Having a clear clinical strategy early on can shave time off overall development projects.

Early Strategizing Tightens Development Timelines

With recent approvals of bispecific antibodies, these complex molecules are fast moving out of the research box and into clinical pipelines.

Bispecific Antibodies are Moving from Research to Clinical Development

Extensive work has greatly expanded the possibilities for fetal bovine serum sourcing.

Considerations for US Fetal Bovine Serum Sourcing

Thanks to technological advances, process chromatography challenges are surmountable, but increasingly complex therapies in the pipeline will demand more efficient processes in the future.

Advancing Process Chromatography in DSP

The complex nature of biologics creates challenges for GMPs in sterile manufacturing, and the EU’s Annex 1 is making an impact.

GMPs for Sterile Manufacturing of Biologics

Precompetitive consortiums seek solutions to industry-wide challenges.

Coming Together to Enable Cell and Gene Therapy Manufacturing

When considering whether to outsource work to a CRO, there are a number of factors to assess, including the type of work that may be outsourced, regulatory considerations, and needs for the study.

When Is It Appropriate to Outsource Bioanalysis Work to a CRO?

Novartis has endorsed the Kigali Declaration on neglected tropical diseases (NTDs) and is committing to an investment of $250 million towards research and development (R&D) to fight against NTDs and malaria.

The Kigali Declaration was adopted to reaffirm commitments to end NTDs and malaria and aims to mobilize political will and secure commitments to achieve the SDG3 target on NTDs and to deliver the targets set out in the World Health Organization’s Neglected Tropical Disease Roadmap (2021–2030).

Novartis’ investment will span over five years (2021–2025) and will fund the advancement of R&D of new treatments to combat NTDs and malaria. $100 million will go towards R&D of its neglected tropical disease program, and $150 million will go to the advancement of its 3 novel drug candidates to combat malaria.

“Over the past decade, great progress has been made against NTDs, but there is still a lot more work to be done. Novartis will continue progressing our longstanding commitment to helping realize a world free of NTDs,” said Vas Narasimhan, CEO of Novartis, in a press release. “Today, by endorsing the Kigali Declaration and pledging to invest USD 250 million, we aim to accelerate progress toward elimination of these diseases, which continue to cause suffering and stigma for millions of people around the globe.”

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Novartis will endorse the Kigali Declaration on neglected tropical diseases and invest $250 million for R&D. 

Novartis to Invest $250 Million for R&D of Neglected Tropical Diseases

GlaxoSmithKline (GSK) announced on June 23, 2022 an investment of £1 billion (approximately US$1.2 billion) over 10 years to accelerate research and development (R&D) toward infectious diseases that disproportionately impact lower-income countries. The focus of the investment will be on malaria, tuberculosis, HIV, neglected tropical diseases, and anti-microbial resistance, which account for more than 60% of the disease burden in many lower-income countries.

GSK Global Health R&D Hubs are progressing more than 30 potential new vaccines and medicines that will target 13 high-burden infectious diseases. The commitment supports the company’s goal to positively impact the health of more than 2.5 billion people in the next 10 years.

“I am delighted to renew our commitment to global health research for the coming decade, consistent with our purpose to unite our science, technology, and talent to get ahead of disease together and our ambition to deliver health impact at scale,” said Thomas Breuer, chief global health officer at GSK, in a press release. “Through our focus on scientific innovation in Global Health, we have delivered the first malaria vaccine, RTS,S, the first radical cure of vivax malaria, tafenoquine, and a new tuberculosis vaccine candidate. GSK now has more than 30 potential new vaccines and medicines (including pre-clinical assets) in 13 high-burden infectious diseases to accelerate, and we must work collectively with urgency to bring these potentially life-saving innovations to people that need them.”

GSK will invest £1 billion in furthering R&D in malaria, tuberculosis, and HIV, as well as neglected tropical diseases.

GSK Announces £1 Billion R&D Investment in Fighting Infectious Diseases in Lower-Income Countries

Kindeva Drug Delivery, a contract manufacturing and development organization (CDMO), completed its acquisition of iPharma Labs, a CDMO focused on inhaled medicines, on June 16, 2022. The entire team at iPharma has joined Kindeva and will continue to operate at their existing location.

iPharma specializes in inhalation formulation and the development of liquid, dry powder, and propellant-based therapies. According to a company press release, iPharma has worked with dry powder inhalation, soft mist inhalers, and nebulizers for both small and large molecules, including biologics, in the early stages of drug development.

“We’re pleased to offer this combination to our customers and their patients: a complete portfolio of respiratory delivery expertise all in one global team,” said Arron Mann, CEO, Kindeva, in the press release. “[The team at iPharma] will help Kindeva accelerate our one-stop CDMO approach: addressing our customers’ desire for a single partner to support feasibility through commercialization without lengthy and risky hand-offs.”

“Joining Kindeva strengthens our ability to craft the best product development strategy for delivery to the patient to and through the lungs,” said Keith Ung, CEO and co-founder, iPharma, in the press release. “Our customers are already excited to benefit from a more complete product development and manufacturing offering. I look forward to leading efforts at Kindeva to expand our clinical and commercial support for dry powder and nebulized respiratory therapies.”

Kindeva’s acquisition of iPharma will increase the company’s inhalation formulation outsourcing capabilities.

Kindeva Drug Delivery Completes Acquisition of iPharma Labs

Aizon, an artificial intelligence and advanced analytics platform company, announced the launch of its good practice compliant data processing product, Aizon Digitize (AD), on June 21, 2022. AD converts data from paper to data with the goal of increasing productivity and avoiding time-consuming, error-prone manual data inputs.

According to a company press release, the product automatically uploads paper records and commonly used file formations to begin the creation of an audit trail; it then immediately enables tracking of changes to the data. It is designed for integration with electronic batch records, laboratory information management systems, quality management systems, and manufacturing execution systems.

“Many pharmaceutical manufacturers still rely on manual entries and processes to perform critical tasks. This very common approach exponentially increases the risk of introducing errors and hindering their ability to optimize the entire development and manufacturing process,” said John Vitalie, CEO, Aizon, in the press release. “[AD] helps [users] avoid unnecessary manual entry and transfer of information which can create volumes of erroneous data resulting in increasing levels of risk. By shifting the manual workload to an automated digital process, our customers can leverage all of the valuable data trapped in paper-based records and spreadsheets. This is the on-ramp to realizing the benefits that compliant Pharma 4.0 technology can bring to any biopharma manufacturing operation.”

Aizon Digitize is designed to help life science manufacturers transition data from paper to digital.

Aizon Launches GxP Compliant Data Processing Solution

 Washington Editor, jillwechsler7@gmail.com.

FDA is at a “pivotal moment” for the nation’s public health and for global well-being due to the potential for realizing important gains in medical treatment based on science, 

Commissioner Robert Califf told the Washington legal and regulatory community in June 2022 at the annual meeting of the Food and Drug Law Institute 

(FDLI). He aims to address these challenges by improving FDA supply chain management in all areas, with digitally integrated systems that support preemptive actions and permit swift reaction to product shortages. Another priority is to counter widespread misinformation about vaccines and medical products through an emphasis on science-based decision making at the agency and to advance medical product development based on appropriate communication between agency experts and those in regulated industry.

Califf emphasized the importance of improved information and data management systems in all FDA operations to support agency oversight and decisions about medical therapies. A priority is to FDA’s foods program, which will require sufficient funds from Congress to support something like a “21st century cures” initiative for food similar to that enacted to modernize and enhance the agency’s oversight of medical products in 2016. Under the current system, Califf noted that food producers don’t even have to tell FDA if they find contaminants in products awaiting shipment, limiting the agency’s ability to anticipate serious supply problems, such as the ongoing infant formula crisis.

At the same time, Califf defended the agency’s “appropriate” engagement with regulated companies as valuable for evaluating “the promise and risk” of new products and technologies. The broad knowledge of FDA staff about the full range of products in development—including many failures—can help guide developers to efficient pathways and prevent wasted and duplicative efforts—including clinical trials that could harm human subjects.

Similarly, Principal Deputy Commissioner Janet Woodcock urged a better understanding of the challenges and difficulties surrounding the many regulatory and scientific decisions made by FDA staff and leaders in another address at FDLI, which acknowledged the agency’s serious error in approving long-release opioids for market. Woodcock noted that a science-based regulatory process, as at FDA, must issue regulations and policies that implement laws, a process that involves interpretation and inevitably generates dissent. Although science aims to assess the facts, many decisions are “surrounded by a cloud of uncertainty,” she said. Consequently, FDA’s critical approval of long-release opioids in 1995 was based on projections of harm and prescribing behavior presented at the time that were way off, Woodcock conceded. However, she acknowledged being pessimistic about this situation improving in the near future unless scientists and policy makers are able to communicate more broadly about the “big picture” of the uncertainty that inevitably surrounds agency decisions.

Califf raised similar concerns about “the growing dissemination of misinformation and disinformation about science, medicine, and the FDA which is putting patients and consumers at risk” at the FDLI conference and also at the BIO International Convention in San Diego. Misinformation, he told biotech leaders, is the leading cause of death in the United States, noting that almost all COVID-19 deaths now are preventable if patients become fully vaccinated and boosted. Misinformation, moreover, hampers the appropriate use of effective treatments for chronic diseases. It is paradoxical, Califf noted at BIO, that even though America is “innovating for the world,” US life expectancy is low, especially compared to other wealthy nations.

Califf observed at FDLI that he agreed to return to FDA last year after a previous stint as the agency’s leader in hopes of helping to modernize agency operations to prevent future critical delays and product shortages. This is a “pivotal moment” for the agency’s future effectiveness, he said, emphasizing that changes are needed to “stay ahead of the industries we regulate and the technology, science, and communications those industries employ.”

FDA is at a pivotal moment because of important gains in medical treatment based on science.

FDA Leaders Seek to Build Public Trust in Science and Gain Support for Regulation

YewMaker and Connect in Pharma have announced that the winners of the new Sustainable Medicines Packaging Award will be announced at an in-person awards ceremony at the Connect in Pharma event in Geneva on Sept. 14, 2022.

The new awards are run by YewMaker, a sustainable healthcare company. The awards will highlight and celebrate sustainable innovations in pharmaceutical packaging under two categories with a gold, silver, and bronze award in each. The two categories will focus on design and processes that reduce the overall impact on the environment.

“Collaboration and innovation are essential to delivering the changes needed to make sustainability core to how pharmaceutical packaging is designed and used,” said Nazneen Rahman, founder and CEO of YewMaker, in a press release. “By announcing the winners of these new awards at the Connect in Pharma conference this September, we can bring a sharper focus on sustainability innovations in the pharmaceutical industry.”

“We are very excited to be partnering with YewMaker to host these important awards at Connect in Pharma in Geneva,” added Renan Joel, divisional director at Connect in Pharma, in the release. “Connect in Pharma is designed to focus on innovation and technology in the pharma and biotech community, and sustainable medicine packaging design is an important area of innovation in the sector. We are delighted to be coupling innovation with sustainability while putting the future of the planet in the spotlight.”

Connect in Pharma will host an awards ceremony to showcase innovations in sustainable pharmaceutical packaging. 

Sustainable Medicines Packaging Award Winners to be Announced at Connect in Pharma

The European Medicines Agency (EMA) has begun a rolling review of the Spikevax vaccine that has been adapted to protect against two variant strains of SARS-CoV-2, the agency announced on June 17, 2022. The review will focus on non-clinical laboratory study data as well as chemistry, manufacturing, and controls (CMC) data relating to the manufacturing of the vaccine. A rolling review allows the agency to begin assessing available data before marketing approval of the vaccine. The agency will review additional data from the sponsor company as it is received.

rolling review of an adapted Comirnaty COVID-19 vaccine 

on June 15, 2022. “The details about the adapted vaccine, for example whether it will specifically target one or more SARS-CoV-2 variants or subvariants, are not yet defined. However, EMA’s review will initially focus on CMC data for the component targeting Omicron subvariants,” the agency stated in a press release.

The agency will review laboratory study and CMC data on the bivalent vaccine that targets two strains of the SARS-CoV-2 virus.

EMA Starts Rolling Review of Adapted COVID-19 Vaccines

An International Coalition of Medicines Regulatory Authorities (ICMRA) workshop, held on May 20, 2022 and co-chaired by Health Canada and the European Medicines Agency (EMA), highlighted lessons learned during the COVID-19 pandemic about the international collaboration on observational research. According to EMA, real-world evidence gathered from observational research assists regulators to understand the safety and effectiveness of medicines used in clinical practice.

Results from three technical ICMRA working groups that focus on vaccines, pregnancy observational research, and building international cohorts of patients were discussed in the workshop. Workshop participants agreed these platforms allowed for timely sharing of data, expertise, and tools.

Experience learned during the performance of observational studies based on COVID-19 real-world data was shared by the participants. It was agreed upon that rapid generation of evidence gained from active interactions between regulators, researchers, and academia is crucial for regulatory decision making in times public health crises.

Participants in the workshop included representatives from the World Health Organization and more than 28 medicines regulatory authorities. The findings from the workshop can be found on the 

Lessons learned from the COVID-19 pandemic about the benefits of international collaboration were discussed in a May 2022 workshop.

International Collaboration on Pharmaceutical Regulations 

Avid Bioservices, a biologics-focused contract development and manufacturing organization, announced the opening of new analytical and process development (AD/PD) suites within their recently built 53,000-ft2 viral vector development and manufacturing facility. Build-out of the suites is currently ongoing and expected to be completed by mid-2023.

According to a June 14, 2022 press release, the new suites will be equipped with complete upstream, downstream, and analytical development capabilities. The suites will be able to support up to 500-L suspension culture in single-use bioreactors and various adherent cell modes. The company's viral vector AD/PD team has worked on technologies for viral vector production and purification processes focused on adeno-associated viruses (AAVs), lentiviruses, oncolytic viruses, and other viruses for gene therapy and vaccine applications.

“We believe that we are uniquely positioned to leverage our deep expertise in the manufacturing of traditional biologics to address the growing demand for high quality manufacturing of cell and gene therapies,” said Nick Green, president and CEO, Avid Bioservices, in the press release. “Today’s launch of our AD/PD labs is a crucial first step in our expansion into the viral vector space as we remain laser focused on smart and sustainable revenue growth for our business.”

Avid Bioservices has expanded its viral vector development and manufacturing facility with the addition of analytical and process development suites.

Avid Bioservices Launches New Analytical and Process Development Suites at Viral Vector Facility

Orbit Discovery, a biopharmaceutical company focused on discovering candidate peptide therapeutics, announced on June 13, 2022 that it had been awarded a National Science and Mathematics Access to Retain Talent (SMART) Grant by Innovate UK, the United Kingdom’s innovation agency. Totaling £472,000 ($498,000 USD), the grant is intended to facilitate the implementation of droplet-based microfluidics for cell-based functional screening and expand the capabilities and throughput of Orbit’s peptide display platform.

The goal of Orbit’s project is to advance the discovery of peptide therapeutic candidates by improving overall process efficiency and reducing risk and associated cost. In this vein, according to a company press release, the grant funds will go toward validating the microfluidic components of Orbit’s peptide discovery platform, a next-generation microfluidic-based functional screen designed to interrogate millions of peptides in cell-based assays.

The platform is being built to directly address difficult-to-access drug targets, in particular multi-membrane spanning proteins like G-protein-coupled receptors. It aims to rapidly identify molecules that generate a therapeutically relevant functional response earlier in the drug discovery process. This will avoid polluting screening outputs with non-functional binders, as is often observed with other platforms.

“This Innovate UK [SMART] Grant will help us further cement our technical leadership in functional screening,” said Anthony Pitt, chief technology officer, Orbit Discovery, in the press release. “The microfluidic platform can elevate our current functional screening capabilities from tens of thousands to millions of peptides. This throughput will help us identify rare functional binders that are typically missed with more traditional screening platforms.”

Orbit Discovery has been awarded the Innovate UK Grant to expand high throughput cell-based functional screening platform capabilities in peptide drug discovery.

Orbit Discovery Awarded Innovate UK Grant

FDA announced that it has issued an Emergency Use Authorization on June 17, 2022 for both the Moderna and Pfizer-BioNTech COVID-19 vaccines in children as young as six months old. The decision was made following an evaluation from FDA’s independent Vaccines and Related Biological Products Advisory Committee, who voted in support of the authorization.

“Many parents, caregivers and clinicians have been waiting for a vaccine for younger children and this action will help protect those down to [six] months of age. As we have seen with older age groups, we expect that the vaccines for younger children will provide protection from the most severe outcomes of COVID-19, such as hospitalization and death,” said Robert Califf, FDA commissioner, in an agency press release. “Those trusted with the care of children can have confidence in the safety and effectiveness of these COVID-19 vaccines and can be assured that the agency was thorough in its evaluation of the data.”

Previously, the Moderna vaccine had only been authorized for adults 18 and older. An FDA analysis of clinical trial data from Moderna found that the vaccine was 50.6% effective in preventing COVID-19 in children aged six months to 23 months two months after their second dose, and 36.8% effective in children aged six months to five years old. Individuals in the age six to 11 and 12 to 17 were found to have immune responses comparable to adults.

The Pfizer-BioNTech vaccine had previously been authorized for individuals five and older. An FDA analysis of clinical trial data from Pfizer found that the immune responses in children five months through four years old was comparable to the immune responses among individuals aged 16 to 25 who received two higher doses of the vaccine.

“As with all vaccines for any population, when authorizing COVID-19 vaccines intended for pediatric age groups, [FDA] ensures that our evaluation and analysis of the data is rigorous and thorough,” said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research, in the release. “In addition to making certain the data for these vaccines met FDA’s rigorous standards, the agency’s convening of an advisory committee was part of a transparent process to help the public have a clear understanding of the safety and effectiveness data supporting the authorization of these two vaccines for pediatric populations.”

FDA has issued an Emergency Use Authorization for the Moderna and Pfizer-BioNTech COVID-19 vaccines in children as young as six months old.

FDA Authorizes COVID-19 Vaccines for Individuals Six Months and Older

Batavia Biosciences announced the expansion of its headquarters and R&D capacity in its Netherlands facility on June 14, 2022. The 22,500-ft² expansion will increase R&D activities for viral vector-based product development, including Lentivirus, adeno-associated virus (AAV), adenovirus, and measles virus vectors. The expansion is expected to be finalized by June 2023.

“The new laboratory floor space for R&D and analytical capabilities will aid the development capacity for customer projects,” said Chris Yallop, chief scientific officer, Batavia Biosciences, in a company press release. “The increased allocation for R&D capacity better positions Batavia Biosciences to serve as pacemakers to the community and to continue to bring promising candidate biopharmaceuticals from bench to clinic.”

According to a company press release, the increased R&D infrastructure is intended to meet increased global development for vaccine development, including live-attenuated, whole killed, vectored, or subunit vaccines.

Batavia Biosciences is expanding its headquarters and R&D capabilities in its Netherlands facility.