3 Niche Healthcare Stocks Tackling Rare Diseases
The healthcare market is undergoing a significant transformation, driven largely by technological advancements and the need to address global health challenges. Increasing demand for innovative healthcare solutions, spurred by aging populations, chronic conditions, and a gradual shift towards value-based care, makes this a pivotal time for investors. In this context, we will delve into three fundamentally strong biotech stocks—Immunocore Holdings plc (IMCR), Catalyst Pharmaceuticals, Inc. (CPRX), and BioCryst Pharmaceuticals, Inc. (BCRX)—that are tackling rare diseases. These companies are well-positioned to leverage trends in personalized medicine, genetic engineering, and sustainable healthcare practices.
The Booming Biotech Landscape
The global biotechnology market is expected to grow at a compound annual growth rate (CAGR) of 6.3% by 2033. This growth trajectory can be attributed to a rising focus on research and development (R&D) in personalized medicine and the ever-increasing innovations in genetic engineering. Additionally, the digital health market is expected to grow at an impressive CAGR of 11.7% by 2034, driven by ongoing advancements in artificial intelligence, the Internet of Things (IoT), and remote monitoring technologies.
Stock #3: Immunocore Holdings plc (IMCR)
Immunocore Holdings plc (IMCR) is a commercial-stage biotechnology company that specializes in developing immunotherapies for cancer, infectious, and autoimmune diseases. The company’s flagship product, KIMMTRAK (tebentafusp), is approved for the treatment of patients with unresectable or metastatic uveal melanoma. On December 3, 2024, IMCR announced that KIMMTRAK received a recommendation for funding through the National Health Service (NHS) in England from the National Institute for Health and Care Excellence (NICE).
In the third quarter of its fiscal year ending September 30, 2024, IMCR reported a remarkable 23.7% year-over-year increase in total revenue, reaching $80.25 million. The company’s net income before taxes grew to $6.09 million, a significant rise compared to the previous year. Analysts anticipate that IMCR’s revenue for the fourth quarter will grow by 20.7% year-over-year to approximately $84.72 million.
Currently, IMCR’s stock price has surged by 11.8% over the past month, closing at $32.61. It holds an overall rating of B in our proprietary POWR Ratings system, which evaluates stocks based on 118 factors. The stock has received a B grade for Growth, Value, and Quality and is ranked #23 out of 330 stocks in the biotech industry.
Stock #2: Catalyst Pharmaceuticals, Inc. (CPRX)
Catalyst Pharmaceuticals, Inc. (CPRX) is a commercial-stage biopharmaceutical company that is dedicated to developing therapies for rare, debilitating neuromuscular, and neurological diseases in the United States. On January 21, 2025, CPRX announced that its sub-licensee in Japan, DyDo Pharma, Inc., launched FIRDAPSE (amifampridine) tablets in Japan to improve muscle weakness in patients with Lambert-Eaton myasthenic syndrome (LEMS).
For the third quarter of 2024, which ended September 30, CPRX reported a 23.2% increase in net product revenue to $126.42 million. Impressively, the company’s non-GAAP net income reached $71.08 million compared to a net loss of $25.64 million in the same quarter last year. Analysts predict a revenue increase of 21.3% in the fourth quarter of 2024, projecting revenues to reach $134.08 million.
CPRX shares have gained 8% over the past month, closing at $23.19. The stock also enjoys an overall rating of B in our POWR Ratings, with an A grade for Sentiment and B grades for Value and Quality, ranking #16 within its industry.
Stock #1: BioCryst Pharmaceuticals, Inc. (BCRX)
BioCryst Pharmaceuticals, Inc. (BCRX) is focused on developing oral small-molecule and protein therapeutics for rare diseases. Its marketed products include peramivir injection for acute uncomplicated influenza and ORLADEYO (berotralstat), an oral medication aimed at hereditary angioedema (HAE). On November 18, 2025, BCRX received positive news when the Health Services Executive (HSE) in Ireland recommended ORLADEYO for routine prevention of recurrent HAE attacks in eligible patients aged 12 and above.
BCRX reported a 35% year-over-year revenue increase for the third quarter of 2024, totaling $117.09 million. Moreover, the company’s income from operations shifted positively to $7.69 million, compared to a loss of $11.92 million in the prior year. As of September 30, 2024, BCRX had cash and investments amounting to $349.44 million, with total assets at $491.25 million.
For the upcoming fiscal year quarter ending December 2024, consensus estimates project revenue to reach $131.10 million, marking a 40.4% increase year-over-year. The company has exceeded consensus revenue estimates in each of the last four quarters, showcasing its consistent performance. With a remarkable 84.3% increase over the last nine months, BCRX’s stock closed at $7.87 with a POWR Rating of B.
Conclusion
As the demand for innovative solutions in healthcare continues to rise, investing in companies that focus on rare diseases offers a promising opportunity for growth. Immunocore Holdings (IMCR), Catalyst Pharmaceuticals (CPRX), and BioCryst Pharmaceuticals (BCRX) are exemplars of companies poised to benefit from favorable industry conditions and technological advancements. As investors seek to align their portfolios with emerging trends in biotech and personalized medicine, these stocks merit serious consideration.
