A new gene therapy has been given a favourable scientific review in Europe paving the way for its use in patients with beta-thalassaemia as early as the second quarter. The treatment, Zynteglo, uses a lentiviral vector to deliver functional copies of a beta-globin gene into a patient’s haematopoietic stem cells in order to correct for a shortage of beta-globin. It was given a positive opinion by the European Medicines Agency on 29 March.
A type of inflammatory arthritis that affects the spine but cannot be detected by x-ray now has an approved treatment. The US Food and Drug Administration has authorised Cimzia (certolizumab pegol) for the treatment of non-radiographic axial spondyloarthritis, a disease that starts in patients under the age of 45 years and can get progressively worse. The fact that the disorder cannot be identified by x-ray sets it apart from other inflammatory conditions in the same disease group.
The US Food and Drug Administration has approved a new drug for the treatment of patients with multiple sclerosis (MS) whose disease gradually worsens over time leading to a disruption of brain function through inflammation and tissue loss. The drug, Mayzent (siponimod), is a small molecule indicated for active secondary progressive MS.
Record sums were invested in biotech companies in 2018 – especially in those at the early stage of development. According to the US industry association BIO, some $12.4 billion in venture capital was raised in the US for biotech, double the amount generated in 2017, while $600 million was raised in Europe.
The US Food and Drug Administration has approved a new device for treating patients with chronic heart failure who are not suited for other interventions such as cardiac resynchronisation therapy. The device is indicated to improve a patient’s ability to perform a six-minute walk and improve his or her quality of life. These are patients who have a marked limitation of physical activity and who remain symptomatic despite receiving optimal medical therapy.
The European Commission is to allocate more than €2 billion for the final two years of an experimental programme intended to nurture entrepreneurs and start-up companies across Europe.
The checkpoint antibody Tecentriq (atezolizumab) received its fifth approval from the US Food and Drug Administration on 8 March – this time for triple-negative breast cancer. The approval is for the use of Tecentriq in combination with chemotherapy for patients with breast cancer whose tumours express the PD-L1 protein. Patients will be identified using a new FDA-approved assay.
The US Centers for Medicare and Medicaid Services (CMS) has proposed to reimburse cancer treatments with chimeric antigen receptor (CAR) T cell therapies under a policy called ‘Coverage with Evidence Development.” Currently there is no national Medicare policy for covering CAR T cell therapy so local Medicare contractors will have discretion over whether to pay for it. The proposed coverage would require Medicare to cover the therapy when it is offered in a CMS-approved registry or clinical study in which patients are monitored for at least two years after treatment.
Scott Gottlieb, commissioner of the US Food and Drug Administration, has resigned his position reportedly in order to spend more time with his family in Connecticut. The FDA job involved a weekly commute to Washington DC, a distance of about 277 miles.
Announced on 5 March, the resignation is expected to take effect in about a month. It was confirmed in a tweet by President Trump who praised Dr Gottlieb for his work leading the drug regulatory authority, a job that he has held since May 2017.
An approach to drug payments, where the price of a new medicine is linked to its effectiveness, may enable patients suffering from cancer to get new treatments faster, according to Cancer Research UK.