Loading
If this is the first time anyone has visited this substance, we may be fetching live data from TGA Product Information, PBS, and state framework sources — that can take up to a minute. Cached substances load in a second or two.
Eculizumab, sold under the brand name Soliris among others, is a recombinant humanized monoclonal antibody used to treat paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, generalized myasthenia gravis, and neuromyelitis optica. In people with paroxysmal nocturnal hemoglobinuria, it reduces both the destruction of red blood cells and need for blood transfusion, but does not appear to affect the risk of death. Eculizumab was the first medication approved for each of its uses, and its approval was granted based on small trials. It is given by intravenous infusion. It is a humanized monoclonal antibody functioning as a terminal complement inhibitor. It binds to the complement C5 protein and inhibits activation of the complement system, a part of the body's immune system. This binding prevents the breakdown of red blood cells in the bloodstream (intravascular hemolysis) in people with paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.
Read the full article on WikipediaAtypical haemolytic uraemic syndrome (aHUS) Clinical criteria: Treatment Phase: Initial treatment Patient must have active and progressing thrombotic microangiopathy (TMA) caused by aHUS, AND Patient must have ADAMTS-13 activity of greater than or equal to 10% on a blood sample taken prior to plasma exchange or infusion; or, if ADAMTS-13 activity was not collected prior to plasma exchange or infusion, patient must have platelet counts of greater than 30x10^9/L and a serum creatinine of greater than 150 mol/L, AND Patient must have a confirmed negative STEC (Shiga toxin-producing E.Coli) result if the patient has had diarrhoea in the preceding 14 days, AND Patient must have clinical features of active organ damage or impairment, AND Patient must not receive more than 4 weeks of treatment under this restriction. Treatment criteria: Must be treated by a prescriber who is either: (i) a haematologist, (ii) a nephrologist; OR Must be treated by a medical practitioner who has consulted at least one of the above mentioned specialist types, with agreement reached that the patient should be treated with this pharmaceutical benefit on this occasion, AND Patient must be undergoing treatment with one C5 inhibitor therapy only at any given time. Evidence of active and progressing TMA is defined by the following: (1) a platelet count of less than 150x10^9/L; and evidence of two of the following: (i) presence of schistocytes on blood film; (ii) low or absent haptoglobin; (iii) lactate dehydrogenase (LDH) above normal range; OR (2) in recipients of a kidney transplant for end-stage kidney disease due to aHUS, a kidney biopsy confirming TMA; AND (3) evidence of at least one of the following clinical features of active TMA-related organ damage or impairment is defined as below: (a) kidney impairment as demonstrated by one of the following: (i) a decline in estimated Glomerular Filtration Rate (eGFR) of greater than 20% in a patient who has pre-existing kidney impairment; and/or (ii) a serum creatinine (sCr) of greater than the upper limit of normal (ULN) in a patient who has no history of pre-existing kidney impairment; or (iii) a sCr of greater than the age-appropriate ULN in paediatric patients; or (iv) a renal biopsy consistent with aHUS; (b) onset of TMA-related neurological impairment; (c) onset of TMA-related cardiac impairment; (d) onset of TMA-related gastrointestinal impairment; (e) onset of TMA-related pulmonary impairment. Claims of non-renal TMA-related organ damage should be made at the point of application for initial PBS-subsidised eculizumab (where possible), and should be supported by objective clinical measures. The prescriber's cover letter should establish that the observed organ damage is directly linked to active and progressing TMA, particularly when indirect causes such as severe thrombocytopenia, hypertension and acute renal failure are present at the time of the initial organ impairment. Serial haematological results (every 3 months while the patient is receiving treatment) must be provided with every subsequent application for treatment. The authority application must be in writing and must include all of the following: (1) A completed authority prescription form(s); (2) A completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice); (3) A detailed cover letter from the prescriber; (4) A measurement of body weight at the time of application; (5) The result of ADAMTS-13 activity on a blood sample taken prior to plasma exchange or infusion; the date and time that the sample for the ADAMTS-13 assay was collected, and the dates and times of any plasma exchanges or infusions that were undertaken in the 2 weeks prior to collection of the ADAMTS-13 assay; (6) In the case that a sample for ADAMTS-13 assay was not collected prior to plasma exchange or infusion, measurement of ADAMTS-13 activity must be taken 7-10 days following the last plasma exchange or infusion. The ADAMTS-13 result must be submitted to Services Australia within 27 days of commencement of eculizumab treatment in order for the patient to be considered as eligible for further PBS-subsidised eculizumab treatment, under Initial treatment - Balance of Supply; (7) A confirmed negative STEC result if the patient has had diarrhoea in the preceding 14 days; (8) Evidence of active and progressing TMA, including pathology results where relevant. Evidence of the onset of TMA-related neurological, cardiac, gastrointestinal or pulmonary impairment requires a supporting statement with clinical evidence in patient records. All tests must have been performed within 4 weeks of application; (9) For all patients, a recent measurement of eGFR, platelets and two of either LDH, haptoglobin or schistocytes of no more than 1 week old at the time of application.
Paroxysmal nocturnal haemoglobinuria (PNH) Clinical criteria: Treatment Phase: Initial treatment - initial 1 (new patient) induction doses Patient must not have received prior treatment with this drug for this condition, AND Patient must have a diagnosis of PNH established by flow cytometry, AND Patient must have a PNH granulocyte clone size equal to or greater than 10%, AND Patient must have a raised lactate dehydrogenase value at least 1.5 times the upper limit of normal, AND Patient must have experienced a thrombotic/embolic event which required anticoagulant therapy; OR Patient must have been transfused with at least 4 units of red blood cells in the last 12 months; OR Patient must have chronic/recurrent anaemia, where causes other than haemolysis have been excluded, together with multiple haemoglobin measurements not exceeding 70 g/L in the absence of anaemia symptoms; OR Patient must have chronic/recurrent anaemia, where causes other than haemolysis have been excluded, together with multiple haemoglobin measurements not exceeding 100 g/L in addition to having anaemia symptoms; OR Patient must have debilitating shortness of breath/chest pain resulting in limitation of normal activity (New York Heart Association Class III) and/or established diagnosis of pulmonary arterial hypertension, where causes other than PNH have been excluded; OR Patient must have a history of renal insufficiency, demonstrated by an eGFR less than or equal to 60 mL/min/1.73m2, where causes other than PNH have been excluded; OR Patient must have recurrent episodes of severe pain requiring hospitalisation and/or narcotic analgesia, where causes other than PNH have been excluded, AND The treatment must be the sole PBS-subsidised therapy for this condition. Treatment criteria: Must be treated by a haematologist; OR Must be treated by a non-specialist medical physician who has consulted a haematologist on the patient's drug treatment details. The authority application must be made via the Online PBS Authorities System, or in writing via HPOS form upload or mail and must include: (1) details of the proposed prescription(s); and (2) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice). At the time of the authority application, details (result and date of result) of the following monitoring requirements must be provided: (i) Haemoglobin (g/L) (ii) Platelets (x109/L) (iii) White Cell Count (x109/L) (iv) Reticulocytes (x109/L) (v) Neutrophils (x109/L) (vi) Granulocyte clone size (%) (vii) Lactate Dehydrogenase (LDH) (viii) the upper limit of normal (ULN) for LDH as quoted by the reporting laboratory (ix) the LDH:ULN ratio (in figures, rounded to one decimal place) must be at least 1.5
“Eculizumab specifically binds to the terminal complement component 5, or C5, which acts at a late stage in the complement cascade. When activated, C5 is involved in activating host cells, thereby attracting pro-inflammatory immune cells, while also destroying cells by triggering pore formation. By inhibiting the complement cascade at this point, the normal, disease-preventing functions of proximal complement system are largely preserved, while the properties of C5 that promote inflammation and cell destruction are impeded.”
“8 to 15 days (mean 11 days)”
Working under the parallel aged-care framework? Aged-care equivalent →