FDA Approves Emcitate for Peripheral Thyrotoxicosis in Patients With MCT8 Deficiency – Egetis Therapeutics
Egetis Therapeutics AB announced that the FDA has approved Emcitate (tiratricol), a thyroid hormone receptor agonist, for the treatment of peripheral thyrotoxicosis in adults and pediatric patients with monocarboxylate transporter 8 (MCT8) deficiency (Allan–Herndon–Dudley syndrome).
Emcitate is not recommended for the treatment of primary hypothyroidism. Emcitate is the first FDA-approved treatment option for patients with MCT8 deficiency in the United States.
MCT8 deficiency is a rare, devastating, life-limiting, X-linked disorder caused by pathogenic mutations in the SLC16A2 gene. These mutations impair the function of MCT8, a critical cell-surface thyroid-hormone transporter responsible for thyroid hormone transport in specific cells, including in the brain. As a result, patients experience disrupted thyroid hormone signaling, characterized by insufficient thyroid hormone activity in the central nervous system and excessive exposure to the active thyroid hormone triiodothyronine (T3) in peripheral tissues. This leads to a complex disorder characterized by severe neurodevelopmental impairment and persistent peripheral thyrotoxicosis. Patients with MCT8 deficiency have a reported median life expectancy of approximately 35 years. For further details, please see ‘About MCT8 deficiency’ below.
The FDA approval of Emcitate was supported by a comprehensive clinical development program evaluating Emcitate in patients with MCT8 deficiency, including ReTRIACt, Triac Trial I, Triac Trial II, Erasmus Medical Center (EMC) Cohort Study, EMC Survival Study and the U.S. Expanded Access Program.
“Today marks a turning point for patients living with MCT8 deficiency and their caregivers, who have waited long for an approved treatment in the United States. Our immediate focus is ensuring that eligible patients can access Emcitate as quickly as possible.” – Nicklas Westerholm, Chief Executive Officer of Egetis Therapeutics.
“We are deeply grateful to the patients, caregivers, investigators, clinicians, and advocacy organizations whose partnership and determination made this achievement possible as well as all Egetis employees and collaborators for their dedicated and hard work. Today also marks a defining milestone for Egetis as we take steps forward in developing medicines that address serious unmet medical needs for patients with rare diseases.” – Nicklas Westerholm, Chief Executive Officer of Egetis Therapeutics.
“Through my experience caring for patients with MCT8 deficiency, I have seen firsthand the profound impact this complex and life-limiting disorder can have on patients and their families. Early diagnosis is critical so that patients can be appropriately evaluated, connected with specialists and receive coordinated multidisciplinary care. The FDA approval of Emcitate provides physicians in the United States with the first approved treatment option for patients and represents an important advance for the MCT8 deficiency community.” – Andrew J. Bauer, M.D., a pediatric endocrinologist and expert in thyroid hormone disorders and Principal Investigator in ReTRIACt trial and Triac Trial II, evaluating Emcitate in MCT8 deficiency.
“The FDA approval of Emcitate is a historic milestone for the MCT8 deficiency community and an important step toward ensuring patients have access to a treatment. We are grateful to Egetis and to the researchers, clinicians, patients, families, and advocates whose dedication helped bring this treatment to patients. This approval is a testament to what is possible when a community comes together with a shared commitment to advancing care. We look forward to continuing to advocate for patients and supporting efforts to expand awareness, access and treatment options for the worldwide MCT8 community.” – the MCT8-AHDS Foundation.
