Selected products in development
Novartis is consistently rated as having one of the industry’s most respected development pipelines, with more than 160 projects in clinical development, as of December 31, 2019. We highlight some promising programs, including new molecules and existing treatments that are under investigation for new indications.
AVXS-101 (onasemnogene abeparvovec, approved in the US as Zolgensma) is a gene replacement therapy for spinal muscular atrophy. It targets the defective or missing gene that causes this fatal disease.
MBG453 is an investigational treatment for myelodysplastic syndrome.
Kymriah is a CAR-T therapy that genetically reprograms a patient’s immune cells to fight certain types of cancer. Kymriah is approved for B-cell acute lymphoblastic leukemia and diffuse large B-cell lymphoma, and is in development for other blood cancers.
SEG101 (crizanlizumab, approved in the US as Adakveo) is a treatment to reduce the frequency of vaso-occlusive crises (VOCs) in patients with sickle cell disease. Novartis continues to study SEG101 for the treatment of VOCs and other complications.
LJN452 (tropifexor) is an investigational treatment for the liver disease nonalcoholic steatohepatitis (NASH). LJN452 is designed to break the cycle of fatty buildup in the liver and harness the body’s built-in mechanisms for coping with excess bile acid.
KAF156 (ganaplacide) is a treatment in development for malaria. This new class of molecules has the potential to clear malaria infections and block parasite transmission.
ZPL389 (adriforant) is an investigational treatment for atopic dermatitis.
CFZ533 (iscalimab) is an investigational treatment for preventing graft rejection in transplant patients and for the treatment of several autoimmune diseases.
177Lu-PSMA-617 is an investigational treatment for metastatic castration-resistant prostate cancer.
SAF312 is a treatment in development for chronic ocular surface pain.
UNR844 is an investigational treatment for presbyopia, the age-related loss of near-distance vision.
ACZ885 (canakinumab) is an antibody treatment in development for non-small cell lung cancer. ACZ885 is approved under the name Ilaris for various inflammatory conditions, including cryopyrin-associated periodic syndrome and some forms of arthritis.
BYL719 (alpelisib, approved in the US as Piqray) is a therapy for use in combination with fulvestrant for the treatment of postmenopausal women, and men, with HR+/HER2-, PIK3CA-mutated, advanced or metastatic breast cancer. It is also in development for other related cancers.
TQJ230 is an investigational treatment for the secondary prevention of cardiovascular events in patients with elevated levels of lipoprotein(a).
LNP023 is a treatment in development for a range of renal diseases.
For more information about the pipeline and progress on individual development programs