Could a Custom-Made Drug from the Netherlands Bring New Hope to 7 Children in India with a Rare Genetic Disorder? In a collaborative effort, Dutch and Indian doctors, pharmacists and civil society organisations have joined forces to arrange an affordable supply of tiratricol for seven children in India suffering from Allan-Herndon-Dudley Syndrome (AHDS), also known as MCT8 deficiency.
Custom-Made Drug Offers an Affordable Treatment Option
The medicine is being prepared by Transvaal Pharmacy in The Hague, Netherlands, through a process called pharmaceutical compounding, in which pharmacists prepare medicines in small quantities based on individual prescriptions.
The compounded tiratricol will cost approximately €1.80 per capsule, making it nearly 99 times cheaper than the commercially marketed version sold in Germany for around €180 per tablet. Currently, one Indian family importing the medicine from Germany faces expenses of approximately ₹3 lakh per month, making lifelong treatment financially difficult.
Understanding Allan-Herndon-Dudley Syndrome
Allan-Herndon-Dudley Syndrome is a rare genetic disorder caused by a defect in the MCT8 gene, which affects the transport of thyroid hormones into brain cells. As a result, the developing brain does not receive adequate thyroid hormone, potentially leading to severe developmental and neurological problems.
Research conducted by Erasmus University Medical Centre (Erasmus MC) established that tiratricol, a thyroid hormone analogue, could help reduce certain physical manifestations of the condition.
Although tiratricol has been known since 1953 and has been available in France for treating thyroid-related conditions since 1974, researchers at Erasmus MC identified its potential application in AHDS in 2014 through research supported by approximately €14 million in public funding.
High Drug Prices Create Access Challenges
Despite its potential benefits, access to tiratricol has remained a major challenge because of its high commercial price. In 2025, Swedish pharmaceutical company Egetis received European marketing authorisation for the medicine, granting it 10 years of market exclusivity under European orphan drug legislation.
Consequently, the price increased substantially, reaching nearly €180 per tablet in Germany in 2026, compared with €0.35 in France in 2017.
To address this affordability challenge, the Netherlands-based Pharmaceutical Accountability Foundation (PAF) is coordinating efforts with Indian paediatricians and access-to-medicine organisations to arrange the compounded medicine for the seven children.
Efforts Underway for Affordable Drug Availability in India
While the custom-compounded medicine provides an immediate solution, it is not intended to be a permanent arrangement. Therefore, PAF is working with access-to-medicine activists and Indian paediatricians to explore affordable production and registration of tiratricol in India.
This initiative could eventually pave the way for generic production, making the medicine more accessible to children who need lifelong treatment. However, establishing local production and obtaining the necessary regulatory approvals could take several years.
As reported by The Times of India, the collaboration between Dutch and Indian healthcare professionals and civil society organisations aims to bridge the immediate treatment gap for seven children while exploring sustainable, affordable access to tiratricol in India.