Indian & Dutch doctors and civil society collaborate for medicine to children with rare thyroid-linked genetic disorder

Tiratricol is not available in India and its international price would translate to about ₹4 crore, per patient, per year

Later this month, seven children in India with a rare thyroid-related genetic disorder will receive a six-month supply of Tiratricol, a drug to treat their condition - through a collaboration between Dutch and Indian physicians, along with civil society support.

Paediatrician Sivaranjani Santosh told businessline, the six-month supply of the medicine is being imported from Netherlands, through legally-approved channels for “personal use”.  She was also going to approach the Indian government for generic versions of this drug, she said, given that the Tiratricol is not available in India and its international price would translate to about ₹4 crore, per patient, per year.  The imported “compounded” drug costs about ₹2.5 lakh a year, she said, adding that she had raised funds to support the patient-family.  

Genetic Defect

Santosh’s patient Arav is about 20 months, and the medicine is to be taken lifelong. The genetic defect prevents certain thyroid hormones from reaching the brain, affecting the child’s development and metabolism, she explained.

Tiratricol, marketed by Swedish company Egetis Therapeutics, is a thyroid hormone receptor agonist used to treat MCT8 (monocarboxylate transporter 8) deficiency also known as Allan–Herndon–Dudley syndrome.

Wilbert Bannenberg, Chairperson with the Pharmaceutical Accountability Foundation, said the drug was an existing one that was repurposed in the Netherlands and sold to the company. Calling it “medicine hijacking”, he said, it needed to be priced much lower.  

Compounded medicine

Tiratricol costs nearly €180 per tablet in Europe, but in Netherlands it can compounded — prepared by a pharmacist — for a fraction of that price, PAF said. Along with Universities Allied for Essential Medicines Netherlands, PAF was also raising funds for the children’s year-long treatment. Chetali Rao, with India’s Working Group on Access to Medicines and Treatments, clarified that compounding was recognized in India, but not actively pursued.  

Pointing out that Tiratricol was known since 1953, PAF said, it was available in France since 1974, for thyroid conditions. In 2014, researchers at Erasmus University Medical Center, through publicly funded research, found that tiratricol could also be used to treat the rare disease AHDS, it added.

“In 2017, Erasmus MC sold the intellectual property rights related to that research to the Swedish pharmaceutical company Egetis. Unfortunately, the contract did not include a “fair price” clause,” PAF said, adding that Tiratricol costs about €441,000 per year, in Germany.

Request to Marketer

An email was sent to Egetis for comment on developments involving its medicine - a response is awaited.

Egetis had done a clinical trial on the drug in India, PAF alleged, but did not accept a subsequent request from Arav’s doctor for compassionate use. The year-long treatment for the seven children costs €14,200, PAF said. “A total of €5,600 has already been raised in India. PAF aims to raise the remaining €8,600 in the Netherlands through crowdfunding,” it added.

Published on November 4, 2026