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Date: 2018-02-13

Type of information: Granting of a Market Authorisation in the EU

Product name: Alkindi® (Infacort®)

Compound: hydrocortisone oral granules

Therapeutic area: Rare diseases - Endocrine diseases - Hormonal diseases

Action mechanism:

  • hormone/corticosteroid. Infacort® is a preparation of hydrocortisone specifically designed for use in children suffering from adrenal insufficiency . It is an immediate-release, oral formulation of hydrocortisone with the potential to significantly improve the lives of children living with adrenal insufficiency.

Company: Diurnal Limited (UK)

Disease:

  • pediatric adrenal insufficiency (0 through 16 years of age)

Latest news:

  • • On February 13, 2018, Diurnal announced that the European Commission (EC) has granted a paediatric use marketing authorisation (PUMA) for Alkindi® (development programme name: Infacort®; hydrocortisone granules in capsules for opening) as replacement therapy of adrenal insufficiency in infants, children and adolescents (from birth to <18 years old), following the positive opinion issued by the European Medicines Agency (EMA) in December 2017. The marketing approval under a PUMA affords 10 years' data and market exclusivity for Alkindi® in Europe.
  • Given the specialist prescribing base, and to retain the full commercial value of the product, Diurnal will commercialise Alkindi® itself in the major European markets. Diurnal has a detailed commercial plan in place for the anticipated first launch of Alkindi® in Q2 2018.
  • The marketing authorisation approval is based upon review of data from the Company's pivotal open-label Phase III clinical trial conducted in 24 subjects before their sixth birthday, requiring replacement therapy for adrenal insufficiency due to congenital adrenal hyperplasia, primary adrenal failure or hypopituitarism. The study successfully met its primary endpoint and no serious adverse events were reported. Based on these data, and a comprehensive dossier from Diurnal, the market approval now extends the product's use to include paediatric patients up to 18 years of age.
  • • On December 15, 2017, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting a paediatric-use marketing authorisation (PUMA) for Alkindi® (hydrocortisone) for the treatment of primary adrenal insufficiency.
  • • On May 13, 2015, the FDA has granted orphan drug designation for Infacort® (hydrocortisone oral granules) for the treatment of pediatric adrenal insufficiency (0 through 16 years of age). Infacort® was recently entered into a pivotal Phase 3 study in Europe which is expected to read out in 2016.

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: 2018-02-13

Favourable opinion UE: 2017-12-15

Favourable opinion USA:

Orphan status USA: 2015-05-13

Orphan status UE: 2007-03-22

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes