Tower Mains Weekly Newsletter
Regulatory Updates
MHRA guidance update: Exceptional use of non-UKCA marked medical devices
On 30th December, the Medicines and Healthcare products Regulatory Agency (MHRA) updated the application form in its guidance on how a manufacturer can apply for approval to supply a non-compliant medical device on humanitarian grounds.
FDA issues final guidance on the format and content of a REMS Document
On 4th January, the US Food and Drug Administration (FDA) issued final guidance outlining updated recommendations for the format and content of a risk evaluation and mitigation strategy (REMS) document for a prescription drug product, including a biological drug product. The agency also issued final guidance detailing updated information on the format of a REMS document.
Industry Updates
Johnson & Johnson acquires Abiomed
On 22nd December, Johnson & Johnson announced its acquisition of Abiomed, Inc., broadening its MedTech portfolio in the high-growth cardiovascular markets.
FDA approves supplemental application for Plan B One-Step
On 23rd December, the FDA approved a supplemental application for Plan B One-Step, an over-the-counter emergency contraceptive. The FDA’s approval of this application removes some of the information on the mechanism of action from the Drug Facts label and updates the mechanism of action information in the Consumer Information Leaflet for Plan B One-Step to reflect the best available science.
Pfizer announces positive results from Phase 3 Study of Hemophilia B gene therapy candidate
On 29th December, Pfizer Inc. announced positive results from the Phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec, an investigational gene therapy containing a bio-engineered adeno-associated virus (AAV) capsid and a high-activity human coagulation FIX gene, for the treatment of adult males with moderately severe to severe hemophilia B. The goal of this gene therapy for those living with hemophilia B, once treated, is that they will be able to produce FIX via this one-time treatment rather than having to regularly receive exogenous FIX. The therapy has been granted breakthrough, regenerative medicines advance therapy (RMAT), and orphan drug designations from the FDA, as well as PRIority MEdicines (PRIME) and orphan drug designation from the European Medicines Agency.
Valneva reports additional booster data for its inactivated COVID-19 vaccine
On 30th December, Valneva SE reported additional heterologous booster data from an exploratory, small clinical study for its inactivated COVID-19 vaccine candidate, VLA2001. The data demonstrated that a booster dose of VLA2001 was well tolerated in previously Pfizer/BioNTech or Moderna-vaccinated participants and duly confirmed the favourable safety profile of VLA2001 across all studies – including in homologous or heterologous booster situations. The company is now seeking regulatory approval for VLA2001 as a homologous booster as well as a heterologous booster in AstraZeneca-primed individuals.
FDA approves treatment for adult patients with relapsing forms of multiple sclerosis
On 4th January, the FDA approved the Briumvi injection for treating patients with relapsing forms of multiple sclerosis (RMS) in adults. Researchers demonstrated Briumvi’s efficacy in two randomised, double-blind, double-dummy, parallel-group, active comparator-controlled clinical trials of identical design, in patients with RMS treated for 96 weeks. Patients were randomised to receive either Briumvi or teriflunomide, the active comparator. The primary outcome of both studies was the annualised relapse rate (ARR) over the treatment period, and in both studies, Briumvi significantly lowered the ARR compared to teriflunomide.
Nirsevimab’s regulatory submission accepted by FDA for prevention of RSV lower respiratory tract disease in infants
On 5th January, AstraZeneca announced its Biologics License Application (BLA) for nirsevimab has been accepted for review by the FDA for the prevention of respiratory syncytial virus (RSV) lower respiratory tract disease in newborns and infants entering or during their first RSV season, and for children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season. If approved, it would be the first single-dose RSV preventative option for the newborn and infant population in the US.
Issue Number: WN00093
Would you like a personal copy of the Tower Mains Weekly Newsletter directly to your mailbox each month? Just send an email to info@towermains.com with the subject “Weekly Newsletter Please”.