Novo Nordisk has received a positive opinion from the EMA's CHMP on its subcutaneous prophylactic haemophilia A/B treatment, Alhemo.
The once-daily, self-administered injectable will be suitable for patients aged 12 years or older.
Alhemo is a monoclonal antibody that acts as an anti-tissue factor pathway inhibitor, (TFPI) and — if approved — will be the first subcutaneous prophylactic treatment for haemophilia A or B with inhibitors.
Currently, the standard of care for haemophilia patients is to receive intravenous clotting factor replacement therapies, which can affect a patient's quality of life, as it requires frequent hospital visits.
Therefore, if approved, Alhemo could offer as a patient-centric alternative to current standard of care strategies.
The CHMP's positive opinion is based on data from the Phase III EXPLORER7 study, which evaluated Alhemo's efficacy and safety.
According to Novo Nordisk, if the drug is approved, it will be distrubuted to patients in a portable, pre-mixed and prefilled pen.
Final word from the European Commission is expected in two months.
Novo Nordisk's Chief Medical Officer for Haemophilia, Stephania Seremetis, commented: “The positive opinion from the CHMP for Alhemo® is a major milestone for patients,”
“If approved, Alhemo would be an important addition to our growing haemophilia portfolio as it offers the potential of everyday prophylaxis to prevent bleeds for people living with haemophilia who have developed the complication of inhibitors."
"It could alleviate the physical, emotional and overall treatment burden for people living with haemophilia, as it is delivered in a pre-filled, multi-use, portable pen that can be stored at room temperature for up to four weeks. This could enable patients to have greater confidence in the pursuit of daily activities, which is especially important for people living with haemophilia B with inhibitors, who currently have very limited treatment options.”
[Image credit: Novo Nordisk]