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.
Pharming Group NV is to get an accelerated review from the European Medicines Agency for leniolisib, its candidate treatment for a rare primary immunodeficiency known as activated phosphoinositide 3-kinase delta syndrome (APDS). A marketing authorisation application is expected to be submitted to the EMA in October, the company announced on 1 August.
The US Food and Drug Administration has launched a plan to support the development of new medicines for rare neurodegenerative diseases including amyotrophic lateral sclerosis, a progressive disease that affects nerves in the brain and spinal cord, causing loss of muscle control. The plan has been developed in line with new US legislation, the Accelerating Access to Critical Therapies for ALS Act that took effect on 23 December 2021.
The European Medicines Agency has recommended the conditional approval of Roctavian (valoctocogene roxaparvovec), a gene therapy for haemophilia A. If approved, it would be the first gene therapy for this disease in Europe. The conditional approval carries with it a requirement that the developer, BioMarin Pharmaceutical Inc, provide follow-up data from ongoing clinical trials to show the therapy continues to be safe and efficacious for patients.
As part of its strategy to incorporate big data into regulatory decision-making, the European Medicines Agency is to invite pharma companies to apply for a pilot project under which they would submit patient data from clinical trials as part of their marketing authorisation applications. The project is expected to start in September and last up to two years. It would cover about 10 regulatory procedures.