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Highlights of the EMA’s human medicine committee meeting, April 2021
The Committee for Medicinal Products for Human Use (CHMP) recommended eight drugs for approval and nine indication extensions.
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Orphan Drugs
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Microbiome biotherapeutic shows promise in acute graft-versus-host disease
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.
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EMA’s transformative treatments of 2020
In this article, we summarise the trial findings for the European Medicines Agency’s 13 most transformative therapies of 2020.
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What impact does disease rarity have on rare and orphan drug pricing?
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.
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FDA grants Orphan Drug Designation to two cancer treatments
The FDA has given small molecules APG-115 and APG-1252, two cancer treatments, Orphan Drug Designation.
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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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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.