Haemophilia injections hailed as ‘real step forward’ for sufferers

Campaigners insist approval of a new, once-weekly injection for Scots suffering from a severe form of haemophilia is a “real step forward”.

Haemophilia Scotland welcomed the decision by the Scottish Medicines Consortium (SMC) to approve the drug marstacimab – also known as Hympavzi – for patients with severe haemophilia B, an inherited bleeding disorder.

With the treatment involving weekly injections, SMC vice chair Dr Robert Peel said it would “bring benefits to patients and their families”.

And Alan Martin, director of Haemophilia Scotland, stated: “This decision is a real step forward for people in Scotland living with severe haemophilia B who have had to rely on frequent intravenous infusions.

“A subcutaneous injection provides more choice so people can work with their healthcare team to pick what treatment plan best fits their needs and lifestyle.”

Over a thousand people in Scotland live with haemophilia, with more than 230 people having haemophilia B – a condition which is usually inherited and predominantly affects men.

Sufferers of the condition lack certain proteins needed for blood clotting, meaning it can take longer than normal for bleeding to stop.

They may also experience spontaneous bleeding in joints and muscles without having had an injury, which can potentially lead to joint damage.

Eleanor Shelley, head of speciality care at the drug company Pfizer UK, said that the SMC decision “expands options for people living with severe haemophilia B in Scotland”

She added: “We are committed to supporting the haemophilia community by working toward solutions that provide choice and address evolving care priorities.”

The SMC also approved new treatments for sickle cell disease, advanced stomach cancer and for advanced bowel cancer, where the disease has undergone a specific genetic mutation.

However, it could not approve the drug serplulimab to be used along with chemotherapy as a first-line treatment for adults with extensive-stage, small cell, lung cancer.

Dr Peel said this was because “the company’s evidence around the cost effectiveness of the treatment, compared to currently available options, was not sufficient”.

He added: “We would welcome a resubmission from the company addressing the issues we have raised.”