Regulation & Policy

With three gene therapies on the US market, the Food and Drug Administration has provided six new guidance documents for developers to clarify the regulatory standards for these new medicines.

Three of the new guidance documents relate to regulatory standards for manufacturing and three are product specific. The product-specific guidelines are intended to assist developers of prospective gene therapies for haemophilia, retinal disorders and rare diseases.

To address the risk of bioterrorism, the US Food and Drug Administration has approved a drug for the treatment of smallpox, an acute contagious viral disease that was declared eradicated in 1980, but could still be reintroduced into the environment at a time of conflict.

“To address the risk of bioterrorism, Congress has taken steps to enable the development and approval of countermeasures to thwart pathogens that could be employed as weapons,” Scott Gottlieb, the FDA commissioner, announced on 13 July.

Ipsen SA received further validation of its in-licensed compound cabozantinib following the publication in the 5 July edition of the New England Journal of Medicine (NEJM) of Phase 3 data showing the drug’s effectiveness in advanced liver cancer. Cabozantinib, which trades commercially as Cabometyx, has already been approved in the US and Europe for advanced kidney cancer. The drug was developed by Exelixis Inc and out-licensed to Ipsen in 2016 for commercialisation outside the US, Canada and Japan. Ipsen later acquired rights to Canada.

A candidate vaccine to prevent infection from the chikungunya virus has been accepted into the European Medicines Agency’s PRIME scheme, a programme of regulatory support for developers of medicines that meet a major public health need.

The vaccine is a biologic developed by Themis Bioscience GmbH of Austria and is currently undergoing Phase 2 studies in 600 patients across the US, the EU and South and Central America. The company hopes to receive marketing approval within three years.

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.