Pre-approval Access to IMPs

Ablynx Policy on Pre-Approval Access to CabliviTM (caplacizumab)

Ablynx’s mission is to create new medicines which will make a real difference to the lives of patients. As part of that mission, Ablynx is firmly committed to advancing, as rapidly as possible, the development of the most promising Nanobodies®, and making them available to patients as soon as permission is granted by regulatory agencies.

Ablynx believes that in general it is in the best interest of patients to progress investigational medicines through the clinical development process and gain approval from regulatory agencies as expeditiously as possible. However, not all patients are able to enrol in clinical trials but may still benefit from treatment with CabliviTM (caplacizumab), the Company’s anti-von Willebrand Factor (vWF) Nanobody® being developed for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP). Therefore, Ablynx has implemented an early access programme for these patients, which will be available until regulatory approval is granted in the respective country.

How do you make a request for access to CabliviTM (caplacizumab)?

A request must always be made by the treating physician who should be experienced in the management of patients with thrombotic microangiopathies.

Each request will be evaluated on a case-by-case basis, taking into account all of the following:

  • The seriousness of the patient condition
  • The fact that there are no satisfactory approved therapies available
  • The evidence that the potential benefit to the patient would likely outweigh the potential risks
  • That access to CabliviTM (caplacizumab) is in accordance with the laws and regulations effective in the country in which the product will be administered

Any communication related to pre-approval access to caplacizumab should be sent to pre-approval@ablynx.com. We will acknowledge receipt of all communications within 24h.