Oligonucleotide drugs, also known as antisense drugs, are a type of medication designed to target specific genes in order to treat or prevent diseases. They work by binding to messenger RNA (mRNA) molecules and blocking their ability to produce certain proteins.
Recently, several oligonucleotide drugs have been approved by the FDA, providing new treatment options for patients. Here are some of the latest developments in this field:
Lumasiran was approved in November 2022 for the treatment of Primary Hyperoxaluria Type 1 (PH1), a rare genetic disorder that causes the buildup of oxalate in the kidneys and other organs. The drug works by targeting and degrading the mRNA that codes for the enzyme responsible for oxalate production.
Givosiran was approved in January 2023 for the treatment of Acute Hepatic Porphyria (AHP), a group of rare genetic disorders that affect the liver and can cause severe abdominal pain, nausea, and vomiting. The drug works by blocking the production of a protein that is involved in the production of heme, a component of hemoglobin.
Milasen was approved in March 2023 for the treatment of Batten disease, a rare and fatal neurodegenerative disorder that affects children. The drug works by targeting the mRNA that codes for a specific protein that is involved in the disease process.
These new drugs offer hope for patients with rare genetic disorders that previously had limited treatment options. As more research is conducted in this field, we can expect to see even more oligonucleotide drugs approved in the coming years.
Milasen was developed for a single patient and is an example of the promise of personalized medicine and the impact of oligonucleotide drugs.