Products

Date: 2012-04-05

Type of information: Granting of the orphan status in the EU

Product name: autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene

Compound: autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene

Therapeutic area:

Action mechanism:

• gene therapy. This medicine is made up of immature haematopoietic cells that are taken from the patient. These cells are able to develop into different types of blood cell. In this gene therapy, the cells are modified by a virus that contains the gene for the gp91(phox) protein, so that this gene is carried into the cells. When these modified cells are transplanted back into the patient, they are expected to populate the bone marrow and produce healthy phagocytes with the gp91(phox) protein, which is lacking in patients with X-linked CGD. The type of virus used in this medicine (lentivirus) is modified in order not to cause disease in humans.

Company: Généthon (France)

Disease: X-linked chronic granulomatous disease

Latest news:

Patents:

Submission of marketing authorization application USA :

Submission of marketing authorization application UE:

Withdrawal of marketing authorization application USA:

Withdrawal of marketing authorization application UE:

US authorization:

UE authorization:

Favourable opinion UE:

Favourable opinion USA:

Orphan status USA:

Orphan status UE: 2012-02-09

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes