Eli Lilly and Company
Eli Lilly and Company works on 37 rare diseases tracked on Trial Friend, including Amyotrophic Lateral Sclerosis, Bullous Pemphigoid, Cholangiocarcinoma and 34 more, with 9 active clinical trials (2 recruiting) and 4 FDA-approved rare disease drugs.
Eli Lilly is a global pharmaceutical company with a growing presence in rare oncology, anchored by its 2019 acquisition of Loxo Oncology. Its rare disease portfolio includes Retevmo (selpercatinib) for RET-driven cancers such as medullary thyroid cancer and RET fusion-positive solid tumors, and Jaypirca (pirtobrutinib) for relapsed or refractory mantle cell lymphoma and chronic lymphocytic leukemia after earlier treatment that included a BTK inhibitor. Lilly also has active trials in ALS, immune thrombocytopenia and, through its Prevail Therapeutics unit, Gaucher disease.
Eli Lilly and Company Drug Pipeline
Eli Lilly and Company has 9 active clinical trials across 3 development stages, with 2 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.
Eli Lilly and Company Clinical Trials (9)
Active and recruiting clinical trials sponsored by Eli Lilly and Company, 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.
Eli Lilly and Company FDA-Approved Drugs (4)
Medications developed or marketed by Eli Lilly and Company 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.
Eli Lilly and Company Trial Locations
Eli Lilly and Company clinical trials are running at 559 sites in 31 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 (37)
Diseases targeted by Eli Lilly and Company'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...
Bullous pemphigoid is an autoimmune blistering disorder caused by autoantibodies against basement membrane proteins, particularly BP180 and BP230. The condition leads to formation of large fluid-fille...
Cholangiocarcinoma is a rare and aggressive cancer that forms in the bile ducts, the thin tubes that carry digestive fluid (bile) from the liver to the small intestine. It can occur inside the liver (...
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...
Dermatomyositis is a rare autoimmune inflammatory disease causing muscle weakness and distinctive skin rashes, particularly over joints. It involves inflammation of muscles and skin blood vessels. Abo...
Desmoplastic small round cell tumor is a rare aggressive sarcoma characterized by EWSR1-WT1 fusion and dense desmoplasia. Most commonly arising in the peritoneum of adolescents and young adults, the c...
Patient Resources
Organizations and resources related to Eli Lilly and Company's rare disease focus areas
Frequently Asked Questions About Eli Lilly and Company
Common questions about Eli Lilly and Company's rare disease programs, clinical trials, and treatments.