Pharmaceutical Technology Editors

Genentech, a member of the Roche Group, announced on Oct. 22nd, 2021, that FDA had approved Susvimo (100 mg/mL ranibizumab injection) for the treatment of wet, or neovascular, age-related macular degeneration (AMD), a condition that can lead to rapid and severe loss of vision. Susvimo is approved for intravitreal use via ocular implant for patients who have previously responded to at least two anti-vascular endothelial growth factor (VEGF) injections.

According to a company press release, Susvimo is the first and only FDA-approved treatment for wet AMD that offers as few as two treatments per year. Patients with wet AMD may need anti-VEGF injections as often as once a month, but Genentech’s treatment delivers ranibizumab continuously following a one-time implant that is refilled approximately every six months.

This approval was made following results from the Archway study, a randomized, multicenter, open-label Phase III study that evaluated Susvimo relative to monthly ranibizumab injections. It measured best-corrected visual acuity scores (the best distance vision a person can achieve when reading letters on an eye chart) following 36–40 weeks of treatment. Per pre-specified study criteria, Susvimo was found to be non-inferior to monthly injections; patients who were given monthly injections gained 0.5 letters on average, while those who received injections via Susvimo gained 0.2 letters.

“Susvimo represents a major advancement in the treatment of retinal disease and is an important new option for patients with wet AMD,” said Carl Regillo, chief of retina service at Wills Eye Hospital in Philadelphia and an Archway study investigator, in the press release. “With Susvimo, my patients now have an option that can help them maintain their vision as well as anti-VEGF injections, but on a more manageable twice-yearly treatment schedule.”

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Genentech’s Susvimo (ranibizumab injection) is approved for intravitreal use via ocular implant for the treatment of neovascular age-related macular degeneration.

FDA Approves Genentech’s Susvimo for Wet AMD

Becton, Dickinson, and Company (BD)’s BD SCF PremiumCoat Plunger Stopperade in partnership with Aptar Pharma, has been designed to support the injection of biologics into subcutaneous tissue with a -mL pre-filled syringe. BD and Aptar Pharma introduced the new stopper in October 2021.

The plunger stopper uses Aptar Pharma’s rubber formulation and film technology that is designed to limit drug component interactions and ensure drug integrity. According to an Oct. 13, 2021 BD press release, the product is designed to deliver improved functional performance, such as reduced glide force and glide force variability, in combination products. Quality of the stopper is verified in part through BD Viosioguard 100% camera inspection, which aims to ensure the product meets customer specifications and reduce the risk of foreign matter contamination.

“Biologics represent a critical and fast-growing segment of the drug market in the fight against serious diseases,” said Eric Borin, worldwide president of BD Medical-Pharmaceutical Systems, in the press release. “Enhanced biologic combination products are needed to ensure that patients and their caregivers can administer these drugs from the comfort of their homes. This partnership with Aptar Pharma will help deliver on that need.”

“The launch of BD SC PremiumCoat Plunger Stopper, merging BD and Aptar Pharma’s expertise, brings a new generation of coated plunger stopper solutions to the market to support the protection of sensitive drugs by reducing the quantity and species of probable leachables,” said Gabriel Zenker, president, Aptar Pharma Injectables, in the press release. “In partnering with BD, we look forward to furthering our support of biotech companies in their efforts to provide safe and easy-to-use drug delivery systems, while we continue to invest in new technologies to address the evolving needs of [the] market.”

Becton, Dickinson, and Company and partner Aptar’s new plunger stopper will support the injection of biologics into subcutaneous tissue.

BD Launches Plunger Stopper in Partnership with Aptar Pharma

Bio-Rad Laboratories, a life science research and clinical diagnostics product provider, announced the launch of the CFX Opus 96 Dx System and CFX Opus 384 Dx System on Oct. 20, 2021. These instruments provide real-time polymerase chain reaction (PCR) detection for 

According to a company press release, the Opus 96 and OIpus 384 offer accurate and precise quantification to improve assay development and workflow efficiencies and productivity for diagnostic testing and research. Additionally, both are open systems that can multiplex up to five targets to provide IVD assay development and testing.

"We continue to see an increasing demand for real-time PCR systems that offer more efficient workflows and improved data management and analysis that meets regulatory requirements in IVD assay development and testing, and we're pleased to introduce the [CFX] Opus Dx Detection Systems to meet these needs," said Steven Blakely, director for Gene Expression and Software Technology, Bio-Rad, in the press release. "Both systems are robust and secure platforms that leverage Bio-Rad's long-standing expertise in genomic tools to better serve both research and clinical diagnostic markets," he added.

Both products have been listed with FDA for IVD testing and meets the requirement for IVD use in Europe. They are also registered with the regulatory bodies of Singapore and Canada.

Bio-Rad will be launching two new real-time PCR detection tools to be used for in-vitro diagnostic testing.

Bio-Rad Launches Real-Time PCR Detection Systems for In-Vitro Diagnostics

MilliporeSigma’s newly launched Millicell DCI Digital Cell Imager instrument helps improve cell culture productivity in the lab by assessing a broad range of parameters, including confluency, morphology, and cell growth trends, which provides a faster and more accurate analysis with reduced user bias, according to a company press release.

Millicell DCI, launched in October 2021, has an intuitive, built-in touchpad display that enables more efficient execution of repetitive daily laboratory techniques associated with cell passaging, screening, or manufacturing of cell-based products. The Millicell Cloud software operates on an off-device subscription and provides automated data analysis. The software also allows for access to the data from remote desktops or mobile devices. The remote feature saves on costs and supports data redundancy for backup as well as protection by automatically sorting and storing data and images. With this instant access to historical data, researchers can track cell culture trends and create consistent cell cultures.

The instrument also removes the requirement for manual manipulation of the cell culture flask, which can result in reduced contamination risk and improved flexibility users. Evaluation is also possible for a wide variety of cell cultures, including adherent cells and cell lines, spheroids, organoids. In addition, the instrument facilitates hemocytometer or in-vessel measurement.

MilliporeSigma’s newly launched Millicell DCI Digital Cell Imager quickly and objectively assesses common cell culture parameters and growth trends for more consistent cell cultures.

MilliporeSigma’s Millicell DCI Digital Cell Imager Offers Quick, Accurate, and Objective Cell Monitoring

Otsuka Pharmaceutical Europe has announced, in an Oct. 29, 2021 press release, that Otsuka Pharmaceutical Netherlands has submitted an initial marketing authorization application to the European Medicines Agency (EMA) for vadadustat.

Vadadustat is an investigational oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor aimed at the treatment of anemia associated with chronic kidney disease (CKD) in adults. The therapy was developed under a collaboration and license agreement with Akebia Therapeutics.

“We are excited to reach this regulatory milestone in collaboration with Akebia, demonstrating our continued commitment to patients with chronic kidney disease” said Andy Hodge, CEO of Otsuka Pharmaceutical Europe Ltd. and Otsuka Pharmaceutical Development and Commercialization Europe GmbH, in the press release.

Otsuka has submitted an initial marketing authorization application to EMA for vadadustat as a treatment of anemia associated with CKD in adults.

Otsuka Applies to EMA for Marketing Authorization of Vadadustat

Vertex Pharmaceuticals and Mammoth Biosciences announced a partnership on Oct. 26, 2021, to develop 

 gene-editing therapies. In a deal that could be worth as much as $691 million, Vertex will use Mammoth’s ultra-small clustered regularly interspaced short palindromic repeats (CRISPR) systems to discover and develop novel 

 gene-editing therapies for two genetic diseases.

Mammoth’s CRISPR platform consists of various novel, ultracompact Cas enzymes, including Cas12 and Casɸ. Because of their small size, they have the potential to increase the scope of 

 gene-editing by facilitating advanced delivery techniques.

Vertex has done significant work with cystic fibrosis in the past, which has led to multiple approved medications. Their current pipeline features small molecule medicines for alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. Additionally, the pipeline also has cell and genetic therapies for sickle cell disease, beta thalassemia, Duchenne muscular dystrophy, and type 1 diabetes mellitus.

“We believe our novel ultra-small CRISPR systems have the potential to be game changers when it comes to systemic and targeted delivery of in vivo gene-editing therapies,” said Peter Nell, chief business officer and head of Therapeutic Strategy at Mammoth Biosciences, in a Vertex press release. “The combination of Mammoth’s unique technology with Vertex’s unmatched experience in serious disease research and development will only accelerate programs with the goal of reaching patients with high unmet medical need.”

“Vertex and Mammoth share the same commitment to developing therapies that have the potential to be transformative for people with serious diseases,” said David Altshuler, chief scientific office of Vertex Pharmaceuticals, in the press release. “We look forward to expanding our cell and genetic therapies capabilities with the addition of Mammoth’s ultra-small CRISPR systems for in vivo genome editing, which will provide us with another set of tools to tackle many of the diseases we’re interested in.”

The $691 million deal will see Vertex use Mammoth’s systems to discover and develop in-vivo gene-editing therapies.