The European Medicines Agency has launched a new project to help academic scientists and those working for non-profit organisations understand the regulatory requirements for developing and eventually commercialising cell, gene and tissue engineered therapies. The project complements the agency’s priority medicines scheme, PRIME, which was launched in 2016 to provide support to developers of medicines that meet a major public health need.
The US Food and Drug Administration has approved a new drug for amyotrophic lateral sclerosis, a rare disease that attacks and kills the nerve cells that control voluntary muscles. In a Phase 2 trial, the drug Relyvrio (sodium phenylbutyrate and /taurursodiol), showed an ability to slow the rate of neuromuscular decline. Moreover in a follow-up study, it extended patient survival compared with a placebo.
A new treatment designed to protect infants from infection from the respiratory syncytial virus (RSV) has received a positive opinion from the European Medicines Agency. The accelerated review was in response to evidence that infections from RSV are on the rise following a relaxation of restrictions that have been in place to prevent Covid-19.
The European Medicines Agency has recommended approval of a bivalent vaccine to protect against two sub-variants of the SARS-CoV-2 virus. The authorisation is for an updated booster shot developed by Pfizer Inc and BioNTech SE to protect people against the omicron subvariants BA.4 and BA.5 in addition to the original strain of SARS-CoV-2. The new vaccine is to be used in people aged 12 years and older who have received at least a primary course of vaccination to prevent Covid-19.
An enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD), a rare genetic disease that can cause premature death, has cleared the US Food and Drug Administration making it the first medicine to treat symptoms of the disease that are not related to the central nervous system. The FDA approval of Xenpozyme (olipudase alfa) was granted to Genzyme, a division of Sanofi SA.
The US Food and Drug Administration has approved two new bivalent vaccines to protect against Covid-19. They are to be administered as an updated single booster dose at least two months after primary or booster vaccination. The decisions were taken in response to the prevalence of the omicron variant of the SARS-CoV-2 virus and specifically to its two new subvariants BA.4 and BA.5.
The first cell-based gene therapy for the treatment of patients with beta thalassaemia, an inherited blood disorder, was approved by the US Food and Drug Administration on 17 August. Zynteglo (betibeglogene autotemcel) is a one-time treatment administered as an ex vivo lentiviral vector gene therapy. A functional beta globin gene is added into a patient’s cells outside of the body and then infused back into the patient. The treatment process is comprised of several steps and will be administered at qualified treatment centres in the US.
Enhertu (trastuzumab deruxtecan) has been approved by the US Food and Drug Administration to treat non-small cell lung cancer whose tumours have activating HER2 mutations. The accelerated approval comes just a week after Enhertu, an antibody-drug conjugate (ADC), was authorised by the FDA for a new breast cancer indication. The decision, announced on 12 August, brings the total number of US regulatory approvals for the drug up to five, all of which are for cancers expressing the HER2 protein.
The US Food and Drug Administration has extended a clinical hold on a proposed Phase 3 clinical trial of a microbiome-based therapeutic developed by MaaT Pharma SA of France because it requires more information about the composition of the faecal-derived product. MaaT Pharma announced the clinical hold extension on 10 August.
The US Food and Drug Administration has approved Enhertu (trastuzumab deruxtecan) for a new breast cancer indication while at the same time reclassifying a breast cancer subtype uniquely treated by the drug. Enhertu is an antibody-drug conjugate that combines a monoclonal antibody conjugated to the chemotherapy deruxtecan. The drug binds to a receptor on cancer cells after which it releases the antibody and toxin into cells to kill the cancer. Enhertu is being developed and commercialised by UK-based AstraZeneca Plc and Daiichi Sankyo Co Ltd of Japan.