Orphan Drug Weekly | Novartis Pancreatic Cancer Antibody Therapy, Bristol-Myers Squibb Protein Degradation Therapy Finalists

Last week, the FDA issued a total of 4 orphan drug qualifications, including 2 small molecule targeted drugs and 2 monoclonal antibodies.

Drug: Cereblon E3 ligase modulator

Research and development company: Bristol-Myers Squibb/Xinji

Treatment of diseases: Acute myeloid leukemia (AML)

Introduction: Cereblon E3 ligase modulator has immunomodulatory and pro-apoptotic activities.

Drug: canakinumab

R&D company: Novartis

Treatment of disease: Pancreatic cancer

Introduction: Canakinumab is a humanized monoclonal antibody that selectively binds to human interleukin 1β (IL-1β) with high affinity and blocks the activity of IL-1β.

Drug: cannabidiol

R&D company: Biom Therapeutics

Treatment of diseases: Angelman syndrome (AS)

Introduction: Angel syndrome (AS) is a neurodevelopmental disorder caused by abnormal expression or functional defects of the UBE3A gene in the 15q11-13 chromosome region of maternal origin.

Reference materials:

[1] Pubchem.

[2] Novartis.

[3] Biom Therapeutics.