UCB
UCB works on 19 rare diseases tracked on Trial Friend, including Amyotrophic Lateral Sclerosis, CDKL5 Deficiency Disorder, Chronic Inflammatory Demyelinating Polyneuropathy and 16 more, with 13 active clinical trials (9 recruiting) and 8 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. 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. 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.
UCB Drug Pipeline
UCB has 13 active clinical trials across 5 development stages, with 9 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.
UCB Clinical Trials (13)
Active and recruiting clinical trials sponsored by UCB, 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.
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UCB FDA-Approved Drugs (8)
Medications developed or marketed by UCB 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 |
|---|---|---|---|
| BIMEKIZUMAB | Bimzelx subcutaneous | Oct 17, 2023 | |
| BRIVARACETAM | BRIVIACT | Feb 18, 2016 | |
| FENFLURAMINE | Fintepla oral | Jun 25, 2020 | |
| LEVETIRACETAM | KEPPRA | Nov 30, 1999 | |
| DOXECITINE AND DOXRIBTIMINE | KYGEVVI oral | Nov 3, 2025 |
UCB Trial Locations
UCB clinical trials are running at 205 sites in 29 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 UCB's clinical trial and drug development programs
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...
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 movemen...
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) is an autoimmune disorder affecting the peripheral nerves, causing progressive weakness and impaired function in the legs and arms. The immune ...
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...
Dravet Syndrome is a severe form of epilepsy that typically begins in infancy with prolonged seizures triggered by fever. Most cases are caused by mutations in the SCN1A gene encoding a sodium channel...
Focal epilepsy is a neurological disorder characterized by recurrent seizures that originate in a specific area of the brain. It is the most common form of epilepsy in adults. Seizures may involve alt...
Patient Resources
Organizations and resources related to UCB's rare disease focus areas
Frequently Asked Questions About UCB
Common questions about UCB's rare disease programs, clinical trials, and treatments.