New Delhi: For 20-month-old Arav, who suffers from Allan-Herndon-Dudley syndrome (AHDS), a rare genetic disorder that affects brain development, access to a medicine that could help manage his condition has been a struggle. His family has been unable to afford the prohibitively expensive drug, tiratricol, which is marketed in Europe at a price that puts it beyond the reach of many patients.
However, a pharmacy in the Netherlands has emerged as a ray of hope for Arav and six other children with the same condition. The pharmacy is preparing the medicine individually for each patient at approximately €1.80 per capsule (Rs 191.16), nearly 99 times cheaper than the price of the commercially marketed drug in Germany. The initiative was announced by the Netherlands-based Pharmaceutical Accountability Foundation (PAF) at a press conference on Wednesday.
The Netherlands-based foundation became aware of the cases after Arav’s mother, Pavitra Sirolkar, began sharing her struggle to access the medicine on social media and reached out to other families facing similar challenges. However, five of the seven children were already known to the doctors involved in their treatment.
AHDS, also known as MCT8 deficiency, is a rare genetic disorder caused by a defect in the MCT8 gene. The defect prevents thyroid hormones from reaching brain cells effectively. These hormones are essential for normal brain development, and inadequate brain levels can cause severe developmental delays and neurological impairment. At the same time, excess thyroid hormones circulating in other parts of the body can cause muscle wasting, weight loss and cardiac complications.
PAF, along with Universities Allied for Essential Medicines Netherlands (UAEM NL), Indian paediatricians and Dutch pharmacists, is working to ensure at least one year of treatment for the seven children. The organisations are also exploring ways to make the medicine more affordable and accessible in India for the long term.
At the German price, a year of treatment can cost about Rs 4 crore, as per PAF. The Dutch-compounded version brings this down to roughly Rs 2.5 lakh a year per child.
The medicine required for the treatment, tiratricol, is marketed by Swedish pharmaceutical company Egetis Therapeutics under the brand name Emcitate. The company charges €178.33 (Rs 18,938.29) per tablet in Germany, according to PAF. The Dutch pharmacy, Transvaal Pharmacy in The Hague, is now preparing the formulation individually for patients based on prescriptions from their Indian doctors.
According to PAF, the intervention follows the struggles of families, including that of Arav’s mother, Pavitra Sirolkar, who had been searching for an affordable supply of the medicine. His paediatrician, Hyderabad-based Dr Sivaranjani Santosh, had also been trying to secure access to tiratricol and sought help through social media and Indian journalists.
Hyderabad-based Sirolkar said she eventually imported a month’s supply from Germany at her own cost.
“Only for one month, I spent Rs 13 lakh. If I had to do it for a year, it would have been a huge burden,” she said. The medicine lasts only 30 to 35 days and has to be taken daily for a lifetime, she told ThePrint.
Dr Praveen at Christian Medical College (CMC) Vellore is caring for five of the seven children. The other two, including Arav, are being treated by Dr Sivaranjani Santosh in Hyderabad.
Egetis had earlier provided tiratricol to Indian patients through a clinical trial but stopped accepting new patients. When one of the parents subsequently approached the company for the medicine under its compassionate-use programme, which allows patients to access drugs free of charge before or outside routine commercial availability, Egetis had rejected the request, PAF said.
The foundation said the Swedish company provides tiratricol free of charge to at least 230 patients in 25 countries, including the Netherlands and the US, through the same programme.
“Egetis is violating ethical principles and its human rights obligations toward Indian patients,” the foundation said.
Hyderabad-based Chinnam Surendra Kumar, father of 19-month-old Chinnam Nandan, one of the seven children, told ThePrint that even finding out about the treatment was difficult.
“I went to several doctors, but nobody gave me any clarity about the disease or even knew about the medicine. Even the endocrinology and genetics departments did not have answers. I had to research the condition myself to understand what it was, where to get the medicine,” he told ThePrint.
He added that the family knew about the medicine but lacked the means and knowledge to import it from Sweden and secure the necessary permissions. “The arrangement through the Netherlands brought a big relief,” he said.
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How tiratricol emerged as a treatment for a rare genetic disorder
Research conducted at Erasmus University Medical Center (Erasmus MC) in the Netherlands found that tiratricol can reduce the physical manifestations of the disease. The medicine does not correct the underlying genetic defect but helps manage the abnormal thyroid hormone levels associated with the condition.
Tiratricol is not a newly discovered drug. It has been known since 1953 and was available in France from 1974 for treating thyroid-related conditions.
The university sold the intellectual property rights related to this research to Egetis in 2017. According to PAF, the agreement did not include a provision requiring the company to maintain an affordable price.
In 2025, Egetis received European marketing authorisation for tiratricol under the brand name Emcitate, giving it 10 years of market exclusivity under European orphan drug legislation — The legislation facilitates the development and authorisation of medicines medicines to treat rare diseases. A disease is considered rare if fewer than 5 in 10,000 people have it.
PAF said the price rose from €0.35 per tablet in France in 2017 to €178.33 in Germany in 2026. Patients typically require lifelong treatment, with doses varying according to body weight and thyroid hormone levels.
The foundation has described this practice as ‘medicine hijacking’.
Egetis, however, disputes the criticism over its pricing and says it has significantly expanded access to tiratricol since acquiring the drug. The company’s director, Nicklas Westerholm, told the Dutch publication that 125 patients worldwide were receiving the medicine, including patients in developing countries where treatment had previously been unavailable. He said Egetis also provided it free of charge in countries without reimbursement arrangements.
“If we had not bought the drug, it would have disappeared from the market. It was only available in France, the production line was old, and there were regular quality issues,” Westerholm said.
He added that Egetis had since upgraded production to meet registration standards and launched an expensive study involving dozens of doctors across seven countries.
On pricing, Westerholm said the company would assess the medicine’s value to patients and healthcare systems, including its impact on survival, quality of life and healthcare costs. “We strive for a reasonable price and we want it to become available to all patients,” he said.
Dutch pharmacy offers interim supply as India explores local access
As an immediate alternative to the commercially marketed drug, PAF worked with Indian paediatricians and Dutch pharmacists to arrange for tiratricol to be compounded individually based on prescriptions from Indian doctors.
Compounding is the process of preparing a medicine by combining or modifying ingredients to meet an individual patient’s specific prescription. It can be used when a commercially available drug does not come in the required dosage or formulation.
In India, compounding by pharmacists is recognised under the Pharmacy Act, 1948. Section 42 stipulates that only registered pharmacists can compound, prepare, mix or dispense medicines against a doctor’s prescription. Rule 65 of the Drugs and Cosmetics Rules, 1945 further requires medicines compounded on licensed pharmacy premises to be prepared under the direct and personal supervision of a registered pharmacist. The rules also prescribe requirements for dispensing and maintaining prescription records.
The families have obtained the necessary import permits, and the medicine is being transported to India with the help of a person assisting the families, according to the foundation.
The estimated cost of one year’s treatment for all seven children is €14,200 (around Rs 15 lakh). Of this, €5,600 has already been raised in India. PAF and UAEM NL are seeking to raise the remaining €8,600 through crowdfunding.
Dr Sivaranjani said most of the families could not have paid for the treatment themselves. “Rs 20,000 per month for lifelong treatment is not feasible. Most of the children are from lower socioeconomic backgrounds,” she told ThePrint.
The foundation is also working with UAEM, Third World Network and Indian paediatricians to explore affordable production and registration of tiratricol in India. This process, however, could take several years.
Dr Sivaranjani said efforts were underway to get an Indian manufacturer on board during the year. “I am confident, not just hoping, that somebody will pick this up and make the drug in India at a reasonable price,” she said.
Kumar said the families’ expectations were modest. “Right now it is around Rs 4 lakh per month, which is an unimaginable situation for a parent. If it is Rs 20,000-25,000, we can even borrow something and manage. I wish we could produce this medicine in India only, like a generic medicine, as early as possible,” he said.
Sirolkar said recognition under the national rare disease policy would also help. “AHDS is not registered under the National Portal for Rare Diseases. If the central health ministry registers this condition there, that itself is an immediate solution, because it is only from the government that we will start getting aid,” she said.
She said the year’s supply had given families breathing room. “Now we have some buffer time to work things out on the government front as well.”
For the seven Indian children, the immediate priority is ensuring uninterrupted access to treatment. The Dutch arrangement offers temporary relief, but affordable and reliable access to the medicine in India remains a longer-term challenge.
(Edited by Anupama Yadav)