Egetis Therapeutics AB announced it has submitted a pre-NDA meeting request to the US Food and Drug Administration to discuss the New Drug Application submission for tiratricol, following the FDA's award of Breakthrough Therapy Designation in July 2025. The designation was granted based on the agency's review of survival data from an international real-world cohort study conducted by Erasmus University Medical Center, demonstrating significant improvement in survival for tiratricol-treated versus untreated patients with MCT8 deficiency.
Breakthrough Therapy Recognition for Rare Disease Treatment
The FDA's Breakthrough Therapy Designation recognizes tiratricol's preliminary evidence of clinical benefit that results in meaningful improvement of key signs and symptoms compared to existing therapies. MCT8 deficiency is a rare and life-threatening genetic disorder affecting fewer than 1,000 individuals globally, with no previously approved treatments available.
"The clinically meaningful evidence available from treating patients with MCT8 deficiency with tiratricol was recognized by the FDA Breakthrough Therapy Designation awarded in July this year," said Nicklas Westerholm, CEO of Egetis. "Receiving a BTD this late in a clinical development program is very encouraging for the forthcoming NDA process, as these designations are typically awarded at an early stage in development."
Clinical Development Progress
The company provided an update on the ReTRIACt study (NCT05579327), which is evaluating withdrawal of tiratricol in patients with MCT8 deficiency. The trial currently has 15 evaluable patients enrolled. Egetis plans to initiate the NDA submission to the FDA during 2025.
Clinical data from the pivotal Triac Trial I demonstrated a 63% reduction in serum T3 (thyroid hormone) levels in patients after 12 months of treatment. This reduction addresses the core pathology of MCT8 deficiency, which causes neurodevelopmental delays and life-threatening thyrotoxicosis.
Regulatory Pathway and Market Position
Tiratricol has received multiple regulatory designations that support its development pathway. The drug received Orphan Drug Designation in 2019, which grants seven years of market exclusivity post-approval. Additionally, it obtained Rare Pediatric Disease Designation in 2020, qualifying Egetis for a Priority Review Voucher valued at over $100 million.
The FDA has established an expanded access program for tiratricol in 13 US hospitals, providing early patient access and building real-world evidence ahead of potential approval. This program supports clinical demand development while the formal approval process proceeds.
European Market Success
Tiratricol treatment for MCT8 deficiency is already included in clinical guidelines by the European Thyroid Association. Emcitate (tiratricol) received approval for MCT8 deficiency treatment in the European Union in February 2025, with the German launch occurring on May 1, 2025. The company has planned additional submissions in Spain and Italy, leveraging the EU's centralized approval system.
Commercial Outlook
With no direct competitors for MCT8 deficiency treatment, tiratricol represents a first-in-class therapy addressing a critical unmet medical need. The rare disease market typically supports premium pricing, with treatments often exceeding $500,000 annually. The combination of Orphan Drug exclusivity and the lack of alternative treatments positions tiratricol for significant commercial potential in the US market.
The pre-NDA meeting with the FDA will help establish the final regulatory pathway and timeline for US approval, potentially leading to market entry by late 2025 or early 2026.
