The global ALS therapeutics market is valued at USD 812 million in 2024 and is projected to reach USD 1,964 million by 2035, growing at an 11.5% CAGR. Growth is driven by innovations in genetic therapies, gene-silencing technologies, improved diagnostics, and increased disease awareness. Key players include Biogen, Ionis Pharmaceuticals, Amylyx Pharmaceuticals, and others developing disease-modifying treatments.

The global amyloidosis therapeutics market is experiencing strong growth, valued at $2.95 billion in 2024 and expected to reach $6.37 billion by 2033 with a 9.20% CAGR. Growth is driven by accelerating ATTR cardiomyopathy diagnosis through imaging technologies, rapid uptake of disease-modifying therapies in North America, and expanded reimbursement in emerging markets like China and Brazil. Five core compounds dominate the market, with chemotherapy remaining the cost-effective backbone for AL amyloidosis treatment.

GSK and Ionis Pharmaceuticals announced positive Phase 3 trial results for bepirovirsen, an investigational antisense oligonucleotide for chronic hepatitis B. The B-Well 1 and B-Well 2 studies, involving over 1,800 patients, met their primary endpoint with statistically significant functional cure rates. If approved, bepirovirsen could become the first finite, six-month therapeutic option for CHB. Regulatory submissions are planned for Q1 2026.

Biogen has acquired Alcyone Therapeutics, a clinical-stage biotechnology company, to enhance its CNS therapy delivery solutions through the ThecaFlex DRx™ drug delivery system, which aims to provide an alternative to repeat lumbar punctures for neurologic disorder treatments.

Ionis Pharmaceuticals reported positive results from a clinical trial of zilganersen for Alexander disease, showing statistically significant stabilization of gait speed and the first evidence of disease modification in this rare neurological condition.

Antisense oligonucleotide (ASO) therapeutics are emerging as a promising treatment approach for rare genetic and neuromuscular disorders, with over 70 pharmaceutical companies actively developing 90+ pipeline therapeutics targeting specific genetic mechanisms.

The FDA approved Ionis Pharmaceuticals' Dawnzera, the first RNA-targeted medicine for preventing hereditary angioedema (HAE) attacks in patients 12 and older, demonstrating significant attack rate reduction in clinical trials.

Biogen will present scientific data on lecanemab and tau-targeted therapies at the 2025 Alzheimer's Association International Conference, showcasing long-term clinical trial results and potential new treatment approaches for Alzheimer's disease.

The angioedema treatment market is expected to grow due to the rising prevalence of allergic conditions and increasing awareness of hereditary angioedema (HAE). The pipeline analysis shows over 15 companies are developing 20+ angioedema drugs, with promising therapies in different clinical trial phases.

Praxis Precision Medicines, a clinical-stage biopharmaceutical company, will present data from its four late-stage programs in epilepsy and movement disorders at the American Academy of Neurology 2025 Annual Meeting. The company's innovative pipeline includes therapies for central nervous system disorders, with a focus on neuronal excitation-inhibition imbalance.

BESREMi, a FDA-approved treatment for polycythemia vera, is gaining market share due to its potential for better tolerability and lower risk of treatment-related malignancies compared to competitors like ruxolitinib and hydroxyurea.

Ionis Pharmaceuticals, a leader in RNA-targeted drug discovery, reported better-than-expected revenue and a smaller-than-expected loss in Q4 2024. The company's recent product launches, including Wainua and Tryngolza, contributed to the positive results, and it plans to continue investing in its pipeline and product launches in 2025.