INSIGHT. FDA’s latest drug approvals provide clear indications of future directions in drug development and point towards more personalised and groundbreaking treatments.
Here are some of the key development areas we’re seeing right now:
Breakthrough in gene editing
The groundbreaking approval of Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics to treat blood disorders beta-thalassemia and sickle cell disease, is an important step forward for gene editing. Casgevy is the first treatment based on CRISPR, a gene editing technique that has received FDA approval, demonstrating FDA’s increased confidence in gene editing as treatment.
Extended applications for existing drugs
Pharmaceutical companies are increasingly focusing on broadening the use of their approved drugs. One example is Dupixent, which, in addition to being approved for treating atopic dermatitis has now received approval for treating the inflammatory disease eosinophilic esophagitis (also known as allergic esophagus) in younger patients. This gives more patients access to necessary treatment while extending the drug’s commercial lifespan.
Progress in precision medicine
FDA’s approval of new, targeted treatments such as Donanemab (Kisunla) for Alzheimer’s disease is an important step forward for precision medicine. Donanemab is designed to reduce harmful amyloid plaque in the brain and slow disease progression. Additionally, FDA has for the first time approved a cell therapy for treating solid tumours – Amtagvi. Cell therapy has previously been used successfully in certain types of blood cancer – being able to show progress in treating solid tumours marks a significant breakthrough, as these account for approximately 90% of all cancer cases. The approval of Amtagvi is also important for the Swedish biotech company Neogap Therapeutics, which is also developing cell therapies for solid tumours but specifically for patients with colorectal cancer.
Focus on rare diseases
FDA continues to prioritise treatments for rare diseases, exemplified by the approval of Vyvgart for the autoimmune muscle disease myasthenia gravis. This provides new treatment options for patient groups with few alternatives and creates incentives for pharmaceutical companies to invest in previously overlooked areas.
In summary, these approvals show how technical advances and better understanding of genetics are driving drug development towards more tailored and effective treatments – a progression that can improve the quality of life for patients worldwide.
