Kayfanda
Opinion
EMA has issued an opinion on this medicine
odevixibat
MedicineHumanOpinion
Overview
On 25 July 2024, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation under exceptional circumstances1 for the medicinal product Kayfanda, intended for the treatment of cholestatic pruritus in patients with Alagille Syndrome (ALGS) in patients aged 6 months or older.
The applicant for this medicinal product is Ipsen Pharma.
Kayfanda will be available as 200 µg, 400 µg, 600 µg and 1200 µg hard capsules. The active substance of Kayfanda is odevixibat, a bile therapy (ATC code: A05AX05). Odevixibat is a reversible and selective inhibitor of the ileal bile acid transporter (IBAT) that acts locally in the distal ileum. It reduces the reuptake of bile acids and increases the clearance of bile acids through the colon.
The benefits of Kayfanda are its clinically relevant effects on pruritus and associated sleep disturbances in ALGS patients. In addition, Kayfanda reduces the concentration of serum bile acids of patients with ALGS. The most common side effects with Kayfanda are diarrhoea, abdominal pain, vomiting, and hepatic enzymes increased.
The full indication is:
Kayfanda is indicated for the treatment of cholestatic pruritus in Alagille syndrome (ALGS) in patients aged 6 months or older (see sections 4.4 and 5.1).
Treatment with Kayfanda must be initiated and supervised by physicians experienced in the management of ALGS.
Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published in the European public assessment report (EPAR) and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.
1 In exceptional circumstances, an authorisation may be granted subject to certain specific obligations, to be reviewed annually. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicinal product, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data.
Product details
- Name of medicine
- Kayfanda
- Active substance
- Odevixibat sesquihydrate
- International non-proprietary name (INN) or common name
- odevixibat
- Therapeutic area (MeSH)
- Pruritus
- Alagille Syndrome
- Anatomical therapeutic chemical (ATC) code
- A05AX05
- EMA product number
- EMEA/H/C/006462
Exceptional circumstances
This medicine was authorised under exceptional circumstances, because the applicant was unable to provide comprehensive data on the efficacy and safety of the medicine under normal conditions of use. This can happen because the condition to be treated is rare or because collection of full information is not possible or is unethical. For more information, see Pre-authorisation guidance.
- Marketing authorisation applicant
- Ipsen Pharma
- Opinion adopted
- 25/07/2024
- Opinion status
- Positive
This page was last updated on