Chemistry:Romiplostim
_250mkg.jpg) | |
| Clinical data | |
|---|---|
| Trade names | Nplate, Romiplate |
| Other names | AMG531 |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a609008 |
| License data | |
| Pregnancy category |
|
| Routes of administration | Subcutaneous |
| ATC code | |
| Legal status | |
| Legal status | |
| Pharmacokinetic data | |
| Elimination half-life | 1 to 34 days |
| Identifiers | |
| |
| CAS Number | |
| IUPHAR/BPS | |
| DrugBank | |
| ChemSpider |
|
| UNII | |
| KEGG | |
| ChEMBL | |
| Chemical and physical data | |
| Formula | C2634H4086N722O790S18 |
| Molar mass | 59085.01 g·mol−1 |
  (what is this?) (verify) | |
Romiplostim, sold under the brand name Nplate among others, is a fusion protein analog of thrombopoietin, a hormone that regulates platelet production.
The drug was developed by Amgen through a restricted usage program called NEXUS.[1] During development and clinical trials the drug was called AMG531.[2]
Medical uses
Romiplostim is indicated as a potential treatment for chronic idiopathic (immune) thrombocytopenic purpura (ITP).[3] Romiplostim was designated an orphan drug by the U.S. Food and Drug Administration (FDA) in 2003, as the chronic ITP population in the USA is under 200,000 (the chronic adult ITP population in the USA is thought to be around 60,000, with women outnumbering men by a factor of two).[4] The wholesale cost of romiplostim if administered weekly is currently estimated at US$55,250 per year.[5]
On August 22, 2008, the FDA approved romiplostim as a long-term treatment for chronic ITP in adults who have not responded to other treatments, such as corticosteroids, intravenous immunoglobulin, Rho(D) immune globulin or splenectomy.[1][6]
Clinical efficacy
In well designed, 24-week, Phase III trials, romiplostim was significantly more effective than placebo in achieving the primary endpoint of a protocol-defined durable platelet response in nonsplenectomized or splenectomized adults with chronic immune thrombocytopenic purpura.[7]
Side-effects
Romiplostim's effect is to stimulate the patient's megakaryocytes to produce platelets at a more rapid than normal rate, thus overwhelming the immune system's ability to destroy them. As doing so involves changes to the bone marrow chemistry, a number of potentially serious side-effects may develop, including death, myalgia, joint and extremity discomfort, insomnia, thrombocytosis, which may lead to potentially fatal clots, and bone marrow fibrosis, the latter of which may result in an unsafe decrease in the red blood count.[citation needed]
Experimental treatment of acute radiation syndrome
Romiplostim may be used to treat acute radiation syndrome.[8] "To reduce radiation-induced bleeding, Nplate stimulates the body’s production of platelets. The drug can be used to treat adults and children."[8]
References
- ↑ 1.0 1.1 "FDA Approvals: Nplate, Aloxi, Vidaza". Medscape. September 4, 2008. http://www.medscape.com/viewarticle/580032. Freely available with registration.
- ↑ "AMG 531, a thrombopoiesis-stimulating protein, for chronic ITP". The New England Journal of Medicine 355 (16): 1672–1681. October 2006. doi:10.1056/NEJMoa054626. PMID 17050891.
- ↑ "Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial". Lancet 371 (9610): 395–403. February 2008. doi:10.1016/S0140-6736(08)60203-2. PMID 18242413.
- ↑ "Amgen to Discuss Romiplostim BLA". drugs.com. March 12, 2008. https://www.drugs.com/nda/romiplostim_080312.html.
- ↑ "Romiplostim: a novel thrombopoiesis-stimulating agent". American Journal of Health-System Pharmacy 66 (9): 817–824. May 2009. doi:10.2146/ajhp080524. PMID 19386944.
- ↑ "FDA Approves Nplate(TM) for Long-Term Treatment of Adult Chronic ITP" (Press release). Amgen. August 22, 2008. Archived from the original on 15 September 2008. Retrieved 2008-09-04.
- ↑ "Romiplostim". Drugs 69 (3): 307–317. 2009. doi:10.2165/00003495-200969030-00006. PMID 19275274.
- ↑ 8.0 8.1 "US splashes $290m on anti-radiation drugs after Putin ups nuclear threats". The Daily Telegraph. 5 Oct 2022. https://www.telegraph.co.uk/world-news/2022/10/05/us-denies-purchase-radiation-injury-drugs-response-russian-nuclear/.
 |  |
