According to the FDA, 53 new drugs received approval in 2020.¹ As the coronavirus disease 2019 (COVID-19) pandemic persists throughout the United States, the FDA is focusing on treatment options for COVID-19. The FDA also issued guidance for the industry regarding conducting clinical trials for investigational drugs during the pandemic to ensure the safety of study participants and the integrity of the data.²

According to a Vizient Drug Price Forecast report, health systems can expect a 3.29% increase for pharmaceutical purchases made from January 1 to December 31, 2021.³ Additionally, a variety of specialty drugs are expected to be approved in 2021.³ Pharmacists should stay up-to-date with the latest drug information so that they are well versed about pipeline drugs for 2021.

Top Pipeline Drugs for 2021

COVID-19 Treatments

On November 21, 2020, the FDA issued an emergency use authorization (EUA) for the monoclonal antibodies casirivimab and imdevimab for the treatment of mild-to-moderate COVID- 19 in adults and pediatric patients 12 years and older weighing at least 40 kg who are at risk of progressing to severe COVID-19 and/or hospitalization.⁴ This therapy should not be used in patients hospitalized due to COVID-19 or who require oxygen therapy due to COVID- 19.⁴ The dose is 1200 mg of casirivimab and 1200 mg of imdevimab administered together as a single intravenous (IV) infusion over at least 60 minutes.⁵ Casirivimab and imdevimab are currently being investigated in the phase 3 RECOVERY trial (NCT04381936).^6,7

The investigational monoclonal antibody bamlanivimab received an EUA on November 9, 2020, for the treatment of mild-to-moderate COVID-19 in adults and pediatric patients 12 years and older weighing at least 40 kilograms who are at high risk of severe COVID-19 and/or hospitalization.⁸ Bamlanivimab is administered as a 700 mg IV infusion over at least 60 minutes, and should not be used in patients hospitalized due to COVID-19 or who require oxygen therapy due to COVID-19.⁸

Additionally, bamlanivimab is currently being investigated in the phase 2/3 ACTIV-2 trial (NCT04518410) in patients with mild-to-moderate COVID-19 who do not require hospitalization and the BLAZE-2 study (NCT04497987) for the prevention of COVID-19 in residents and staff at long-term care facilities.^9,10 The ACTIV-3 clinical trial (NCT04501978) revealed that bamlanivimab was unlikely to help hospitalized COVID-19 patients.¹¹

HIV Prevention

Cabotegravir is an investigational, long-acting medication that was recently granted breakthrough therapy designation by the FDA for HIV pre-exposure prophylaxis (PrEP).¹² This designation was based on the efficacy and safety results from HPTN 083 (NCT02720094). HPTN 083 was a phase 2b/3 randomized, multicenter, double-blind trial that compared long-acting, injectable cabotegravir with daily oral emtricitabine 200 mg and tenofovir disoproxil fumarate 300 mg (Truvada) for HIV prevention.¹² The results showed that cabotegravir was 66% more effective than emtricitabine/tenofovir at preventing HIV. Cabotegravir is given as an intramuscular injection every 8 weeks, which could offer another option that may enhance adherence.¹² Trial safety results showed that cabotegravir was well tolerated, and the most common adverse effects (AEs) were injection site reactions.¹³

Duchenne Muscular Dystrophy

Casimersen is an investigational drug known that uses a proprietary phosphorodiamidate morpholino oligomer chemistry for the rare disease Duchenne muscular dystrophy in patients who have a genetic mutation that is amenable to skipping exon 45 of the dystrophin gene.¹⁴ An interim analysis of the double-blind, placebo-controlled phase 3 ESSENCE trial (NCT02500381) demonstrated a statistically significant increase in dystrophin production with casimersen.^14,15 This ongoing clinical trial is collecting additional safety and efficacy data.¹⁵

Multiple Myeloma

Idecabtagene vicleucel (ide-cel; bb2121) is an investigational drug for the treatment of multiple myeloma in adult patients, and it is the first anti-BCMA chimeric antigen receptor T-cell therapy.¹⁶ The biologics license application (BLA) is based on positive safety and efficacy results from the phase 2 KarMMa study (NCT03361748), which were presented at the American Society of Clinical Oncology 2020 Virtual Scientific Program.¹⁶

Hypercholesterolemia

Evinacumab is an investigational monoclonal antibody for the treatment of patients with a rare inherited form of high cholesterol known as homozygous familial hypercholesterolemia (HoFH). VelocImmune technology was used to develop evinacumab, and it works by binding to and blocking the function of angiopoietin-like 3 in patients with HoFH.¹⁷ Positive efficacy and safety results from the phase 3 ELIPSE trial (NCT03399786) were presented at the American College of Cardiology’s Annual Scientific Session. These data supported the BLA submission to the FDA.¹⁸ In the ELIPSE trial, 65 patients were randomized to receive either evinacumab 15 mg/ kg intravenously every 4 weeks plus other lipid-lowering therapies versus lipid-lowering therapies alone. Patients in the evinacumab group had reduced their low-density lipoprotein cholesterol from baseline by 49% compared with placebo at week 24 (P < .0001). Evinacumab was well tolerated, and the most common AEs were flu-like illness and rhinorrhea.¹⁸

Migraine

The novel drug dihydroergotamine mesylate (INP104) or DHE is for the treatment of migraine headaches in adults with or without aura. This medication is administered via Precision Olfactory Delivery technology, which is the first and only delivery system to use the vascular-rich upper nasal space for fast and lasting migraine relief at a lower dose than previous FDA-approved products. The phase
3 STOP 301 trial (NCT03557333) evaluated the safety and efficacy of INP104 in 360 patients, and over 5650 migraine attacks were treated. AEs were mild, and the most common ones reported were nasal congestion, nausea, nasal discomfort, and abnormal taste. Overall, 66.3% of patients reported pain relief, 38% had pain freedom, and 52% reported freedom from their most bothersome migraine symptoms 2 hours following their first dose of INP104.¹⁹

Biosimilars

Biosimilars are expected to lower drug costs by $100 billion over the next 5 years.²⁰ The biosimilar AVT02 is highly similar to adalimumab (Humira), demonstrated by the pharmacokinetics study following subcutaneous administration.²¹ Additionally,SB11 is a proposed biosimilar referencing ranibizumab (Lucentis) for retinal vascular disorders.²²

References

1. New drug approvals for 2020. FDA. Updated November 9, 2020. Accessed November 22, 2020. https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2020 
2. FDA guidance on conduct of clinical trials of medical products during COVID-19 public health emergency. FDA. Updated September 21, 2020. Accessed November 22, 2020. https://www.fda.gov/media/136238/download 
3. Vizient. Drug price forecast 2020. Summer 2020. Accessed November 22, 2020. https://www.vizientinc.com/-/media/documents/sitecorepublishingdocuments/public/Jul2020DPF_DrugPriceForecast_Public_RXDPF220.pdf 
4. Coronavirus (COVID-19) update: FDA authorizes monoclonal antibodies for treatment of COVID-19. New release. FDA; November 21, 2020. Accessed November 23, 2020. https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-fda-authorizes-monoclonal-antibodies-treatment-covid-19 
5. Fact sheet for health care providers: Emergency use authorization (EUA) of casirivimab and imdevimab. Regeneron. November 2020. Accessed November 24, 2020. https://www.fda.gov/media/143892/download 
6. RECOVERY trial data monitoring committee recommends continuing evaluation of REGN-COV2 in all hospitalized patients. News release. Regeneron; November 5, 2020. Accessed November 25, 2020. https://investor.regeneron.com/news-releases/news-release-details/recovery-trial-data-monitoring-committee-recommends-continuing. 
7. Randomised evaluation of COVID-19 therapy (RECOVERY). ClinicalTrials.gov. Updated December 1, 2020. Accessed December 1, 2020. https://clinicaltrials.gov/ct2/show/NCT04381936 
8. Coronavirus (COVID-19) update: FDA authorizes monoclonal antibody for treatment of COVID-19. New release. FDA; November 9, 2020. Accessed November 25, 2020. https://www.fda.gov/news-events/press-announcements/coronavirus-covid-19-update-fda-authorizes-monoclonal-antibody-treatment-covid-19. 
9. ACTIV-2: A study for outpatients with COVID-19. NIH. Updated November 30, 2020. Accessed November 25, 2020. https://clinicaltrials.gov/ct2/show/NCT04518410 
10. A study of LY3819253 (LY-CoV555) in preventing SARS-CoV-2 infection and COVID-19 in nursing home residents and staff (BLAZE-2). ClinicalTrials.gov. Updated November 10, 2020. Accessed November 25, 2020. https://clinicaltrials.gov/ct2/show/NCT04497987 
11. Lilly statement regarding NIH’s ACTIV-3 clinical trial. News release. Eli Lilly; October 26, 2020. Accessed November 25, 2020. https://www.lilly.com/news/stories/statement-activ3-clinical-trial-nih-covid19. 
12. ViiV Healthcare receives FDA Breakthrough Therapy designation for investigational, long-acting cabotegravir for HIV prevention. News release. ViiV Healthcare; November 17, 2020. Accessed November 27, 2020. https://viivhealthcare.com/en-gb/media/press-releases/2020/november/viiv-healthcare-receives-fda-breakthrough-therapy-designation-fo/ 
13. Important HIV prevention, treatment and cure research unveiled at AIDS 2020: Virtual. News release. AIDS 2020 23rd International AIDS Conference; July 7, 2020. Accessed November 27, 2020. https://www.aids2020.org/important-hiv-prevention-treatment-and-cure-research-unveiled-at-aids-2020-virtual/ 
14. Sarepta Therapeutics announces FDA acceptance of casimersen (SRP-4045) new drug application for patients with Duchenne muscular dystrophy amenable to skipping exon 45. News release. Sarepta Therapeutics; August 25, 2020. Accessed November 27, 2020. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-fda-acceptance-casimersen-srp 
15. Study of SRP-4045 and SRP-4053 in DMD patients (ESSENCE). ClinicalTrials.gov. Last updated July 8, 2020. Accessed November 27, 2020. https://clinicaltrials.gov/ct2/show/NCT02500381 
16. U.S. Food and Drug Administration (FDA) accepts for priority review Bristol Myers Squibb and bluebird bio application for anti-BCMA CAR T cell therapy idecabtagene vicleucel (Ide-cel, bb2121). News release. Bristol Myers Squibb; September 22, 2020. Accessed November 27, 2020. https://news.bms.com/news/details/2020/U.S.-Food-and-Drug-Administration-FDA-Accepts-for-Priority-Review-Bristol-Myers-Squibb-and-bluebird-bio-Application-for-Anti-BCMA-CAR-T-Cell-Therapy-Idecabtagene-Vicleucel-Ide-cel-bb2121/default.aspx 
17. FDA accepts evinacumab Biologics License Application for priority review as a treatment for patients with HoFH, an ultra-rare inherited form of high cholesterol. News release. Regeneron; August 12, 2020. Accessed November 27, 2020. https://newsroom.regeneron.com/news-releases/news-release-details/fda-accepts-evinacumab-biologics-license-application-priority 
18. Regeneron announces American College of Cardiology presentation of positive phase 3 evinacumab results in patients with severe inherited form of high cholesterol. News release. Regeneron; March 30, 2020. Accessed November 27, 2020. https://investor.regeneron.com/index.php/news-releases/news-release-details/regeneron-announces-american-college-cardiology-presentation 
19. Impel NeuroPharma announces FDA submission of new drug application for INP104 for the acute treatment of migraine. News release. Impel NeuroPharma; November 9, 2020. Accessed November 27, 2020. https://impelnp.com/2020/11/09/impel-neuropharma-announces-fda-submission-of-new-drug-application-for-inp104-for-the-acute-treatment-of-migraine 
20. Biosimilars in the United States 2020-2024. IQVIA. September 29, 2020. Accessed November 28, 2020. https://www.iqvia.com/insights/the-iqvia-institute/reports/biosimilars-in-the-united-states-2020-2024 
21. Alvotech announces that the U.S. FDA and EMA have accepted regulatory submissions for AVT02, a proposed biosimilar to Humira (adalimumab). News release. Alvotech; November 19, 2020. Accessed November 28, 2020. https://www.alvotech.com/newsroom/alvotech-announces-that-the-u.s.-fda-and-ema-have-accepted 
22. Samsung Bioepis and Biogen announce FDA filing acceptance of SB11, a proposed biosimilar referencing Lucentis (ranibizumab). News release. Biogen; November 18, 2020. Accessed November 28, 2020. https://investors.biogen.com/news-releases/news-release-details/samsung-bioepis-and-biogen-announce-fda-filing-acceptance-sb11