Simoctocog alfa (trade name Nuwiq) has been approved since July 2014 for people with type A haemophilia, an inherited disorder that impairs blood clotting. The German Institute for Quality and Efficiency in Health Care (IQWiG) examined in a dossier assessment whether this new drug offers an added benefit over the appropriate comparator therapy. Such an added benefit cannot be derived from the dossier, however, because the drug manufacturer did not submit any suitable data.

The manufacturer presented two randomized controlled trials (RCTs) with crossover design for the direct comparison with octocog alfa. Their primary outcome criterion was pharmacokinetics such as ingestion and excretion of the drug. In these two studies, the patients received a single dose of simoctocog alfa or octocog alfa and – after a wash-out phase – a second single dose of the respective other drug.

However, a single dose is insufficient to assess the added benefit. Patients with haemophilia require longterm treatment and prevention of bleeding. European Medicines Agency (EMA) guidelines therefore recommend a minimum duration of six months for studies that aim to test the advantages and disadvantages of haemophilia drugs used for prophylaxis. Incidentally, the manufacturer itself also derived no advantage of its drug from these two studies.

For more details, go to: https://www.iqwig.de/en/press/press-releases/press-releases/simoctocog-alfa-for-haemophilia-a-no-suitable-data.6593.html