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Licensing agreement deals decline in rare diseases space, says report
New research has shown that complex collaborations and partnerships are increasing, while licensing agreements are decreasing in the rare disease space.
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Orphan Drugs
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Tinostamustine granted Orphan Drug Designation by European Commission
Tinostamustine has been granted Orphan Drug Designation by the European Commission to the treatment of T-cell prolymphocytic leukaemia (T-PLL).
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Comparison of health technology assessments and time to reimbursement for Orphan Drugs
This article delves into four health technology assessment agencies, analysing the opportunities and challenges for orphan drugs in France, Germany, England and Scotland.
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FDA grants Orphan Drug Designation to phenylketonuria treatment
A new treatment called APR-OD031 has been given Orphan Drug Designation (ODD) by the FDA, for the treatment of phenylketonuria.
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Developers request revocation of remdesivir’s ODD for COVID-19
Gilead Sciences say they are confident they can maintain an expedited regulatory review timeline for remdesivir as a treatment for COVID-19 without Orphan Drug Designation (ODD).
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EC grants Orphan Drug Designation to FLT190, a Fabry Disease treatment
FLT190 gene therapy has been granted Orphan Drug Designation based on preliminary trial data and the positive opinion of an EMA committee.
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FDA approves Isturisa (osilodrostat) treatment for Cushing’s disease
The FDA has approved Isturisa for Cushing’s disease treatment after it demonstrated significant efficacy in clinical trials.
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Orphan Drug Designations are declining, says market analysis
A report has suggested that number of Orphan Drug Designations in both the US and Europe is declining, possibly due to prices and political changes.
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FDA approves Tepezza for the treatment of thyroid eye in adults
The US FDA approved Tepezza (teprotumumab-trbw), the first treatment for thyroid eye, on the basis of two trials in which the drug improved eye protrusion in patients.
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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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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.