Pharmaceutical Technology Editors

Global contract development and manufacturing organization (CDMO), ten23 health, has acquired filling technology and drug product manufacturing expert, swissfillon, it was announced in an Oct. 25, 2021 press release.

The acquisition will enhance ten23 health’s integrated offering of sterile drug product development and manufacturing of biologics, challenging molecules, and dosage forms. The swissfillon brand will continue to be used for operations.

“We look forward to joining ten23 health’s world-class team. There is a great strategic fit between our profound expertise in sterile drug product manufacturing for complex pharmaceuticals and ten23 health’s focus on the development and manufacturing of injectable treatments,” said Daniel Kehl, current CEO of swissfillon, in the press release. “By pooling our profound expertise, we will be able to further expand our market position for injectable treatments. The swissfillon team looks forward to continue advancing under the ten23 health umbrella and providing our combined group of customers with excellent support and customizable solutions.”

“Sterile fill/finish services are expected to experience significant growth over the coming years. The rising demand is driven by expanded drug development pipelines, incorporating more complex, large-molecule products and therapies that require specific expertise for both development and sterile production,” added Hanns-Christian Mahler, CEO of ten23 health, in the press release. “This is precisely why we expect ten23 health´s services, now including swissfillon, to be in great demand. At ten23 health, we are very pleased that we can now offer this sought-after know-how to biotech startups and pharmaceutical companies from one single source.”

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Global CDMO, ten23 health, has acquired filling technology and drug product manufacturing expert, swissfillon, enhancing its integrated offering.

ten23 health Acquires swissfillon to Enhance its Integrated Offering

The National Institutes of Health (NIH) announced the formation of the Bespoke Gene Therapy Consortium (BGTC) on Oct. 27th, 2021. In partnership with FDA, ten pharmaceutical companies, and five non-profits, this coalition is aimed at accelerating development of gene therapies for rare diseases.

According to a NIH press release, while there are over 7000 rare diseases (diseases which affect less than 200,000 people) that affect as many as 30 million Americans, only two heritable diseases have FDA-approved gene therapies. BGTC intends to donate approximately $76 million over the course of five years to support various projects aimed at providing treatments for these underserved populations.

BGTC plans to support between four and six clinical trials that are each focused on a different rare disease. To qualify, the condition must be a rare, single-gene disease with no gene therapies or commercial programs in development. It must also have substantial groundwork in place such that preclinical and clinical studies can be started quickly.

“Rare diseases affect 25 to 30 million Americans, but because any given rare disorder affects so few patients, companies often are reluctant or unable to invest the years of research and millions of dollars necessary to develop, test and bring individualized gene therapy treatments for a single disease to market,” said Joni L. Rutter, acting director of NIH’s National Center for Advancing Translational Sciences, in the press release. “The BGTC aims to make it easier, faster and less expensive to pursue bespoke gene therapies in order to incentivize more companies to invest in this space and bring treatments to patients.”

“Most rare diseases are caused by a defect in a single gene that could potentially be targeted with a customized or ‘bespoke’ therapy that corrects or replaces the defective gene,” said NIH director Francis S. Collins in the press release. “There are now significant opportunities to improve the complex development process for gene therapies that would accelerate scientific progress and, most importantly, provide benefit to patients by increasing the number of effective gene therapies.”

The National Institutes of Health (NIH), FDA, and fifteen private organizations are partnering together to form the Bespoke Gene Therapy Consortium.

NIH and FDA Lead Coalition to Accelerate Gene Therapy Development for Rare Diseases

The second part of the CPhI Annual Report, released on Oct. 25, 2021, has predicted an accelerated rate of change in pharma manufacturing and supply over the coming five years, with adoption of automated cobots, artificial intelligence (AI), real-time-release, and rapid growth in areas such as messenger RNA vaccines.

Released ahead of the 2021 event, which is being held as a hybrid event with the in-person show taking place in Milan, Italy between Nov. 9–11, 2021, the report predicts that China and the United States will lead innovation by 2030, followed by European nations and Japan. Emerging economies are expected to be nurturing new R&D centers in the near future.

“Our experts suggest that the rate of change in pharma manufacturing and supply is likely to accelerate over the next five-years and we will see adoption that might have otherwise taken 10-years to achieve [pre-pandemic],” said Orhan Caglayan, brand director at CPhI Worldwide, in a press release. “Pharma is seeking new partners, ingredients providers, and contracts services organizations—with the supply chain competing to align not only with the new therapies coming through discovery, but also with next-generation technologies that will enable better and potentially cheaper medicines.”

One of the experts who provided insights for the report, Bikash Chatterjee, CEO at Pharmatech Associates (a USP Company), suggests that manufacturing is now producing better-quality products at potentially lower costs thanks to the combined adoption of continuous processing, process analytical technology (PAT), and process analytical chemistry (PAC). Chatterjee also emphasizes in the report that the adoption of newer manufacturing approaches has been sped up by the macro changes that have happened over the past year along with efforts from governments to reshore manufacturing and secure supply chain resiliency for essential medicines.

“The integrity of the strategic national stockpile (SNS) will be a springboard for the development and refinement of advanced manufacturing technologies. For industry, the chief benefit from broader adoption of such approaches is to take advantage of lower cost of goods by reducing waste and the cost of poor quality (COPQ), and that in itself may increase the industry’s comfort with principles such as real time release (RTR),” said Chatterjee in the press release. “In a market likely to continue to encounter consistent downward pricing pressures, the adoption of these elements will allow manufacturers to lower their costs and still manage a profit as they provide improved, higher quality drug therapies to patients—this is a win-win in anyone’s book.”

Furthermore, Chatterjee anticipates that the industry is on the cusp of a standards revolution, with COPQ factoring in development and methodology, AI used in process selection, and new PAT and PAC standards likely being defined by regulatory bodies or the International Council for Harmonisation.

Aurelio Arias, engagement manager, Thought Leadership, IQVIA, who also provided expert insight for the CPhI report, reveals that therapeutic indications in the pipeline will also change by 2030, in addition to the adjustments expected in manufacturing processes. “By 2030 we expect the leading therapeutic indications accompanying oncology will be in crossover areas like inflammation and cancer vaccines. Growth potential will also be sourced from rare diseases and niche immunology areas. Some therapies will experience a revival, like cardiovascular, liver disease, and central nervous system—with or without Alzheimer’s—already showing huge potential to be treated using digital therapeutics,” Arias said in the press release. “Significantly, the modalities in which we can tackle these disease areas will increase with the development of platform technologies, more specifically in RNA, Cell and Gene, Digital and Microbiome.”

Both experts highlighted increased digital innovation in the supply chain, which can transform manufacturing efficiencies and improve patient adherence. However, a challenge in the form of regulations may be witnessed, as regulators may not be able to keep pace with the speed of change, which could hinder innovation.

Moreover, the innovative manufacturing approaches are enabling more personalization of medicine at scale, which the experts specify will mean that pharma companies need to rebalance book and divert costs from traditional engagements to leaner, smarter ways of operating. “The top pharma companies in 2030 will be those that can navigate this sea change by ruthlessly creating a path of prioritizing investments in promising countries and disease areas,” added Arias in the press release.

The full report is available to those attending CPhI Worldwide.

The second part of the CPhI Annual Report has predicted an accelerated rate of change in pharma manufacturing and supply over the coming five years.

CPhI Report Predicts Accelerated Rate of Change in Pharma Manufacturing and Supply Over Next Five Years

The Medicines and Healthcare products Regulatory Agency (MHRA) has granted a license to Gedeon Richter for Ryeqo (relugolix 40 mg, estradiol 1 mg, and norethisterone acetate 0.5 mg) for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age.

MHRA’s decision was based on the outcomes from a Phase III clinical program, the data for which have been published in the 

 With this decision, Ryeqo (relugolix combination therapy) becomes the first oral gonadotropin-releasing hormone (GnRH) receptor antagonist to be licensed in Great Britain for the specified patient population.

“Over a quarter of women of reproductive age develop uterine fibroids, a chronic condition that can cause debilitating symptoms and significantly impact quality of life,” said David Jordan, medical director United Kingdom and Ireland, Gedeon Richter, in an Oct. 26, 2021 press release. “Currently in Great Britain, there are limited therapies available for use, and many women have to decide whether to undergo surgery to alleviate their symptoms. The MHRA’s decision represents a significant milestone. We have now moved a step closer to providing an important new treatment option which has been shown to be effective in treating moderate to severe symptoms of uterine fibroids, such as heavy menstrual bleeding and pain, while offering eligible women a once-daily tablet.”

Gedeon Richter has started discussions with the National Institute of Health and Care Excellence and the Scottish Medicines Consortium about the potential availability of the combination therapy. Results of these discussions are expected to be published in 2022.

“The granting of a license for relugolix combination therapy is fantastic news and we are delighted that the value of this non-invasive treatment has been recognised,” added Tamas Neubauer, managing director UK and Ireland, Gedeon Richter, in the press release. “We want to redefine care for women living with uterine fibroids and we are engaging with the relevant health authorities throughout Great Britain with a view to securing NHS reimbursement as soon as possible. Our hope is that women suffering from symptoms with this common condition may benefit from greater treatment choice in the future.”

MHRA has granted a license to Gedeon Richter for Ryeqo to treat moderate to severe symptoms of uterine fibroids in adult women of reproductive age.

MHRA Grants License to Gedeon Richter for Combination Therapy to Treat Uterine Fibroids

An agreement has been reached between Ajinomoto and its subsidiary company, Ajinomoto Genexine, and Insilico Biotechnology aimed at accelerating and improving process development and production of biologics using digital twin technology.

According to an Oct. 26, 2021 press release, the companies will employ Insilico’s digital twins technology and Ajinomoto’s cell-culture media platform to speed up and improve the development of cell culture processes. Through the joint offering, Ajinomoto Genexine expects to meet the rising demand for cell culture media in the Asia Pacific region (APAC).

“We have found a wonderful partner in Ajinomoto. Their strength is in developing and producing quality media quickly, whereas our strength lies in fast and superior optimization of media compositions including the feeding strategy,” said Klaus Mauch, CEO of Insilico, in the press release. “Together, we can offer rapid process development to existing and new customers.”

“Biomanufacturing in the APAC region has unique needs. Aggressive timelines are one aspect,” added Yuta Nakai, president of Ajinomoto Genexine, in the press release. “With Insilico’s Digital Twins plugged into our high-quality media development technology, we aim to shorten timelines by up to 50%. We will continue to fine tune our offerings based on our customers’ voice.”

“This collaboration excites me because it makes digitalization accessible to a larger number of customers in the APAC region,” said Shilpa Nargund, director of Insilico’s APAC operations, in the press release. “The Digital Twins ability to virtually screen media together with Ajinomoto’s quick turnaround on media production give customers the option to test tailored media for their cell lines and clones.”

Ajinomoto and its subsidiary company, Ajinomoto Genexine, have partnered with Insilico Biotechnology to accelerate and improve process development and production of biologics using digital twin technology.



Partnership Aims to Accelerate and Improve Smart Bioprocess Development Solutions

Pfizer announced that the FDA Vaccines and Related Biological Products Advisory (VRBPAC) Committee voted to recommend that FDA grant Emergency Use Authorization (EUA) for the Pfizer-BioNTech COVID-19 vaccine in children aged 5 to 11 years old. The committee voted 17 to 0, with one member abstaining.

randomized, controlled trial that included approximately 4500 children aged 5 years to less than 11 years. According to a Pfizer press release, the vaccine efficacy rate was 90.7% in participants who were given a two-dose regimen of 10-µg of the Pfizer-BioNTech vaccine administered 21 days apart (one-third of the dosage given to individuals aged 12 years and older).

If approved, this would make the Pfizer-BioNTech vaccine the first available for the 5 years to under 12 years age demographic. FDA is expected to make a decision on the EUA in the coming days.

“We appreciated the opportunity to present our clinical data demonstrating the safety and high efficacy of our COVID-19 vaccine in children 5 to under 12 years of age,” said Kathrin U. Jansen, senior vice-president and head of Vaccine Research & Development, Pfizer, in the press release. “COVID-19 is an ongoing threat for the more than 28 million young children in this age group in the US, as they remain at risk for this infection. About 10% of all weekly US cases occur in children 5 to under 12 years of age with a potential risk of complications. In addition, immunizing children will help to get us closer to herd immunity, with the potential to stem the pandemic sooner. We thank the FDA advisory committee for their review and positive recommendation in support of [EUA] to help protect this young population.”

“We are committed to support the ongoing efforts to reduce infections and COVID-19 cases around the world by expanding the population of people protected against COVID-19,” said Özlem Türeci, co-founder and chief medical officer of BioNTech, in the press release. “The clinical data reviewed underline that our vaccine induces a strong immune response in children when Delta was the prevalent strain and thus may contribute to help address this public health crisis.”

The panel voted 17 to 0 to recommend that FDA grant Emergency Use Authorization for Pfizer-BioNTech’s COVID-19 vaccine in children aged 5 to 11 years old.