Peptides are short chains of amino acids, which are the fundamental building blocks of proteins. These molecules are typically composed of two to fifty amino acids linked together. In the human body, peptides play various roles, acting as signaling molecules, hormones, or even antimicrobial agents. While proteins are generally larger, usually containing more than 100 amino acids, peptides are smaller and can exert potent effects on cells and tissues.
What FDA Approval Means for Peptides
The U.S. Food and Drug Administration (FDA) approval process ensures that a peptide product is safe and effective for its intended medical use. This rigorous evaluation relies on extensive scientific evidence gathered through preclinical research and human clinical trials. The approval signifies that the drug’s benefits outweigh its known and potential risks for the target patient population.
The FDA’s review also guarantees the product’s quality and purity, confirming that it is manufactured consistently and meets strict standards. Companies seeking approval must submit comprehensive data on the drug’s composition, manufacturing processes, and the results from all testing phases. This meticulous process helps to ensure that approved peptides can be trusted by healthcare providers and patients alike.
Therapeutic Peptides Approved by the FDA
Diabetes Management
Insulin was the first peptide approved by the FDA in 1922 for treating diabetes mellitus. More recently, glucagon-like peptide-1 (GLP-1) receptor agonists like semaglutide (Ozempic, Rybelsus) are approved for type 2 diabetes and sometimes weight management. Tirzepatide (Mounjaro) is another FDA-approved peptide that activates both GIP and GLP-1 receptors, improving glycemic control in adults with type 2 diabetes.
Cancer Treatment
For cancer treatment, several peptide-based therapies have gained approval. Gonadotropin-releasing hormone (GnRH) analogs and somatostatin analogs are examples of peptides used to manage certain types of cancer, including breast and prostate cancers. Peptide-drug conjugates (PDCs) deliver cytotoxic agents directly to cancer cells, minimizing systemic toxicity. Lutathera, a radiolabeled peptide, is approved for gastroenteropancreatic neuroendocrine tumors, and Pepaxti for multiple myeloma.
Pain and Hormone Conditions
In pain management, ziconotide, marketed as Prialt, is an FDA-approved peptide used for severe chronic pain in adult patients who have not responded to other treatments. Pentosan polysulfate (PPS) is another approved oral medication with anti-inflammatory properties that can be used for pain associated with osteoarthritis. Peptides also play a role in hormone-related conditions, with teriparatide approved for osteoporosis and somatropin used as a growth hormone. Trofinetide, known as Daybue, received FDA approval in 2023 for treating Rett syndrome, marking a notable advancement for this rare neurodevelopmental disorder.
Diagnostic and Other Approved Peptides
Beyond direct therapeutic applications, peptides also serve important functions in medical diagnostics. These approved peptides often assist in visualizing specific tissues or disease markers within the body. For instance, flotufolastat F 18 is an FDA-approved radioactive diagnostic agent used in positron emission tomography (PET) imaging for prostate cancer. This peptide binds to prostate-specific membrane antigen (PSMA) overexpressed by prostate cancer cells, allowing for their detection.
Some peptides are designed as theragnostic agents, meaning they combine both diagnostic and therapeutic capabilities. These innovative peptides can help identify disease presence and then deliver a targeted treatment to the same area. The development of such agents highlights the versatility of peptides in medical applications that extend beyond traditional drug therapies.
Why FDA Approval Matters
FDA approval is crucial for medical products like peptides. Without this regulatory oversight, products may lack scientific backing, reliable quality control, or have unknown risks and side effects. This protects public health by promoting the availability of reliable and beneficial medical treatments.