In this article, we will take a detailed look at the Top 20 Breakthrough Pharmaceutical Drugs of the Decade with insights into peak sales estimate at the time of approval by FDA. For a quick overview of the 5 breakthrough pharmaceutical drugs, read our article Top 5 Breakthrough Pharmaceutical Drugs of the Decade.
The pharmaceutical industry has made significant progress in the past decade, developing many novel and breakthrough drugs that have revolutionized treatment of a wide range of diseases. These drugs have improved the lives of millions of people worldwide and potentially saved even more lives in the years to come. Breakthrough drugs, often referred to as breakthrough therapies, represent significant advancements in treating specific medical conditions. These drugs are characterized by their potential to provide substantial benefits over existing therapies or to address previously unmet medical needs.
To facilitate the development of novel, breakthrough, and therapeutic biological products, the FDA's Center for Drug Evaluation and Research (CDER) offers invaluable counsel to drug developers regarding essential components of study design and requisite data for a thorough evaluation within drug applications. CDER's expertise is grounded in its comprehension of scientific underpinnings behind novel product development, intricacies of testing and production protocols, and specific diseases and conditions targeted by these innovative products.
Likewise, post-pandemic era is an alluring prospect for everyone after the disruptions of the past three years. While global COVID-19 vaccinations are expected to be the largest driver of medicine spending through 2027, innovation will continue to drive spending on medicines, offset by losses of exclusivity and lower costs of generics and biosimilars. Biotech will represent 35% of the expenditure globally in 2027, including breakthrough cell and gene therapies and a maturing biosimilar segment.
Consequently, prompted by a rise in the utilization of prescription medications and the transition from older to new pharmaceutical products, U.S. healthcare spending on prescription drugs reached $603 billion in 2021, with retail drugs accounting for $421 billion. Non-retail drug spending grew faster (25%) than retail drug spending (13%).
A Look Into the Pharmaceutical Industry
Eli Lilly & Co. (NYSE:LLY), a healthcare company founded in 1876, is a pioneer in the development and marketing of human healthcare products, including world's first commercially available insulin product, Iletin. Its shares soared 18% to a record high.Eli Lilly (NYSE:LLY) has revised its annual projections upward due to a significant uptick in demand for its recently launched diabetes medication, Mounjaro. This increase in demand coincides with an imminent decision regarding its potential application as a treatment for weight loss.
Eli Lilly & Co. (NYSE:LLY) is suing 10 U.S. health pharmacies, including medical spas, wellness centres, and compounding pharmacies, regarding scrutiny for marketing products that purportedly contain tirzepatide, the primary component found in Eli Lilly's (NYSE:LLY) diabetes medication, Mounjaro. Eli Lilly is seeking court orders to stop the defendants from selling tirzepatide and paying unspecified damages. Eli Lilly & Co. (NYSE:LLY) also accuses the defendants of trademark infringement for advertising compounded tirzepatide as Mounjaro.
Bristol Myers Squibb Co. (NYSE:BMY), a global biopharmaceutical company, has a market cap of $123.42 billion. Bristol Myers Squibb Co. (NYSE:BMY) grew at an annual average rate of 7.8%, from $19.4 billion in 2016 to $22.6 billion in 2018. It has made 71 investments, including its latest investment in ROME Therapeutics and its 2022 acquisition of Turning Point Therapeutics. Through the Turning Point acquisition, Bristol Myers Squibb Co. (NYSE:BMY) gained a pipeline of investigational medicines targeting the most common mutations associated with oncogenesis, including repotrectinib, which BMS expects to be approved in the U.S. in the second half of 2023 and become a new standard of care for patients with ROS1-positive NSCLC in first-line setting.
Genentech Inc (NYSE:DNA), a biotechnology company dedicated to developing life-saving medicines, has invested over $250 million in a new biological facility that will reduce its environmental footprint. The Clinical Supply Center and new manufacturing facility in Oceanside, California, are important steps towards Genentech's corporate sustainability goals to reduce greenhouse gas emissions by 40%, water use by 20%, total landfill waste by 20%, and plastic waste by 20% at its South San Francisco campus by 2025.
Orionis Biosciences, a private life sciences company, and Genentech Inc (NYSE:DNA), a Roche company, announced a multi-year collaboration to discover novel small molecule medicines for challenging targets in major disease areas. Orionis will be responsible for finding and optimizing molecular glues for Genentech Inc's (NYSE:DNA) designated targets, while Genentech will be responsible for subsequent later-stage preclinical, clinical development, regulatory filing, and commercialization of such small molecules. Orionis will receive upfront and milestone payments that could exceed $2 billion and a tiered royalty on sales.
Breakthrough Pharmaceutical Drugs of the Decade
Methodology
To rank the top 20 breakthrough Pharmaceutical Drugs of the decade, i.e. 2013 - 2022, we have used the parameter of peak sales estimate at the time of approval by the FDA. Peak sales estimate of the drug represents how much the drug is effective and in demand.
The data for peak sales estimate of drugs has been taken from Fierce Pharma. In our list, we presented the drugs in ascending order of peak sales estimate at the time of approval.
Top 20 Breakthrough Pharmaceutical Drugs of the Decade
Voquezna
Peak Sales Estimate at the Time of Approval (USD Billion): 0.2
Disease: Helicobacter pylori infection
Company: Phathom Pharmaceuticals
Phathom Pharmaceuticals entered the commercial market in 2022, a few years after spinning off from Takeda. Its anti-infective therapy, Voquezna, was approved in two combinations: Voquezna Triple Pak and Voquezna Dual Pak. Before the approvals, Phathom secured $260 million in funding to support the commercial launch and continued R&D.
Terlivaz
Peak Sales Estimate at the Time of Approval (USD Billion): 0.3
Disease: Hepatorenal syndrome
Company: Mallinckrodt
Mallinckrodt's terlipressin, the standard of care for hepatorenal syndrome (HRS) in Europe since 2010, was finally approved in the U.S. in 2022. HRS is an acute and life-threatening condition in people with advanced liver disease and rapid reduction in kidney function.
Spevigo
Peak Sales Estimate at the Time of Approval (USD Billion): 0.35
Disease: Generalized pustular psoriasis flares
Company: Boehringer Ingelheim
Generalized pustular psoriasis (GPP) flares are rare and life-threatening skin conditions that can cause painful blisters and trigger heart failure, renal failure, or sepsis. Spevigo, an IL-36 receptor inhibitor, is the first drug approved to treat GPP. The most common adverse reactions are (≥5%) asthenia, fatigue, nausea, vomiting, headache, pruritus, prurigo, infusion site hematoma and bruising, and urinary tract infection.
Priorix
Peak Sales Estimate at the Time of Approval (USD Billion): 0.364
Disease: Measles, Mumps
Company: GSK
Measles outbreaks have increased globally in recent years, with over 400,000 cases confirmed in 2019, ending progress towards elimination in many countries. Priorix, a measles, mumps, and rubella vaccine, was approved in 2023 based on comparable efficacy and safety to M-M-R II. Priorix, Priorix Tetra, and chickenpox Varilrix contributed £260 million ($325.99 million) to GSK's vaccine turnover in 2021.
Enjaymo
Peak Sales Estimate at the Time of Approval (USD Billion): 0.5
Disease: Cold agglutinin disease
Company: Sanofi
Sanofi's stock price is up 7.1% in the past year, compared to a 16.3% increase for the industry. Its drug Enjaymo, a classical complement inhibitor for cold agglutinin disease in adults, costs $1,800 per vial in the U.S. Enjaymo emerged from Sanofi's $11 billion acquisition of Bioverativ was approved in 2023 to treat cold agglutinin disease, a rare blood disorder.
Xenpozyme
Peak Sales Estimate at the Time of Approval (USD Billion): 0.5
Disease: Niemann-Pick
Company: Sanofi
Xenpozyme (olipudase alfa) is the first and only approved therapy for ASMD, a rare group of genetic disorders in which a fatty substance called sphingomyelin builds up in cells, causing them to die and disrupting tissue and organ function. The company has set a U.S. list price of $7,142 per vial for Xenpozyme, equating to an annual median cost of treatment of approximately $780,000 per year before any discounts or rebates. With such a small patient population, Xenpozyme is not expected to be a big seller.
Pyrukynd
Peak Sales Estimate at the Time of Approval (USD Billion): 1
Disease: Hemolytic anaemia associated with pyruvate kinase deficiency
Company: Agios
Agios generated $11.7 million in net U.S. product revenue from sales of PYRUKYND in 2022. R&D expenses increased to $279.9 million in 2022 from $257.0 million in 2021, primarily due to increased costs for PYRUKYND.
Vonjo
Peak Sales Estimate at the Time of Approval (USD Billion): 1.01
Disease: Hemolytic anaemia associated with pyruvate kinase deficiency
Company: CTI BioPharma
The Seattle-based pharmaceutical company, CTI BioPharma, navigated a challenging journey to FDA approval. TheyThey received FDA approval in late February in late February for their JAK2 and IRAK1 inhibitor, Vonjo. Vonjo exceeded revenue expectations, achieving $54 million in total net product revenue for 2022, with $21.1 million generated in the fourth quarter alone. This represented a notable 16% increase compared to the third quarter.
Daxxify
Peak Sales Estimate at the Time of Approval (USD Billion): 1.02
Disease: Glabellar lines
Company: Revance
Revance reported total revenue of $58.1 million for the second quarter ending June 30, 2023, marking a substantial increase of 104.9% compared to last year. This significant growth can be attributed primarily to increased sales of the RHA Collection of dermal fillers and successful commercialization of DAXXIFY.
Quviviq
Peak Sales Estimate at the Time of Approval (USD Billion): 1.05
Disease: Insomnia
Company: Idorsia
Idorsia's Quviviq generated CHF 11.8 million in net sales in the first half of 2023. The company had set a target to become profitable in 2025 with global revenue above CHF 1 billion but has suspended this target due to a slower-than-expected ramp-up of Quviviq sales, a portfolio review, and ongoing discussions with potential partners.
Pluvicto
Peak Sales Estimate at the Time of Approval (USD Billion): 1.05
Disease: Prostate Cancer
Company: Novartis
Novartis' Pluvicto, a PSMA-targeted radioligand therapy, generated $240 million in sales in Q2 2023, up 14% from Q1 2023 and exceeding expectations. With the approval of two new radiopharmaceutical manufacturing sites, Novartis can ramp up Pluvicto's commercial production and meet growing demand.
Vtama
Peak Sales Estimate at the Time of Approval (USD Billion): 1.05
Disease: Psoriasis
Company: Dermavant
VTAMA’s net product revenue of $28 million for the fiscal year ended March 31, 2023, representing a 25% gross-to-net yield. Over 170,000 VTAMA prescriptions have been written by approximately 11,000 unique prescribers for psoriasis as of June 2023, according to IQVIA data. Coverage has been expanded to 125 million U.S. commercial lives, representing 76% of the affected people.
Cibinqo
Peak Sales Estimate at the Time of Approval (USD Billion): 1.05
Disease: Refractory atopic dermatitis
Company: Pfizer
Cibinqo, an effective once-daily pill for moderate-to-severe atopic dermatitis, is a breakthrough made possible by Pfizer researchers and clinical trial participants. Pfizer CEO Albert Bourla projected $3 billion in peak sales, while SVB Leerink analysts projected $2 billion by 2027.
Sotyktu
Peak Sales Estimate at the Time of Approval (USD Billion): 3.01
Disease: Moderate to severe plaque psoriasis
Company: Bristol Myers Squibb
Bristol Myers Squibb's Sotyktu outperforms Otezla, validating the company's decision to prioritize its development. BMS acquired Sotyktu for $74 million in 2019 and sold Otezla to Amgen for $13.4 billion due to competition concerns. Analysts forecast $2.9 billion in sales for Sotyktu by 2029.
Vabysmo
Peak Sales Estimate at the Time of Approval (USD Billion): 3.6
Roche's new eye drug, Vabysmo, generated nearly $500 million in sales in Q1 2023, outpacing all other Roche drugs. Vabysmo is dosed every four months and has a list price of $6,570 per year, or $2,190 per shot, which is competitive with Lucentis biosimilars.
