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UCB

Drugmaker with rare disease work · based in Brussels, Belgium · founded 1928 · ticker UCB · ucb.com

UCB works on 21 rare diseases we track, including Amyotrophic Lateral Sclerosis, CDKL5 Deficiency Disorder and 19 more. It has 13 active clinical trials, and 9 are looking for volunteers now. It also has 7 FDA-approved rare disease drugs.

UCB is a Belgian pharmaceutical company headquartered in Brussels, founded in 1928 and listed on Euronext Brussels. It employs more than 9,000 people in about 40 countries and reported EUR 7.7 billion in revenue for 2025, most of it from immunology medicines like Bimzelx and Cimzia.

UCB pipeline

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

Approved
Drugs the FDA has approved.
Phase 3
Large studies, usually the last step before a company asks the FDA for approval.
Phase 2
Mid-size studies that check whether a drug works and at what dose.

Non-cystic Fibrosis Bronchiectasis recruiting

Phase 1
First studies in people, mostly checking safety.
After approval
Studies that follow people already taking an approved drug.
Other studies
Studies with no phase listed, such as registries and natural history studies.

Myasthenia Gravis recruiting · Ankylosing Spondylitis (AS) / Radiographic Axial SpA (r-axSpA) recruiting

UCB clinical trials

These are UCB 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.

9 trials looking for volunteers now

Looking for volunteersPhase 2Recently updatedNCT07720375

A Study of 24 to 52 Weeks Treatment to Evaluate The Efficacy And Safety of Galvokimig in Adult Study Participants With Non-Cystic Fibrosis Bronchiectasis

Tests Galvokimig, Placebo

For Non-cystic Fibrosis Bronchiectasis

The purpose of the study is to investigate the efficacy of galvokimig versus placebo on the time to the first pulmonary exacerbation in study participants with non-cystic fibrosis bronchiectasis (NCFB)

Ages 18 Years - 80 Years6 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT07463521

A Study to Evaluate the Efficacy and Safety of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis

Tests Rozanolixizumab, Placebo

For Ocular Myasthenia Gravis

The purpose of the study is to demonstrate the efficacy, safety and tolerability of rozanolixizumab compared with placebo in the treatment of adult study participants with Ocular Myasthenia Gravis.

Ages 18 Years+29 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT07465289

A Study to Evaluate The Long-Term Safety And Efficacy of Rozanolixizumab in Adult Participants With Ocular Myasthenia Gravis

Tests Rozanolixizumab

For Ocular Myasthenia Gravis

The purpose of the study is to assess the long-term safety and efficacy of rozanolixizumab in adult study participants with ocular myasthenia gravis.

Ages 18 Years+7 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT07503444

A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome

Tests fenfluramine hydrochloride, Placebo

For Rett Syndrome

The purpose of this study is to investigate the efficacy of fenfluramine hydrochloride (HCl) versus placebo in study participants with Rett syndrome (RTT).

Ages 5 Years - 35 Years33 locations
View full study
Looking for volunteersPhase 2/3Recently updatedNCT06055959

A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

Tests Zilucoplan

For Generalized Myasthenia Gravis

The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).

Ages 12 Years - 17 Years9 locations
View full study
Looking for volunteersPhase 4Recently updatedNCT07246564

Phase 4 Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Chinese Participants With Generalized Myasthenia Gravis

Tests Rozanolixizumab

For Myasthenia Gravis

The purpose of the study is to assess the clinical efficacy of rozanolixizumab in adult Chinese participants with generalized myasthenia gravis (gMG) in the first Treatment Cycle.

Ages 18 Years+12 locations
View full study
Looking for volunteersPhase 3Recently updatedNCT07215949

Zilucoplan for Severe gMG Exacerbations

Tests Zilucoplan®

For Generalized Myasthenia Gravis (gMG)

This is an open-label, multicenter, interventional phase 3b study in participants with AChR+ gMG and severe exacerbation that require hospitalization. Patients will receive subcutaneous zilucoplan injections daily for 12 weeks. Participation in the study will last for approximately 18 weeks.

Ages 18 Years+3 locations
View full study
Looking for volunteersHasn't posted an update in over a yearNCT06866483

Validation and Scaling of Screening Program for Undiagnosed Myasthenia Gravis-Social Media Campaign Paired With a Self-moderated Assessment

Tests Social media recruitment, Self-Assessment, Follow-up

For Myasthenia Gravis, Neuromuscular Disease and Neuromuscular Manifestations

This study expands and validates the pilot study NCT06381284. It is a fully remote, site-less, prospective, observational study enrolling adults in the United States (excluding U.S. territories) with undiagnosed neuromuscular symptoms. The primary objective is to determine the validity of a self-assessment tool in encouraging undiagnosed participants, recruited through a social media campaign, to seek medical evaluation for suspected myasthenia gravis (MG).

Ages 18 Years+1 location
View full study
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FDA-approved drugs from UCB

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

Briviact brivaracetam

For Focal Epilepsy. Approved Feb 18, 2016.

Fintepla fenfluramine

For Dravet Syndrome. Approved Jun 25, 2020.

Keppra levetiracetam

For Focal Epilepsy. Approved Nov 30, 1999.

Kygevvi doxecitine and doxribtimine

For Thymidine Kinase 2 Deficiency. Approved Nov 3, 2025.

Rystiggo rozanolixizumab

For Myasthenia Gravis. Approved Jun 26, 2023.

Vimpat lacosamide

For Focal Epilepsy. Approved Oct 28, 2008.

Zilbrysq zilucoplan

For Myasthenia Gravis. Approved Oct 17, 2023. Taken by subcutaneous route.

More about UCB

Its U.S. operations are based near Atlanta, with global clinical development run out of Research Triangle Park, North Carolina, and in March 2026 the company announced a $2 billion biologics plant in Georgia, its first U.S. manufacturing site. For the rare disease community, UCB matters because of two franchises: myasthenia gravis and severe childhood epilepsies.

In generalized myasthenia gravis, an autoimmune disease in which antibodies interfere with the signal between nerves and muscles, UCB launched two new medicines within four months of each other in 2023. Rystiggo (rozanolixizumab), approved June 26, 2023, blocks a receptor called FcRn so the body clears harmful IgG antibodies faster, and it was the first therapy approved for both anti-AChR and anti-MuSK antibody-positive patients.

Zilbrysq (zilucoplan), approved October 17, 2023, is a once-daily self-injection that blocks complement protein C5 for anti-AChR positive adults. Together they brought in EUR 549 million in 2025 and were being used by more than 3,700 patients by year end.

UCB is now running Phase 3 studies of rozanolixizumab in ocular myasthenia gravis (started mid-2026, results expected in 2029) and in MOG antibody-associated disease, a rare inflammatory condition of the optic nerves, brain, and spinal cord, where the fully enrolled cosMOG trial is expected to read out in the second half of 2027. Both myasthenia drugs are also in Phase 2/3 trials in children.

Keep reading about UCB

A newer approach, a T-cell engager called cizutamig that came with the $2 billion acquisition of Candid Therapeutics in June 2026, is planned to enter Phase 2 in myasthenia gravis by the end of 2026.

The epilepsy side of the business grew out of UCB's long history with Keppra, Vimpat, and Briviact for focal seizures, and its 2022 acquisition of Zogenix brought Fintepla (fenfluramine), approved for seizures in Dravet syndrome in 2020 and Lennox-Gastaut syndrome in 2022.

After the GEMZ Phase 3 study met its goal in 2025, UCB filed Fintepla for CDKL5 deficiency disorder with the FDA in March 2026, and a Phase 3 trial of fenfluramine in Rett syndrome started in 2026 with results expected in 2029.

On November 3, 2025, the FDA approved Kygevvi (doxecitine and doxribtimine), the first and only treatment for thymidine kinase 2 deficiency, a rare mitochondrial disease that causes progressive muscle weakness; in the pivotal analysis the therapy was associated with roughly an 86% reduction in the risk of death compared with matched untreated patients, and Europe followed with approval in March 2026.

UCB also acquired Neurona Therapeutics in June 2026 for its cell therapy rezanecel, which is expected to begin a Phase 3 trial in drug-resistant mesial temporal lobe epilepsy in the first half of 2027.

Not everything has worked. A Phase 2 study of rozanolixizumab in fibromyalgia was stopped in 2024 after missing its progression criteria, and a study in a rare autoimmune encephalitis was ended early. Bepranemab, an antibody against tau protein for Alzheimer's disease, missed the primary endpoint of its Phase 2 study, though UCB plans a follow-up trial in 2027 based on a signal in patients with lower tau levels.

Where UCB trials take place

The trials above run at 222 study sites in 29 countries. Tap a country to see its states, cities and hospitals.

United States
50▼
Japan
25▼
Spain
23▼
China
21▼
Italy
18▼
Germany
12▼
France
10▼
United Kingdom
10▼
Poland
8▼
Belgium
6▼
Turkey (Türkiye)
5▼
Georgia
4▼

Rare diseases UCB works on

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

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

CDKL5 Deficiency Disorder

CDKL5 deficiency disorder is a severe genetic condition that causes early-onset seizures, usually beginning within the first few months of life, along with significant developmental delays and...

How common: Approximately 1 in 40,000 to 60,000 live births

Chronic Inflammatory Demyelinating Polyneuropathy

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) is an autoimmune disorder affecting the peripheral nerves, causing progressive weakness and impaired function in the legs and arms.

How common: About 2.8 per 100,000 people in a 2019 pooled analysis of 9 studies (Broers et al., Neuroepidemiology); NORD estimates 5 to 7 per 100,000, and NIH's GARD estimates about 50,000 people 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

Focal Epilepsy

Focal epilepsy is a neurological disorder characterized by recurrent seizures that originate in a specific area of the brain.

How common: Approximately 1.5 million people in the U.S. have focal epilepsy; about 60% of all epilepsy cases

Gastroparesis

Gastroparesis is a condition in which the stomach empties food too slowly even though nothing is blocking it, causing early fullness, nausea, vomiting, bloating and upper belly pain.

How common: In a Minnesota population study cited by the NIDDK, about 10 men and 40 women per 100,000 had diagnosed gastroparesis

Generalized Pustular Psoriasis

Generalized pustular psoriasis (GPP) is the rarest form of psoriasis and behaves like a different disease.

How common: Roughly 2 to 7 new cases per million people a year worldwide (NORD); published estimates range from 2 to 124 per million, lower in Europe and higher in parts of Asia; about half of people with GPP also have ordinary plaque psoriasis

Immune Thrombocytopenia

Immune thrombocytopenia (ITP) is a rare autoimmune disorder in which the immune system destroys platelets, the blood cells that stop bleeding, and slows their production.

How common: Estimated 9 to 20 per 100,000 people; children often have a temporary form that resolves within months

Lennox-Gastaut Syndrome

Lennox-Gastaut Syndrome is a severe childhood epilepsy characterized by multiple types of seizures that are often resistant to medication, intellectual disability, and characteristic EEG findings...

How common: Approximately 1-2% of all childhood epilepsy cases; estimated 1 in 30,000 children

MOG Antibody-Associated Disease

MOG antibody-associated disease (MOGAD) is a rare autoimmune condition in which antibodies attack a protein on the myelin coating of nerves in the optic nerves, spinal cord and brain, causing...

How common: Roughly 0.5 to 3.4 people per 100,000 (Genentech); the MOG antibody is found in 40% to 58% of children with ADEM and in 7.4% to 39% of people with NMOSD who test negative for the aquaporin-4 antibody

Multiple Sclerosis

Multiple sclerosis is a chronic autoimmune disease where the immune system attacks the protective myelin sheath surrounding nerve fibers in the brain and spinal cord.

How common: Approximately 1 million people in the U.S.; about 1 in 300

Myasthenia Gravis

Myasthenia gravis is a chronic autoimmune neuromuscular disorder causing muscle weakness and rapid fatigue.

How common: 1 in 5,000 people

Narcolepsy Type 1

Narcolepsy type 1 is a chronic neurologic disorder caused by loss of hypocretin-producing neurons, resulting in severe daytime somnolence and cataplexy.

How common: 1 in 2,000 to 3,000 people; often undiagnosed

Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal Nocturnal Hemoglobinuria is a rare, life-threatening blood disorder where complement-mediated destruction of red blood cells occurs due to acquired mutations in the PIGA gene.

How common: Estimated at 1 to 5 per million people (MedlinePlus Genetics); NORD gives 0.5 to 1.5 per million

Primary Biliary Cholangitis

Primary Biliary Cholangitis (PBC) is a chronic autoimmune liver disease in which the immune system mistakenly attacks the small bile ducts in the liver, leading to bile duct destruction and...

How common: About 14.6 per 100,000 people worldwide in a 2021 pooled analysis of 47 population studies, highest in North America (21.8 per 100,000) and lowest in the Asia-Pacific region (9.8 per 100,000)

Progressive Supranuclear Palsy

Progressive supranuclear palsy is a rare atypical Parkinsonian disorder characterized by progressive vertical supranuclear gaze palsy, postural instability, and cognitive decline.

How common: About 6 in 100,000 people worldwide, though the exact number is unknown (MedlinePlus Genetics)

Rett Syndrome

Rett syndrome is a rare genetic neurological disorder affecting brain development, almost exclusively in girls.

How common: 1 in 9,000 to 15,000 girls

Sjögren's Syndrome

Sjögren's syndrome is a chronic autoimmune disorder in which the immune system attacks the moisture-producing exocrine glands, primarily the salivary and lacrimal (tear) glands.

How common: Estimated 1 to 4 million Americans (including undiagnosed); approximately 1 in 1,000 adults based on physician-diagnosed prevalence

Spinal Muscular Atrophy

Spinal muscular atrophy is an autosomal recessive genetic disorder affecting motor neurons in the spinal cord, causing progressive muscle weakness and atrophy.

How common: 1 in 10,000 to 14,000 births; 1 in 25 people are carriers

Thymidine Kinase 2 Deficiency

Thymidine Kinase 2 Deficiency (TK2d) is an ultra-rare inherited mitochondrial disease caused by mutations in the TK2 gene, which produces an enzyme essential for making and maintaining mitochondrial...

How common: Estimated worldwide prevalence is roughly 1.64 cases per 1,000,000 people, making TK2d an ultra-rare disease

Where to get support

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

Questions about UCB