Acquired thrombotic thrombocytopenic purpura (aTTP)

Active Ingredient: Caplacizumab

Indication for Caplacizumab

Population group: only adults (18 - 65 years old)

Caplacizumab is indicated for the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression.

For this indication, competent medicine agencies globally authorize below treatments:

10 mg once daily

Route of admnistration

Intravenous

Defined daily dose

10 - 10 mg

Dosage regimen

From 10 To 10 mg once every day

Detailed description

First dose

Intravenous injection of 10 mg of caplacizumab prior to plasma exchange.

Subsequent doses

Daily subcutaneous administration of 10 mg of caplacizumab after completion of each plasma exchange for the duration of daily plasma exchange treatment, followed by daily subcutaneous injection of 10 mg of caplacizumab for 30 days after stopping daily plasma exchange treatment.

If at the end of this period there is evidence of unresolved immunological disease, it is recommended to optimise the immunosupression regimen and continue daily subcutaneous administration of 10 mg of caplacizumab until the signs of underlying immunological disease are resolved (e.g. sustained normalisation of ADAMTS13 activity level).

In the clinical development program, caplacizumab has been administered daily for up to 65 days. No data on re-treatment with caplacizumab are available.

Missed dose

If a dose of caplacizumab is missed, it can be administered within 12 hours. If more than 12 hours have passed since the dose was to have been given, the missed dose should NOT be administered and the next dose should be administered per the usual dosing schedule.

Dosage considerations

The first dose of caplacizumab is to be administered as an intravenous injection. Subsequent doses are to be administered via subcutaneous injection in the abdomen.

Injections into the area around the navel should be avoided and consecutive injections should not be administered in the same abdominal quadrant.

Active ingredient

Caplacizumab

Caplacizumab is a humanised bivalent Nanobody that consists of two identical humanised building blocks (PMP12A2hum1), genetically linked by a three-alanine linker, targeting the A1-domain of von Willebrand factor and inhibiting the interaction between von Willebrand factor and platelets. As such, caplacizumab prevents the ultralarge von Willebrand factor-mediated platelet adhesion, which is characteristic of aTTP. It also affects the disposition of von Willebrand factor, leading to transient reductions of total von Willebrand factor antigen levels and to concomitant reduction of factor VIII:C levels during treatment.

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