American multinational pharmaceutical company headquartered in Wilmington, Delaware; develops and manufactures prescription biopharmaceutical medications across oncology, inflammation, and autoimmunity therapeutic areas.
Clinical Trials
721
293 active
Approvals
12
Total approvals
Agencies
2
Regulatory bodies
Founded
1991
Active, not recruiting
283
39.3%
Approved For Marketing
3
0.4%
Available
2
0.3%
Completed
157
21.8%
No Longer Available
5
0.7%
Not yet recruiting
10
1.4%
Recruiting
181
25.1%
Terminated
66
9.2%
Withdrawn
14
1.9%
- The US FDA approved Mirum Pharmaceuticals' once-daily pill zilurgisertib, branded Atebrioz, for patients aged 12 and older with fibrodysplasia ossificans progressiva. - Atebrioz blocks ALK2, a protein abnormally active in most FOP patients that drives bone formation outside the skeleton, at a recommended 100 milligram dose. - Approval was based on a 63-patient study in which Atebrioz significantly reduced new bone formation versus placebo at week 24. - Mirum expects to launch the drug in October, with the company having licensed it from Incyte earlier in 2026 for US$16 million upfront plus milestones and royalties.
- The FDA approved Eli Lilly's once-daily oral JAK inhibitor Olumiant (baricitinib) for pediatric patients 12 years and older with severe alopecia areata, expanding its existing US label. - The decision rests on 36-week data from BRAVE-AA-PEDS, the first and largest Phase 3 trial designed specifically for pediatric severe alopecia areata. - At Week 36, 42% of patients on Olumiant 4 mg and 27% on 2 mg achieved at least 80% scalp hair coverage versus 5% on placebo. - The pediatric safety profile matched that seen in adults with severe alopecia areata, and the label carries a boxed warning for serious infections, mortality, malignancy, MACE and thrombosis.
- The FDA approved Atebrioz (zilurgisertib) tablets at 100 mg once daily to reduce total new heterotopic ossification volume in patients aged 12 and older with FOP. - In the Phase 2 PROGRESS study, mean total new HO lesion volume fell 3.2 cm3 with zilurgisertib versus a 24.6 cm3 increase with placebo at Week 24. - Zilurgisertib was generally well tolerated, with headache, arthralgia, upper respiratory tract infection, epistaxis and nausea as the most common adverse reactions. - Mirum licensed zilurgisertib from Incyte for worldwide development; an EMA marketing authorization application is under review and pediatric cohorts continue enrolling.
- Ryvu Therapeutics SA has terminated its Phase 2 POTAMI-61 study of RVU120 in intermediate- or high-risk primary or secondary myelofibrosis. - The open-label trial tested oral RVU120 as monotherapy and combined with the JAK inhibitor ruxolitinib, with no placebo arm and non-random allocation. - The study record, first submitted 2024-04-29 and last updated 2026-09-15, is now listed as terminated before planned completion, pressuring the blood cancer pipeline.
- China's NMPA approved Lumirix® (ruxolitinib phosphate cream) on 2 September 2026 for mild-to-moderate atopic dermatitis in non-immunocompromised adults and pediatric patients aged 2 years and older. - In a Phase III trial, Lumirix® met its primary endpoint with 63.0% of patients achieving IGA 0/1 versus 9.2% for placebo (P < 0.001), and EASI 75 in 78.0% versus 15.4% (P < 0.001). - The approval expands Lumirix® beyond its January 2026 vitiligo indication, making it the first topical JAK inhibitor approved in China for vitiligo. - The indication addresses a large unmet need, with over 54 million AD patients in China as of 2024, of whom mild-to-moderate cases represent over 52.5 million.
- Knight Therapeutics submitted a supplemental application to ANVISA seeking approval for MINJUVI (tafasitamab) plus lenalidomide added to R-CHOP as first-line treatment for previously untreated DLBCL and HGBL. - The supplemental application was selected for review under Project Orbis, reflecting a coordinated regulatory pathway across participating agencies. - The submission builds on results from the Phase 3 frontMIND trial, which enrolled 899 adults and evaluated tafasitamab plus lenalidomide added to R-CHOP versus R-CHOP alone. - The filing addresses a significant unmet need, as roughly 40% of DLBCL patients do not respond to initial therapy or relapse thereafter.
- A Nature correspondence argues that pre-clinical data interpretation must be grounded in clinical context to avoid misleading translational conclusions. - The exchange involves researchers from The Institute of Cancer Research, The Royal Marsden NHS Foundation Trust, and The University of Chicago. - The reply clarifies that the Matters Arising discussion concerns the broader clinical implications of findings rather than mechanistic aspects. - Authors disclose extensive industry relationships, including consulting, funding, and patent interests relevant to radiation oncology and cancer research.
- Minjuvi (tafasitamab) combined with rituximab and lenalidomide becomes the first chemotherapy-free CD19/CD20 dual-targeted immunotherapy listed on Australia's PBS for relapsed or refractory follicular lymphoma. - The pivotal Phase 3 inMIND trial demonstrated a 57% reduction in risk of disease progression, relapse, or death, with median PFS of 22.4 months versus 13.9 months in the control arm (HR: 0.43; P<0.0001). - This marks the first new therapy reimbursed on the PBS for relapsed or refractory follicular lymphoma in nine years, effective 1 August 2026. - Follicular lymphoma affects over 10,000 Australians, with approximately 1,500 new diagnoses annually, representing the second most common form of non-Hodgkin lymphoma.
- The AI Protein Design Market was valued at USD 1.58 billion in 2025 and is projected to reach USD 13.70 billion by 2035, growing at a 24.1% CAGR. - Drug Discovery & Therapeutics dominated applications with a 59.30% share in 2025, while De Novo Protein Generation is the fastest-growing technology segment at a 30.60% CAGR. - Isomorphic Labs closed a USD 2.1 billion Series B round, and Chai Discovery signed a licensing agreement with Pfizer for its de novo antibody design model Chai-3. - North America led the market with a 39.60% regional share in 2025, while Asia Pacific is expected to grow fastest at a 28.30% CAGR.
- The CHMP has issued a positive opinion recommending EU approval of Acadia's Daybue (trofinetide) for Rett syndrome following a successful re-examination after an initial rejection in February. - If approved by the European Commission, Daybue would become the first therapy specifically authorized in the EU for the neurobehavioral symptoms of Rett syndrome. - Acadia's Q1 2025 Daybue revenue reached $101 million, representing 20% year-over-year growth, and the company's 2028 sales guidance of $700 million includes European revenue. - The FDA first approved Daybue for Rett syndrome in pediatric and adult patients in 2023, and the drug faces little to no real competition in this indication.
