Genetic disorders

On this page:

• Cerdelga^O
• Galafold^O
• Haegarda^B,O
• Kanuma^B,C,O
• Procysbi^P,O
• Replagal^B,C,O
• Spinraza^C,O

Cerdelga^O

(new active substance)

Medical ingredient:

Eliglustat Tartrate

Indication:

Cerdelga is a medicine used for treating adult patients with Gaucher disease type 1.

For more information, see the Regulatory Decision Summary.

Galafold^O

(new active substance)

Medical ingredient:

Migalastat Hydrochloride

Indication:

Galafold is used for the long-term treatment of Fabry disease in adults who have certain genetic mutations (changes) in an enzyme called alpha-galactosidase A (α-Gal A).

For more information, see the Regulatory Decision Summary.

Haegarda^B,O

Medical ingredient:

C1 Esterase Inhibitor (Human)

Indication:

Haegarda is an injectable medicine used to prevent swelling and/or painful attacks in adults and adolescents with Hereditary Angioedema (HAE).

For more information, see the Regulatory Decision Summary.

Kanuma^B,C,O

(new active substance)

Medical ingredient:

Sebelipase Alfa

Indication:

Kanuma is used to treat infants, children and adults with lysosomal acid lipase deficiency (LAL deficiency)- a condition that affects an individual's ability to breakdown and use fats and cholesterol. It is used to treat infants, children and adults with lysosomal acid lipase deficiency (LAL deficiency).

For more information, see the Regulatory Decision Summary.

Procysbi^P,O

(new active substance)

Medical ingredient:

Cysteamine Bitartrate

Indication:

Procysbi is used for treatment of nephropathic cystinosis, a rare metabolic disease.

For more information, see the Regulatory Decision Summary.

Replagal^B,C,O

Medical ingredient:

Agalsidase Alfa

Indication:

Replagal is used to treat patients with a confirmed diagnosis of Fabry Disease.

For more information, see the Regulatory Decision Summary.

Spinraza^C,O

(new active substance)

Medical ingredient:

Nusinersen Sodium

Indication:

Spinraza is used to treat a genetic disease called 5q Spinal Muscular Atrophy (SMA). Spinal Muscular Atrophy- a disease of the nervous system that affects voluntary muscle movement.