Process and plasmid design optimization, disposable equipment, and flexible platform processes all play important roles.

Achieving High Yields in Upstream Processing of Plasmids

Decreasing vein to vein time saves lives.

High-titer Vector Producing Cells

There is growing pressure for robust and economically scalable viral-vector manufacturing technologies.

Tactics and Strategies for Designing an Ideal Lentiviral Vector Platform

Shifts in pharmaceutical packaging have spurred tremendous growth in the pre-filled syringe fill/finish industry.

Automated Inspection of Pre-filled Syringes and Biologics During Fill/Finish

Considerable efforts have been made over the years to resolve the key issues of stability and delivery of RNA-based therapeutics.

The Marriage of RNA and Mass Spectrometry

The bio/pharma outsourcing industry continues to be integral in the development and manufacture of medicines.

Outsourcing Still Vital

The trends shaping the growth of the biologics outsourcing industry demand attention.

The Logistics of Outsourcing Biologics

Moderna announced on March 23, 2023 a collaboration with Generation Bio, a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, to combine Moderna’s biological and technical expertise with core technologies of Generation Bio’s non-viral genetic medicine platform. The collaboration aims to expand the application of each company’s technology by developing novel nucleic acid therapeutics, including those capable of reaching immune cells, to accelerate their respective pipelines of non-viral genetic medicines. Moderna will fund all research and development activities under the collaboration.

Under the agreement, Generation Bio will receive a $40 million upfront cash payment, a pre-payment of research funding, plus a $36 million equity investment from Moderna, with the potential for additional milestones, fees, and royalties. Moderna has also acquired an option to license Generation Bio’s cell-targeted lipid nanoparticle (ctLNP) and close-ended DNA (ceDNA) technology for two immune cell programs and two liver programs, with an additional option for a third immune cell or liver program.

"Moderna continues to invest in innovative technology to enable us to develop a breadth of transformative medicines for patients," said Rose Loughlin, Moderna's senior vice-president for research and early development, in a company press release. "Through this collaboration, which builds on Generation Bio's non-viral genetic medicines platform, we have the potential to target immune cells with diverse nucleic acid cargos and the liver for gene replacement. We are excited to have Generation Bio as our partner as we continue to broaden our therapeutic pipeline and extend the potential benefit of nucleic acid therapeutics to more patients."

"Non-viral DNA therapeutics may offer durable, redosable, titratable genetic medicines to patients suffering from rare and prevalent diseases on a global scale," said Phillip Samayoa, chief strategy officer of Generation Bio, in the release. "This collaboration represents a foundational investment in our platform science, both deepening our pipeline of rare and prevalent liver disease programs beyond hemophilia A and accelerating our work to reach outside of the liver with nucleic acid therapies. We are thrilled to collaborate with Moderna to extend genetic medicines to new tissues and cell types through the joint development of noveltargeting for our stealth ctLNPs to reach immune cells."

Articles

Moderna and Generation Bio have announced a strategic collaboration to develop non-viral genetic medicines. 

Moderna and Generation Bio Announce Strategic Collaboration

Curia announced on March 23, 2023 that it is collaborating with Corning to expand and accelerate continuous-flow development and manufacturing programs for the chemical and biopharmaceutical industries globally. The collaboration marks the first global installation of Corning’s G1 production system to support higher-quality API-chemical production using flow-chemistry technology.

Continuous flow chemistry is crucial for the development and manufacture of pharmaceutical intermediates and APIs and provides advantages compared with traditional batch processing. It can deliver a faster and more robust material production with a higher selectivity of desired products, according to a company press release. The collaboration will combine Curia’s expertise and global network of facilities with Corning’s continuous flow technology to help drive business efficacies as well as improve patients’ lives.

“Innovation in drug development and production calls for safety at high speed,” said Christopher Conway, president, R&D, Curia, in the release. “The implementation of Corning’s G1 Production Reactor at our Albany facility expands our capability to provide scalable solutions that address complex development and manufacturing requirements facing the pharmaceutical industry. Using advanced technology and standardized workflows, Curia offers continuous flow chemistry from targeted development to commercial scale globally.”

Curia is collaborating with Corning to advance biopharmaceutical continuous-flow development and manufacturing programs.

Curia and Corning Collaborate to Advance Biopharmaceutical Continuous-Flow Development

 Washington Editor, jillwechsler7@gmail.com.

The escalating attack on too-high prices for prescription medicines has heightened debate over the government’s authority to revoke licenses for medical products that are developed with support of public funds. The Department of Health and Human Services (HHS) and Department of Commerce (DOC) 

 a broad review of the “march-in” authority provided in the Bayh-Dole Act of 1980 in the wake of its controversial decision to let stand licensing rights to a widely used cancer drug.

The review will be conducted by a new Interagency Working Group for Bayh-Dole, which is charged with updating the framework for altering march-in provisions spelled out in that legislation. A main issue is whether a high price for a product provides sufficient reason to revoke a manufacturer’s license. HHS will hold a workshop later this year to discuss when it might be appropriate to implement march-in rights, with input from patient groups, industry, academic, nonprofit organizations, and legal and policy experts. And the discussion will be informed by a recent publication from National Institute of Standards and Technology (NIST) that proposes changes to streamline and clarify licensing procedures for federally funded inventions.

This issue hit the headlines recently when the National Institutes of Health (NIH)

 to initiate march-in proceedings for the prostate cancer drug Xtandi (enzalutamide), marketed by Pfizer and Astellas Pharmaceuticals. Public interest groups had filed a petition in 2021 that called on NIH to take ownership of the patents on the drug due to its much higher cost in the United States than in Canada and other countries, which limited patient access to the therapy.

At the same time, Congressional leaders highlighted the substantial NIH funding of Moderna’s COVID-19 vaccine in challenging the company’s plan to raise the price of the preventive when federal subsidies end in May. In testimony before the Senate Health, Education, Labor and Pensions (HELP) Committee on March 22, 2023, Moderna CEO Stephane Bancel 

emphasized the years of research and billions

 invested by his company to devise methods for quickly testing and producing such a vaccine. Committee chair Bernie Sanders (I-Vt) 

highlighted the $1.2 billion invested by the federal government

 in the project in blasting the company’s plan to boost the price from $26 to $130 per shot with the end of the COVID health emergency.

The Bayh-Dole Act of 1980 provides a pathway to facilitate commercialization of inventions, including biomedical products, that are developed with support from federal agencies such as NIH. The program permits the government to “march in” to rescind licensing rights if it determines that the benefits of such inventions are not available to the public on reasonable terms. Over the years, various organizations have petitioned NIH and other agencies to initiate march-in proceedings when a product’s high cost might be considered a barrier to access. But federal officials have rejected such pleas, as with several petitions related to Xtandi.

In rejecting the latest filing on Xtandi, NIH stated that the aim of Bayh-Dole is to promote commercialization and public availability of government-funded inventions, and not necessarily to ensure a low price for a covered medical product. NIH noted that Xtandi is widely available to patients, and that instituting march-in rights was unlikely to lower the price. Sen. Sanders and patient advocates clearly disagreed with the NIH decision, pointing out that Xtandi can be purchased in Canada for one-fifth the US price. But manufacturers and industry experts counter that without private sector investment and know-how, most government-funded research would never move from the laboratory to the marketplace—at any price.

A new Interagency Working Group for Bayh-Dole will review the government’s authority to revoke licenses for medical products.

Feds to Review March-In Authority on Drug Patents

FDA released the framework for the Use of Digital Health Technologies (DHTs) in Drug and Biological Product Development on March 23, 2023. The recent document will guide the use of DHT-derived data in regulatory decision-making for drugs and biological products.

engage stakeholders and include workshops and demonstration projects

establish internal processes to support DHT evaluation for use in drug development

promote shared learning and consistency for DHT-based policy, procedure, and analytic tool development

facilitate the publication of relevant guidance documents.

Further, DHTs can help conduct decentralized clinical trials, where data can be remotely recorded and analyzed directly from participants as a part of everyday tasks at places such as school, home, work, or outdoors. DHTs can also benefit with data collection from participants who are unable to report their experiences, like infants or cognitively impaired individuals. This kind of data collection can expand access to underrepresented and diverse patient populations and improve trial retention rates by improving convenience for trial participants and reducing caregiver burden.

The recent document will guide the use of DHT-derived data in regulatory decision-making for drugs and biological products.

Digital Health Technologies Document Outlines Framework for FDA

Cheryl Barton is director of PharmaVision, 

For more than a decade, manufacturers have been exploring new ways to improve the efficiency of cell therapy production to make it more cost-effective and accessible to those in need. Many companies have been working in this field, including US-based CAMP4 Therapeutics and Stealth Biotherapeutics’ and France-based TreeFrog Therapeutics SAS, and innovative approaches have now been developed to provide viable stem cells, at scale. 

In 2018, Erwan Bezard, Jean-Luc Treillou, Kevin Alessandri, Laurent Cognet, Maxime Feyeux, and Pierre Nassoy co-founded Bordeaux-based TreeFrog Therapeutics with the sole mission to establish a secure cell production process based on their proprietary 3D C-Stem scalable technology to improve the accessibility and affordability of induced pluripotent stem cell (iPSC)-based therapeutics (1).

In July 2022, Peter Nell, PhD, joined TreeFrog as an independent board member. He previously worked at Bayer Healthcare and has extensive knowledge about business development and licensing and clustered regulatory interspaced short palindromic repeats (CRISPR) technology (2).

In January 2022, the company was recognized as one of the top 25 BioTech start-ups (1). Since its conception, it has attracted US$86.9 million (€80.8 million) in series A and B funding from private equity investors, including Leonard Green and Partners, XAnge, AQUITI Gestion, BNP Paribas Développement, and Bpifrance, as well as Bristol Myers Squibb, Aquitaine Science Transfer, and the European Commission (1).

The company has utilized this funding to leverage its C-Stem technology to generate a pipeline of innovative products which include iPSC dopaminergic neurons to treat Parkinson’s diseases (enter Phase I in 2024); and preclinical programmes using hematopoietic stems cells for bone marrow transplant, cardiomyocytes for myocardial infarction, iPSCs platform and NK and T cells for immune‑oncology (3).

TreeFrog’s lead programme targets Parkinson’s disease via the generation of three-dimensional (3D) neurospheres which contain mature dopaminergic neurons. Following promising pre-clinical studies, the company is expecting to enter first-in-human (FIH) trials in 2024 (3).

On 11 Oct. 2022, TreeFrog’s collaboration with the contract development and manufacturing organization (CDMO) Invetech, came to fruition, and the first good manufacturing practice (GMP)-grade cell encapsulation device was installed and will be used to produce TreeFrog’s PD cell therapy candidate.

The device combines microfluidics and C-Stem cell biology to generate biomimetic alginate capsules seeded with iPSCs. The encapsules iPSCs rapidly expand and differentiate in 3D culture to produce ready-to-transplant microtissues. The capsule helps to reduce the impact impeller shear stresses exerted on iPSCs cells in large-scale bioreactors which can generate more than 1000 capsules per second, and are suited to seed reactors from 200 mL to 10 L (4).

In 2023, the GMP-grade cell encapsulation device will be transferred from TreeFrog to the CDMO to manufacturer clinical grade batches for Treefrog Parkinson’s Disease programme, and three additional GMP encapsulation devices will be delivered to Invetech to support TreeFrog’s pipeline programme.

TreeFrog taps into new technologies through strategic partnerships

TreeFrog has forged several partnerships to tap into new gene editing technologies by leveraging its C-Stem technology.

In June 2022, Treefrog teamed up with iPSC engineering Umoja Biopharma, USA, to use it synthetic rapamycin-activated cytokine receptor (RACR) technology in combination with TreeFrogs C-Stem platform to generate off-the-shelf iPSC-derived allogenic products that target immune cells in solid and haematological malignancies (5).

Geographical expansion to drive C-Stem adoption

TreeFrog has ambitious plans to drive adoption of its C-Stem technology across the globe. The company has started expanding its workforce and facilities; in 2022, the company aimed to double its laboratory capacity for the development and production of iPSC therapies.

During 2022, it opened technological hubs in Boston in the United States and Kobe, Japan, and aimed to establish strategic alliances with leading academic and biopharma players.

In 2022, TreeFrog awarded several Stem Cell SpaceShot grants to leading academic institutions including the University of Sheffield, UK; MCR Laboratory of Molecular Biology, Cambridge, UK; Harvard University, USA; University of California, USA; R&D Center for Cell Therapy, Kobe, Japan; and CNRS, France, which brought together experts in stem cell, gene editing and biophysics to drive further innovations in this field (6).

Investors will be watching this unicorn stock closely to see if the company delivers on its business goals, providing three additional GMP-grade devices to Invetech in 2023 and initiating a FIH trial for iPSC targeting Parkinson’s Diseasein 2024.

Additionally, it is anticipated to bring more news during 2023/2024 regarding further partnerships/licensing deals across the globe, as the company leverage its C-Stem technology with leading academic institutes and biopharma companies.

Competition in this field will increase as new technologies come to the forefront and investment in competitor companies such as CAMP4 Therapeutics (7) and Stealth Biotherapeutics’ (8) have products nearing the market.

1. Crunchbase. TreeFrog Therapeutics, Company Profile & Funding. 

 (accessed 13 Dec. 2022).
2. TreeFrog Therapeutics. Peter Nell joins TreeFrog Therapeutics as Independent Board Member. Press Release, 25 July 2022 (accessed 13 Dec. 2022).
3. TreeFrog Therapeutics. Cell therapy pipeline. 

 (accessed 13 Dec 2022).
4. TreeFrog Therapeutics. Treefrog Therapeutics Moves to GMP Manufacturing with Cell Encapsulation Device Delivered by Invetech. Press Release. 11 Oct. 2022.
5. Umoja Biopharma. Umoja Biopharma and TreeFrog Therapeutics Announce Collaboration to Address Current Challenges Facing Ex Vivo Allogeneic Therapies in Immuno-Oncology. Press Release. 10 June 2022.
6. TreeFrog Therapeutics. 3 PhD Students from King’s College London Win the $100,000 Stem Cell SpaceShot Grant. Press Release. 20 Oct. 2022.
7. CAMP4. CAMP4 Secures $100 Million Series B Financing to Accelerate Expansion of its Novel Regulatory RNA-Targeting Platform and Advance Lead Programs into the Clinic. Press Release. 20 July 2022.
8. Stealth BioTherapeutics. Programs and Pipeline. 

TreeFrog Therapeutics is leaping ahead in cell therapies through 
resources such as new technologies and investor partnerships.


Frontrunners in the Latest Stem Cell Technology

Editor of Pharmaceutical Technology Europe

There are myriad factors influencing the drug delivery market, such as the rising prevalence of chronic conditions, an ageing population, and technological advancements in drug development, leading analysts to predict continued growth in the sector at a compound annual rate of 3.7% between 2022 and 2030 (1). A significant driver of growth within drug delivery has been highlighted as the advancement of the biopharmaceutical industry, which is giving rise to new and innovative drugs for previously untreatable or incurable diseases and, hence, the need for ways to deliver these novel therapies.

To learn more about influential drug delivery trends, market drivers, aspects of drug delivery that require greater research, and how partnering with an outsourcing company can help in this sector, 

® spoke with Torkel Gren, senior director, science and technology officer, and Uwe Hanenberg, head of product development, Oral Solid Dose, both at Recipharm.

 Over the past decade, what, in your opinion, have been the most influential developments in alternative drug delivery formulation?

 The most influential development in recent years has been the advance in solid lipid nanoparticles that allowed the development of mRNA [messenger RNA] vaccines to tackle COVID-19. [This advancement] opens up the opportunity to develop many new pharmaceutical products with increased efficacy in the future. There is plenty of potential to harness mRNA technology to create exciting new vaccines and other therapies to tackle previously untreatable conditions over the next decade.

 What trends in alternative drug delivery formulation do you think may be seen during the next decade?

 There will be increasing investment in new delivery technology for targeted and or simplified drug delivery. The effect/side effect ratio of a large number of drug substances can be significantly improved when delivered directly to the site of action. Administration by injection will play a key part here, alongside other delivery routes—topical, pulmonary, and ophthalmic, for example.

Integration of dosing with smart devices will also be a major theme in the coming decade as a means of increasing patient adherence. By monitoring biodata, such as blood glucose, these systems can provide both patient and HCP [healthcare professional] with valuable information to ensure that medication is used optimally to meet the unique needs of individual patients.

It is expected that the next decade will see advances allowing oral delivery of a greater number of biologic formulations. Efforts are already being made to develop a platform technology to support absorption of orally administered biopharma treatments, with exciting potential to further improve patient convenience.

 Could you elaborate on any of the market factors that are driving growth in the alternative drug delivery formulation sector?

 Rising demand for new biologics has created the need for special formulations for these treatments. It is important to note that the term ‘biologics’ refers to a large number of different modalities, from monoclonal antibodies, nucleotides, CAR-T [chimeric antigen receptor T] cells, and microbiome products. These all have their specific scientific challenges, each requiring a different formulation approach.

Interest in improved treatment for small indications has also contributed to greater demand for alternative drug delivery, as these often call for some special drug delivery solutions.

 How have these drivers changed over recent years?

 The trend toward more advanced biological treatments with a higher potential to cure the disease rather than just its symptoms has implications for drug delivery. Technologies that ensure an effective treatment by improving the stability of the active ingredient and that can be administered easily (e.g., orally) are drivers and will enhance patient compliance This could increase the demand for specialized drug delivery solutions in the coming years.

 Are there any areas in alternative drug delivery formulation approaches that still require a significant amount of research?

 [There have been] impressive developments in new biological medicines in recent years, with a number of new therapies entering the global market. However, the cost of these products has undermined their impact on public health and wellbeing. More work needs to be done to reduce development and manufacturing costs of these biopharma treatments while optimizing their efficacy.

New drug delivery technology may help solve this problem in the future. Better formulation approaches, for example, have the potential to improve stability, resulting in longer shelf life and reduced need for cold storage. This can cut distribution costs significantly, especially in countries with warm climates and limited infrastructure. Currently, biologics that have exceeded their shelf life or been transported outside their allowed temperature interval have to be disposed of—which is also very costly.

The fact that most biologics today need to be administered by injection is also contributing to their relatively high overall treatment cost, as this usually means they must be administered to patients by HCPs. There is some interesting work on alternative administration routes (orals, buccal, and pulmonary, for example), but it is likely that injection will continue to dominate in the future, due to the unique stability and absorption characteristics of biopharma. Injectable delivery systems, such as auto-injectors, which allow self-administration by the patient in the home, can help reduce costs by eliminating the need for a trip to a clinic to be treated by a HCP. Depot formulations allowing monthly dosing instead of daily injection can also reduce the burden on HCPs and clinics.

 What is the role of an outsourcing partner in the alternative drug delivery formulation market?

 Many different types of drug delivery technologies will be needed for different modalities, administration routes, and markets. As such, it is almost impossible for pharmaceutical companies to fill all their needs in-house. Outsourcing drug development and manufacturing to contract development and manufacturing organization partners can address this issue. By working with partners that have experience in a particular alternative technology, companies can benefit from specialist expertise and infrastructure, helping to smooth their development journey.

Pharmaceutical Drug Delivery Market (by Route of Administration: Oral, Pulmonary, Ocular, Nasal, Injectable, Topical, and Others; by Application: Cardiovascular, Diabetes, Cancer, Infectious Diseases, and Others; by End Users: Hospitals, Ambulatory Services, and Home Healthcare)—Global Industry Analysis, Size, Share, Growth, Trends, Regional Outlook, and Forecast 2021–2030

Felicity Thomas is the European/senior editor for 

Advancements in therapeutic modalities are necessitating change in drug delivery to help negate some of the expense of development and manufacturing.

Influencing Change in Drug Delivery

Feliza Mirasol is the science editor for 

On March 22, 2023, Invitae, a US-based medical genetics company, and Deerfield Management, a US-based healthcare investment firm, announced that they are partnering to advance genetics-based drug discovery and development in rare diseases. Under the agreement, the partnership will harness genetics and clinical data from millions of patients and apply that data to Deerfield's drug discovery and development expertise.

The two parties aim to address research gaps in the drug discovery process by drawing insights from Invitae's data platform. The platform includes sizable cohorts of rare disease patients. Through collaboration, the companies will analyze data from the more than 3.6 million genetic tests delivered by Invitae that are linked to longitudinal clinical data, which will strictly follow patients' preferences for sharing of their data. The companies believe that by combining Invitae’s data with Deerfield’s analytics expertise, they will be well-suited to uncover insights needed to discover and validate new rare disease targets, according to a company press release.

"Invitae's core objectives include expanding access to genetic information to improve healthcare for patients. Through this partnership, we intend to serve millions of families affected by rare diseases with high unmet needs," said Farid Vij, general manager of patient network and data at Invitae, in the release. "Our genetic and clinical data platform is highly enriched with patients possessing rare disorders, enabling a deeper understanding of the natural history and genetic basis of these diseases, and improving the success rate of new clinical programs."

"Despite significant research efforts, there are thousands of rare diseases without a line of sight toward a viable therapeutic intervention," said Matt Nelson, head of genetics and genomics at Deerfield Management, in the release. "Invitae's unique data and analytics capabilities position it to interrogate genetic effects on rare and many other diseases. With Deerfield's extensive experience in rare diseases and drug discovery, we believe there is strong potential for this partnership to uncover valuable patient insights and create important therapies for a range of currently untreatable conditions."

Under a new partnership, Invitae and Deerfield Management will harness genetic and clinical testing data from patients to discover potential novel therapeutics for treating rare diseases.

Invitae and Deerfield Management Team Up on Novel Therapeutics for Rare Diseases

Susan Haigney is managing editor of BioPharm International, susan.haigney@ubm.com.

Scorpius BioManufacturing, a contract research and contract development and manufacturing organization based in Texas, announced on March 21, 2023 that the company has appointed Gary Welch as interim president. Welch will lead the company in serving clients with mammalian and microbial biologics and new modalities. Welch, who has previously worked with AbbVie, Agensys, and CytomX, comes to the company with more than 30 years of experience.

The company also announced that it will open new microbial capacity to supplement the mammalian suites at its San Antonio facility. In addition, the company plans to construct a commercial-scale facility in Manhattan, Kan. to help their clients that are creating new microbial and mammalian modalities to start projects quickly and utilize Scorpius throughout the lifecycle of those modalities.

In addition to a new interim president, the company is opening new microbial capacity at its San Antonio facility and has plans to construct a commercial-scale facility in Manhattan, Kan.

Scorpius BioManufacturing Announces New Microbial Capacity and New Interim President

TrakCel announced on March 17, 2023 that their collaboration with Lonza has reached a new milestone, resulting in a secure, reliable combination of TrakCel’s cell orchestration platform OCELLOS and Lonza’s manufacturing MODA-ES(R) platform. This integration will translate into critical manufacturing steps, checks, and data flow directly into the Chain of Custody and Chain of Identity within the cell and gene therapy supply chain without the opportunity of being missed or not connecting with the patient journey.

For developers, this will assist in them gaining additional visibility and control while reducing product loss, delayed treatment, recalls, and poor-quality product. The combined solutions are currently available on the market.

The collaboration between TrakCel and Lonza includes a multi-phase rollout of connectivity with OCELLOS to other Lonza systems like SAP and LIMs. Other integrations are planned to follow the MES proof of concept and are likely to be completed in the second half of 2023.

The combined solutions are currently available on the market.

TrakCel and Lonza Reach Integration Milestone to Combine Cell and Gene Therapy Manufacturing, Orchestration Solutions 

Gyros Protein Technologies, a provider of peptide synthesizers and reagents, announced on March 21, 2023 the launch of three service offerings. These are intended to support the development and manufacturing of peptide-based drugs, and they are based on the company’s PurePrep EasyClean (PEC) orthogonal peptide purification technology.

“PEC-grade peptide service,” which is intended for custom synthesis of PEC-grade peptides

“Custom PEC process and kit development,” which is for scale-up and scale-out of the technology

“Enabling new modalities manufacturing with PEC,” which will help to accelerate the developments of novel and complex peptide-based drug modalities.

The new technology is now fully integrated into Gyros Protein Technologies’ offering via two kit types and the newly launched services, and the integration will help the company support customers throughout the whole workflow from synthesis to final purified product, which will accelerate drug development and manufacturing processes.

The services are based on the company’s PurePrep EasyClean (PEC) orthogonal peptide purification technology.

PurePep EasyClean Services Set to Accelerate Development and Manufacturing of Peptide-based Drugs 

AstraZeneca and MSD, known as Merck & Co., Inc., Rahway, NJ, USA in the United States and Canada, announced on March 20, 2023 that the Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorization for Lynparza (olaparib) in the United Kingdom for use as combination therapy with abiraterone and prednisone or prednisolone for the treatment of adult patients with metastatic castration-resistant prostate cancer (mCRPC) when chemotherapy is not clinically indicated.

The decision was based on results from the PROpel Phase III trial, which showed that AstraZeneca and MSD’s Lynparza, in combination with abiraterone, significantly improved radiographic progression-free survival (rPFS) versus abiraterone alone as a first-line treatment for patients with mCRPC regardless of their biomarker status.

The results additionally show that combination therapy reduced the risk of disease progression or death by 34% versus abiraterone alone. As for adverse reactions, Lynparza has been associated with adverse reactions generally of mild or moderate severity and generally not requiring treatment discontinuation.

As for the most frequently observed adverse reactions across clinical trial in patients receiving Lynparza monotherapy were nausea, fatigue/asthenia, anemia, vomiting, diarrhea, decreased appetite, headache, neutropenia, dysgeusia, cough, leukopenia, dizziness, dyspnea, and dyspepsia.

We are delighted that olaparib has been authorised by the MHRA today, opening a new chapter in prostate cancer treatment. This additional therapy option supports our overall ambition to reduce the national burden of prostate cancer and we are continuing to work with NICE and NHS England to secure access as quickly as possible for patients. Medical advances like these offer hope of further progressing the UK as a leader in life science,” said Ed Piper, Medical and Scientific Affairs director, AstraZeneca UK, in a press release.

“Prostate cancer is the most common cancer in men in the UK and advanced prostate cancer is associated with a significant mortality rate. This approval from the MHRA marks important progress in advancing a new treatment option to address the significant unmet need of patients with metastatic castration-resistant prostate cancer,” said David Long, Head of Oncology at MSD UK, in the release.

The decision was based on results from the PROpel Phase III trial.

Lynparza Approved in UK as Combination Therapy for Treatment of Metastatic Castration-Resistant Prostate Cancer 

The European Commission (EC) has approved Dupixent (dupilumab) in the European Union to treat severe atopic dermatitis in children six months of age to five years of age who are candidates for systemic therapy. This approval makes the drug the first and only targeted medicine indicated to treat this population of children in Europe and the United States.

Dupixent is a subcutaneous injection at different injection sites and is intended for use under the guidance of a healthcare professional. It can be given in a clinic or at home by self-administration after training by a healthcare professional.

The approval is based on data from a Phase III trial that evaluates Dupixent every four weeks plus low-potency primarily topical corticosteroids (TCS) or TCS alone in 162 children between six months of age to five years of age with moderate-to-severe atopic dermatitis. The medicine improved skin clearance and reduced overall disease severity and itch at 16 weeks compared to placebo in the overall enrolled population.

In addition, Dupixent improved sleep quality, skin pain, and health-related quality of life compared to placebo in both the overall and severe populations, and the long-term efficacy data showed the clinical benefit at 16 weeks was sustained through 52 weeks, according to the Sanofi.

Common side effects across all indications include injection site reactions, conjunctivitis allergic, arthralgia, oral herpes, and eosinophilia.

This approval makes the drug the first and only targeted medicine indicated to treat this population of children in Europe and the United States.