European Medicines Agency - Human medicines - European public assessment reports
New designations March 2012
Treatment of congenital hyperinsulinism
Glucagon
Treatment of systemic amyloidosis caused by beta-2 microglobulin
Doxycycline hyclate
Treatment of soft tissue sarcoma
(1-methyl-2-nitro-1H-
Treatment of pulmonary arterial hypertension
Sodium nitrite
Treatment of pneumonia caused by Staphylococcus aureus
Human monoclonal antibody targeting Staphylococcus aureus alpha-toxin
Treatment of amyotrophic lateral sclerosis
6-ethynyl-1-(pentan-3-yl)-1H-
Treatment of carbamoyl-phosphate synthase-1 deficiency
Heterologous human adult liver-derived stem cells
Treatment of hereditary inclusion body myopathy
Sialic acid
New designations January 2012
Treatment of haemophilia B
Adeno-associated viral vector containing the human factor IX Gene
New marketing authorisations
Vyndaqel [tafamidis]
ICON Clinical Research Limited
United Kingdom
What is Vyndaqel?
Vyndaqel is a medicine that contains the active substance tafamidis. It is available as capsules (20 mg).
What is Vyndaqel used for?
Vyndaqel is used to delay nerve damage caused by transthyretin amyloidosis, a hereditary disease in which fibres called amyloid build up in tissues around the body including around the nerves. Vyndaqel is used in adult patients with the early stage of nerve disease (stage 1).
Because the number of patients with transthyretin amyloidosis is low, the disease is considered 'rare', and Vyndaqel was designated an 'orphan medicine' (a medicine used in rare diseases) on 28 August 2006.
The medicine can only be obtained with a prescription.
Plenadren [hydrocortisone]
DuoCort Pharma AB
Sweden
What is Plenadren?
Plenadren is a medicine that contains the active substance hydrocortisone. It is available as modified-release tablets (5 mg and 20 mg).
What is Plenadren used for?
Plenadren is used to treat adrenal insufficiency in adults. Adrenal insufficiency (including primary insufficiency or Addison's disease) is a condition where the adrenal glands (located just above the kidneys) do not produce enough of a steroid hormone called cortisol (also known as the stress hormone because it is released in response to stress). Symptoms include weight loss, muscle weakness, fatigue, low blood pressure, and sometimes darkening of the skin. Adrenal insufficiency can require life-long treatment to replace the missing cortisol.
Because the number of patients with adrenal insufficiency is low, the disease is considered 'rare', and Plenadren was designated an 'orphan medicine' (a medicine used in rare diseases) on 22 May 2006.
The medicine can only be obtained with a prescription.
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