Patisiran, sold under the brand name Onpattro, is a

medication A medication (also called medicament, medicine, pharmaceutical drug, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy (pharmacotherapy) is an important part of the medical field and re ...

used for the treatment of

polyneuropathy Polyneuropathy ( poly- + neuro- + -pathy) is damage or disease affecting peripheral nerves (peripheral neuropathy) in roughly the same areas on both sides of the body, featuring weakness, numbness, and burning pain. It usually begins in the hand ...

in people with

hereditary transthyretin-mediated amyloidosis Familial amyloid polyneuropathy, also called transthyretin-related hereditary amyloidosis, transthyretin amyloidosis abbreviated also as ATTR (hereditary form), or Corino de Andrade's disease, is an autosomal dominant neurodegenerative disease. It ...

, a fatal rare disease that is estimated to affect 50,000 people worldwide. It is the first small interfering RNA-based drug approved by the U.S.

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respon ...

(FDA) and the first drug approved by the FDA to treat this condition. It is a gene silencing drug that interferes with the production of an abnormal form of transthyretin. Patisiran utilizes a novel approach to target and reduce production of the TTR protein in the liver via the RNAi pathway. Patisiran was developed and is marketed by

Alnylam Alnylam Pharmaceuticals, Inc. is an American biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics for genetically defined diseases. The company was founded in 2002 and is he ...

. The FDA considers it to be a first-in-class medication.

History

Patisiran was granted

orphan drug status An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment o ...

fast track designation Fast track is a designation by the United States Food and Drug Administration (FDA) of an investigational drug for expedited review to facilitate development of drugs that treat a serious or life-threatening condition and fill an unmet medical ne ...

, priority review and

breakthrough therapy designation Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's "br ...

due to its novel mechanism and the rarity of the condition it treats. It was approved for medical use in the United States and in the European Union in August 2018. The per-patient cost is between and per year, depending on the number of vials needed.

Formulation

The siRNA active component of Patisiran is formulated into

lipid nanoparticles lipid nanoparticles (LNPs), are nanoparticles composed of lipids. They are a novel pharmaceutical drug delivery system (and part of nanoparticle drug delivery), and a novel pharmaceutical formulation. LNPs as a drug delivery vehicle were f ...

, which protect the

RNA Ribonucleic acid (RNA) is a polymeric molecule essential in various biological roles in coding, decoding, regulation and expression of genes. RNA and deoxyribonucleic acid ( DNA) are nucleic acids. Along with lipids, proteins, and carbohydra ...

and facilitate its delivery to target tissues. The lipid nanoparticle formulation includes buffer components, as well as the

lipid Lipids are a broad group of naturally-occurring molecules which includes fats, waxes, sterols, fat-soluble vitamins (such as vitamins A, D, E and K), monoglycerides, diglycerides, phospholipids, and others. The functions of lipids include ...

components DLin-MC3-DMA,

Distearoylphosphatidylcholine Distearoylphosphatidylcholine is a phosphatidylcholine, a kind of phospholipid. It is a natural constituent of cell membranes, eg. soybean phosphatidylcholines are mostly different 18-carbon phosphatidylcholines (including minority of saturated DS ...

cholesterol Cholesterol is any of a class of certain organic molecules called lipids. It is a sterol (or modified steroid), a type of lipid. Cholesterol is biosynthesized by all animal cells and is an essential structural component of animal cell mem ...

, and the PEGylated lipid

DMG-PEG 2000 DMG-PEG 2000 is a synthetic lipid formed by the PEGylation of myristoyl diglyceride. It is used to manufacture lipid nanoparticles that are used in mRNA vaccines, and in particular forms part forms part of the drug delivery system for the Moderna ...

Society and culture

Economics

As of 2020, there were 1050 people globally receiving patisiran, generating $65.5M in net-revenues for Alnylam Pharmaceuticals.

References

External links

* {{Portal bar , Medicine Biopharmaceuticals Orphan drugs RNA interference