CSL Behring

In 2024, the European Medicines Agency received a record number of marketing authorization applications for advanced therapy medicinal products. Notable filings include UCB’s orphan drug for thymidine kinase 2 deficiency and Novo Nordisk’s Alhemo for hemophilia. The FDA approved 50 novel agents and 11 biologics. EMA recommended several new products for EU approval, including treatments for hereditary angioedema and transthyretin amyloidosis. Sanofi’s rilzabrutinib and Alvotech/Advanz Pharma’s golimumab are under review. EMA also reconsidered Eisai/Biogen’s Alzheimer’s drug for approval. The UK approved Eli Lilly’s Alzheimer’s drug, despite NICE's reservations.

CAR T-cell therapies, a breakthrough in Hematology and Oncology, show high efficacy but cause toxicities like cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). ICANS involves neurological symptoms and is linked to blood-brain barrier permeability and CNS inflammation. Understanding ICANS's pathophysiology is crucial for safer, broader CAR T-cell therapy application.

In 2024, Hemophilia News Today highlighted key advancements in hemophilia treatment, including gene therapies like BBM-H803 for hemophilia A, Mim8's efficacy, and the launch of Beqvez for hemophilia B. Roctavian showed significant bleeding reduction, and the FDA cleared a CRISPR therapy trial for hemophilia B, marking a year of progress in hemophilia care.

In 2024, the pharmaceutical industry saw significant patent expirations, leading to increased generic competition. Blockbuster drugs like Sprycel, Tysabri, Myrbetriq, Victoza, and others faced generics, impacting revenues but improving medication access. Companies focused on new drug launches to offset losses, highlighting the industry's ongoing adaptation to patent cliffs.

In 2024, Angioedema News highlighted top advancements in angioedema treatment, including deals for donidalorsen, NTLA-2002's 95% attack reduction, Takeda's partnerships, Takhzyro's efficacy in adolescents, deucrictibant's Phase 3 trials, FDA's lift on deucrictibant hold, Ionis' FDA application for donidalorsen, garadacimab's safety, Haegarda's real-world effectiveness, and Orladeyo's long-term benefits in Japan.

The Myasthenia Gravis Treatment Market is set to grow significantly by 2034, driven by improved diagnostics, emerging therapies, and increased awareness. Key companies like Horizon Therapeutics and Amgen are developing novel treatments. The market size reached USD 4,950 million in 2023, with the U.S. seeing 129 thousand cases.

DelveInsight's 2024 report highlights over 50 companies developing 50+ therapies for Systemic Sclerosis, detailing drug profiles, clinical trials, and mechanisms of action. Key players include Eicos Sciences and Corbus Pharmaceuticals, with promising therapies like GS-248 and Lenabasum in development.

The 'Myositis - Pipeline Insight, 2024' report by ResearchAndMarkets.com offers insights into 18+ companies and 20+ pipeline drugs for Myositis, covering drug profiles, therapeutic assessments, and pipeline development activities.

The Inflammatory Myositis market is set to grow significantly by 2034, driven by increasing disease prevalence and new therapies. Key companies include Merck KGaA, Pfizer, and others, with treatments like Brepocitinib and PF-06823859 expected to revolutionize the market. The US led the market in 2023, with a projected CAGR of 11.7% across major markets.

EMA's CHMP recommended garadacimab for HAE, ages 12+, as a once-monthly preventive treatment. The European Commission will review the recommendation, with a decision expected in Q1 2025. Garadacimab, designed to block FXII activation, reduced swelling attack rates by over 85% in a Phase 3 trial, with ongoing extension study showing 95% reduction and half of patients free from attacks. Common side effects were injection site reactions.