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With almost every aspect of pharmaceutical regulation currently under review across Europe and the proposal for changes expected in December 2022, in this article, Maarten Meulenbelt, Partner at Sidley Austin LLP, discusses with European Pharmaceutical Review some of the more controversial amendments under consideration and emphasises why industry participation is…
According to a new report, the orphan drug market is growing more than twice as fast as the non-orphan market and, by 2026, orphan drug sales will account for 20 percent of all prescription drug sales.
The US Food and Drug Administration (FDA) approves Besremi (ropeginterferon alfa-2b-njft) injection to treat adults with polycythemia vera.
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.
In this article, we summarise the trial findings for the European Medicines Agency’s 13 most transformative therapies of 2020.
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.
