FDA grants orphan drug designation to elamipretide for LHON
1 May 2018 | By Dr Zara Kassam (European Pharmaceutical Review)
The FDA granted Orphan Drug Designation to elamipretide, for the treatment of Leber's hereditary optic neuropathy...
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Orphan Drugs
FDA approves first therapy for rare inherited form of rickets, x-linked hypophosphatemia
20 April 2018 | By Dr Zara Kassam (European Pharmaceutical Review)
The FDA has approved Crysvita, the first drug approved to treat adults and children ages 1 year and older with x-linked hypophosphatemia, a rare, inherited form of rickets...
KL1333 receives FDA ODD for mitochondrial diseases
20 April 2018 | By Dr Zara Kassam (European Pharmaceutical Review)
NeuroVive Pharmaceutical AB has been granted Orphan Drug Designation for its project KL1333 for treatment of inherited mitochondrial respiratory chain diseases...
FDA expands approval of Blincyto for treatment of B-cell precursor ALL
5 April 2018 | By European Pharmaceutical Review
The FDA has granted accelerated approval to Blincyto to treat adults and children with B-cell precursor acute lymphoblastic leukaemia (ALL)...
FDA approves Lutathera for somatostatin receptor-positive GEP-NETs
29 January 2018 | By Dr Zara Kassam (European Pharmaceutical Review)
The FDA has approved Lutathera for the treatment of a type of cancer that affects the pancreas the gastrointestinal tract...
Enzyme replacement therapy to treat alpha-mannosidosis
29 January 2018 | By Dr Zara Kassam (European Pharmaceutical Review)
The EMA has recommended granting a marketing authorisation in the EU for Lamzede a long-term enzyme replacement therapy for alpha-mannosidosis...
EC grants orphan drug designation for Gilteritinib for the treatment of AML
24 January 2018 | By Dr Zara Kassam (European Pharmaceutical Review)
The European Commission has issued Orphan Designation to gilteritinib for the treatment of patients with acute myeloid leukaemia...
FDA accepts IND for the treatment of mucopolysaccharidosis type IIIA
23 January 2018 | By Dr Zara Kassam (European Pharmaceutical Review)
The FDA has issued a Study may proceed letter for the first study in humans, thereby accepting the investigational new drug application for the drug candidate SOBI003...
FDA approves Nucala for Eosinophilic Granulomatosis with Polyangiitis
15 December 2017 | By Dr Zara Kassam (European Pharmaceutical Review)
The FDA has expanded the approved use of Nucala to treat a rare autoimmune disease that causes vasculitis, an inflammation of the wall of blood vessels of the body...
EMA grants marketing authorisation for Prevymis against cytomegalovirus
10 November 2017 | By
The EMA has recommended granting a marketing authorisation Prevymis, an antiviral medicine that prevents CMV reactivation and disease...
FDA approves Zelboraf for certain patients with Erdheim-Chester disease
7 November 2017 | By Dr Zara Kassam (European Pharmaceutical Review)
The FDA has expanded the approval of Zelboraf to include the treatment of certain adult patients with Erdheim-Chester Disease, a rare cancer of the blood...
FDA grants orphan drug status to Cellect’s ApoGraft
5 September 2017 | By Dr Zara Kassam (European Pharmaceutical Review)
The FDA has granted orphan drug designation for ApoGraft for the prevention of acute and chronic graft versus host disease...
FDA approves Mylotarg for treatment of acute myeloid leukemia
4 September 2017 | By Dr Zara Kassam (European Pharmaceutical Review)
The FDA has approved Mylotarg for the treatment of adults with newly diagnosed acute myeloid leukaemia whose tumours express the CD33 antigen...
FDA approves new targeted treatment for acute myeloid leukemia
2 August 2017 | By Dr Zara Kassam (European Pharmaceutical Review)
The U.S. Food and Drug Administration has granted the approval of Idhifa to Celgene Corporation and RealTime IDH2 Assay to Abbott Laboratories...
UK bioscience companies form Rare Disease Industry Group
24 July 2017 | By Dr Zara Kassam (European Pharmaceutical Review)
The BIA is committed ensuring patient access to treatments for rare and very rare conditions, sometimes referred to as orphan and ultra-orphan medicines...
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