The European Medicines Agency is recommending approval of caplacizumab, a new drug for treating the rare blood disorder, acquired thrombotic thrombocytopenic purpura (aTTP). The disorder leads to extensive micro-clot formation in the small blood vessels and potential damage to vital organs.
The European Medicines Agency has given positive opinions for two chimeric antigen receptor (CAR) T cell therapies to treat blood cancers – bringing these medicines closer to the market for patients in the EU.
The US Food and Drug Administration has approved Epidiolex, a treatment for severe forms of epilepsy that is based on cannabidiol, a purified drug substance derived from marijuana. Epidiolex was developed by GW Pharmaceuticals Plc.
It has been approved to treat Lennox-Gastaut syndrome and Dravet syndrome in patients two years of age and older.
The drug is the first approved treatment containing cannabidiol, a chemical component of the Cannabis sativa plant. It is also the first drug approved for patients with Dravet syndrome.
A gene therapy for a rare, genetic eye disorder has been selected by the US Food and Drug Administration for its Regenerative Medicine Advanced Therapy Designation in recognition of the product’s potential to treat an unmet medical need.
The European Medicines Agency is restricting the use of two checkpoint inhibitor drugs as first-line treatments for urothelial cancer, or cancer of the bladder and urinary tract, because they may not work as well as chemotherapy in this group of patients. The drugs are Keytruda (pembrolizumab), the programmed cell death 1 (PD-1) inhibitor, and Tecentriq (atezolizumab), which inhibits the protein programmed cell death-ligand 1 (PD-L1). Both drugs are monoclonal antibodies that block a protective mechanism of cancer cells, allowing the immune system to destroy these cells.
The US Food and Drug Administration has approved the marketing of an artificial intelligence algorithm which is part of a diagnostic intended to help physicians detect wrist fractures. Called OsteoDetect, the product is a computer-aided detection and diagnostic software package that uses an artificial intelligence algorithm to analyse two-dimensional X-ray images for signs of distal radius fracture, a common type of wrist fracture. The software marks the location of the fracture on the image to assist the healthcare provider with detection and diagnosis.
The European Medicines Agency has issued a positive opinion for Aimovig (erenumab), an antibody treatment for migraine prevention, only weeks after it was given approval by the US Food and Drug Administration. Both agencies highlighted the novelty of the drug which is the first preventive treatment for migraine.
It works by blocking the activity of calcitonin gene-related peptide, a molecule that is involved in migraine attacks.
The US Food and Drug Administration has approved the first stand-alone prosthetic iris, a surgically implanted device to treat adults and children whose irises are completely missing or damaged due to aniridia, a congenital condition, or other damage to the eye.
The device, CustomFlex Artificial Iris, is made of thin, foldable medical-trade silicone and is custom-sized and coloured for each individual patient. It is implanted by a surgeon and held in place by the anatomical structures of the eye or, if necessary, by sutures.
With the introduction on 25 May of data protection regulations for companies inside the European Community and the outside companies that trade with Europe, a new entity called the European Data Protection Board (EDPB) began operations by holding a press conference in Brussels.
The data protection board consists of heads of national data protection agencies and has powers that are independent of the European Commission as it was created by an act of the European Parliament.
The US Food and Drug Administration has requested permanent injunctions against stem cell clinics in two regions of the country to prevent them from administering unapproved stem cell treatments to patients with cancer and other diseases. The move comes nearly 10 months after it issued warning letters to the clinics.