3 Niche Healthcare Stocks Tackling Rare Diseases
The healthcare market is experiencing a significant transformation driven by technological advancements and global health challenges. With an ever-increasing demand for innovative solutions arising from aging populations, chronic conditions, and a greater emphasis on value-based care, healthcare stocks that focus on rare diseases are becoming more appealing to investors. This article examines three fundamentally strong stocks in this niche: Immunocore Holdings plc (IMCR), Catalyst Pharmaceuticals, Inc. (CPRX), and BioCryst Pharmaceuticals, Inc. (BCRX).
The Growing Biotech Landscape
The biotech sector is undergoing remarkable growth due to several factors including heightened research and development (R&D) aimed at personalized medicine, advancements in genetic engineering, and increasing demand for sustainable agriculture. According to reports, the global biotechnology market is anticipated to expand at a compound annual growth rate (CAGR) of 6.3% by 2033. In parallel, the digital health market is expected to enjoy growth driven by ongoing technological innovations, with a projected CAGR of 11.7% through 2034.
Stock #3: Immunocore Holdings plc (IMCR)
Immunocore Holdings plc (IMCR) is a commercial-stage biotechnology company focused on developing immunotherapies for various diseases, including cancer and autoimmune disorders. The company’s primary product, KIMMTRAK (tebentafusp), is designed for patients suffering from unresectable or metastatic uveal melanoma. In a notable development, on December 3, 2024, IMCR announced that KIMMTRAK received a funding recommendation from the National Health Service (NHS) in England.
For the third quarter ending September 30, 2024, IMCR reported a remarkable 23.7% year-over-year increase in total revenue, reaching $80.25 million. Their net income before taxes soared to $6.09 million, a considerable rise from the previous year. Additionally, the net income per share grew significantly to $0.17. Analysts project a revenue increase of 20.7% year-over-year for the fourth quarter (ending December 2024), estimating it to reach $84.72 million. Recently, IMCR’s shares gained 11.8%, closing at $32.61.
In terms of ratings, IMCR has an overall POWR Rating of B, indicating a Buy in our proprietary system. The stock earns B grades for Growth, Value, and Quality, and is ranked #23 out of 330 stocks within the Biotech industry.
Stock #2: Catalyst Pharmaceuticals, Inc. (CPRX)
Catalyst Pharmaceuticals, Inc. (CPRX) is a biopharmaceutical company dedicated to developing and commercializing therapies for rare and debilitating chronic neuromuscular and neurological diseases within the United States. On January 21, 2025, CPRX reported that its sub-licensee in Japan, DyDo Pharma, Inc., launched FIRDAPSE (amifampridine) Tablets 10 mg for treating muscle weakness in patients with Lambert-Eaton myasthenic syndrome (LEMS).
In the third quarter of 2024, CPRX saw a 23.2% year-over-year increase in net product revenue to $126.42 million. Its non-GAAP net income reached $71.08 million compared to a substantial net loss of $25.64 million in the corresponding quarter of the previous year. Furthermore, analysts expect CPRX to achieve revenue and earnings per share (EPS) growth of 21.3% and 17.2% respectively in the fourth quarter. Stock performance for CPRX has been strong, showing an 8% increase over the past month.
CPRX’s prospects are reflected in its POWR Ratings, with the stock receiving an overall rating of B, equating to Buy. It boasts an A grade for Sentiment and B grades for Value and Quality, landing it at #16 within the same industry.
Stock #1: BioCryst Pharmaceuticals, Inc. (BCRX)
BioCryst Pharmaceuticals, Inc. (BCRX) is a biotech firm specializing in developing oral small-molecule and protein therapeutics for rare diseases. Among its offerings are peramivir injection for treating acute uncomplicated influenza and ORLADEYO (berotralstat), an oral therapy for hereditary angioedema (HAE). On November 18, 2025, BCRX announced a significant recommendation from the Health Services Executive (HSE) in Ireland to make ORLADEYO accessible for eligible HAE patients aged 12 and older.
BCRX reported substantial growth in total revenues for the third quarter of 2024, which increased by 35% year-over-year to $117.09 million. It also showed improved operational income of $7.69 million compared to a loss of $11.92 million the previous year. As of September 30, 2024, BCRX had $349.44 million in cash and investments with total assets amounting to $491.25 million. With a projected revenue of $131.10 million for the upcoming fiscal year quarter ending December 2024, representing a year-over-year growth of 40.4%, the stock has surged 84.3% over the past nine months.
Reflecting its positive outlook, BCRX has an overall POWR Rating of B, indicating a Buy, further boosted by its satisfactory performance in revenue estimates during previous quarters.
Conclusion
In conclusion, with the ever-expanding needs in the healthcare industry, particularly for innovative solutions addressing rare diseases, stocks like Immunocore Holdings plc (IMCR), Catalyst Pharmaceuticals, Inc. (CPRX), and BioCryst Pharmaceuticals, Inc. (BCRX) are well-positioned for investment. Their strong financials, growth prospects, and commitment to addressing pressing health challenges make them attractive options for investors seeking opportunities in the biotech sector.