New drug review

Emcitate

Every table value is a verbatim quote from the prescribing information cited beneath it; a fact the label does not carry is quoted below its table, with its own source. · 3 sources

Review section

Field	Value
Drug	Emcitate (tiratricol)
Indication reviewed	MCT8 deficiency
Therapeutic category	Thyroid hormone receptor agonists
Manufacturer	Egetis Therapeutics
Regulatory status	Approved September 28, 2026
Data as of	September 29, 2026
Prepared by	To be completed by the reviewing plan
Review status	Draft for pharmacy and therapeutics committee review

Indications

Table 1. FDA-approved indications

Indications	Emcitate (tiratricol)
“treatment of peripheral thyrotoxicosis in adults and pediatric patients with monocarboxylate transporter 8 (MCT8) deficiency (Allan-Herndon-Dudley syndrome)”	✓

Limitation of use

“EMCITATE is not recommended for the treatment of primary hypothyroidism.” (opens the prescribing information at this quote)

Boxed warning and contraindication

“WARNING: NOT FOR TREATMENT OF OBESITY OR FOR WEIGHT LOSS” (opens the prescribing information at this quote)
“Thyroid hormones, including EMCITATE, either alone or with other therapeutic agents, should not be used for the treatment of obesity or for weight loss” (opens the prescribing information at this quote)
“EMCITATE should not be taken for weight reduction as it may cause symptoms of thyrotoxicosis that could be serious or life-threatening.” (opens the prescribing information at this quote)
“EMCITATE is contraindicated in patients with primary hyperthyroidism” (opens the prescribing information at this quote)

Age range of the indication

“The safety and effectiveness of EMCITATE for the treatment of peripheral thyrotoxicosis have been established in pediatric patients with MCT8 deficiency. Use of EMCITATE in pediatric patients is supported by a single-arm trial and a double-blind, placebo-controlled, randomized withdrawal trial that included pediatric patients with MCT8 deficiency ages 10 months to less than 2 years (n=5), ages 2 years to less than 12 years (n=38), and ages 12 years to less than 17 years (n=12)” (opens the prescribing information at this quote)
“MCT8 deficiency is largely a disease of children and young adults; therefore, there are insufficient data on the use of EMCITATE in patients aged 65 years and older to determine whether they respond differently from younger patients.” (opens the prescribing information at this quote)

Pharmacokinetics

Table 2. Pharmacokinetic parameters

Generic Name(s)	Bioavailability	Protein Binding	Metabolism	Excretion	Half-Life
Tiratricol	Not reported	“Tiratricol plasma protein binding is 99%.”	“Tiratricol is metabolized by stepwise deiodination, sulfation and glucuronidation, mainly in the liver.”	“Tiratricol and its metabolites are predominantly eliminated in urine as inorganic iodine and in bile.”	“The steady state half-life is approximately 12 hours for a 70 kg patient and 9.4 hours for a 25 kg patient.”

Absorption, food effect, distribution, and clearance

“The area under the plasma concentration-time curve (AUC) and maximal plasma concentration (Cmax) of tiratricol increased dose proportionally over the dose range studied (175 mcg to 1050 mcg) after oral administration. Administration of EMCITATE via an enteral tube compared to standard oral administration did not affect tiratricol pharmacokinetics.” (opens the prescribing information at this quote)
“The absorption of tiratricol is rapid with a median time to maximum plasma concentration (Tmax) of 0.5 hours (range 0.25 to 1 hour).” (opens the prescribing information at this quote)
“Administration of EMCITATE in healthy volunteers at doses of 175 mcg and 1050 mcg with a high-fat high-calorie meal (956 kcal energy, 35 g protein, 65 g carbohydrate and 61 g fat (with 57% energy from fat)) resulted in a delayed Tmax (1 hour), reduced Cmax (67%-70%), and unchanged AUC, compared to fasted conditions. These changes are not clinically significant” (opens the prescribing information at this quote)
“The apparent volume of distribution is 364 L for a 70 kg patient and 130 L for a 25 kg patient.” (opens the prescribing information at this quote)
“The apparent clearance of tiratricol is 21 L/hr for a 70 kg patient and 10 L/hr for a 25 kg patient.” (opens the prescribing information at this quote)

Specific populations

“No clinical studies have been performed in subjects with renal and hepatic impairment.” (opens the prescribing information at this quote)
“Pediatric patients (5 to 16 years of age) with a lower body weight had a lower clearance and volume of distribution as compared to adult patients.” (opens the prescribing information at this quote)

Exposure-response and QT interval

“Tiratricol reduces elevated T3 concentrations in patients with MCT8 deficiency in a concentration-dependent manner, with greater T3-lowering effect at higher tiratricol exposures; however, this effect plateaus at or above mean tiratricol concentrations of approximately 746 ng/dL in patients 5 years and older. Following discontinuation of EMCITATE, total T3 concentrations increased toward pretreatment levels within one week.” (opens the prescribing information at this quote)
“There is insufficient information to characterize the effect of tiratricol on the QTc interval.” (opens the prescribing information at this quote)

Drug interactions

Table 3. Major drug interactions

Generic Name(s)	Interaction	Mechanism
Tiratricol	Thyromimetic products or products used to treat thyroid conditions (levothyroxine, propylthiouracil, carbimazole)	“Taking EMCITATE in combination with thyromimetic products or products used to treat thyroid conditions (e.g., levothyroxine, propylthiouracil, and carbimazole) may increase the risk of thyrotoxicosis or hypothyroidism and should be avoided.”
Tiratricol	Bile acid sequestrants and ion exchange resins	“Concurrent use may affect the efficacy of EMCITATE by binding and altering absorption.”“Bile acid sequestrants and ion exchange resins decrease thyroid hormone exposure by binding and delaying or preventing absorption.”“Concurrent use may reduce the efficacy of EMCITATE.”“Avoid concomitant use of bile acid sequestrants and ion exchange resins with EMCITATE.”
Tiratricol	Iron and calcium supplements	“Iron and calcium may interfere with the absorption of tiratricol.”“Iron and calcium supplements can reduce tiratricol exposure. If concomitant use of iron or calcium supplements is necessary, administer iron or calcium supplements consistently at the same time each day relative to EMCITATE dosing.”
Tiratricol	Proton pump inhibitors	“Changes in gastric pH associated with PPI therapy may increase the absorption of tiratricol.”“Serum concentrations of T3 should be monitored and dose adjustments of EMCITATE considered when initiating, modifying or discontinuing PPI treatment.”
Tiratricol	Oral estrogen containing products	“Changes in serum plasma protein levels can alter concentrations of thyroid hormones (total T3 levels).”“These drugs may increase serum thyroxine-binding globulin (TBG) concentration and increase total T3 serum levels.”“Monitor clinical symptoms and evaluate free T3 levels.”
Tiratricol	Anabolic steroids and glucocorticoids	“Changes in serum plasma protein levels can alter concentrations of thyroid hormones (total T3 levels).”“These drugs may decrease serum TBG concentration and decrease total T3 serum levels.”“Monitor clinical symptoms and evaluate free T3 levels.”
Tiratricol	Salicylates (more than 2 g/day)	“Changes in serum plasma protein levels can alter concentrations of thyroid hormones (total T3 levels).”“Salicylates inhibit binding of T3 to TBG and transthyretin and may inhibit binding of tiratricol to transthyretin.”“Monitor clinical symptoms and evaluate free T3 levels.”
Tiratricol	Drugs that may increase hepatic metabolism of tiratricol	“Stimulation of hepatic drug-metabolizing enzyme activity may cause increased hepatic degradation of tiratricol and thyroid hormones, resulting in an increased EMCITATE dose requirement.”“Administration of drugs that increase hepatic drug-metabolizing enzyme activity may increase the hepatic degradation of thyroid hormones and tiratricol.”“Serum concentrations of thyroid hormones should be monitored and dose adjustments of EMCITATE considered when initiating, titrating or discontinuing treatment with enzyme inducing agents.”
Tiratricol	Oral anticoagulants	“EMCITATE may increase the response to oral anticoagulant therapy, which may increase the risk of hemorrhage. Therefore, a reduction in the dose of anticoagulant may be warranted if administered concomitantly with EMCITATE. Closely monitor coagulation tests to permit appropriate and timely dosage adjustments of the anticoagulant.”
Tiratricol	Psychostimulants (caffeine, NDRIs, amphetamines)	“Administration of psychostimulants (e.g., caffeine, norepinephrine–dopamine reuptake inhibitors (NDRIs), and amphetamines) in combination with high doses of EMCITATE may lead to increased heart rate and blood pressure. Concomitant use of psychostimulants and EMCITATE is not recommended.”
Tiratricol	T3 immunoassays (drug-laboratory interference)	“An LC-MS/MS method should be used for total T3 measurement due to interference of EMCITATE with T3 immunoassays. An FDA-authorized LC-MS/MS test for the measurement of total T3 is not currently available. Currently available LC-MS/MS tests used to measure total T3 may vary in accuracy”“Tiratricol can cross-react with T3 immunoassays leading to unreliable T3 results. When present, such cross-reactivity causes an overestimation of T3.”
Tiratricol	CYP450 and UGT enzyme substrates and transporter substrates (in vitro)	“Tiratricol is unlikely to inhibit or induce CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6 and CYP3A4 at clinically relevant doses.”“Tiratricol is unlikely to inhibit UGT1A1, UGT1A3, UGT1A4, UGT1A6, UGT1A9, UGT2B7, UGT2B15 and UGT2B17 at clinically relevant doses.”“Tiratricol is not a substrate for P-gp, BCRP, OAT1, OAT3, OCT1, OCT2, OATP1B1, OATP1B3, MATE1 or MATE2-K.”“Tiratricol is unlikely to inhibit BCRP, OAT1, OAT3, OATP1B1 transporters at clinically relevant doses.”

Abbreviations: BCRP=breast cancer resistance protein; CYP=cytochrome P450; LC-MS/MS=liquid chromatography tandem mass spectrometry; MATE=multidrug and toxin extrusion protein; NDRIs=norepinephrine-dopamine reuptake inhibitors; OAT=organic anion transporter; OATP=organic anion transporting polypeptide; OCT=organic cation transporter; P-gp=P-glycoprotein; PPI=proton pump inhibitor; T3=triiodothyronine; TBG=thyroxine-binding globulin; UGT=uridine diphosphate glucuronosyltransferase

Basis of the interaction statements

“Many drugs can exert effects on thyroid hormone pharmacokinetics and may alter the therapeutic response. No clinical drug interaction studies have been conducted with EMCITATE. Tables 2 to 4 provide a summary of expected potential clinically significant drug interactions with EMCITATE.” (opens the prescribing information at this quote)
“No clinical drug-drug interaction studies have been performed with tiratricol.” (opens the prescribing information at this quote)

Adverse drug events

Table 4. Adverse drug events reported in more than 5% of patients in Study 1 and Study 2

Adverse Event	Study 1: Emcitate (N = 20), n (%)	Study 2: Emcitate (N = 46), n (%)
Diarrhea	“0”	“6 (13)”
Vomiting	“2 (10)”	“5 (11)”
Rash	“2 (10)”	“4 (9)”
Hyperhidrosis	“1 (5)”	“3 (7)”

Studies in the safety evaluation

“The safety of EMCITATE in patients with MCT8 deficiency was evaluated in two studies. Study 1 (NCT05579327) was a 3-period, multicenter study with an up to 16-week, open-label, titration period followed by a 30-day randomized double-blind, placebo-controlled withdrawal period, then a 6-week open-label treatment period. Study 1 enrolled 20 male patients with MCT8 deficiency. Study 2 (NCT02060474) was an open-label, single-arm, multicenter study of 12 months duration that enrolled 46 male patients with MCT8 deficiency” (opens the prescribing information at this quote)

Signs and symptoms of thyrotoxicosis

“In Study 2, transient increases in heart rate” (opens the prescribing information at this quote)
“and systolic blood pressure” (opens the prescribing information at this quote)
“were observed in 35% and 26% of participants, respectively, during the titration period. In addition, 30% of participants experienced at least one adverse reaction potentially related to thyrotoxicosis during the trial (diarrhea, hyperhidrosis, irritability, insomnia, nightmares), with most of these adverse reactions occurring during the titration period” (opens the prescribing information at this quote)
“Signs and symptoms of thyrotoxicosis, such as increased heart rate, elevated blood pressure, diarrhea, hyperhidrosis, irritability, insomnia, and nightmares, have occurred in patients receiving EMCITATE” (opens the prescribing information at this quote)
“These symptoms may occur if the EMCITATE dose is further increased despite T3 normalization, due to a drug-induced thyromimetic effect” (opens the prescribing information at this quote)
“Signs and symptoms of thyrotoxicosis may also occur during the dose titration period due to a combined thyromimetic effect from endogenous T3 levels that have not yet declined and EMCITATE.” (opens the prescribing information at this quote)

Lens findings in animals and vision assessment in young children

“For pediatric patients less than 8 years of age, consider periodic vision assessment during treatment with EMCITATE to rule out lens abnormalities (e.g., cataract formation)” (opens the prescribing information at this quote)
“Beagle dogs treated orally for 13 weeks with 0, 0.15, 0.5, or 5 mg/kg/day tiratricol had lens opacities and lens fiber degeneration in all animals treated at the highest dose (approximately 24-fold clinical exposures based on AUC estimates).” (opens the prescribing information at this quote)
“The risk of ocular toxicity from tiratricol use in humans is unknown, but is considered low based on the absence of findings in dogs at clinically relevant exposures.” (opens the prescribing information at this quote)

Pregnancy and lactation

“There are no available data on the use of tiratricol for the treatment of MCT8 deficiency during pregnancy to inform a drug-related risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Animal reproduction studies have not been conducted with tiratricol.” (opens the prescribing information at this quote)
“There are no data regarding the presence of tiratricol in human or animal milk, the effects on the breastfed infant, or the effects on milk production.” (opens the prescribing information at this quote)

Dosing and administration

Table 5. Usual dosing regimens

Generic Name	Usual Adult Dose	Usual Pediatric Dose	Availability
Tiratricol	“EMCITATE dosage is individualized based on weight, total T3 levels, and signs and symptoms of thyrotoxicosis”“Administer the EMCITATE total daily dosage orally or by enteral feeding tube once daily or in two to three divided doses spread throughout the day.”Starting dosage: “10 kg or less: 175 mcg/day”“Greater than 10 kg: 350 mcg/day”Maximum dosage: “Less than 10 kg: 100 mcg/kg/day”“10 kg to less than 40 kg: 80 mcg/kg/day”“40 kg to less than 60 kg: 60 mcg/kg/day”“60 kg and above: 50 mcg/kg/day”	“The safety and effectiveness of EMCITATE for the treatment of peripheral thyrotoxicosis have been established in pediatric patients with MCT8 deficiency.”“EMCITATE dosage is individualized based on weight, total T3 levels, and signs and symptoms of thyrotoxicosis”Starting dosage: “10 kg or less: 175 mcg/day”“Greater than 10 kg: 350 mcg/day”Maximum dosage: “Less than 10 kg: 100 mcg/kg/day”“10 kg to less than 40 kg: 80 mcg/kg/day”“40 kg to less than 60 kg: 60 mcg/kg/day”“60 kg and above: 50 mcg/kg/day”“For pediatric patients less than 8 years of age, consider periodic vision assessment during treatment with EMCITATE to rule out lens abnormalities (e.g., cataract formation)”	“Tablets for oral suspension: 350 mcg. White oblong tablet with functional scoring on both sides.”“NDC 87216-0100-1: PVC/Aluminum blisters, 60 tablets for oral suspension. Each outer carton contains 6 blister cards of 10 tablets each.”

See the current prescribing information for full details.

Dose titration and total T3 measurement

“Measure individual total T3 levels using an LC-MS/MS method. Tiratricol cross-reacts with T3 when assessed by immunoassay, which may cause unreliable test results. An FDA-authorized LC-MS/MS test for the measurement of total T3 is not currently available.” (opens the prescribing information at this quote)
“Based on total T3 levels, increase the dosage approximately every two weeks in the following increments for patients weighing:” (opens the prescribing information at this quote)
“10 kg or less: 175 mcg/day.” (opens the prescribing information at this quote)
“Greater than 10 kg: 350 mcg/day.” (opens the prescribing information at this quote)
“Increase the dosage until a total T3 level below the midpoint of the normal range for age has been achieved (the maintenance dose) or until the maximum recommended dosage based on weight is achieved” (opens the prescribing information at this quote)
“If total T3 is within the normal range, the dosage may be further adjusted up to the maximum recommended dosage based on the patient’s heart rate and blood pressure. Monitor total T3 levels approximately two weeks after these further increases in dosage and assess clinically for new or worsening thyrotoxicosis” (opens the prescribing information at this quote)
“Once on a stable dose of EMCITATE, reassess total T3 levels once every 6 months and modify the dosage as clinically required.” (opens the prescribing information at this quote)

Dosage reduction for new or worsening thyrotoxicosis

“If signs or symptoms of thyrotoxicosis either occur for the first time or worsen, and do not resolve within two weeks, reduce the EMCITATE dosage by 175 mcg for patients with a body weight equal to or less than 10 kg or by 350 mcg for patients with a body weight greater than 10 kg” (opens the prescribing information at this quote)
“Continue to reduce the dosage by these amounts every two weeks, if needed, until the signs or symptoms of thyrotoxicosis have resolved.” (opens the prescribing information at this quote)
“Following the resolution of thyrotoxicosis-related adverse reactions, titrate EMCITATE dosage to achieve target total T3 levels” (opens the prescribing information at this quote)

Missed dose, vomiting, and discontinuation

“If a dose is missed and there are more than 4 hours to the next scheduled dose, the missed dose should be taken as soon as possible. If a dose is missed and the next dose is scheduled within 4 hours, the dose should be skipped and the next dose taken according to the regular schedule. Doses should not be doubled.” (opens the prescribing information at this quote)
“If vomiting occurs within 30 minutes after administration, administer a new dose. Instruct patients or caregivers to contact their healthcare provider if vomiting occurs a second time.” (opens the prescribing information at this quote)
“Do not abruptly stop EMCITATE treatment in order to avoid occurrence or worsening of symptoms of thyrotoxicosis” (opens the prescribing information at this quote)
“For patients weighing 10 kg or less, reduce EMCITATE by 175 mcg/day once every two weeks.” (opens the prescribing information at this quote)
“For patients weighing greater than 10 kg, reduce EMCITATE by 350 mcg/day once every two weeks.” (opens the prescribing information at this quote)

Preparation and administration

“EMCITATE tablets for oral suspension must be dispersed in room temperature drinking water directly in a 10-12 mL syringe and given as a suspension only. Do not prepare with other liquids, food vehicles or enteral nutrition formulas.” (opens the prescribing information at this quote)
“Do not swallow, chew, or crush the tablets.” (opens the prescribing information at this quote)
“If more than 3 tablets are needed for a single dose, divide the dose into multiple, separate preparations not to exceed 3 tablets per preparation.” (opens the prescribing information at this quote)
“Discard the suspension 30 minutes after preparation if not used.” (opens the prescribing information at this quote)
“The prepared EMCITATE suspension may be administered with or without food” (opens the prescribing information at this quote)
“Prepared EMCITATE suspension can be administered through an enteral feeding tube directly into the stomach. Either nasogastric (NG) tubes (6 French and above) or gastrostomy (G) tubes (12 French and above) can be used.” (opens the prescribing information at this quote)

Storage and split tablets

“Store refrigerated at 2°C to 8°C (36°F to 46°F), and keep protected from light.” (opens the prescribing information at this quote)
“EMCITATE tablets for oral suspension have a functional score line and can be split into equal halves. If dosing requires half a tablet, store the unused half in the original blister pack and outer carton (or in the light-protective packaging from the pharmacy) to protect from light and refrigerate at 2°C to 8°C (36°F to 46°F).” (opens the prescribing information at this quote)

Effectiveness

Table 6. Comparative clinical trials

Study and Drug Regimen	Study Design and Demographics	Study Size and Duration	End Points	Results
Study 1 (NCT05579327): “10 participants in Cohort A, who were already receiving EMCITATE treatment, and 10 participants in Cohort B, who were started on EMCITATE 350 mcg daily. The EMCITATE dose was titrated by 350 mcg daily every 2 weeks based on serum total T3 to achieve the stable dose criterion.”“randomized (1:1) to receive placebo or continue EMCITATE treatment for 30 days or until the rescue criterion, defined as the serum total T3 above the upper limit of normal (ULN), was met.”	“an international, multicenter study (Study 1; NCT05579327) that consisted of three study periods as follows:”“Screening Period (Run-in/Titration period): 6-week run-in period for participants already treated with EMCITATE (Cohort A) and a 16-week dose-titration period for participants newly started on EMCITATE (Cohort B).”“Randomized Treatment Period (RTP): 30-day double-blind, placebo-controlled, randomized withdrawal period, which provided data for the primary efficacy endpoints.”“Follow-up Period: 6-week open-label treatment period.”“The stable dose criterion was defined as at least 4 weeks of treatment with a fixed daily dose of EMCITATE and at least 2 consecutive serum total T3 results within the target range (between 80% of the lower limit of normal and no higher than the 75th percentile of the normal range).”“The median age of the 20 participants enrolled in the study was 11 years, ranging from 5 to 31 years. All subjects were male. There were 65% White, 15% Black or African Americans, 5% Asian, 5% American Indian or Alaska Native and 10% other races.”	“Twenty participants with MCT8 deficiency were enrolled during the Screening Period”“Fifteen participants (8 participants from Cohort A and 7 participants from Cohort B) achieved the stable dose criterion and were randomized”“After completion of the RTP, 14 participants entered a 6-week follow-up period”“The mean duration of treatment with EMCITATE during this period was 86 days in Cohort A and 77 days in Cohort B.”	“The two primary efficacy endpoints were: 1) Rate of change from baseline in serum total T3 during the 30-day Randomized Treatment Period; 2) Proportion of participants who met the rescue criterion during the 30-day Randomized Treatment Period.”“Secondary efficacy endpoints assessing mean changes in heart rate and systolic blood pressure from screening until pre-randomization and from screening until end of study”	T3 rate of change [95% CI], placebo (N = 8): “1.6 [1.2; 2.1]”T3 rate of change [95% CI], Emcitate (N = 7): “1.1 [0.8; 1.3]”Ratio of T3 rate of change, placebo/tiratricol [95% CI]: “1.5 [1.0; 2.2] (p-value=0.034)”“Note: Estimates from random effects model”“The mean (SD) change from baseline to the end of the Randomized Treatment Period in total T3 levels was 64.6 (12.7) ng/dL from a baseline of 109.1 ng/dL in the placebo arm and -0.2 (12.5) ng/dL from a baseline of 100.5 ng/dL in the tiratricol arm. The treatment difference (95% CI) in total T3 change from baseline at the end of randomized treatment period was 64.8 ng/dL (44.7, 84.8) for placebo versus tiratricol arm.”Rescue criterion met, Emcitate (N = 7), n (%): “1* (14.3)”Rescue criterion met, placebo (N = 8), n (%): “4 (50.0)”Difference in proportions (placebo-tiratricol): “0.36”95% CI: “-0.16, 0.77”“One participant who discontinued prematurely during the Randomized Treatment Period was considered to have met the rescue criterion.”“showed a trend towards numerical improvements in participants in Cohort B (treatment-naïve), while heart rate and blood pressure remained stable in participants in Cohort A (previously-treated).”
Study 2 (NCT02060474): “The starting dose of EMCITATE was 350 mcg daily in participants with a body weight of”“175 mcg daily in participants with a body weight of < 10 kg.”“The EMCITATE dose was titrated in 350 mcg daily (175 mcg daily for participants < 10 kg) increments every 2 weeks to achieve serum total T3 levels within the normal range for adults of 91 ng/dL to 163 ng/dL.”“The mean maintenance dose of EMCITATE was 741 mcg.”	“a multicenter, single-arm, open-label study”“The median age of participants was 7.1 years (range: 10 months to 66.8 years). The majority (96%) of participants were White, 2% were Asian and 2% were other races.”	“enrolled 46 male participants with MCT8 deficiency”“12 months duration”	“The primary efficacy endpoint was the change from baseline to end of study in mean serum total T3 concentration.”“Secondary efficacy endpoints evaluated the effect of EMCITATE on signs that could reflect thyrotoxicosis, such as systolic blood pressure, heart rate and body weight.”	Serum total T3, ng/dL (N = 45), baseline mean (SD): “323.4 (100.8)”Month 12 mean (SD): “118.3 (44.6)”Difference, mean [95% CI]: “-205.2 [-235.7; -174.6]”“Data were imputed as Last Observation Carried Forward for patients who discontinued treatment prior to Month 12 visit.”“At Month 12, there was a mean (95% confidence interval) change of: -4.1 (-8.1, -0.1) mmHg from a baseline of 107.1 mmHg in systolic blood pressure (N = 35 participants); -8.9 (-15.6, -2.3) beats per minute (bpm) from a baseline of 112.4 bpm in heart rate (N = 34 participants), 0.22 (-0.01, 0.45) from a baseline of -2.85 in body weight-for-age Z-score (N = 45 participants) and 0.51 (0.25, 0.76) from a baseline of 0.46 in body weight-for-age Z-score in an untreated MCT8 patient population (N = 36 participants).”

Abbreviations: CI=confidence interval; RTP=randomized treatment period; SD=standard deviation; T3=triiodothyronine; ULN=upper limit of normal

Study 2 as published (Triac Trial I): registration and co-primary endpoints, which the prescribing information does not report

“This trial is registered with ClinicalTrials.gov, number NCT02060474.” (opens the source at this quote in a new tab)
“The prespecified primary endpoint was the change in serum T3 concentrations from baseline to month 12. The co-primary endpoints were changes in concentrations of serum thyroid-stimulating hormone (TSH), free and total thyroxine (T4), and total reverse T3 from baseline to month 12.” (opens the source at this quote in a new tab)
“Serum T3 concentration decreased from 4·97 nmol/L (SD 1·55) at baseline to 1·82 nmol/L (0·69) at month 12 (mean decrease 3·15 nmol/L, 95% CI 2·68-3·62; p<0·0001), while serum TSH concentrations decreased from 2·91 mU/L (SD 1·68) to 1·02 mU/L (1·14; mean decrease 1·89 mU/L, 1·39-2·39; p<0·0001) and serum free T4 concentrations decreased from 9·5 pmol/L (SD 2·5) to 3·4 (1·6; mean decrease 6·1 pmol/L (5·4-6·8; p<0·0001).” (opens the source at this quote in a new tab)

Neurodevelopmental outcome, which the prescribing information does not report

“Emcitate did not improve neurodevelopmental delay.” (opens the source at this quote in a new tab)