In 2023, biotechnology and pharmaceutical companies leverage cutting-edge technology and significant investments in research and development (R&D) to continue pushing the boundaries of innovation in the healthcare sector, transcending the challenges posed by the pandemic. This groundwork ensures a more efficient and effective approach to combatting diseases in the future. Now, let’s delve into the list of the top 20 fastest growing biotech and pharma companies in 2023, ranked by their compound annual growth rate (CAGR). These companies were identified through The Americas’ Fastest-Growing Companies 2023 list by the Financial Times.
Related: Top 30 Pharma Companies in 2023: Statistics and Trends
1) Moderna
Compound annual growth rate: 415 percent
Moderna, headquartered in Massachusetts, is a prominent biotechnology company specializing in RNA therapeutics, particularly mRNA vaccines. Its remarkable success is primarily credited to its innovative mRNA platform, which is dedicated to developing therapies for a wide spectrum of medical conditions, such as infectious diseases, cancer, cardiovascular ailments and rare diseases.
In 2023, Moderna is poised to achieve sales figures ranging from $6 billion to $8 billion, contingent on the prevailing COVID-19 vaccination rates in the US. The company’s sustained investment in R&D has led to a rapid expansion of its pipeline in 2023, with notable milestones such as four infectious disease vaccines in Phase III trials, including the recently submitted respiratory syncytial virus (RSV) vaccine for regulatory approval. Additionally, individualized neoantigen therapy for melanoma is currently in Phase III and is expected to launch between 2024 and 2026.
Modern is making significant strides in advancing its mRNA technology and diversifying its product portfolio to address a broad spectrum of medical conditions. Notably, R&D expenditures for the second quarter of 2023 surged by 62 percent to reach $1.1 billion, in contrast to the same quarter in 2022.
The company has recently presented clinical data showcasing the strong neutralization and cross-reactivity of its monovalent XBB.1.5 vaccine, mRNA-1273.815. Moreover, it has submitted global marketing authorization applications for mRNA-1345, a vaccine designed to prevent RSV-associated lower respiratory tract disease (RSV-LRTD) and acute respiratory disease (ARD), supported by favorable data from a predefined interim analysis. In collaboration with Merck, Moderna has also announced the commencement of a pivotal Phase III global trial to evaluate the safety and efficacy of mRNA-4157 (V940) in conjunction with Keytruda for individuals with resected high-risk (stage IIB-IV) conditions. This trial is anticipated to enroll approximately 1,089 patients at more than 165 sites spanning over 25 countries.
2) Selecta Biosciences
Compound annual growth rate: 355 percent
Established in 2008, Selecta Biosciences Inc., a clinical-stage biotechnology company, is harnessing its well-established ImmTOR® platform to pioneer the development of antigen-specific tolerogenic therapies. With its capacity to potentially induce tolerance to exceptionally immunogenic substances, ImmTOR® seeks to rejuvenate the innate equilibrium of the immune system. In doing so, it aims to re-imagine immunotherapy for autoimmune disorders and unlock the latent potential of gene therapy while enhancing the effectiveness of biological therapy.
Since its inception, the company has primarily focused on advancing its technology and product candidates through research and development, recruiting skilled management and technical personnel, procuring operational assets and securing necessary capital. Selecta Biosciences reported a net income of $35.4 million, a significant leap from a net loss of $25.7 million in 2021, while the total revenue for 2022 was reported to be $110.8 million, up from $85.1 million in 2021.
3) Dynavax Technologies
Compound annual growth rate: 277 percent
Dynavax Technologies, headquartered in California, is a thriving commercial-stage biotech company dedicated to the development and commercialization of groundbreaking vaccines. The company is unwavering in its commitment to creating and introducing innovative vaccines that safeguard the global population against infectious diseases. It achieves this goal by harnessing proven and inventive adjuvant technology.
Dynavax boasts two successful commercial products in its portfolio. First, there is the HEPLISAV-B vaccine (hepatitis B vaccine, recombinant, adjuvanted), which has received approval in the US, the European Union (EU) and Great Britain. This vaccine is designed to prevent infections caused by all known subtypes of the hepatitis B virus in adults aged 18 and older. Additionally, the company produces the CpG 1018 adjuvant, which is presently utilized in several adjuvanted COVID-19 vaccines.
Recent financial reports from Dynavax indicate substantial growth, with quarterly net product revenue of $56 million generated by the HEPLISAV-B vaccine, marking a remarkable 73 percent year-over-year increase. Consequently, the full-year revenue guidance for HEPLISAV-B has been revised to a range of $200 million to $215 million, surpassing the prior estimate of $165 million to $185 million. This financial success is a testament to Dynavax’s enduring dedication to R&D.
In the second quarter of 2023, the company saw a notable increase in R&D expenses, which reached $13.0 million, compared to $9.7 million in the same quarter of 2022. This surge is primarily attributed to ongoing investments in product candidates that leverage the CpG 1018 adjuvant through preclinical and clinical collaborations, as well as additional discovery efforts. These efforts encompass programs related to the shingles vaccine, Tdap vaccine and plague vaccine, reinforcing Dynavax’s commitment to advancing the field of vaccines and medicine.
4) Fulgent Genetics
Compound annual growth rate: 260 percent
Fulgent Genetics, headquartered in California, is a technology-driven company with a strong presence in clinical diagnostics and therapeutic development. The company’s clinical diagnostic division provides a wide array of services, including molecular diagnostic testing, comprehensive genetic testing and high-quality anatomic pathology laboratory services. Renowned as a leader in the genetic and genomic testing industry, Fulgent Genetics offers one of the most extensive collections of clinical genetic tests globally, encompassing over 22,000 single gene tests, 900+ preset panels, rearrangement testing, clinical exome/trios, whole exome/trios, whole genome sequencing and the comprehensive all-in-one reflex test.
The year 2022 has proven to be a momentous period for Fulgent Genetics, marked by significant expansion of its product portfolio across various medical conditions. The company achieved core revenues of $67 million, reflecting a remarkable 48 percent year-over-year growth. Furthermore, Fulgent has raised its full-year 2023 core revenue guidance to $260 million while simultaneously narrowing its financial losses. This impressive growth can be attributed to Fulgent’s distinctive chemistries and advanced proprietary bioinformatics pipeline, which have been instrumental in fueling the company’s rapid advancement in recent years.
Fulgent Genetics continues to make strides in its core genetics business, maintaining momentum in three primary areas: precision diagnostics, anatomic pathology and pharmaceutical services. Additionally, in its therapeutic development segment, the company recently received promising Phase Ib data for its leading drug candidate, FID-007, in various cancer types. These results were presented at the American Society of Clinical Oncology annual meeting, and Fulgent is eagerly preparing for the initiation of Phase II studies by the end of 2023, highlighting its commitment to advancing the field of therapeutic development.
5) Arcus Biosciences
Compound annual growth rate: 258 percent
Arcus Biosciences, a clinical-stage biopharmaceutical company focused on cancer research, reported $112 million in revenue in 2022. Since its founding in 2015, Arcus has demonstrated remarkable speed in progressing its molecules from program initiation to investigational new drug (IND) filing, achieving this milestone in as little as 18 months. Furthermore, the company has expedited platform and signal-seeking studies, enabling the transition from the proof-of-concept Phase I stage to randomized Phase II and registrational Phase III studies in just a few years.
6) Alpine Immune Sciences
Compound annual growth rate: 222 percent
Alpine Immune Sciences, Inc. stands at the forefront as a leading clinical-stage immunotherapy company with a keen focus on pioneering treatments for autoimmune and inflammatory diseases. Notably, the company reported revenues of $8.6 million and $18.0 million for the first three and six months ending June 30, 2023, respectively, compared to $5.3 million and $18.9 million for the corresponding periods in 2022. Bolstered by world-class R&D capabilities, a highly productive scientific platform and an established management team, Alpine is dedicated to crafting first- or best-in-class multifunctional immunotherapies through distinctive protein engineering technologies, all aimed at enhancing the quality of life for patients.
Alpine Immune Sciences has recently announced an augmentation in its R&D investment. For the three and six months ending June 30, 2023, R&D expenses reached $19.2 million and $38.8 million, respectively, as compared to $17.6 million and $33.9 million for the same intervals in 2022. This substantial investment is accelerating the growth of their new drug pipeline.
Notably, the company has achieved significant milestones in the development of povetacicept, a potentially best-in-class dual BAFF/APRIL inhibitor currently progressing in multiple autoimmune diseases. Alpine has expanded its clinical studies, including patient enrollment in the RUBY-4 study focusing on povetacicept in autoimmune cytopenias. Additionally, the company is particularly encouraged by the high level of interest in the RUBY-3 study of povetacicept in autoimmune glomerulonephritis. Initial data is expected to be available later this year, and Alpine Immune Sciences is actively planning a comprehensive and expedited development program for povetacicept across multiple indications in 2024.
7) Ocular Therapeutix
Compound annual growth rate: 180 percent
Ocular Therapeutix, Inc. is a biopharmaceutical firm primarily focusing on creating, advancing and bringing groundbreaking treatments for eye-related diseases and conditions to market. They achieve this through their exclusive bioresorbable hydrogel-based formulation technology called Elutyx. Its impressive total net revenue for the year 2022 was $51.5 million versus $43.5 million in 2021, an increase of 18 percent. It already has Dextenza (dexamethasone ophthalmic insert) in the market and has three products (OTX-TKI: axitinib intravitreal implant; OTX-TIC: travoprost intracameral implant; OTX-DED: dexamethasone intracanalicular insert) in clinical trials.
8) EyePoint Pharmaceuticals (formerly pSivida Corp)
Compound annual growth rate: 132 percent
Eyepoint Pharmaceuticals is a company focused on developing therapeutics for retinal diseases. For the full year ended December 31, 2022, total net revenue was $41.4 million compared to $36.9 million for the entire year ended December 31, 2021.
The company’s product pipeline utilizes its exclusive bioerodible Durasert E technology for prolonged intraocular drug delivery. The pipeline includes EYP-1901, an experimental sustained-release intravitreal anti-VEGF therapy undergoing Phase II clinical trials. The company has FDA-approved treatment Dexycu for the treatment of inflammation following ocular surgery.
9) Alnylam Pharmaceuticals
Compound annual growth rate: 124 percent
Founded in 2002 on the groundbreaking Nobel Prize-winning discovery of RNAi, Alnylam had a visionary mission to harness this biological breakthrough for silencing disease-causing genes that lie upstream of today’s medical treatments. Headquartered in Cambridge, US, Alnylam stands as the world’s foremost RNAi therapeutics company and holds the distinction of being the first and only organization to successfully bring RNAi-based medicines to market.
In the second quarter of 2023, Alnylam reported global net product revenues of $306 million, marking an impressive 43 percent year-over-year growth compared to the second quarter of 2022. This remarkable success is largely driven by the robust and ongoing launch of Amvuttra (vutrisiran).
The year 2023 is shaping up to be a pivotal year for Alnylam as the company continually expands its product portfolio to encompass a broad spectrum of medical conditions. Notably, the global net product revenues for Onpattro (patisiran) and Amvuttra in the second quarter reached $91 million and $132 million, respectively, reflecting a 46 percent total TTR reported year-over-year growth compared to the second quarter of 2022. Additionally, the global net product revenues for Givlaari (givosiran) and Oxlumo (lumasiran) in the same quarter were $58 million and $24 million, respectively, demonstrating a remarkable 37 percent total ultra-rare reported year-over-year growth compared to the second quarter of 2022.
Alnylam is also intensifying its commitment to R&D by increasing investments in various products. During the second quarter of 2023, R&D expenses witnessed an uptick compared to the previous year. This increase primarily stemmed from higher development costs linked to the KARDIA-1/KARDIA-2 (zilebesiran) clinical studies, as well as augmented compensation and related expenses due to an expanded headcount aimed at bolstering support for the R&D pipeline.
10) OptiNose
Compound annual growth rate: 119 percent
Optinose is a specialized pharmaceutical company dedicated to developing and introducing inventive products tailored for patients under the care of ear, nose and throat (ENT) and allergy specialists. Their patented Bi-Directional Exhalation Delivery Systems (EDS) uniquely deliver a potent steroid deep into the nose where inflammation can be hard to reach. The drug-device combination system used to provide Flonase is named Xhance, which received FDA approval in 2017. In September 2019, Optinose announced a license agreement with Currax Pharmaceuticals, which grants Currax the exclusive rights to particular Optinose intellectual property to market Onzetra Xsail (sumatriptan nasal powder) in the US, Canada and Mexico. The company reported $76.3 million in net revenue from sales of Xhance, an increase of 4 percent compared to $73.7 million during the twelve months ended December 31, 2021.
11) Xoma
Compound annual growth rate: 93 percent
Xoma is a distinctive player in the biotechnology landscape, specializing in the aggregation of biotech royalties, with a mission to aid biotech firms in their pursuit of advancing human health. Xoma’s unique approach involves acquiring future economic interests tied to pre-commercial therapeutic candidates that have been licensed to pharmaceutical or biotech companies. Through this acquisition, the seller receives non-dilutive, non-recourse funding, which can be directed toward advancing their internal drug candidate(s) or for general corporate purposes.
Xoma is increasingly gaining recognition for its pivotal role in empowering innovative biotech enterprises to unlock value, particularly in a challenging financing environment. In the second quarter of 2023, Xoma reported total revenues of $1.7 million, compared to $1.0 million for the same period in 2022. The company boasts an expansive portfolio of assets that grant rights to future potential royalty and milestone payments.
12) Karyopharm Therapeutics
Compound annual growth rate: 91 percent
Karyopharm Therapeutics, a commercial-stage pharmaceutical company founded in 2008, primarily concentrates on developing innovative drugs to provide potential treatments for individuals afflicted by specific blood cancers or solid tumors. In short, it already has one FDA-approved product and four unique drug candidates in clinical development.
It reported an impressive 22 percent increase in US net product revenue for 2022 ($120.4 million) compared to that revenue for 2021 ($98.4 million). Karyopharm’s primary selective inhibitor of nuclear export (SINE) compound, Xpovio (selinexor), is an innovative, first-in-class oral inhibitor of exportin 1 (XPO1). It is approved and marketed by the company in three oncology indications in the US.
Furthermore, it has garnered regulatory approvals for numerous indications in an expanding array of international regions and nations, including Europe, the United Kingdom (branded as Nexpovio) and China. Karyopharm initiated a Phase III study of selinexor as a maintenance therapy following systemic therapy in patients with TP53 wild-type advanced or recurrent endometrial cancer. Additionally, it entered into a global collaboration with Foundation Medicine Inc. to develop FoundationOne CDx, a TP53 wild-type companion diagnostic for selinexor.
13) Ultragenyx Pharmaceutical
Compound annual growth rate: 90 percent
Ultragenyx is a biopharmaceutical company dedicated to delivering innovative products to patients suffering from severe rare and ultra-rare genetic diseases. The company boasts a diverse portfolio of approved therapies and product candidates aimed at addressing conditions characterized by a high unmet medical need and a clear biological basis for treatment, often lacking approved therapies that target the underlying disease.
In the second quarter of 2023, Ultragenyx reported a total revenue of $108.3 million, marking a 21 percent increase compared to the same period in 2022. Notably, the company achieved $61.3 million in Crysvita (burosumab-twza) revenue in North America during this quarter, reflecting a 19 percent growth compared to the corresponding period in 2022. Despite the transition of commercialization responsibilities for Crysvita in North America from Ultragenyx to its collaboration partner Kyowa Kirin in April 2023, growth in the region continued along its established trajectory.
The year 2023 has been a momentous period for Ultragenyx, marked by a significant expansion of its product portfolio across a wide range of medical conditions. In June 2023, the company announced positive data from the dose-selection Phase II portion of the Phase II/III Orbit study, revealing that setrusumab rapidly stimulated bone production in patients with osteogenesis imperfecta (OI). Additionally, Ultragenyx recently revealed that the US Food and Drug Administration (FDA) has reviewed and agreed to a protocol amendment for the Phase I/II study of GTX-102 in pediatric patients with Angelman syndrome, enabling the harmonization of dose ranges in the US with those used in ex-US cohorts of the study.
14) Organicell Regenerative
Compound annual growth rate: 80 percent
Organicell Regenerative, headquartered in Miami, is a leading clinical-stage biopharmaceutical and regenerative medicine company with a focus on pioneering innovative biological therapeutics for treating chronic diseases and delivering related services. The company’s proprietary products are sourced from perinatal origins and are carefully manufactured to preserve the natural bioactive exosomes, hyaluronic acid and proteins, without the addition or mixture of any other substances or diluents.
Organicell is actively engaged in researching the safety and effectiveness of Zofin in individuals at various stages of COVID-19 infection. Clinical studies represent the groundbreaking use of a human perinatal-derived nanoparticle biologic as a potentially safe and effective therapeutic solution for acute respiratory distress syndrome induced by COVID-19 infection. Building upon these promising results, Organicell has announced its plans to advance into larger Phase II trials for multiple chronic conditions in 2024.
Another noteworthy product, Patient Pure (PPX), is the first of its kind, a concentrated autologous exosome solution containing nanoparticles and proteins extracted from a patient’s own blood. This unique formulation has the ability to reduce components of cellular inflammation and foster tissue healing. The distinctive exosomes present in PPX feature multiple specific microRNA markers, targeting damaged cells, reducing inflammation and promoting cellular-level regeneration, ultimately facilitating long-term healing and repair.
15) Aravive
Compound annual growth rate: 76 percent
Aravive, Inc. is a late-stage oncology company dedicated to developing targeted therapeutics for the treatment of metastatic disease. Demonstrating remarkable financial performance, Aravive reported strong results for the first quarter of 2023, with a total revenue of $1.5 million, compared to $1.1 million for the same period in 2022. These revenues were exclusively generated through a collaboration and license agreement with 3D Medicines, which was initiated in November 2020 for the development and commercialization of batiraxcept in oncology indications in China.
The enduring success of Aravive can be attributed to its ongoing investment in R&D and the commercialization of innovative new medicines. Recently, the company announced promising results from the platinum-resistant ovarian cancer (PROC) Phase III trial for the candidate drug batiraxcept in mid-2023. It is now progressing towards the submission of a Biologics License Application (BLA) by the end of 2023.
In clinical studies conducted thus far, batiraxcept has exhibited a favorable safety profile and compelling early clinical activity in cancer patients. Its impressive safety and tolerability profile suggest its potential suitability for use in combination with various anti-cancer drugs or as a maintenance therapy.
16) TherapeuticsMD
Compound annual growth rate: 75 percent
TherapeuticsMD, Inc. is now a royalty pharmaceutical company dedicated to promoting women’s health and advocating for greater awareness of their healthcare concerns. Their products address the distinct changes and challenges women encounter during the different phases of their lives.
Therapeutics MD posted a noteworthy transition in its financial performance, reporting a net income of $1.1 million in 2022, as opposed to a significant net loss of $79.3 million in 2021. The company licensed its products to Mayne Pharma for $153 million and reduced its workforce to streamline the operations. They completed the transition to a virtual business model that solely collects royalties from Mayne Pharma and other licensees.
17) Twist Bioscience
Compound annual growth rate: 73 percent
Twist Bioscience is a leading and rapidly advancing synthetic biology and genomics company that has introduced a groundbreaking DNA synthesis platform designed to streamline the engineering of biology. At the heart of this platform lies a proprietary technology that pioneers a novel method for manufacturing synthetic DNA by “writing” DNA on a silicon chip.
In the third quarter of 2023, the company’s revenue surged to $63.7 million, marking a significant increase from the $56.1 million recorded during the same period in the fiscal year 2022. Anticipating a full fiscal year 2023 revenue in the range of approximately $241 million to $242 million, in contrast to the previous guidance of $235 million to $238 million, Twist Bioscience attributes this robust revenue growth to its core business, encompassing SynBio and next-generation sequencing (NGS).
The substantial investment in R&D is expediting the expansion of their new product pipeline. Powered by silicon, the DNA synthesis platform enables customers to miniaturize the chemistry required for DNA synthesis, effectively addressing the inherent limitations in terms of throughput, scalability and cost associated with traditional DNA synthesis methods. Notably, Twist Bioscience continues to enhance its NGS portfolio by introducing a new RNA sequencing portfolio, catering to the extensive research market. This state-of-the-art RNA sequencing tool empowers targeted or whole transcriptome research across a spectrum of fields, including precision medicine, biomarker discovery and immuno-oncology research.
18) Elite Pharmaceuticals
Compound annual growth rate: 62 percent
With a simple, straightforward strategy of developing generic versions of controlled-release drug products using proprietary know-how and technology, Elite Pharmaceuticals reported a 27 percent increase in revenues for the fiscal year 2022. The increase was primarily attributed to revenues from generic immediate-release Adderall, generic extended-release Adderall, generic Loxapine capsules and substantial revenues relating to Naltrexone tablet sales.
Elite is actively working on the development of distinctive niche generic products to drive future expansion. The company’s primary emphasis is on challenging formulations and excellent oral dose products, including extended-release items and those they believe offer substantial competitive benefits.
19) Rigel Pharmaceuticals
Compound annual growth rate: 50 percent
Rigel Pharmaceuticals is a biotechnology firm committed to the exploration, creation and delivery of innovative therapies that substantially improve the well-being of individuals grappling with hematologic disorders and cancer. The total revenue for 2022 reached $120.2 million, exhibiting a remarkable 20 percent increase compared to the $63 million reported in 2021.
They currently have two products in the market: Tavalisse (fostamatinib disodium hexahydrate) is the only oral spleen tyrosine kinase (SYK) inhibitor for the treatment of adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment and Rezlidhia (olutasidenib) capsules which are indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia with a susceptible IDH1 mutation as detected by an FDA-approved test.
20) JA Saúde Animal
Compound annual growth rate: 47 percent
JA Saúde Animal is a veterinary pharmaceutical company that is entirely Brazilian-owned and operated. Their core expertise lies in the R&D of innovative animal health solutions, complemented by a substantial manufacturing and commercial infrastructure.
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