Pipeline review

semaglutide /
Novo Nordisk
Treatment of diabetes mellitus, type II This form of semaglutide is an oral version of Ozempic, an injectable insulin analogue given weekly. Oral semaglutide will be dosed once daily and may be preferred as an alternative to weekly self-injections. Route of administration: Oral
Benefit coverage: Pharmacy
Anticipated FDA decision: 9/2019
US sales forecast in 2024: 2,252M
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: $490M
bempedoic acid /
Second-line treatment of individuals with elevated cholesterol (LDL)PM Bempedoic acid is the first cholesterol-lowering drug that works by inhibiting ATP citrate lyase (ACL), which results in reduced production of fatty acids and cholesterol. It's role as a second-line agent for treating individuals who do not achieve an adequate reduction in LDL despite the use of current treatments may depend on the product's pricing compared to the PCSK9 inhibitors Praluent and Repatha. If approved, Esperion also plans to launch a combination product that includes bempedoic acid with ezetimibe/Zetia. Route of administration: Oral
Benefit coverage: Pharmacy
Anticipated FDA decision: 1Q2020
US sales forecast in 2024: $1,021M
idecabtagene /
bluebird bio
Treatment of advanced multiple myeloma that has failed or not responded to other therapies Idecabtagene is a chimeric antigen receptor T-cell therapy (CAR-T) immuno-oncology agent.PM CAR-T therapy is a new type of immuno-oncology that harnesses patient’s own immune system to eliminate cancer cells. If approved, it will be the first CAR-T therapy for use in multiple myeloma. Route of administration: IV injection
Benefit coverage: Medical
Anticipated FDA decision: Mid-2020
US sales forecast in 2024: $378M
valoctocogene 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: Mid-2020
US sales forecast in 2024: $659M

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