Rare bleeding disorder patients in India to get 1st preventive treatment

New Delhi: A US FDA-approved treatment that may prevent haemophilia, a rare bleeding disorder, has been launched in India for the first time, global biopharmaceutical company Takeda Pharmaceutical said on Monday.

A genetic condition that prevents blood from clotting, haemophilia causes bleeding for a long time after injury or surgery and painful swelling of the joints either after injury or even without injury.

In India, more than 1,36,000 people have been diagnosed with the disorder that causes lifelong bleeding, and has so far no known cure.

The prophylaxis treatment is named Adynovate and is an innovative extended half-life recombinant Factor VIII (rFVIII) treatment, using established technology (controlled PEGylation), for haemophilia A patients.

Adynovate, in combination with MYPKFIT, is an FDA approved application that offers personalised and interactive prophylaxis treatment option that enables both healthcare professionals (HCPs) and patients in real-time monitoring, the company said in a statement.

Alerts are sent to patients on prophylaxis when their estimated factor VIII levels are low, and reminds them when their infusions are due, thereby providing excellent prophylactic coverage.

The drug is available in India post all necessary clinical trials, the company said, adding it has also taken requisite approvals including from Indian regulatory bodies such as the DCGI, and the CDSCO.

"Individuals with severe haemophilia have recurrent hemarthrosis - fragmentation of joint cartilage, bone destruction, and crippling, which can be effectively reduced through prophylaxis as opposed to on-demand therapy," Dr. Sandeep Arora, Head of Medical Affairs and Patient Services, Takeda India, in a statement.

Adynovate has demonstrated "favourable safety and efficacy results that offer effective bleed resolution, better joint health, and almost zero spontaneous bleeds in majority", he added.