Ultomiris

Ultomiris Uses, Dosage, Side Effects, Food Interaction and all others data.

Ultomiris is considered a long-acting complement 5 (C5) inhibitor that has been undergoing clinical trials for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) as of 4 February, 2016. A drug similar to ravulizumab (ALXN1210), called eculizumab, is currently approved for the treatment of PNH in 46 countries under the brand name Soliris®. Ultomiris is considered by Alexion Pharmaceuticals Inc. to be a "next-generation" eculizumab molecule. Ultomiris was subsequently approved by the US FDA in December of 2018 for a variety of beneficial characteristics that make it an advanced, next-generation agent in comparison to eculizumab . In particular, ravulizumab is currently the first and only long-acting C5 complement inhibitor that can be administered every eight weeks for the treatment of adult patients with PNH whereas eculizumab is a bi-weekly treatment . Moreover, virtually all of the phase 3 trial results for ravulizumab have demonstrated the equivalent efficacy and safety established by eculizumab and that patients transition safely and effectively from using eculizumab to ravulizumab . Subsequently, whereas PNH patients may have needed to previously plan their lives rather strictly around the bi-weekly infusion administrations of eculizumab, with ravulizumab such patients can find a more relaxed dosing schedule of only six or seven infusions over an entire year .

Just as the US FDA permitted a timely and expedited approval of ravulizumab ahead of the Prescription Drug User Fee Act (PDUFA) date of February 18, 2019 following the use of a rare disease priority review voucher by Ultomiris (ravulizumab) developer Alexion for many of the aforementioned beneficial treatment reasons, regulatory authorities in the European Union (EU) and Japan have currently accepted and are reviewing applications for the approval of Ultomiris (ravulizumab) as a treatment for adults with PNH as well .

Immediate and complete inhibition of serum-free complement protein C5 (concentration of less than 0.5 mcg/mL) was observed by the end of the first ravulizumab infusion and sustained throughout the entire 26-week treatment period in all patients, both complement-inhibitor naïve and previously treated with eculizumab .

Ultomiris
Uses
Dosage
Side Effect
Precautions
Interactions
Uses during Pregnancy
Uses during Breastfeeding
Accute Overdose
Food Interaction
Half Life
Volume of Distribution
Clearance
Interaction With other Medicine
Contradiction
Storage

Trade Name	Ultomiris
Availability	Prescription only
Generic	Ravulizumab
Ravulizumab Other Names	Ravulizumab, ravulizumab-cwvz
Related Drugs	Ultomiris, Empaveli, pyridostigmine, neostigmine, Mestinon, Soliris, Vyvgart, eculizumab, pegcetacoplan
Weight	10mg/ml, 100mg/ml,
Type	Infusion, Intravenous Solution
Protein binding	Readily accessible data regarding the protein binding of ravulizumab is not available.
Groups	Approved, Investigational
Therapeutic Class
Manufacturer	Alexion Pharma UK Ltd
Available Country	United Kingdom, United States,
Last Updated:	September 19, 2023 at 7:00 am

Uses

Ultomiris is a monoclonal antibody used to treat paroxysmal nocturnal hemoglobinuria.

Ultomiris is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) .

Ultomiris is also used to associated treatment for these conditions: Paroxysmal Nocturnal Haemoglobinuria (PNH)

How Ultomiris works

Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive, debilitating and life-threatening ultra-rare blood disorder characterized by hemolysis (destruction of red blood cells) that is mediated by the uncontrolled activation of the complement system, a component of the body’s immune system .

Ultomiris is subsequently a terminal complement inhibitor that specifically binds to the particular complement protein C5 with high affinity, thereby inhibiting its cleavage to C5a (the proinflammatory anaphylatoxin) and C5b (the initiating subunit of the terminal complement complex [C5b-9]) and preventing the generation of the terminal complement complex C5b9 . Ultomiris inhibits terminal complement-mediated intravascular hemolysis in patients with PNH .

Toxicity

Although PNH in pregnancy is associated with adverse maternal outcomes, including worsening cytopenias, thrombotic events, infections, bleeding, miscarriages, increased maternal mortality, and adverse fetal outcomes like fetal death and premature delivery, there are no available data on ravulizumab use in pregnant women to inform a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes .

Although there are currently no human reproductive studies, human immunoglobulins like ravulizumab are known to cross the human placental barrier, and thus may potentially cause terminal complement inhibition in the fetal circulation .

There are no data on the presence of ravulizumab in human milk, the effect on the breastfed child, or the effect on milk production . Since many medicinal products and immunoglobulins are secreted into human milk, and because of the potential for serious adverse reactions in a nursing child, breastfeeding should be discontinued during treatment and for 8 months after the final dose .

The safety and efficacy of ravulizumab in pediatric patients and geriatric use have not yet been established .

Genotoxicity studies have not been conducted with ravulizumab .

Effects of ravulizumab upon fertility have not been studied in animals . Intravenous injections of male and female mice with a murine anti-C5 antibody at up to 0.8-2.2 times the equivalent of the clinical dose of ravulizumab had no adverse effects on mating or fertility .

Food Interaction

No interactions found.

Ultomiris Disease Interaction

Major: meningitisModerate: infections, severe renal impairment

Volume of Distribution

The mean (SD) volume of distribution at steady state was 5.34 (0.92) L .

Elimination Route

It has been demonstrated that mean ravulizumab Cmax and AUC∞ increase in a dose-proportional manner and that a single 400 mg intravenous dose of ravulizumab administered to subjects reach or exceed the threshold level of 100 µg/mL .