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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: 26 October 2026.

Name
Indication
Clinical phase
Study
Altuvoct
efanesoctocog alfa
Synovitis in people with haemophilia A on efanesoctocog alfa prophylaxis.
Phase 4
SHINE
Gamifant
emapalumab
Sepsis
Phase 2
EVEREST
Gamifant
emapalumab
Cytokine release syndrome (CRS) prophylaxis in CAR-T therapy
Phase 2
Gamifant
emapalumab
HLH/MAS in Still's disease
Registration
In Europe
Gamifant
emapalumab
Malignancy-associated HLH (mHLH)
Phase 3
SAPPHIRE
Lacutamab
Cutaneous T-cell lymphoma (CTCL) and Sézary syndrome
Phase 2
NASP
nanoencapsulated sirolimus plus pegadricase
Uncontrolled gout
Registration
DISSOLVE I & DISSOLVE II
Pozdeutinurad
AR882
Progressive gout
Phase 3
REDUCE 1 & REDUCE 2
Tryngolza
olezarsen
Severe hypertriglyceridemia (sHTG)
Registration
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