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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Tinostamustine has been granted Orphan Drug Designation by the European Commission to the treatment of T-cell prolymphocytic leukaemia (T-PLL).
This article delves into four health technology assessment agencies, analysing the opportunities and challenges for orphan drugs in France, Germany, England and Scotland.
A new treatment called APR-OD031 has been given Orphan Drug Designation (ODD) by the FDA, for the treatment of phenylketonuria.
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).
FLT190 gene therapy has been granted Orphan Drug Designation based on preliminary trial data and the positive opinion of an EMA committee.
The FDA has approved Isturisa for Cushing’s disease treatment after it demonstrated significant efficacy in clinical trials.
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.
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.
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.
The US Food and Drug Administration has awarded APR-TD011 Orphan Drug Designation, for the treatment of a rare disease.
The FDA and EMA have granted Orphan Drug Designation to zotiraciclib, currently the subject of two separate Phase Ib clinical trials in glioblastoma.
The US FDA has awarded pemigatinib Priority Review, after the drug met its primary and secondary endpoints in a Phase II trial.
Givlaari (givosiran) has been approved by the FDA for the treatment of adult patients with the rare genetic disorder, acute hepatic porphyria.