SANOFI B.V.

It is used to treat patients with MPS I (Mucopolysaccharidosis I), acting on the non-neurological manifestations of the disease by replacing a deficient natural enzyme.

Dosage form: solution for infusion, concentrate

ATC: A16AB05

It is used to treat adults and children aged 5 years and older suffering from a type of medullary thyroid carcinoma with a RET gene mutation that cannot be surgically removed or that has already spread to other parts of the body.

Dosage form: tablets, film-coated

ATC: L01EX04

Cerdelga is used for the long-term treatment of type 1 Gaucher disease in adult patients and children aged 6 years and older weighing at least 15 kg.

Dosage form: capsules, hard gelatin

ATC: A16AX10

It is used for the treatment of patients with Gaucher disease (Type 1 or Type 3) presenting with symptoms such as anemia, tendency to bleed, enlargement of the spleen or liver, or bone disease.

Dosage form: powder for concentrate for infusion solution

ATC: A16AB02

It is used for the treatment of acute lymphoblastic leukemia (ALL) in children (from 1 year of age and older), adolescents, and young patients up to 21 years of age, for whom previous treatments have not worked or have ceased to be effective.

Dosage form: solution for infusion, concentrate

ATC: L01BB06

It is used as long-term enzyme replacement therapy for patients with a confirmed diagnosis of Fabry disease.

Dosage form: solution for infusion, powder for preparation

ATC: A16AB04

It is used for the treatment of B-cell chronic lymphocytic leukemia (CLL) in patients who have sufficient production of healthy blood cells.

Dosage form: solution for injection or infusion

ATC: L01BB05

It is used to promote the release and collection of stem cells from the blood, which can then be stored for a future transplant. It is indicated for adults with lymphoma or multiple myeloma and for children between 1 and 18 years of age with lymphoma or solid tumors.

Dosage form: solution for injection

ATC: L03AX16

It is used for the treatment of adults, children, and adolescents with a confirmed diagnosis of Pompe disease, a condition characterized by reduced levels of the enzyme alpha-glucosidase.

Dosage form: powder for concentrate for infusion solution

ATC: A16AB07

It is used to treat people of all ages affected by Pompe disease. The drug replaces a missing enzyme in the body to help reduce the accumulation of glycogen that damages the muscles.

Dosage form: powder for concentrate for injectable/infusion solution

ATC: A16AB22

This drug is an immunosuppressant used to prevent or treat the rejection of transplanted organs (such as kidney, liver, pancreas, or heart). It is also used to prevent graft-versus-host disease (GvHD) in patients who have received a bone marrow or stem cell transplant, and it can be used to treat aplastic anemia if other treatments have not been effective.

Dosage form: solution for infusion, powder for preparation

ATC: L04AA04

Xenpozyme is used to treat acid sphingomyelinase deficiency (ASMD), a hereditary disorder, in children and adults. It works by replacing the missing natural enzyme to reduce the accumulation of sphingomyelin in organs such as the spleen, liver, heart, and lungs.

Dosage form: powder for concentrate for injectable/infusion solution

ATC: A16AB25