Related coverage linked through entity extraction aliases.
Mar 25, 2026
Italian pharmaceutical company Recordati reported 2025 revenues of €2.6 billion and adjusted net profit of €651.1 million, with rare disease therapies driving 29.7% growth. The company projects 2026 core earnings of €995 million to €1.03 billion, anticipating continued momentum in its specialized rare disease treatments.
Mar 24, 2026
The AI-driven precision medicine market is projected to grow from $0.7 billion in 2025 to $9.7 billion by 2035 at a 26.8% CAGR. North America dominates with 62-67% market share, while machine learning accounts for 39% of the market. The market is moderately consolidated with the top five players controlling over 64% share.
Mar 23, 2026
Sarepta Therapeutics received FDA feedback supporting supplemental applications for AMONDYS 45 and VYONDYS 53, while New Zealand approved a Phase 1 trial for Huntington's Disease candidate SRP-1005. The company faces significant share price declines despite these developments, with analysts showing wide disagreement on valuation targets ranging from $5 to $80.
Mar 20, 2026
The global AI in clinical trials market is expected to grow from $2.09 billion currently to $18.62 billion by 2040, driven by enhanced patient recruitment, optimized trial designs, and automation that reduces time and costs in drug development.
Mar 17, 2026
Global orphan drug sales are forecast to reach $409 billion by 2032, representing one-fifth of prescription drug sales, with Johnson & Johnson and Argenx leading the market amid regulatory uncertainty.
Mar 17, 2026
Diagnosing rare diseases can take 3 to 15 years, while about 95% lack approved treatments. Families are advocating for regulatory changes to speed access to therapies as new digital tools aim to improve clinical trials.
Mar 15, 2026
Regulatory incentives for rare disease treatments, including market exclusivity and development subsidies, are spurring pharmaceutical innovation across the US, Europe, Japan, and Australia, with multiple companies advancing therapies for conditions affecting limited patient populations.
Mar 12, 2026
The global next generation drug conjugates market is projected to grow from $3.9 billion in 2026 to $10.9 billion by 2033 at 15.3% CAGR, while the radionuclide drug conjugate segment is forecast to reach $20.22 billion by 2032 at 9.6% CAGR.
Mar 12, 2026
New AI platforms are emerging to address rare disease diagnosis and epidemiology, as global costs reach up to $8.6 trillion annually affecting 300 million patients worldwide across more than 7,000 distinct conditions.
Mar 11, 2026
The Consolidated Appropriations Act, 2026 enacted February 3 narrows orphan drug exclusivity to specific approved uses rather than entire disease categories, overriding a 2021 court decision and codifying FDA's longstanding interpretation.
Mar 11, 2026
The FDA has established a "plausible mechanism pathway" to approve personalized genome editing and RNA-based therapies for rare and ultra-rare diseases without requiring large randomized controlled trials, streamlining access to individualized treatments.
Mar 11, 2026
The FDA is expected to issue draft guidance reducing testing requirements for biosimilar drugs, potentially lowering development costs by USD 20 million. The move comes as the biosimilars market shows strong growth, with major launches in 2025.
Mar 03, 2026
Multiple organizations submitted comments opposing CMS's proposed GLOBE and GUARD models, which would tie Medicare drug reimbursement to international reference prices. Critics argue the models function as price controls that could undermine pharmaceutical innovation.
Mar 02, 2026
The pharmaceutical industry is experiencing recovery driven by innovation in drug development, aggressive M&A activity, and expansion of drug development services. The sector faces both opportunities and headwinds including pipeline setbacks and regulatory pressures.
Mar 02, 2026
Over 100 children with rare diseases in India have exhausted the Rs 50 lakh financial assistance limit under the National Policy for Rare Diseases 2021, forcing treatment halts and prompting urgent appeals for continued support.
Mar 01, 2026
Artificial intelligence is revolutionizing rare disease diagnosis, cutting diagnostic timelines from years to weeks. Rare Disease Day on February 28 highlights challenges faced by over 300 million people worldwide living with more than 7,000 distinct rare conditions.
Feb 26, 2026
Three new market reports project significant expansion in pharmaceutical R&D outsourcing, the U.S. pharmaceutical market, and biotechnology services outsourcing, driven by innovation, aging populations, and cost efficiency needs through 2035.
Feb 24, 2026
Health systems are learning that successful cell and gene therapy programs require early pharmacy involvement, strong payer partnerships, and flexible infrastructure to navigate complex contracts, workflows, and financial realities.
Feb 25, 2026
The global stem cell therapy market is projected to grow from $18.13 billion in 2025 to $59.70 billion by 2035 at a 12.66% CAGR, driven by chronic disease prevalence and regenerative medicine advances.
Feb 26, 2026
Orphan drug development faces structural uncertainty due to small patient populations and varying global definitions, with regulators balancing early access against evidence requirements through accelerated pathways and post-market monitoring.
