Emcitate

Treatment should be initiated and monitored by physicians who are experienced in the management of patients with rare genetic disorders such as MCT8 deficiency. Posology Dosing of Emcitate should be titrated on an individual patient basis based on the patient’s thyroid hormone levels.

The dose should be increased stepwise approximately every two weeks during a titration period until a maintenance dose has been reached. It is generally recommended to titrate the dose until the serum T3 level is below the midpoint of the normal range for age.

The dose may be further adjusted based on the patient’s response to treatment on clinical features of MCT8 deficiency. 4). TSH and (F)T4 levels may provide further information to guide individual dosing. 3 Adults, adolescents, children, and infants with a body weight of 10 kg or above Dose titration and adjustment The recommended starting dose for patients with a body weight of 10 kg or above is 350 micrograms daily.

A recommended dose titration regimen is shown in Table 1. The daily dose should be gradually increased by 350 micrograms every two weeks until a maintenance dose has been reached. It is generally recommended to titrate the dose until the serum T3 level is below the midpoint of the normal range for age.

Smaller dose escalation steps (half tablets) may be used when a patient is approaching target serum T3 levels, as appropriate. The dose may be further adjusted based on the patient’s response to treatment on clinical features of MCT8 deficiency.

g. morning, midday, evening). Table 1. Recommended dose titration regimen in patients with a body weight of 10 kg or above Titration Total daily dose (micrograms) Number of tablets/day Starting dose 350 1 Week 2 700 2 Week 4 1 050 3 Week 6 1 400 4 Week 8 1 750 5 Week 10 2 100 6 Dose titration should continue in increments of 350 micrograms until a maintenance dose has been reached.

It is not recommended to exceed a daily dose of 80 micrograms/kg in patients with a body weight between 10 and 40 kg; 60 micrograms/kg in patients with a body weight between 40 and 60 kg; and 50 micrograms/kg in patients with a body weight above 60 kg.

Children and infants with a body weight below 10 kg Dose titration and adjustment The recommended starting dose for patients with a body weight below 10 kg is 175 micrograms (a half tablet) daily. A recommended dose titration regimen is shown in Table 2.

Summary of the safety profile The most commonly reported adverse reactions associated with the use of tiratricol treatment were hyperhidrosis (7%), diarrhoea (6%), irritability (2%), anxiety (2%), and nightmares (2%). These reactions usually occurred at the start of treatment and/or when the dose was increased, and generally resolved within a few days.

Tabulated list of of adverse reactions The safety assessment of tiratricol is based on data from clinical trials. Adverse reactions are listed by MedDRA system organ class and frequency convention as follows: very common (≥ 1/10); common (≥ 1/100 to < 1/10); uncommon (≥ 1/1 000 to < 1/100); rare (≥ 1/10 000 to < 1/1 000); very rare (< 1/10 000); not known (cannot be estimated from the available data).

Table 3. Adverse reactions System organ class Adverse reaction Frequency category Psychiatric disorders Irritability Anxiety Nightmares Insomnia Common Common Common Not known 9 Cardiac disorders Tachycardia Not known Gastrointestinal disorders Diarrhoea Common Skin and subcutaneous tissue disorders Hyperhidrosis Common General disorders and administration site conditions Hyperthermia Not known Description of selected adverse reactions Hypermetabolic signs and symptoms In clinical trials in patients with MCT8 deficiency, the onset of the observed adverse reactions hyperhidrosis, irritability, anxiety, and nightmares coincided with treatment initiation or dose modification.

In all cases, these reactions were mild and resolved spontaneously. 4). Paediatric population Safety data were evaluated in 63 patients between 0 and 17 years of age, in Triac Trial I and Triac Trial II combined. Thirty (30) patients were below 2 years of age at start of treatment, 25 patients were between 2 and 11 years of age and 8 patients were between 12 and 17 years of age.

There is no indication from clinical trial data that the safety profile in any subset of the paediatric population is different from the safety profile in adult patients. Reporting of suspected adverse reactions Reporting suspected adverse reactions after authorisation of the medicinal product is important.

8). These signs and symptoms are usually transient and resolve spontaneously within a few days. 2). Following the resolution of hypermetabolic signs and symptoms, dose titration may be resumed, as clinically appropriate. 8). Interference with laboratory tests Tiratricol cross-reacts with T3 if assessed by immunoassay, which may cause unreliable test results.

It is recommended to use an LC/MS/MS method to measure T3 levels. Care should be taken if an immunoassay method is used. 2). 5). 6 Hepatic impairment The safety and efficacy of Emcitate in patients with hepatic impairment have not been studied.

2). Renal impairment The safety and efficacy of Emcitate in patients with renal impairment have not been studied. 2). Misuse for weight reduction Tiratricol should not be taken for weight reduction. 5 under “Orlistat”). Lactose Emcitate tablets contain lactose.

Patients with rare hereditary problems of galactose intolerance, total lactase deficiency or glucose-galactose malabsorption should not take this medicine.

1. g. Grave’s Disease). 6).