Science & Pipeline
Trusted Science, Responsibly Delivered
Evidence sets our direction, and responsibility shapes how we put our expertise to work. At Egetis, we concentrate our efforts on advancing innovative therapies for rare disease with significant unmet medical need. with MCT8 deficiency as our immediate priority.
Scientific evidence, not assumption, guides the opportunities we pursue and the decisions we make. We work alongside healthcare professionals, researchers, patients and caregivers to connect emerging science with lived experience and support better-informed care.
Our Scientific Approach
Understanding the underlying biology of rare diseases is the foundation of every development program at Egetis. We focus our efforts on diseases where scientific innovation has the potential to improve outcomes and address significant unmet medical needs.
Our work is driven by rigorous research, collaboration with the scientific and medical community, and a commitment to generating evidence that can support patients, healthcare professionals and decision-makers.
From Research to Patient Access
Developing treatments for rare diseases requires more than scientific discovery alone. It also demands clinical expertise, regulatory excellence and a clear focus on patient access.
At Egetis, we apply an integrated approach that spans the full development pathway, from scientific research and clinical studies to regulatory engagement and commercialization. This helps ensure that scientific advances can be translated into real-world benefits for patients and families.
Our Pipeline
Our pipeline reflects our commitment to addressing serious rare diseases where treatment options remain limited. Through focused development programs, we seek to deliver innovative therapies that have the potential to improve the lives of patients and families affected by rare diseases.
Explore our current development programs and learn more about the science that drives our approach.
Candidate
EMCITATE® (tiratricol) EU
MCT8 deficiency
MAA/NDA
- EC approval received Feb 12, 2025
- Launched in Germany May 1, 2025
EMCITATE® (tiratricol) U.S.
MCT8 deficiency
MAA/NDA
- NDA accepted Mar 27, 2026
- Priority Review granted
- FDA approved Sep 28, 2026
tiratricol
RTH-beta*
Preclinical
- ODD granted by FDA & EMA in 2022
- Finalizing development plan
EC: European Commission; EMA: European Medicines Agency; MAA: Marketing Authorization Application (EU); MCT8: Monocarboxylate Transporter 8; NDA: New Drug Application (USA); ODD: Orphan Drug Designation; RTH-beta: Resistance to Thyroid Hormone beta.
*tiratricol in RTH-beta is an investigational product and has not been approved for use in any market
Why Rare Diseases
Rare diseases collectively affect an estimated 300 million people worldwide, yet most still lack approved treatment options. For patients and families, this often means years of uncertainty, limited treatment choices and significant daily challenges.
This reality is why we care for the rare.
By focusing our expertise and resources on rare diseases, we strive to advance therapies that can make a meaningful difference for patients while contributing to scientific innovation in areas of high unmet medical need.
Research Approach
Learn more about the scientific principles, research strategy and expertise that drive the development of therapies at Egetis.
Explore