Rare Disease Biotechs Show Strong 2025 Performance with Cashflow and R&D Expansion
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Jan 14 2026
0mins
Source: Yahoo Finance
- Performance Breakthrough: Alnylam Pharmaceuticals achieved nearly $3 billion in sales in 2025 and projects sales of up to $5.3 billion in 2026, demonstrating strong growth potential in the rare disease sector.
- R&D Progress: Ultragenyx anticipates two gene therapy approvals in 2026 despite facing clinical trial setbacks in 2025, indicating ongoing investment and risk management in its development pipeline.
- Regulatory Support: The FDA's recent implementation of the Rare Disease Evidence Principles framework allows for therapy approvals based on single-arm studies, showcasing support for rare disease developers and potentially accelerating new therapies to market.
- Financial Outlook: PTC closed 2025 with $1.94 billion in cash reserves and plans to continue the global launch of its flagship product Sephience in 2026, aiming for break-even cash flow, reflecting robust development in the market.
Analyst Views on ALNY
Wall Street analysts forecast ALNY stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for ALNY is 500.28 USD with a low forecast of 351.00 USD and a high forecast of 570.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.
20 Analyst Rating
16 Buy
4 Hold
0 Sell
Strong Buy
Current: 361.350
Low
351.00
Averages
500.28
High
570.00
Current: 361.350
Low
351.00
Averages
500.28
High
570.00
About ALNY
Alnylam Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company. The Company is engaged in discovering, developing, manufacturing and commercializing novel therapeutics based on ribonucleic acid interference (RNAi). Its marketed products include AMVUTTRA (vutrisiran) for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR) with polyneuropathy in adults; ONPATTRO (patisiran) for the treatment of the polyneuropathy of hATTR amyloidosis in adults; GIVLAARI (givosiran) for the treatment of adults with acute hepatic porphyria; OXLUMO (lumasiran) for the treatment of primary hyperoxaluria type 1, and Leqvio (inclisiran), which is being developed and commercialized by its partner, Novartis AG, for the treatment of adults with hypercholesterolemia or mixed dyslipidemia. The Company’s clinical development programs include Cemdisiran, Fitusiran, Zilebesiran, Elebsiran, Mivelsiran and ALN-HTT02. It is developing Cemdisiran to treat complement-mediated diseases.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
