While big pharma holds a sizable stake in the healthcare sector, several biotechs and mid-sized pharma companies fuel both the cutting-edge research and growing revenues in the pharmaceutical space.

In this feature, we take a look at the leading emerging companies in the US ranked based on their publicly disclosed revenues. The companies on this list are all headquartered in US and have a market cap of less than or equal to $10bn.

While having a relatively smaller footprint than big pharma companies, cumulatively, the top ten emerging pharmaceutical companies in the US employed a total of 17,170 people in 2023 data. Additionally, the annual revenues of these top ten emerging companies surpassed $12.5bn in the same year.

From Organon & Co to Denali Therapeutics, Pharmaceutical Technology lists the top ten emerging US companies in 2023, based on their revenues.

1. Organon & Co- $6.26bn

2. Exelixis - $1.83 bn

3. PTC Therapeutics - $937.8m

4. Acadia Pharmaceuticals - $726.4m

5. Supernus Pharmaceuticals - $607.5

6. Ultragenyx Pharmaceutical- $434.3 m

7. Amicus Therapeutics - $399.4m

8. Apellis Pharmaceuticals - $396.6m

9. Beam Therapeutics - $377.7m

10. Denali Therapeutics - $330.5m

1. Organon & Co- $6.26bn

Organon & Co (Organon) is the leading emerging pharmaceutical company in the US in 2023, reaching $6.26bn in annual revenues. The FY 2023 revenue of $6.26bn was 1% higher than the previous year and by 3% at constant currencies.

Organon is a pharmaceutical company that develops biosimilars and medicines with a focus on women’s health. The company provides over 60 medications and products within the field of women’s health, alongside an expanding biosimilar pipeline across various therapeutic areas such as respiratory, cardiovascular and dermatology.

The women’s health division’s revenue rose by 7% in 2023 and by 8% in Q4 2023 compared to Q4 2022, excluding foreign currency impacts. This observed growth was mainly driven by robust performance in the company's fertility products, especially Follistim AQ (follitropin beta injection), which experienced a 63% increase, excluding foreign exchange impacts during the final quarter of 2023.

2. Exelixis- $1.83 bn

The second emerging pharmaceutical company on our list is Exelixis, with total revenues reaching $1.83bn in 2023 in contrast to $1.61bn in the previous year.

The increased net product revenues were mainly attributed to growth in sales volume and a rise in the average net selling price of products. Additionally, the company’s collaboration revenues for license and services revenues increased from $46.5m in 2022 to $50.3m in 2023. The rise mainly stemmed from increased royalty earnings on the sales of oncology drug, Cabometyx (cabozantinib), outside the US facilitated by Exelixis’ collaboration partners, Ipsen Pharma and Takeda Pharmaceutical.

3. PTC Therapeutics- $937.8m

The next emerging US company on our list is PTC Therapeutics, which posted a 34% revenue growth in 2023. According to PTC Therapeutics’ CEO, Matthew Klein, the company “closed 2023 with strong revenue performance in the fourth quarter,” based on a company’s press release. 

PTC’s total revenues reached $937.8m last year and its Duchenne muscular dystrophy (DMD) franchise played a significant role in the company's revenue growth. Specifically, the DMD franchise revenue totaled $611m, driven by the sales of Translarna (ataluren) and Emflaza (deflazacort) which generated revenues of $355m and $255m respectively. The increase in generated revenue resulted from the rise in patient numbers and expansion into new regions for Translarna, alongside ongoing new prescriptions and enhanced access for Emflaza.

Lastly, PTC Therapeutics experienced some clinical milestones last year, such as the positive outcomes of the Phase III APHENITY clinical trial of the small molecule sepiapterin in phenylketonuria (PKU) treatment, which could represent a $1bn global commercial opportunity, and the PIVOT-HD study of PTC-518 for patients with Huntington's disease.

4. Acadia Pharmaceuticals- $726.4m

Acadia Pharmaceuticals experienced a staggering 40% revenue growth in 2023. The company’s total net product sales rose to $726.4m in 2023 compared to $517.2m that was reported in the previous year.

Acadia attributed this significant increase to the successful launch of their commercial product, Daybue (trofinetide), the first approved treatment for Rett syndrome. Rett syndrome is a rare, X-linked neurodevelopmental disorder leading to a range of cognitive, motor, and autonomic symptoms. Additionally, the company’s existing Nuplazid (pimavanserin) franchise experienced a 6% growth.

With Nuplazid, which is an FDA-approved treatment for hallucinations and delusions associated with Parkinson's disease psychosis, sales increased by 5% to $143.9m for the fourth quarter of 2023. Additionally, Nuplazid recorded net product sales of $549.2m in FY 2023, marking a 6% rise from 2022. This observed increase in net product sales of Nuplazid was attributed to a rise in unit sales combined with a higher average net selling price in 2023 compared to 2022.

Lastly, Daybue earned $177.2m since its April 2023 launch.

5. Supernus Pharmaceuticals- $607.5m

Supernus Pharmaceuticals posted an 8.9% decrease in revenue from 2022. The company announced a total revenue of $607.5m in 2023—a drop from $667.2m recorded in the previous year. Net product sales also dropped from $649.4m to $573.9m in FY 2023.

This decrease in net product sales is mainly attributed to the decline in anticonvulsant therapy, Trokendi XR (topiramate ER) sales. However, decreased Trokendi XR sales were somewhat balanced by a rise primarily in Qelbree (viloxazine hydrochloride) and Gocovri (amantadine hydrochloride) net product sales. Gocovri is a therapeutic agent for the treatment of Parkinson’s disease and dyskinesias.

In Q4 2023, net sales of Qelbree, a non-stimulant option for the treatment of ADHD,  increased by 97% to $46.4m compared to Q4 2022. Furthermore, FY 2023 net sales of Qelbree rose tremendously by 129% to $140.2m in comparison to FY 2022. According to the company, IQVIA’s prescription data shows a 91% rise in Qelbree prescriptions in 2023 compared to the previous year.

6. Ultragenyx Pharmaceutical - $434.3 m

Ultragenyx Pharmaceutical, a biopharmaceutical company dedicated to developing innovative treatments for severe rare and ultra-rare genetic disorders, displayed a 20% revenue growth compared to the previous year. 

These 2023 results were driven by the increased demand for both Crysvita (burosumab) and Dojolvi (triheptanoin) which reached $328m and $71m in revenue respectively. Crysvita is a monoclonal antibody marketed for the treatment of osteomalacia and Dojolvi is a triglyceride indicated for the treatment of pediatric and adult patients with long chain fatty acid oxidation disorders. Crysvita’s FY 2023 revenue showed a 17% growth compared to the same period in 2022.

One of the highly anticipated milestones for 2024 for the company is the readout of the Phase I/II/III trial (NCT04884815) for UX-701 (rivunatpagene miziparvovec) in Wilson’s disease. Emil Kakkis, CEO and president of Ultragenyx, noted that the company achieved multiple regulatory and reimbursement milestones for their commercial products, aiming to sustain a revenue growth rate of around 20% in 2024.

7. Amicus Therapeutics- $399.4m

In February this year, Amicus Therapeutics reported a 21% increase in total revenues amounting to $399.4m for 2023. Amicus is a biotech company developing advanced therapies for a range of devastating rare diseases.

The net product sales of Galafold (migalastat)— the first and only oral therapy for adults with Fabry disease—reached $387.8m for FY 2023, reflecting an 18% year-over-year growth. In 2024, the company expects Galafold revenue to grow propelled by sustained demand from both switch and treatment-naïve patients. This growth is also projected to be driven by expansion into new geographical regions, label extensions, continued diagnosis of new Fabry disease patients, and effective commercial strategies in key markets like the US, EU, Japan and UK.

In 2023 the company also successfully launched its second commercial therapy, Pombiliti + Opfolda (cipaglucosidase alfa + miglustat) in the three largest Pompe disease markets. Pompe disease is a rare genetic disorder that causes progressive muscle weakness to the heart. The drug earned $2.8m in net product sales in Q3 2023 in Germany and the UK and marketing is also underway in the US and EU.

8. Apellis Pharmaceuticals- $396.6m

Closely following Amicus Therapeutics, is Apellis Pharmaceuticals which generated $396.6m in revenue in 2023. Apellis possesses a strong ophthalmology pipeline, focusing on complement medicine, with two approved therapies targeting complement C3 in the protein cascade.

The company’s revenues were driven by the sales of Syfovre (pegcetacoplan injection) ($275m) and Empaveli (pegcetacoplan) ($91m) for FY 2023.

Syfovre, a treatment for geographic atrophy (GA) resulting from age-related macular degeneration (AMD), was launched successfully in 2023 and met with high demand. Empaveli, a drug for the treatment of paroxysmal nocturnal hemoglobinuria was approved in October 2023 and displays continued high patient compliance rates of 97%.

9. Beam Therapeutics - $377.7m

Beam Therapeutics’ financial results for 2023 exceeded analyst expectations due to a significant jump of $316.8m in revenue from the previous year. More specifically, the company’s total revenue rose from $60.9m in 2022 to $377.7m in 2023.

The biotechnology company is developing precision genetic medicines through base editing using a proprietary platform. The year 2024 represents a critical one, as the company is expected to initiate its first in vivo clinical studies as well as report the first in-human data from ex vivo base editing clinical programs. This year, the BEACON Phase I/II trial of BEAM-101 for severe sickle cell disease (SCD) is estimated to complete.

At the end of 2023, the company wrapped up with a substantial rise in cash reserves, amounting to $1.2bn, projected to sustain operations until 2027.

Notably, Beam Therapeutics announced a net profit of $142.8m for Q4 2023, a significant change from the net loss of $38.3m that was observed in the corresponding period of 2022, reflecting the company's strategic development and improved operational effectiveness.

10. Denali Therapeutics- $330.5m

The last company on our list is Denali Therapeutics, which reported a net loss of $145.2m for FY 2023. However, these financial results displayed an improvement from 2022’s net loss of $326m. Denali managed to bring in a revenue of $330.5m, which was primarily attributed to revenue recognised under the Biogen Collaboration Agreement on the LRRK2 Program for Parkinson’s Disease. Despite the net loss, the company still maintains a stable position with approximately $1.03bn in cash reserves.

Denali Therapeutics is a biopharmaceutical company developing a portfolio of therapeutic candidates that cross the blood-brain barrier (BBB) to treat neurodegenerative diseases and lysosomal storage diseases. Last year the company showed an increased investment in R&D, with expenses rising to $423.9m for FY 2023.

Note: The 19th paragraph has been updated to reflect the correct description of the drug Qelbree.