The last day of February is International Rare Disease Day®, which aims to raise awareness of rare diseases to make a difference. Diseases with a prevalence of less than 1/2000 people are classified as rare diseases. About 70% of rare diseases occur in childhood, and nearly one in five is cancer.1More than 7,000 rare diseases are known, many of which are fatal. Due to the small number of patients with rare diseases, most rare diseases often do not receive adequate attention, research, or funding. In the United States, the Orphan Drug Act was signed into law in 1983 and incentivizes the development of rare disease drugs through tax breaks, user fee waivers, and market-exclusive incentives. In addition, the U.S. FDA provides funding for rare disease research,2In 2022, the Center for Drug Evaluation and Research (CDER) launched the Accelerated Rare Disease Therapy (ARC) program to accelerate the development of treatments for rare diseases.3

Peptides are essentially advantageous as a therapeutic and detection of disease, including cell-permeability, receptor binding, and tumor targeting.

Some of the peptides in clinical development for rare diseases include:

· PTH analogues used in the treatment of hypoparathyroidism4。

· Insulin-like growth factor-binding protein 2 (IGFBP2) peptide for the regulation of fat and glucose metabolism4。

· A peptide vaccine against CMV antigen for the treatment of newly diagnosed high-grade glioblastoma in children5。

· Peptides used in the treatment to improve degenerative retinopathy.

· Broad-spectrum antibiotic peptides for the treatment of prosthetic joint infections (PJI)6。

· Rusfertide, an iron-stimulating mimetic used to treat iron overload (e.g., polycythemia vera).7。

Some of the drugs that have been approved for rare diseases include:

· Ziconotide, an omega-conotoxin derived from conotoxin, interferes with pain signals and is approved for long-term pain relief.

· Afamelanotide for the prevention of phototoxicity in adult erythropoietic protoporphyria.

· Carfilzomib for multiple myeloma in adults.

· Ciclosporin, for severe spring keratoconjunctivitis in children and adolescents over 4 years of age.

· Lutetium Lu 177 dotatate injection is used for the treatment of gastroenteropancreatic neuroendocrine tumors with somatostatin receptor positive in vivo.

· Pasireotide (Pasireotide), which is used to treat hyperpituitary growth hormone Cushing's disease.

· Teduglutide, a GLP-2 analogue that promotes mucosal growth, is used to treat short bowel syndrome.

Go Top can support companies developing peptides for rare diseases by manufacturing and supplying peptides in clinical development or approved commercial products for the treatment, prevention, and diagnosis of a wide range of rare diseases through cGMP. As a peptide contract development and manufacturing organization (CDMO) providing cGMP peptide APIs, AmbioPharm has R&D to commercial-scale capabilities to actively partner with innovative biopharmaceutical companies to develop best-in-class and breakthrough peptide technologies that leverage AmbioPharm's peptide manufacturing expertise and deep scientific experience in novel and traditional peptide chemistry.