June 2019

Pipeline review

DRUG NAME/ MANUFACTURER PROPOSED USE HOW IT WORKS WHAT’S IMPORTANT
bb2121 / bluebird bio Treatment of advanced multiple myeloma that has failed or not responded to other therapies bb2121 is a chimeric antigen receptor T-cell therapy (CAR-T) immune-oncology agent.  CAR-T therapy is a new type of immuno-oncology that harnesses patient’s own immune system to eliminate cancer cells. Route of administration: IV injection
Benefit coverage: Medical
Anticipated FDA decision: Early 2020
US sales forecast in 2024: $378M
luspatercept / Acceleron Pharma, Inc. Treatment of Beta-Thalassemia, an ultra-rare genetic blood disorder that causes severe anemia Luspatercept is the first treatment designed to repair the red blood cell (RBC) production defect that causes Beta-Thalassemia and restore RBC production. It works by targeting specific proteins involved in late-stage red blood cell production and significantly reducing or eliminating the need for frequent and lifelong blood transfusions. Route of administration: Subcutaneous (SC) self-administered injection
Benefit coverage: Pharmacy
Anticipated FDA decision: 4Q2019
US sales forecast in 2024: $544M
valoctogene roxaparvovec / BioMarin Pharmaceutical, Inc. Treatment of hemophilia A Valoctogene is a one-time gene therapy treatment designed to replace the missing gene that causes hemophilia A, thereby restoring normal blood clotting function and eliminating the need for lifelong factor replacement therapy. Route of administration: IV injection
Benefit coverage: Medical
Anticipated FDA decision: Early 2020
US sales forecast in 2024: $657M
aducanumab / Biogen, Inc. Treatment and/or prevention of early Alzheimer’s Disease The memory loss and functional decline of Alzheimer’s Disease has been linked to the formation of amyloid plaques, abnormal protein deposits that build up in the brain.  Aducanumab binds to and may reduce amyloid plaques from the brain, potentially slowing the progress of Alzheimer’s Disease.  Clinical studies of aducanumab have included individuals between the ages of 50 and 85 with mild to moderate cognitive impairment. Route of administration: IV injection
Benefit coverage: Medical
Anticipated FDA decision: 2020
US sales forecast in 2024: $1.9B

"Notes:
U.S. sales forecast provided by EvaluatePharma. www.evaluatepharma.com Accessed June 17, 2019
Benefit coverage is based on currently available information and could change pending final FDA-approved prescribing information.

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