Pipeline review

DRUG NAME/ MANUFACTURER	PROPOSED USE	HOW IT WORKS	WHAT’S IMPORTANT
vericiguat / Bayer/Merck	Chronic heart failure	Soluble guanylate cyclase stimulator for oral use in combination with other heart failure therapies to reduce the risk of cardiovascular death and heart failure hospitalization.	Route of administration: Oral Benefit coverage: Pharmacy Anticipated FDA decision: 1/20/2021 Cost Estimate: Unknown
casimersen / Sarepta	Duchenne muscular dystrophy (DMD)	In DMD, a mutation in the gene for dystrophin, a muscle protein, causes progressive muscle wasting. Exons are bits of DNA or RNA that contain the information needed to produce specific proteins. Bypassing (skipping) the defective exon, allows a partly-working form of dystrophin to assemble. Casimersen would be indicated for those with genetic mutations amenable to skipping exon 45 of the Duchenne gene.	Route of administration: Intravenous infusion Benefit coverage: Medical, Specialty Anticipated FDA decision: 2/25/2021 Cost Estimate: $300,000+ per patient per year
aducanumab / Biogen/Eisai	Alzheimer's disease	Monoclonal antibody for the treatment of Alzheimer's disease that targets beta amyloid. One of the pathological hallmarks of Alzheimer's disease are beta amyloid plaques which cause atrophy of the brain and surrounding blood vessels.	Route of administration: Intravenous infusion Benefit coverage: Medical, Specialty Anticipated FDA decision: 3/7/2021 Cost Estimate: $50,000-$100,000 per patient per year
idecabtagene vicleucel (Ide-cel) / Bluebird Bio/Bristol Myers Squibb	Multiple myeloma	CAR-T therapy for treatment of patients with relapsed or refractory aggressive large B-cell non-Hodgkin lymphoma. One time IV infusion of the patient's own genetically modified T-cells.	Route of administration: Intravenous infusion Benefit coverage: Medical, Specialty Anticipated FDA decision: 3/29/2021 Cost Estimate: $475,000 per patient
abrocitinib / Pfizer	Atopic dermatitis	Janus kinase 1 (JAK1) inhibitor for the treatment of patients with moderate-to-severe atopic dermatitis	Route of administration: Oral Benefit coverage: Pharmacy, Specialty Anticipated FDA decision: 4/25/2021 Cost Estimate: $55,000 per patient per year
tralokinumab / LEO Pharma	Atopic dermatitis	Anti-interleukin-13 for the treatment of moderate to severe atopic dermatitis. Atopic dermatitis (AD) is a chronic skin disease characterized by inflammation of the skin and skin barrier defects.	Route of administration: Subuctaneous injection Benefit coverage: Pharmacy, Specialty Anticipated FDA decision: 4/27/2021 Cost Estimate: $40,000 per patient per year
pegunigalsidase alfa / Protalix BioTherapeutics	Fabry disease	Enzyme replacement therapy designed to be a more stable enzyme than currently marketed enzyme replacement therapies	Route of administration: Intravenous infusion Benefit coverage: Medical, Specialty Anticipated FDA decision: 4/27/2021 Cost Estimate: $300,000 per patient per year

Notes:
Benefit coverage is based on currently available information and could change pending final FDA-approved prescribing information.