The Committee for Medicinal Products for Human Use (CHMP) recommended eight drugs for approval and nine indication extensions.
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MaaT013 was safe and generated complete or very good partial responses in a third of patients with steroid-refractory, gastrointestinal-predominant acute graft-versus-host disease.
Bhavesh Patel, Cécile Matthews and Owen Male at CRA’s Life Sciences Practice assess the relationship between drug pricing for rare disease therapies and disease prevalence in Europe, taking a closer look at payer assessments of orphan drugs in Germany.
The FDA has given small molecules APG-115 and APG-1252, two cancer treatments, Orphan Drug Designation.
New research has shown that complex collaborations and partnerships are increasing, while licensing agreements are decreasing in the rare disease space.
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.