List view / Grid view
The FDA has awarded 11 grants equating to about $25 million of funding to clinical trials for the development of rare disease treatments.
In its most recent meeting, the CHMP recommended nine new medicines and concluded its review of Vaxzevria’s thrombosis risk.
FDA approves mobocertinib as the first oral therapy for patients with non-small cell lung cancer with EGFR Exon20 insertion mutations.
Moderna have announced that the first patient has been dosed in Phase I/II study of mRNA-3705 for methylmalonic acidemia (MMA).
GW Pharmaceuticals received approval for Epidyolex®(cannabidiol) for the treatment of seizures linked to tuberous sclerosis complex (TSC) in the UK.
The Committee for Medicinal Products for Human Use (CHMP) recommended two medicines for approval and provided a negative opinion on Nouryant.
The Committee for Medicinal Products for Human Use (CHMP) recommended eight drugs for approval and nine indication extensions.
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.