FDA approves 55 new drugs in 2023, one of the highest numbers in a decade
Infectious diseases, neurological diseases, type 2 diabetes in children, oncology and cardiac disorders are just some of the areas that benefited from the innovation drive last year.
More than half of these innovations have orphan drug designation, i.e. they are intended for patients with rare diseases
36% have been classified as “first in class” because they address unmet medical needs or have a new mechanism of action.
Farmaindustria.es
Biomedical innovation and the commitment of pharmaceutical companies to new drug research and patients continues apace. The drug regulatory agency’s (FDA) Center for Drug Evaluation and Research approved 55 drugs based on new active ingredients in 2023, according to its annual report on the previous year’s progress.
The approvals include treatments for infectious diseases, including Covid-19, respiratory syncytial virus (RSV) and HIV; neurological conditions, such as amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and migraine; type 2 diabetes in children; different types of anaemia; and chronic weight management, among other drugs for heart, blood, kidney and endocrine disorders. “These approvals, which cover a wide range of diseases and health conditions, are intended to help many people live better lives and achieve a longer life expectancy,” the FDA explains in its report.
In addition, more than half of the new drugs (28 of the 55 approvals) are intended to prevent, diagnose or treat a rare disease and therefore received orphan drug designation, confirming the innovative pharmaceutical industry’s drive for research in this area of rare diseases, a constant demand among healthcare professionals and patients and their families.
In addition to ALS, approvals have come for Friedreich’s ataxia, an inherited degenerative disease that damages the nervous system; for paroxysmal nocturnal haemoglobinuria, a disease that causes red blood cells to rupture; and for activated phosphoinositide 3-kinase delta syndrome, a genetic disorder that damages the immune system. For rare cancers, therapies are already available for mantle cell lymphoma, an aggressive form of non-Hodgkin’s lymphoma, and nasopharyngeal carcinoma, a cancer of the head and neck.
For lung diseases, new medicines have been approved for asthma attacks and cystic fibrosis. For gastrointestinal conditions, drugs for inflammatory bowel disease and paediatric functional constipation, while in oncology, approvals included treatments for colorectal, prostate, lung and low-grade cancers (less aggressive tumours that start in the brain). In women’s health, the FDA approved treatments for postpartum depression, hot flushes caused by menopause, and over-the-counter oral contraceptives.
Similarly, the FDA’s Center for Drug Evaluation and Research identified 20 of the 55 new drugs (36%) as first-in-class, i.e. drugs that have different mechanisms of action from existing therapies or treat unmet medical needs.
Over the past ten years, the US agency has approved an average of 46 new treatments each year, and these 55 approvals are the second highest in a decade. “The availability of more and better treatments has a direct impact on society as a whole, because in addition to curing and controlling diseases, reducing symptoms and improving patients’ quality of life, they boost the effectiveness and efficiency of the healthcare system, enable greater labour productivity and generate a significant contribution to the national economy,” recalls Isabel Pineros, director of Farmaindustria’s Access Department.
“But innovation only matters if it reaches patients, so we need to push for faster, fairer and more sustainable access to innovative medicines and for these FDA-approved innovations to reach Europe as well, for the benefit of our society,” Pineros says.
In this sense, the vast majority of these new medicines have not yet reached the European region, largely due to the size of the US market, the receptiveness of innovation and the speed of FDA procedures compared to those of the European Medicines Agency (EMA). It is essential that the reform of European pharmaceutical legislation includes incentives for innovation that will improve Europe’s attractiveness to companies, make the EMA more streamlined in its procedures, and create the conditions for companies to choose to invest in research, development and manufacturing in Europe.
“The FDA approvals demonstrate that the work of our companies to continue to innovate does not cease and that new treatment hopes are opening up for many patients and their families in numerous therapeutic areas, many of them hitherto unanswered. This is undoubtedly the best news so that our society can continue to move forward,” concludes the director of Farmaindustria’s Access Department.
