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Summarising 2019: a year of firsts for the EMA
The EMA was a proactive force in 2019, granting marketing authorisation for a range of drugs. This article highlights some of the most important advancements made by the agency last year.
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Orphan Drugs
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FDA grants Avatrombopag Orphan Drug Designation for thrombocytopenia
The US FDA has granted avatrombopag (Doptelet) ODD as around 10 percent of US cancer patients per year experience chemotherapy-induced thrombocytopenia (CIT) with no available treatment.
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FDA grants Orphan Drug Designation to Epidermolysis Bullosa treatment
The US Food and Drug Administration has awarded APR-TD011 Orphan Drug Designation, for the treatment of a rare disease.
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Zotiraciclib granted Orphan Drug Designation by FDA and EMA
The FDA and EMA have granted Orphan Drug Designation to zotiraciclib, currently the subject of two separate Phase Ib clinical trials in glioblastoma.
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Priority Review granted to pemigatinib for treatment of cholangiocarcinoma
The US FDA has awarded pemigatinib Priority Review, after the drug met its primary and secondary endpoints in a Phase II trial.
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First treatment for inherited rare disease approved by the FDA
Givlaari (givosiran) has been approved by the FDA for the treatment of adult patients with the rare genetic disorder, acute hepatic porphyria.
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The 10 latest FDA Orphan Drug Designations for cancer treatments
The FDA grants Orphan Drug Designation to treatments for rare diseases in R&D; here are the 10 most recent for oncologic conditions.
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Multiple myeloma treatment granted Orphan Drug Designation
Orphan Drug Designation has been given to bispecific antibody candidate, GBR 1342, for the treatment of multiple myeloma.
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Multiple myeloma treatment receives Orphan Drug Designation
Orphan Drug Designation has been granted to the CT053 anti-BCMA CAR-T programme for multiple myeloma treatment.
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FDA grants Orphan Drug Designation to DYN101 treatment
A treatment for Centronuclear Myopathies has received Orphan Drug Designation from the FDA and will begin Phase I/II trials.
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FDA grants Orphan Drug Designation for narcolepsy treatment
The FDA has given the XWL-008 compound Orphan Drug Designation for the treatment of narcolepsy.
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FDA approves first gene therapy for paediatric spinal muscular atrophy
The FDA has given approval to AveXis for the first treatment of paediatric spinal muscular atrophy (SMA).
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Eurofins Amatsigroup ǀ Your cDmo partner
Frédéric Gaussens from Eurofins Amatsigroup (CDMO) giving an overview of the services they provide...
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FDA grants orphan drug designation to elamipretide for LHON
The FDA granted Orphan Drug Designation to elamipretide, for the treatment of Leber's hereditary optic neuropathy...
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FDA approves first therapy for rare inherited form of rickets, x-linked hypophosphatemia
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...