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In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended eight new medicines for approval, including an enzyme replacement therapy for a rare disease.
The European Commission has approved Enjaymo®, the first-and-only approved therapeutic for haemolytic anaemia in adults with cold agglutinin disease.
The Committee for Medicinal Products for Human Use (CHMP)’s November meeting consisted of approval recommendations for four medicines, including a biosimilar for osteoporosis and a new COVID-19 vaccine.
Research for rare diseases including ALS will be supported over the next four years by $38 million in FDA funding.
The FDA has granted ODD to Telitacicept, produced by RemeGen, for the treatment of myasthenia gravis.
Hansizhuang (serplulimab), the world's first anti-PD-1 monoclonal antibody (mAb) treatment for extensive-stage small cell lung cancer (ES-SCLC), enhanced survival rates compared to chemotherapy, and is projected to be the leading immunotherapy for the disease in the next five years.
A Phase I trial revealed that Bioheng Biotech’s chimeric antigen receptor (CAR) T-cell therapy drug RD13-01 was safe for use and that it could reduce malignant tumours.
With a growing number of therapies under development for rare diseases, ICON’s Dr William Maier discusses how real-world evidence (RWE) can be effectively used as a historical control (HC), overcoming challenges presented in clinical development.
In its latest meeting, the Committee for Medicinal Products for Human Use (CHMP) recommended 12 medicines for approval, including those for rare diseases, osteoporosis and cancer.
European approval of BioMarin’s ROCTAVIAN (valoctocogene roxaparvovec) one-time gene therapy represents a breakthrough in the treatment of adults with severe haemophilia A.
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.
