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Pipeline

We see innovation as essential to our vision of being a global leader in rare diseases. We need to reinvest in research and development to continue providing innovative treatments, creating new evidence, and expanding the use of our medicines.

Major ongoing clinical studies and medicines in registration in a major region or country.

Updated: 16 July 2025.

Name
Indication
Clinical phase
Study
Aspaveli/Empaveli
pegcetacoplan
C3G and primary IC-MPGN, C3 glomerulopathy and immune-complex membranoproliferative glomerulonephritis
Registration
Registration based on VALIANT study
Doptelet
avatrombopag
Immune thrombocytopenia, ITP
Registration
Registration in Japan and for paediatric indication in US
Gamifant
emapalumab
Interferon-gamma-driven sepsis – IDS
Phase 2
EMBRACE
Gamifant
emapalumab
Cytokine release syndrome (CRS) prophylaxis in CAR-T therapy
Phase 2
Kineret
anakinra
Still’s disease
Phase 3
In Japan
NASP
formerly SEL-212
Uncontrolled gout
Registration
DISSOLVE I and DISSOLVE II
Vonjo
pacritinib
VEXAS (Vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic)
Phase 2
PAXIS
Vonjo
pacritinib
Myelofibrosis with severe thrombocytopenia
Phase 3
PACIFICA (confirmatory study)
Vonjo
pacritinib
Chronic myelomonocytic leukemia (CMML)
Phase 2
Zynlonta
loncastuximab tesirine
DLBCL, Diffuse large B-cell lymphoma (confirmatory & extension into second line or earlier in combination)
Phase 3
LOTIS 5