Japan-based Takeda, a global biopharmaceutical company that specialises in drugs for rare diseases, announced on Monday that it is introducing in India three therapies — Velaglucerase Alfa for treatment of Gaucher, Agalsidase Alfa for Fabry, and Idursulfase for Hunter Syndrome.

It has taken 9-13 years for these crucial drugs to reach the Indian market since their launch in other global markets.

But the launch means little, if the government, which is re-drafting its Rare Disease Policy, does not include these costly drugs in their access programmes, said experts. “As of now, the government is not providing access to patients. The launch means that the drugs will now be available in the Indian market, while earlier, patients could import them individually,” said Prasanna Shirol, co-founder and director, Organization for Rare Diseases, India.

Access to life-saving drugs

While the government earlier said that a ₹100-crore fund had been created for patients with rare diseases, it recently backtracked before a court saying it had not been put in place. “Patients with rare diseases should be able to access life-saving drugs and a separate fund should be set aside for this. One cannot argue that because many more children are dying from malnutrition, those with rare diseases cannot be provided costly drugs.” said Ari Zimran, Director of Gaucher Clinic at Shaare Zedek Medical Centre in Jerusalem.

Fabry disease interferes with the body’s ability to break down a specific fatty substance called ‘Gb3’ and affects the heart and the kidneys. Nearly 10,000 people worldwide have been affected by Fabry. “In India, nearly 100 Fabry patients have been identified, but estimated occurrence is one case in 20,000-30,000. There may be many undiagnosed cases in India,” said Atul Mehta, Consultant Haematologist and Physician at the Royal Free Hospital in London.

While Takeda launched Agalsidase Alfa to tackle Fabry in 2001 under the brand name Replagal, Sanofi and Genzyme launched Agalsidase Beta under the brand name Fabrazyme. “Both drugs have been available in the UK since 2001, and their results in patients are equivalent,” said Mehta. They cost around £1,00,000 or ₹85 lakh per patient per year.

Improved platelet count

In the case of Gaucher, a rare inherited metabolic condition which affects approximately one in a lakh persons, Zimran observed that Takeda’s Velaglucerase Alfa under the brand name VPRIV has shown improved results in at least 40 per cent cases compared to Cerezyme supplied by Sanofi and Genzyme. “Certain patients switched to VPRIV from Cerezyme recorded better haemoglobin and platelet counts, and so VPRIV derived from human enzymes had a booster effect and was also safe to use as possibility of adverse reactions was lower,” said Zimran.

“India has reported close to 500 Gaucher patients, but these numbers are grossly under-reported as children with improper care succumb early and deaths are not recorded. The cost of treatment for Gaucher is close to ₹30 lakh per child per year,” said Shirol.

Takeda has a compassionate drug-use programme in place for Indian patients, a company official said. “Doctors forward applications to procure drugs under this programme to a global committee which evaluates the need of the patient. If the committee approves the use, we provide it,” said the official.

Hunter Syndrome affects muscles, bones, heart and breathing. In 2006, the US Food and Drug Administration granted Takeda (erstwhile Shire Pharmaceuticals) approval to market Idursulfase. “Since then, over 95 per cent of Hunter patients have benefited from this drug globally. The use of Idursulfase has shown an increased life expectancy of 11.8 years with 54 per cent reduction in mortality for patients,” said a company spokesperson.

“There are close to 200 reported cases of Hunter Syndrome in India and treatment costs around ₹40 lakh annually,” said Shirol.

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