New drug for haemophilia A patients with inhibitors

Most of us would stop bleeding quite quickly after a finger prick, as seen in this filepic; however, those with haemophilia A will bleed uncontrollably due to their inability to clot blood.

Haemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding.

Said consultant haematologist Dr Jameela Sathar: “According to the report of the World Federation of Haemophilia’s Annual Global Survey 2017, there are 158,225 persons with haemophilia A worldwide.

“In Malaysia, up to 1,295 persons had haemophilia A in 2017.”

She added: “People with haemophilia A can bleed into their muscles and joints, causing difficulty in using them. They can even bleed spontaneously.

“Without treatment, the quality of life of people with severe haemophilia may be affected.

“They may find it find it difficult to go to school or work regularly, become they become physically disabled and have trouble walking or doing simple activities.”

The main treatment for haemophilia A is factor VIII replacement therapy.

However, nearly one in three people with severe haemophilia A can develop antibodies towards this therapy.

These antibodies, known as inhibitors, neutralise the factor concentrate given to patient, resulting in ineffective treatment.

Complications include higher risk of life-threatening bleeds or repeated bleeding episodes that can cause long-term joint damage or disease, and other complications from bleeding, including death.

Said consultant haematologist Dr Veena Selvaratnam: “Although the prevalence of haemophilia A with inhibitors in Malaysia was 83 persons in 2017, patients face significant challenges in preventing bleeds and typically require infusions of bypassing products multiple times a week, which can be especially difficult for young children and their families.”

However, a new medicine called Hemlibra (emicizumab) was recently introduced in Malaysia by pharmaceutical company Roche Malaysia to prevent and reduce bleeding episodes in people with haemophilia A and factor VIII inhibitors.

This is the first new medicine in more than 20 years to be introduced for the treatment of such patients, whether children or adults.

Hemlibra is administered via a subcutaneous injection once a week, which is more convenient for patients, and their caregivers and families.

The Health Ministry approved Hemlibra based on two of the largest clinical studies in people with haemophilia A and factor VIII inhibitors.

In the Haven 1 study, which had 109 adults and adolescents (aged 12 and above) with haemophilia A and inhibitors, the usage of Hemlibra as prophylaxis (prevention) showed a statistically significant reduction in treated bleeds of 87%, compared to no prophylaxis.

Usage of Hemlibra also resulted in a bleeding rate that was significantly lower by 79%, compared to the bleeding rate with the currently-used bypassing-agent prophylaxis.

Meanwhile, interim results from the Haven 2 study, which has 57 patients aged less than 12 years, 54 out of 57 (94.7%) patients had zero treated bleeds.

The US Food and Drug Administration (FDA) approved Hemlibra in November 2017 and the European Commission in February 2018.

“The approval of Hemlibra in Malaysia represents an important advancement for people with haemophilia A with inhibitors here, who have struggled to manage their bleeding disorder and haven’t had a new medicine in nearly 20 years, ” said Roche Malaysia general manager Lance Duan.

“We believe Hemlibra will improve protection against bleeds and reduce the treatment administration burden for people with haemophilia A with inhibitors, and we are committed to helping them access this medicine.”

Patients can speak to their doctors about Hemlibra, which will be made available through hospitals in Malaysia.

Dr Jameela and Dr Veera were both speaking at the launch of Hemlibra in Malaysia in September 2019.

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