The European Medicines Agency’s main scientific committee has recommended approval of a new hybrid medicine for managing chronic pulmonary infection due to the bacteria Pseudomonas aeruginosa in patients with cystic fibrosis.
Novartis has agreed to offer Kymriah (tisagenlecleucel) the cell-based gene therapy, for an undisclosed discount to patients under the age of 25 years following a negotiation with UK authorities, including the National Institute for Health and Care Excellence (NICE). Supplies of Kymriah will be financed through the Cancer Drugs Fund, a special fund for promising treatments, NICE announced on 16 November.
A Chinese scientist’s reported use of the Crispr gene editing tool to alter a gene in two embryos which were implanted into a mother’s womb has created an uproar in the scientific community. The edit was performed when the embryos were just a day old and involved alterations to the CCR5 gene in order to prevent the offspring from contracting HIV. They were reported on YouTube videos, rather in a scientific journal, after twin girls bearing the DNA changes were born.
A new precision medicine for patients whose cancers have a specific genetic feature has been approved by the US Food and Drug Administration – the second time the agency has based a decision on a common biomarker rather than the location of a tumour in the body.
The US Food and Drug Administration has approved a new oral treatment for acute myeloid leukaemia (AML) in adults who are 75 years or older and who have other chronic conditions that may preclude the use of intensive chemotherapy. The drug, Daurismo (glasdegib), is an inhibitor of the Hedgehog pathway which when abnormal, is thought to contribute to the development of cancer stem cells. Daurismo was developed by Pfizer Inc.
The European Commission has cleared Takeda Pharmaceutical Company Ltd’s proposed $62 billion takeover of Shire Plc, subject to its disposal of an investigational drug in Shire’s portfolio for the treatment of inflammatory bowel disease (IBD). The Shire compound overlaps with Entyvio, a drug marketed by Takeda for the same disease.
Takeda has offered to divest the experimental treatment, including the rights to its development, manufacture and marketing to a purchaser that would have an incentive to produce a competitor to Entyvio.
A new treatment for sleeping sickness, a life-threatening tropical disease, has received a positive review by the European Medicines Agency, paving the way for its approval in sub-Saharan Africa where the illness is endemic. The medicine, Fexinidazole Winthrop (fexinidazole), was developed through a collaboration between Sanofi SA and the non-profit organisation, Drugs for Neglected Diseases initiative (DNDi).
The antibody-drug conjugate Adcetris (brentuximab vedotin) has received a new lymphoma indication from the US Food and Drug Administration under a procedure that allows the agency to study key clinical data for the compound prior to its formal regulatory application.
The US Food and Drug Administration is proposing a waiver to the legal requirement on pharma companies to obtain informed patient consent for certain low-risk clinical trials. The proposal would amend a provision of the 21st Century Cures Act to add an exception to the consent requirement.
The proposed rule, if finalized, would allow the institutional review board (IRB) responsible for the review and approval of clinical research to waive or alter certain elements of informed consent, or to waive the requirement entirely, under limited circumstances.
The European Medicines Agency has recommended the approval of Dengvaxia (dengue tetravalent vaccine) for the prevention of dengue, the most common mosquito-borne viral disease affecting people worldwide. The vaccine is for the prevention of dengue caused by the virus serotypes 1,2,3 and 4 in people between the ages of nine and 45 and who have already had a prior infection.