Oncology drugs that promise to meet a major public health need may in future be eligible for a clinical development pathway that is both simpler and less costly than traditional methods, the US Food and Drug Administration announced on 10 August.
The US Food and Drug Administration has approved an RNA therapeutic developed by Alnylam Pharmaceuticals Inc to treat patients with a rare genetic disease characterised by the build-up of abnormal amyloid protein in peripheral nerves and other organs.
A candidate treatment for Huntington’s disease has been selected by the European Medicines Agency for PRIME, a programme of regulatory support for developers of medicines that meet a major public health need. The drug, RG6042, is an antisense oligonucleotide.
It is being developed jointly by Roche and Ionis Pharmaceuticals Inc to slow or stop the progress of Huntington’s disease, a rare genetic disorder that causes nerve cells in the brain to break down. Over time, people with the disease can develop personality changes and have difficulty walking and swallowing.
Darzalex (daratumumab) is being proposed as a first-line treatment for newly diagnosed multiple myeloma in combination with three other cancer drugs for patients who are ineligible for an autologous stem cell transplant.
With Brexit looming, the European Medicines Agency now believes that it could lose up to 30% of its staff as the agency prepares to move from its current location in London to new premises in Amsterdam. The move is scheduled to take place in March 2019.
As an agency of the European Union, the EMA must be located in an EU member state. London will no longer be eligible after Britain leaves the EU which is set for 23.00 BST on 29 March 2019.
An RNA therapeutic developed by Alnylam Pharmaceuticals Inc is the newest drug to be recommended for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), a life-threatening disease caused by mutations in the transthyretin (TTR) gene.
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.