Pipeline Report February 2021 Pipeline Report February 2021 © 2021 Envolve. All rights reserved. Page 1 This quarterly at-a-glance publication is developed by our Clinical Pharmacy Drug Information team to increase your understanding of the drug pipeline, Table of Contents ensuring you’re equipped with insights to prepare for shifts in pharmacy benefit management. In this issue, you’ll learn more about key themes and notable drugs referenced in the following points. COVID-19 1 > Veklury is currently the only agent that is FDA-approved for the treatment of COVID-19. Three additional therapeutics and two vaccines have been granted Emergency Use Authorization (EUA), and at least three more vaccines are Recent Specialty Drug Approvals1 4 expected to receive an EUA in the relatively near future. > The previous quarter noted the approval of several breakthrough therapies for rare or ultra-rare conditions, which previously had no available FDA-approved Recent Non-Specialty Drug Approvals 9 treatments — Zokinvy for Hutchinson-Gilford progeria syndrome and progeroid laminopathies, Oxlumo for primary hyperoxaluria type 1, and Imcivree for genetically mediated obesity. Upcoming Specialty Products 10 > Other notable approvals include: Lupkynis — the first oral therapy approved for lupus nephritis; Orladeyo — the first oral therapy approved as prophylaxis of hereditary angioedema attacks;Cabenuva – the first long-acting injectable antiretroviral therapy intended as maintenance treatment of HIV; and Breyanzi — Upcoming Non-Specialty Products 18 the third CAR-T therapy to market for treatment of large B-cell lymphomas, with a favorable adverse effect profile relative to the two previously approved agents Kymriah and Yescarta. > Other potentially impactful FDA approvals are on the horizon, including teplizumab which would be the first Biosimilars 20 disease-modifying therapy for delaying the onset of type 1 diabetes in high-risk patients, arimoclomol as the first approved therapy for Niemann-Pick disease type C, andcasimersen as the first treatment for Duchenne muscular dystrophy that has mutations amenable to exon 45 skipping. Meanwhile, the FDA’s three-month Generic Specialty Agents 22 delay on the approval decision for aducanumab for mild or prodromal Alzheimer’s disease hints at the willingness to continue its review of the drug, providing hope for the therapy to ultimately be approved. Generic Non-Specialty Agents 23 To prepare this report, our team accesses a wide range of clinical resources. This information is then analyzed, resulting in updates across multiple disease states including recent and anticipated drug approvals, key changes in the biosimilar agent landscape, and notes on recent and anticipated generic product launches. Our pipeline report is just one of many ways we’re committed to providing helpful tools and resources to our clients and partners. We Data is current as of 2.22.2021 look forward to sharing more updates with you in the months ahead. Ross Hoffman, MD To provide comments, feedback or requests for report enhancements, please email us at [email protected]. Pipeline Report COVID-19 February 2021 FDA-APPROVED AGENTS FDA Drug Name Manufacturer(s) Approval Therapeutic Class Comments Cost (WAC) Date • Indicated for the treatment of adults and pediatric patients $520 for a 100 mg ≥ 12 years old and weighing ≥ 40 kg requiring hospitalization for single dose vial Veklury COVID-19 $3,120 for a 5-day remdesivir Gilead 10/22/20 Treatment - antiviral course intravenous infusion • Veklury should only be administered in a hospital or healthcare setting capable of providing acute care comparable to inpatient $5,720 for a 10-day hospital care course AGENTS GRANTED FDA EMERGENCY USE AUTHORIZATION (EUA) EUA Drug Name Manufacturer(s) Approval Therapeutic Class Comments Cost (WAC) Date Original: • Current EUA is for the use of high titer convalescent plasma for Convalescent U.S. Dept of Health and 8/23/20 Treatment - blood the treatment of hospitalized patients early in the disease course plasma Unknown at this time Human Services Revised: product and to those hospitalized patients who have impaired humoral intravenous infusion 2/4/21 immunity and cannot produce an adequate antibody response • EUA is for the treatment of mild to moderate COVID-19 in adults and pediatric patients with positive results of direct SARS-CoV-2 $1,250 single dose LY-CoV555 viral testing who are ≥ 12 years old and weighing ≥ 40 kg, and Treatment - infusion bamlanivimab Eli Lilly 11/9/20 who are at high risk for progressing to severe COVID-19 and/or monoclonal antibody $309.60 intravenous infusion hospitalization administration cost • Outpatient only - no cost to patients, although healthcare facilities may charge fees related to administration • EUA for use in combination with Gilead’s Veklury (remdesivir) for treating hospitalized patients with COVID-19 infection in patients Olumiant Treatment - acute aged two years or older, with suspected or laboratory confirmed $2,553 for 14-day baricitinib Eli Lilly 11/19/20 respiratory distress COVID-19 requiring supplemental oxygen, invasive mechanical course oral tablet syndrome (ARDS) ventilation, or extracorporeal membrane oxygenation $91.17 per 2 mg tablet • 4 mg once daily for 14 days or until hospital discharge © 2021 Envolve. All rights reserved. Continued Page 1 Pipeline Report COVID-19 February 2021 EUA Drug Name Manufacturer(s) Approval Therapeutic Class Comments Cost (WAC) Date • EUA is for the treatment of mild to moderate COVID-19 in adults and pediatric patients with positive results of direct SARS-CoV-2 REGN-COV2 viral testing who are ≥ 12 years old and weighing ≥ 40 kg, and $1,500 single dose casirivimab + Treatment - infusion Regeneron 11/21/20 who are at high risk for progressing to severe COVID-19 and/or imdevimab monoclonal antibody hospitalization $309.60 intravenous infusion administration cost • Outpatient only - no cost to patients, although healthcare facilities may charge fees related to administration BNT162b2 • mRNA vaccine that expresses SARS-CoV2 spike protein tozinameran Pfizer 12/11/20 Vaccine $19.50 per dose* intradermal injection • No cost to patients mRNA 1273 • mRNA vaccine that expresses SARS-CoV2 spike protein Moderna 12/18/20 Vaccine $32-37 per dose* intradermal injection • No cost to patients • EUA is for the emergency use of bamlanivimab and etesevimab administered together for the treatment of mild to moderate bamlanivimab: $1,250 LY-CoV555 + LY- coronavirus disease 2019 (COVID-19) in adults and pediatric single dose infusion CoV016 Treatment - patients ( ≥ 12 years of age and weighing ≥ 40 kg) with positive etesevimab: pending bamlanivimab + Eli Lilly 2/9/21 monoclonal antibody results of direct SARS-CoV2 viral testing, and who are at high risk launch etesivimab for progressing to severe COVID-19 and/or hospitalization $309.60 intravenous infusion • Outpatient only - no cost to patients, although healthcare administration cost facilities may charge fees related to administration PIPELINE AGENTS Anticipated Date of Drug Name Manufacturer(s) Therapeutic Class Comments Cost (WAC) EUA or FDA Approval • Non-replicating adenovirus 26-vector dsDNA expressing SARS- Ad26.COV2-S Projected $10 per J&J/Janssen 2/26/21 Vaccine CoV-2 spike protein intradermal injection dose* • No cost to patients *Initially, the federal government will supply the vaccine at no cost to providers. © 2021 Envolve. All rights reserved. Page 2 Pipeline Report COVID-19 February 2021 Anticipated Date of Drug Name Manufacturer(s) Therapeutic Class Comments Cost (WAC) EUA or FDA Approval • Non-replicating chimpanzee adenovirus-vector dsDNA expressing AZD 1222 AstraZeneca Q1 2021 Vaccine SARS-CoV-2 spike protein Projected $3 per dose* intradermal injection • No cost to patients NVX-CoV2373 • SARS-CoV-2 spike glycoprotein nanoparticle + adjuvant vaccine Projected $16 per Novavax Q2 2021 Vaccine intradermal injection • No cost to patients dose* © 2021 Envolve. All rights reserved. Page 3 Pipeline Report Recent Specialty Drug Approvals February 2021 FDA AcariaHealth and Drug Name Manufacturer(s) Indication(s) Approval PANTHERx Access Comments Cost (WAC) Date Status CARDIOVASCULAR • Approved for use as an adjunct to other low- density lipoprotein-cholesterol (LDL-C) lowering Evkeeza Homozygous familial therapies for the treatment of adult and evinacumab-dgnb Regeneron hypercholesterolemia 2/11/21 Pending pediatric patients aged ≥ 12 years $450,000/year intravenous infusion (HoFH) • Administered as monthly intravenous infusions • Repatha, Juxtapid, and Kynamro are also FDA- approved for HoFH ENDOCRINOLOGY • Approved for use in patients ≥ 12 months of age with a body surface area of ≥ 0.39 m2 for either Hutchinson-Gilford progeria syndrome, or for processing-deficient Progeroid laminopathies with either: heterozygous LMNA mutation Hutchinson-Gilford with progerin-like protein accumulation or Zokinvy progeria syndrome homozygous or compound heterozygous $785,000- lonafarnib Merck 11/20/20 Pending ZMPSTE24 mutations $1,300,000/year oral capsule Progeroid laminopathies • Progeria is an ultra-rare and fatal disease that causes premature aging in children • There are currently no other FDA approved therapies • Projected impact: cost increase • Administered as monthly injections AcariaHealth: limited • Current treatment options for advanced disease Oxlumo 15 kg child: access are very limited, and include frequent renal lumasiran Primary hyperoxaluria $440,000/year Alnylam 11/23/20 dialysis or combined