 3 FDA-approved drugs with a rare disease use that we track, as of November 2026. Zanvastro (zilganersen), approved 2026: Alexander Disease. Tryngolza (olezarsen), approved 2024: Familial Chylomicronemia Syndrome. Dawnzera (donidalorsen), approved 2025: Hereditary Angioedema. Still in development: Obudanersen (Phase 3). Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.

Does Ionis Pharmaceuticals offer copay help or free medicine for its rare disease drugs?

Yes, for 3 of its FDA-approved rare disease medicines that we track, each checked on Ionis Pharmaceuticals' own website. Zanvastro: Ionis Every Step, 1-844-399-5544. Offers copay help with private insurance, infusion cost help and insurance help. Checked October 5, 2026. Tryngolza: Ionis Every Step, 1-844-789-8744. Offers copay help with private insurance and insurance help. Checked September 29, 2026. Dawnzera: Ionis Every Step, 1-844-444-4305. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked September 24, 2026. Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page. Eligibility is decided by each program, so call the number listed before a prescription is filled.

When was Ionis Pharmaceuticals founded and where is it based?

Ionis Pharmaceuticals was founded in 1989 and is headquartered in Carlsbad, United States. The company has been active for 37 years. It trades publicly under the ticker symbol IONS.

Ionis Pharmaceuticals

Drugmaker with rare disease work · based in Carlsbad, United States · founded 1989 · ticker IONS · ionis.com

Ionis Pharmaceuticals works on 20 rare diseases we track, including Acromegaly, Alexander Disease and 18 more. It has 15 active clinical trials, and 8 are looking for volunteers now. It also has 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.

Ionis Pharmaceuticals pipeline

Each row lists the diseases Ionis Pharmaceuticals is studying at that stage, from the ones closest to approval down to the earliest work.

Approved

Drugs the FDA has approved.

Dawnzera (donidalorsen) · Tryngolza (olezarsen sodium) · Zanvastro (zilganersen)

Phase 3

Large studies, usually the last step before a company asks the FDA for approval.

Familial Chylomicronemia Syndrome 3 trials recruiting · Hereditary Angioedema 2 trials recruiting · Angelman Syndrome 2 trials recruiting · Alexander Disease · Amyotrophic Lateral Sclerosis

Phase 2

Mid-size studies that check whether a drug works and at what dose.

Dravet Syndrome recruiting · Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome recruiting · Angelman Syndrome

Phase 1

First studies in people, mostly checking safety.

Multiple System Atrophy recruiting

Other studies

Studies with no phase listed, such as registries and natural history studies.

Leukodystrophy recruiting · Alexander Disease recruiting

Ionis Pharmaceuticals clinical trials

These are Ionis Pharmaceuticals trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.

8 trials looking for volunteers now

Looking for volunteersRecently updatedNCT03047369

The Myelin Disorders Biorepository Project

For Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome and Adrenoleukodystrophy

The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago. Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.

Ages not specified24 locations

View full study

Looking for volunteersPhase 1/2Recently updatedNCT07531745

ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome

Tests ION337

For Dravet Syndrome

The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).

Ages 2 Years - 12 Years7 locations

View full study

Looking for volunteersPhase 3Recently updatedNCT07727538

A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants

Tests Olezarsen

For Familial Chylomicronemia Syndrome

The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.

Ages 2 Years - 17 Years6 locations

View full study

Looking for volunteersPhase 3Recently updatedNCT07298447

Donidalorsen Treatment in Children With Hereditary Angioedema

Tests Donidalorsen

For Hereditary Angioedema (HAE)

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).

Ages 2 Years - 11 Years18 locations

View full study

Looking for volunteersPhase 3Recently updatedNCT07782827

CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)

Tests obudanersen

For Angelman Syndrome

The primary purpose of the study is to evaluate efficacy of obudanersen in participants with AS due to uniparental disomy or imprinting defects (UPD/ID) as measured through expressive communication.

Ages 2 Years - 50 Years1 location

View full study

Looking for volunteersPhase 1/2Recently updatedNCT06430385

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

Tests ION440, Sham procedure

For Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

The primary purpose of this study is to evaluate the safety and tolerability of ION440.

Ages 2 Years - 65 Years12 locations

View full study

Looking for volunteersPhase 1No updates in a whileNCT04165486

Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)

Tests ION464, Placebo

For Multiple System Atrophy

The primary objectives are to evaluate the safety and tolerability of multiple doses of ION464 administered via intrathecal (IT) injection (Part 1) and to evaluate the long-term safety and tolerability of ION464 (Part 2) in participants with multiple system atrophy (MSA). The secondary objectives are to evaluate the pharmacodynamic (PD) effect of ION464 on the level of a potential biomarker of target engagement (Parts 1 and 2) and to evaluate the pharmacokinetic (PK) profile of ION464 in serum (Part 1).

Ages 40 Years - 70 Years15 locations

View full study

Looking for volunteersNo updates in a whileNCT02714764

Evaluation of Outcome Metrics in Alexander Disease

For Alexander Disease

The purpose of this study is to define the natural history of Alexander Disease, a leukodystrophy that causes neurological dysfunction. Investigators will obtain clinical outcome assessments to measure how the disease affects a patient's gross motor, fine motor, speech and language function, swallowing, and quality of life. Specimens are collected to measure glial fibrillary acidic protein (GFAP) levels in cerebrospinal fluid (CSF) and blood. The data obtained from this study will be used for the design of future treatment trials.

Ages not specified1 location

View full study

Ask about Ionis Pharmaceuticals's trials

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Trial Alerts, when a new trial opens or a trial changes
Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month

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FDA-approved drugs from Ionis Pharmaceuticals

From the openFDA database. Tap a drug for side effects, dosing and help paying.

Dawnzera donidalorsen

For Hereditary Angioedema. Approved Aug 21, 2025. Taken by subcutaneous route.

Tryngolza olezarsen sodium

For Familial Chylomicronemia Syndrome. Approved Dec 19, 2024. Taken by subcutaneous route.

Zanvastro zilganersen

For Alexander Disease. Approved Sep 3, 2026.

More about Ionis Pharmaceuticals

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.

Where Ionis Pharmaceuticals trials take place

The trials above run at 262 study sites in 27 countries. Tap a country to see its states, cities and hospitals.

United States

117▼

Canada

20▼

Italy

19▼

United Kingdom

14▼

Spain

13▼

Germany

12▼

France

11▼

Australia

9▼

Israel

6▼

Poland

5▼

Netherlands

5▼

Japan

4▼

Rare diseases Ionis Pharmaceuticals works on

Each disease page lists every open trial, not only Ionis Pharmaceuticals's, plus approved treatments.

Acromegaly

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.

How common: About 50 to 70 people per million, according to NORD, with 3 to 11 new cases per million people each year

Alexander Disease

Alexander disease is a rare genetic leukodystrophy caused by mutations in the GFAP gene affecting astrocyte function.

How common: Unknown; about 500 cases reported since 1949 (MedlinePlus Genetics)

Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord.

How common: About 5,000 new cases per year in the U.S.; CDC's National ALS Registry estimates about 33,000 Americans were living with ALS in 2022, a number projected to pass 36,000 by 2030

Angelman Syndrome

Angelman Syndrome is a rare neurological disorder caused by loss of function of the UBE3A gene on the maternal chromosome 15.

How common: Approximately 1 in 12,000 to 20,000 people

ATTR Amyloidosis (Transthyretin Amyloidosis)

ATTR amyloidosis is a rare progressive disease where abnormal transthyretin protein accumulates as amyloid deposits in the heart, nerves, and other organs.

How common: Approximately 5,000 to 7,000 new cases diagnosed annually in the U.S

Cystic Fibrosis

Cystic fibrosis is an autosomal recessive genetic disorder affecting the CFTR protein, which normally regulates chloride transport.

How common: Nearly 40,000 children and adults in the U.S.; 1 in 2,500 to 3,500 births among Caucasians

Dravet Syndrome

Dravet Syndrome is a severe form of epilepsy that typically begins in infancy with prolonged seizures triggered by fever.

How common: Approximately 1 in 16,000 to 40,000 people, accounting for 5-10% of early infantile epileptic encephalopathies

Familial Chylomicronemia Syndrome

Familial Chylomicronemia Syndrome is a rare inherited metabolic disorder where the body cannot break down certain fats (triglycerides) normally, causing dangerously high blood fat levels.

How common: 1 to 2 per 1,000,000 people globally

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Ionis Pharmaceuticals's own site is ionis.com, and our help paying for treatment page lists drugmaker programs and charity funds.

NIH GARD - Alexander Disease
Alexander Disease and Cystic Fibrosis and 3 more
Acromegaly Community Inc.
Acromegaly
National Organization for Rare Disorders (NORD)
Acromegaly
Alexander Disease Info & Support
Alexander Disease
The ALS Association
Amyotrophic Lateral Sclerosis
ALS Therapy Development Institute
Amyotrophic Lateral Sclerosis
National Organization for Rare Disorders (NORD)
Angelman Syndrome
NIH Genetic and Rare Diseases Information Center
Angelman Syndrome

Ionis Pharmaceuticals

Ionis Pharmaceuticals pipeline

Ionis Pharmaceuticals clinical trials

8 trials looking for volunteers now

The Myelin Disorders Biorepository Project

ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome

A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants

Donidalorsen Treatment in Children With Hereditary Angioedema

CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)

Evaluation of Outcome Metrics in Alexander Disease

FDA-approved drugs from Ionis Pharmaceuticals

More about Ionis Pharmaceuticals

Where Ionis Pharmaceuticals trials take place

Rare diseases Ionis Pharmaceuticals works on

Where to get support

Other companies working on the same diseases

Questions about Ionis Pharmaceuticals