FDA approved Tepezza (teprotumumab) for Thyroid Eye Disease Regardless of Activity or DurationFDA approved New Drug for Thyroid Eye Disease, Including Longstanding Cases

Teprotumumab is an insulin-like growth factor-1 receptor inhibitor.

TEPEZZA is indicated for the treatment of Thyroid Eye Disease regardless of Thyroid Eye Disease activity or duration. In the clinical studies, patients had a clinical diagnosis of Thyroid Eye Disease with symptoms and were euthyroid or had thyroxine and free triiodothyronine levels less than 50% above or below normal limits. Prior surgical treatment for Thyroid Eye Disease was not permitted. Proptosis ranged from 16 to 33 mm, and 125 patients (73%) had diplopia at baseline. The median age was 52 years (range 20 to 79 years); 86% were White, 9% were Black or African-American, 4% were Asian, and 1% identified as Other. The majority (73%) were female, and 27% were smokers at baseline.

The recommended dose is an intravenous infusion of 10 mg/kg for the initial dose, followed by 20 mg/kg every three weeks for 7 additional infusions. Administer the diluted solution intravenously over at least 90 minutes for the first two infusions. If well tolerated, the minimum time for subsequent infusions can be reduced to 60 minutes. If not well tolerated, the minimum time for subsequent infusions should remain at 90 minutes, and pre-medication is recommended for subsequent infusions. Do not administer as an intravenous push or bolus. TEPEZZA should not be infused concomitantly with other agents. Reconstitution: each vial contains 500 mg of teprotumumab; reconstitute with 10 mL Sterile Water for Injection, USP, to yield a total volume of 10.5 mL (47.6 mg/mL). Dilute in 0.9% Sodium Chloride Injection, USP: use 100 mL bags for doses less than 1800 mg and 250 mL bags for doses equal to or greater than 1800 mg. The combined storage time of reconstituted and diluted solution is up to 4 hours at room temperature (20°C to 25°C) or up to 48 hours refrigerated (2°C to 8°C), protected from light. Do not freeze. No incompatibilities observed with polyethylene, polyvinyl chloride, polyurethane, or polyolefin bags and intravenous administration sets.

TEPEZZA was evaluated in 2 randomized, double-masked, placebo-controlled studies in 171 patients with Thyroid Eye Disease: Study 1 (NCT01868997) and Study 2 (NCT03298867). Patients were randomized 1:1 to TEPEZZA or placebo. The proptosis responder rate at week 24 was defined as the percentage of patients with at least 2 mm reduction in proptosis in the study eye from baseline, without deterioration in the non-study eye (at least 2 mm increase). In Study 1, the proptosis responder rate was assessed in 42 TEPEZZA and 45 placebo patients; in Study 2, 41 TEPEZZA and 42 placebo patients. The difference and its 95% confidence interval were based on a weighted average of the difference within each randomization stratum (tobacco user, [text truncated in label]). Additional evaluations included signs and symptoms of Thyroid Eye Disease such as pain, gaze evoked orbital pain, swelling, eyelid erythema, redness, chemosis, inflammation, clinical activity score, and assessments of functional vision and patient appearance. Specific responder rates and confidence intervals are not fully reported in the provided label text.

Not reported in label.

TEPEZZA provides a targeted systemic therapy for Thyroid Eye Disease regardless of disease activity or duration, as stated in the indication. It is administered as a series of eight intravenous infusions over approximately