Biggest Novel Drug Approvals in 2020

FDA Approval_Compressed

2020 was a peculiar year for drug approvals, partly because the ongoing COVID-19 pandemic delayed many clinical trials. Another reason is because several of the highly anticipated drug approvals for the year were surprising failures, such as Novartis’ inclisiran for hyperlipidemia in adults, FibroGen’s roxadustat for anemia in chronic kidney disease, and Gilead Sciences’ filgotinib in rheumatoid arthritis. Some of them may make a comeback in 2021 or later, but they did not hit the mark for one reason or another in 2020.

On the other hand, the pandemic and the astonishing innovations of numerous pharmaceutical companies created drugs to treat COVID-19 in record time. Here’s a look at the top 10 novel drug approvals of 2020, loosely based on projected earnings in the upcoming years.

Roche’s Evrysdia for Spinal Muscular Atrophy

Roche’s Evrysdi (risdiplam) was approved on August 10 by the FDA for spinal muscular atrophy (SMA) in adults and children two months of age and older. The drug is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. It is administered daily at home in liquid form by mouth or feeding tube.

SMA is a severe, progressive neuromuscular disease that can be fatal. Without the SMN protein, nerve cells do not function correctly, which leads to muscle weakness over time. Depending on the type of SMA, the disease can affect the patient’s strength and their ability to walk, eat or breathe.

EvaluatePharma projects sales of $803 million by 2024. Some analysts project it could bring in $2.5 billion to $3 billion at its peak.

Immunomedics’ Trodelvy for Metastatic Triple-Negative Breast Cancer

Immunomedics’ Trodelvy (sacituzumab govitecan-hziy) for adults with metastatic triple-negative breast cancer (TNBC) who have at least two previous therapies for metastatic disease in April 2020. The drug is an antibody-drug conjugate (ADC) directed against Trop-2, a cell-surface protein found on many solid cancers. Trodelvy binds to Trop-2 and delivers SN-38, an anti-cancer drug.

 

TNBC is an aggressive form of breast cancer, making up to 20% of all breast cancers. It is more frequently diagnosed in younger and premenopausal women and is highly prevalent in African Americans and Hispanic women. TNBC cells don’t have estrogen or progesterone hormone receptors or the human epidermal growth factor receptor 2 (HER2-).

 

This was ranked as one of EvaluatePharma’s top five biggest drug approvals in the first half of 2020. They it projected to bring in $2.3 billion in 2026 sales.

 

Esperion’s Nexletol for LDL-Cholesterol

The FDA approved Esperion’s Nexletol (bempedoic acid) in February 2020 for lowering LDL-cholesterol (LDL-C). Nexletol is indicated as an adjunct to diet and maximally tolerated statin therapy for adults with heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease (ASCVD) who require additional lowering of LDL-C.

The approval was supported by a global pivotal Phase III LDL-C lowering program run in more than 3,000 patients. In them, the drug provided an average of 18% placebo corrected LDL-C lowering when used with moderate or high intensity statins.

EvaluatePharma projects it will have 2026 annual sales of $1.8 billion.

Gilead’s Veklury (Remdesivir) for COVID-19

At the beginning of 2020, nobody knew how bad COVID-19 would be or that Gilead Sciences’ remdesivir, which had failed to work in Ebola, would turn out to be the first drug granted Emergency Use Authorization (EUA) for COVID-19 in spring 2020 and then full approval on October 22, 2020. At the time of the EUA, it was really the only available treatment, but few physicians or analysts were particularly impressed with the drug’s effectiveness.

Back in April 2020, when the world was desperate for any drug that might help, Gilead reported encouraging results from its clinical trial of Veklury in COVID-19, with topline results from the Phase III SIMPLE trial evaluating 5-day and 10-day dosing in hospitalized patients with severe disease. It also reported positive data from the National Institute of Allergy and Infectious Diseases’ (NIAID) trial.

The results were promising, but not dazzling. One of the bigger takeaways was that the 5-day regimen appeared to have similar results to the 10-day regimen, which was good news because supply of the drug was limited. In terms of clinical improvement, the trial showed the time to clinical improvement for 50% of patients was 10 days in the 5-day cohort and 11 days in the 10-day cohort.

But a clinical trial by the World Health Organization (WHO) reported that the drug does not have any particular effect on a patient’s survival. The WHO has since taken remdesivir off their list of recommended treatments for COVID-19.

Gilead has set a price of $3,120 for a course of treatment of Veklury for private insurers and $2,340 for government payers. The price was met with mixed opinions. The Institute for Clinical and Economic Review’s own estimate had been $2,800 as a fair price and said Gilead’s price was a “responsible pricing decision.” But that was based on if the drug ultimately demonstrated it significantly decreased death rates, which has not happened.

Others, such as Public Citizen, a consumer group, argue that the prices are too high given that the federal government invested $70 million in the drug and sponsored the only major trial comparing it with a placebo; $70 million is a minuscule amount of money compared to what the government’s Operation Warp Speed, the program to advance therapies and vaccines against COVID-19, has spent. Congress directed almost $10 billion toward Operation Warp Speed through supplemental funding and has appropriated other funds as well.

Gilead stated that it had already invested more than $1 billion into the drug’s development and expects to invest even more next year into scaling up manufacturing globally. It is also working to develop an inhaled version of the drug.

With the Pfizer-BioNTech and Pfizer vaccines rolling out, it’s entirely possible that Gilead will be left with a drug for which there is no market. But meanwhile, it’s making a lot of money. The drug has brought in $873 million, making it Gilead’s second-best-selling drug for the third quarter of 2020. Statista projects peak sales in 2021 of $3.074 billion, with sales dropping afterwards to $1.729 billion in 2026.

Bristol Myers Squibb’s Zeposia

Bristol Myers Squibb’s Zeposia (ozanimod) was approved by the FDA in March 2020, the first approval after it completed its merger with Celgene. The drug was approved for the treatment of adults with relapsing forms of multiple sclerosis (RMS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary disease. It is the only approved spingosine-1-phosphate (S1P) receptor modulators for RMS patients with no genetic test and no label-based first-dose observation required for patients.

Estimated sales by 20224 are $1.598 billion. The drug was a significant reason for Bristol Myers Squibb’s acquisition of Celgene—part of the payout to Celgene shareholders is related to FDA approvals for three drugs, including Zeposia.

Eli Lilly’s Bamlanivimab for COVID-19 and Regeneron’s REGN-COV2

These two are being presented together because they represent a peculiarity of the COVID-19 market and the unpredictability of therapies for the disease.

On November 9, 2020, the FDA granted Eli Lilly’s neutralizing antibody bamlanivimab EUA for mild to moderate COVID-19 in adults and pediatric patients 12 years and older who are at high risk for progressing to severe COVID-19 and/or hospitalization. The SUA was based on data from BLAZE-1, a Phase II trial in patients with recently diagnosed mild to moderate COVID-19 in the outpatient setting.

On November 22, the FDA granted Regeneron Pharmaceuticals’ antibody cocktail casirivimab and imdevimab, together REGN-COV2, EUA for mild to moderate COVID-19 in adults and pediatric patients at least 12 years of age and weighing at least 40 kg.

Similarly to Gilead’s remdesivir, the long-term sales potential of the neutralizing antibodies are unpredictable because of the distribution of apparently effective vaccines by Pfizer-BioNTech and Moderna.

SVB Leerink projects peak sales of about $1.3 billion for Regeneron, probably in 2021, with sales falling every year after that. Bernstein analysts projected the total market for anti-COVID antibodies of about $3 billion in 2021, with Lilly and Regeneron splitting them, but overall sales are expected to plunge quickly to about $805 million.

Horizon’s Tepezza for Thyroid Eye Disease

In January 2020, the FDA approved Horizon Therapeutics’ Tepezza (teprotumumab-trbw) for adults with thyroid eye disease. That is a rare disease where the muscles and fatty tissues behind the eye become inflamed. This causes the eyes to bulge outwards and forward. It is the first drug approved for thyroid eye disease.

The drug was approved based on two trials of 170 patients with active thyroid eye disease. Of the patient receiving Tepezza, 71% in Study 1 and 83% in Study 2 demonstrated a greater than 2-millimeter reduction in eye protrusion compared to 20$ and 10% in the placebo groups, respectively. The drug was approved under Priority Review, Fast Track and Breakthrough Therapy Designation, as well as Orphan Drug designation.

EvaluatePharma project 2026 annual sales of $1.4 billion.

Aimmune’s Palforzia

Aimmune’s Palforzia [Peanut (Arachis hypogaea) Allergen Power-dnfp] was approved in January 2020 as the first approved treatment for patients with peanut allergy. Palforzia is an oral immunotherapy indicated for the mitigation of allergic reactions, including anaphylaxis, that might happen from accidental exposure to peanut. It was approved for use in patients with a confirmed diagnosis of peanut allergy, for patients aged 4 through 17 years.

In August, Nestlé acquired Aimmune for $2.6 billion, and became the point pharmaceutical business for Nestlé Health Science. Aimmune’s pharmaceutical expertise and infrastructure complements Nestlé Health Science’s existing focus on nutrition science, and a holistic approach to treat food allergies. Estimated sales for 2024 are $1.28 billion.

Novartis’ Kesimpta for Relapsing Multiple Sclerosis

The FDA approved Novartis’ Kesimpta (ofatumumab), the first and only self-administered, targeted B-celll therapy for patients with relapsing multiple sclerosis (RMS) in August 20. The approval includes clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease. It is self-administered once a month at home via the Sensoready autoinjector pen.

Ofatumumab was first approved by the FDA in 2009 for chronic lymphocytic leukemia (CLL) as an intravenous infusion. It was then studied in a completely new development program in RMS because B-cells play a critical role in autoimmune diseases, such as MS. The program took 10 years and involved more than 2,300 patients globally. The approval was based on data from the Phase III ASCLEPIOS I and II trials.

Clarivate projects $1.26 billion in peak sales.

Biohaven Pharmaceutical’s Nurtec for Acute Migraine

On February 28, the FDA approved Biohaven Pharmaceutical’s Nurtec ODT (Rimegepant) for the acute treatment of migraine in adults. It was the first FDA-approved product for the company. It is a single quick-dissolving tablet provides fast relief within one hour and delivers sustained efficacy up to 48 hours for many patients. It is not indicated for prevention of migraine, although the company is running clinical trials for that.

There are currently three marketed CGRP drugs for migraine on the market, Amgen and Novartis’ Aimovig, Teva’s Ajovy and Eli Lilly’s Emgality, but all are injections. Biohaven’s Nurtec has a definite advantage there. Allergan’s Ubrelvy (ubrogepant) is an oral therapy for migraine, but many analysts believe Nurtec is a more successful sales opportunity. Evaluate Pharma has projected estimated 2024 sales of $897 million.

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