Christopher Cole

An antiviral used to treat hepatitis B and C | Image Credit © luchschenF

 (pharma&) has received a regulatory variation from the UK’s 

Medicines and Healthcare products Regulatory Agency

 (MHRA), authorizing its wholly owned subsidiary, 

 GmbH, as an approved manufacturing site for the API in Pegasys (peginterferon alfa-2a), according to a May 28, 2025 news release from the company (1). This regulatory update marks a significant milestone in stabilizing the long-term supply chain of Pegasys in the United Kingdom and enables the company to begin product replenishment across the region.

Following the MHRA decision, pharma& expects that eligible patients in the UK will begin to see improved access to Pegasys in the coming weeks.

“We are delighted that the MHRA has granted approval for the production of Pegasys API at Loba biotech GmbH. This marks a critical step forward, building on the recent EMA [European Medicines Agency] approval, securing the path forward for Pegasys replenishment across the United Kingdom,” said E

, founder and managing director of pharma& (1).

·MHRA approval of Loba Biotech enables in-house Pegasys API production, reinforcing pharma&'s control over its biologics manufacturing process.

·UK Pegasys supply chain gains long-term stability as pharma& scales bio-manufacturing capacity at its Loba Biotech facility.

·pharma&’s investment in Pegasys API production reflects growing industry focus on supply chain resilience for essential biologic therapies.

Strategic Manufacturing Investment Supports Global Pegasys Continuity

The decision to invest in internal API manufacturing capabilities followed pharma&'s acquisition of Pegasys from F. Hoffmann-La Roche AG in 2021 (2). Roche had announced in 2019 that it would cease global commercialization of the product (1). Since then, pharma& has prioritized uninterrupted patient access to Pegasys, with a particular focus on markets affected by supply shortfalls.

Following the acquisition, pharma& observed a surge in demand for Pegasys, leading the company to invest in bio-manufacturing infrastructure at Loba biotech GmbH. The regulatory approval for this site underscores the company's commitment to securing reliable access to critical therapies.

“Since acquiring Pegasys, pharma& has remained dedicated to maintaining continuous access to this medicine for eligible patients,” Zagler added (1). “The MHRA’s approval, along with our investment in Loba biotech, represents pharma&’s ongoing commitment to preserving the availability of essential medicines and foster their development worldwide to leave no patient behind.”

While supply restoration in the UK and Europe is advancing, pharma& also continues to work closely with regulatory bodies in other regions, including the United States, to support expanded access for eligible patients.

Pegasys mechanism of action and indications

Pegasys is a pegylated type I interferon (IFN), primarily interferon alfa-2a, according to the release (1). Type I interferons—including IFN-α, IFN-β, IFN-ε, IFN-κ, and IFN-ω—are a class of alpha-helical cytokines central to innate and adaptive immune responses, with evolutionary origins tracing back approximately 500 million years. These proteins exert antiviral effects by binding to the interferon-α receptor (IFNAR), composed of IFNAR1 and IFNAR2 subunits.

Pegylation of interferon alfa-2a involves the covalent attachment of a branched polyethylene glycol (PEG) molecule, resulting in the formulation known as Pegasys (1). The 40-kilodalton PEG moiety enhances pharmacokinetics by acting as a protective barrier and extending systemic exposure.

In the UK, Pegasys has been authorized for the treatment of chronic hepatitis B (CHB) in adults and children aged three years and older, as well as chronic hepatitis C (CHC) in adults and children aged five years and older (1). CHC treatment in pediatric patients is approved in combination with ribavirin.

Full safety information and the Summary of Product Characteristics for Pegasys are accessible through the 

pharma& Receives MHRA Approval for Pegasys Manufacturing Site Variation, Adding Loba biotech for API Production and Ensuring a Reliable, Long-term Supply in the UK

Medicines & Healthcare products Regulatory Agency. 

Articles

MHRA approves Loba Biotech to manufacture Pegasys API, enabling pharma& to strengthen UK supply and ensure continued access for eligible patients.

pharma& Secures MHRA Approval for Pegasys API Production at Loba Biotech

Sanofi logo at one of skyscraper. Sanofi is a French biopharma company | Image Credit: © Robert - stock.adobe.com

 announced via a news release dated May 21, 2025, that it has signed an agreement to acquire 

., a clinical-stage biotechnology company specializing in therapies for neurodegenerative diseases (1). The acquisition aligns with Sanofi’s strategic emphasis on neurology and is intended to enhance its capabilities in early-stage drug development within this therapeutic area.

Vigil’s lead candidate, VG-3927, is an oral small molecule that acts as a TREM2 agonist. This compound is being prepared for a Phase II clinical trial in Alzheimer’s disease, according to the release (1). TREM2 activation is hypothesized to enhance the neuroprotective functions of microglia by promoting their migration to sites of neural injury, increasing their phagocytic activity, and supporting cell survival and proliferation. These functions are considered critical in mitigating chronic inflammation and debris accumulation in neurodegenerative conditions.

·Sanofi acquires clinical-stage biotech Vigil Neuroscience to expand its neurology drug pipeline focused on neurodegenerative disease therapies.

·VG-3927, Vigil's lead candidate, is a TREM2 agonist targeting microglial function; Sanofi plans to advance it into Phase II trials for Alzheimer’s.

·The deal reflects Sanofi’s strategic use of early investments to secure biotech assets and accelerate central nervous system drug development and manufacturing.

“This acquisition is fully supporting Sanofi’s strategic focus on neurology and on advancing science and leveraging our expertise in immunology to solve areas of critical unmet need,” said 

, head of research and development at Sanofi (1). TREM2 represents a compelling target at the intersection of immune dysregulation and neurodegeneration, particularly in people living with Alzheimer’s because they face devastating cognitive decline with limited treatment options. Vigil’s expertise is complementary to our capabilities in neurology and reinforces our dedication to developing innovative medicines to improve people’s lives. Vigil’s team are a welcome addition, and we look forward to working with them and the patient community.”

The news release notes that Vigil’s research is focused on the role of microglia in central nervous system disorders (1). In neurodegenerative diseases, microglial dysfunction contributes to progressive neuronal damage. Current Alzheimer’s treatments offer limited efficacy and stringent eligibility, underscoring the need for novel therapeutic approaches.

Sanofi’s acquisition follows a strategic $40 million investment made in June 2024, which included the exclusive right of first negotiation for VG-3927 (2). The decision to proceed with the full acquisition reflects Sanofi’s broader strategy of early equity investments to secure potential assets in critical areas of unmet medical need (1).

“We are incredibly proud of the extraordinary and diverse team at Vigil and the progress we have made in developing innovative TREM2-based therapeutics. We look forward to joining forces with Sanofi's team to leverage Sanofi’s resources, broad platform and far-reaching network to fully unlock and accelerate the development of VG-3927 for the potential treatment of Alzheimer’s disease—a devastating disease with significant unmet need,” said 

, president and chief executive officer of Vigil.

, Vigil’s second investigational molecule. The deal is expected to close in the third quarter of 2025 and will not impact Sanofi’s financial guidance for the year.

Sanofi to Acquire Vigil Neuroscience, Inc., Adding a New Investigational Medicine to Treat Alzheimer’s Disease to the Neurology Pipeline

Vigil Neuroscience Announces $40 Million Strategic Investment from Sanofi

Sanofi is acquiring Vigil Neuroscience, a biotech firm focused on novel neurodegenerative disease therapies, to boost its early-stage neurology pipeline.

Sanofi to Acquire Vigil Neuroscience to Expand Neurodegenerative Disease Pipeline

Data technology background. Abstract background. Connecting dots and lines on dark background. 3D rendering. 4k. | Image Credit: © Dmitry

Emerson has announced a new AI-driven approach aimed at enhancing the reliability and performance of mission-critical manufacturing operations, including those in the pharmaceutical sector (1). The initiative builds on the company’s acquisition of Aspen Technology and leverages decades of domain-specific automation expertise, according to the company’s news release (1).

·Emerson's new Project Beyond platform supports deployment of AI tools across embedded, edge, and cloud systems in mission-critical environments.

·Localized, first-principles-based AI models aim to improve reliability, safety, and interpretability in industrial and pharmaceutical operations.

·Emerson's AI tools assist with system modernization, facility design, and sustainability planning, minimizing risks in regulated manufacturing settings.

At the Emerson Exchange 2025 (2) conference on May 20 in San Antonio, the company introduced 

, a software-defined, operational technology (OT)-ready platform developed to manage Emerson’s growing portfolio of AI models and applications. Designed for deployment across embedded, edge, and cloud environments, the platform integrates industrial AI with contextualized data to support operations where safety, reliability, and regulatory compliance are paramount.

AI has reached a critical stage in its evolution, with foundational models and open-source frameworks becoming increasingly accessible, Emerson noted in the release (1). While these developments offer potential benefits for automation and workflow optimization, Emerson notes that public generative AI (GenAI) tools remain unsuitable for industrial settings, where reliability and security are essential.

To address this gap, Emerson has developed an AI portfolio centered on localized, fit-for-purpose models (1). These tools, the company says, are built with embedded guidance grounded in first-principles physics and engineering, aiming to eliminate the types of inaccurate or unsafe outputs sometimes generated by general-purpose AI systems. Localized implementation also avoids the risks associated with transferring sensitive operational data to public cloud environments.

“Generic large language models are necessary for AI, but they are insufficient for a live, mission-critical industrial plant that cannot afford unreliable or unsafe results,” said 

, chief operating officer at Emerson (1). “Emerson’s solutions leverage industrial AI, built on decades of industry expertise and first principles constraints, to deliver correct, actionable guidance users can count on to make better operational and business decisions that will safely and rapidly drive competitive advantage.”

For pharmaceutical manufacturers, where automation plays a critical role in quality control, batch consistency, and regulatory compliance, Emerson’s suite of AI-enabled tools is designed to support high-availability applications (1). These include:

AI Equipment Layout, which uses GenAI to generate multiple facility design options based on user-defined requirements. This tool is intended to streamline layout planning for both greenfield and brownfield manufacturing sites, including consideration of safety, proximity, and buffer zones—key factors in regulated environments.

 which supports the modernization of legacy control and safety systems. By analyzing data from thousands of prior modernization efforts, the tool accelerates the migration process to the DeltaV platform with a focus on reducing risk and improving configuration accuracy.

AspenTech Strategic Planning for Sustainability Pathways

 which applies GenAI to evaluate decarbonization scenarios and develop long-term sustainability strategies—a growing priority for pharmaceutical companies under ESG mandates.

(AVA), an embedded AI advisor that provides real-time operational guidance to users. Currently integrated with Aspen PIMS and Aspen DMC3 software, AVA enables users to query system performance in natural language, supporting decision-making related to production optimization and process bottlenecks.

Additional virtual advisors within Emerson’s 

 further support diagnostics and visualization of control system operations (1). These tools aim to reduce complexity for plant personnel and improve response times to system anomalies.

“Emerson is the only company with the wide range of domain expertise, deep stores of first principles models, vast array of fit-for-purpose technology solutions, and history of industry-shaping innovation necessary to deliver industrial AI tools that modern manufacturers can trust in a mission-critical environment,” Krishnan added in the news release (1). “With Emerson’s expertise, data and Boundless Automation vision, we are perfectly positioned to advance AI solutions that actually deliver on their promises.”

By focusing on AI models that align with specific industrial needs and constraints, Emerson aims to provide pharmaceutical and other manufacturers with tools that enhance automation reliability, reduce downtime, and improve operational insights without compromising data security or system integrity.

Emerson’s Expanded AI Portfolio Paves the Way for More Optimized Autonomous Operations.

Project Beyond: Shaping the Future of Automation

Emerson’s purpose-built industrial AI solutions are meant to enhance accessibility and reliability, enabling manufacturers to maximize efficiency and performance from automation systems.

Emerson Unveils AI Platform for Mission-Critical Industrial Applications

The world logistics background or transportation Industry or shipping business, Container Cargo shipment , truck delivery, airplane , import export Concept | Image Credit: © sittinan.

In a move FDA says is aimed at improving drug affordability without compromising safety or efficacy, the administration is refining its regulatory pathway to support state- and Native American tribe-led importation of certain prescription medications from Canada, according to a news release on May 21, 2025 (1). These enhancements fall under FDA’s implementation of the Presidential Executive Order 

Lowering Drug Prices by Once Again Putting Americans First

 (2) and are intended to reduce pharmaceutical costs for American patients while upholding strict quality standards.

·FDA announced plans to streamline importation proposals, offering pre-reviews and guidance tools to help states develop compliant programs for drug imports from Canada.

·Regulatory burden is reportedly set to be reduced via informal meetings and simplified cost analysis aimed at accelerating approval of state importation plans.

·High drug quality and safety standards remain central to the plans, ensuring imported drugs meet US manufacturing and compliance expectations despite lower costs.

Section 804 of the Federal Food, Drug, and Cosmetic Act

The update centers on Section 804 of the Federal Food, Drug, and Cosmetic Act, which permits states and tribes to import select prescription drugs from Canada. FDA is optimizing this framework to remove administrative barriers and encourage broader participation.

“For too long, Americans have been getting taken advantage of. Drug prices in the US are sometimes 5-10 times higher than in wealthy European countries,” said FDA Commissioner 

 in the news release (1). “Today’s actions will support the ability to import drugs at much lower prices while also maintaining the high quality and safety of medicines that Americans expect and deserve.”

To support this goal, FDA will now allow states and tribes to submit draft proposals for informal pre-review. These preliminary reviews, followed by optional meetings with FDA representatives, are intended to provide early feedback, reduce the regulatory burden, and help sponsors craft more robust and technically sound 

A new, user-friendly tool is also in development to assist stakeholders in preparing proposals that align with FDA requirements (1). This tool will provide structured guidance, helping applicants meet submission standards more efficiently and thereby shortening the agency’s review timeline.

In parallel, FDA is working on options to simplify the required cost savings analysis. This includes clarifying the types of data states can use to project consumer savings, which remains a central component in SIP proposal approval.

The agency plans to meet with interested states this fall to discuss these improvements, provide technical guidance, and gather feedback on the revised process. These discussions aim to foster collaboration and innovation while maintaining FDA’s commitment to public health and safety.

Importation of Prescription Drugs Final Rule

 (4) from the Secretary of Health and Human Services notes that importation program proposals may be submitted in “certain future circumstances” by pharmacists and wholesalers in addition to states and tribes. Importation program proposals must explain how the programs pose no additional risk to public health and safety and explain how the sponsor will ensure significant reductions in American consumer costs for products covered under the program. Drugs included in a program must also be approved by Canada’s Health Products and Food Branch, or HPFB, and meet the conditions of an FDA-approved new drug application or abbreviated new drug application. Additional information is available via the 

Importation of Certain FDA-Approved Human Prescription Drugs, Including Biological Products, and Combination Products under Section 801(d)(1)(B) of the Federal Food, Drug, and Cosmetic Act, Guidance for Industry

FDA Takes Steps to Enhance State Importation Programs to Help Lower Prescription Drug Prices

Importation Program under Section 804 of the FD&C Act

, updated May 21, 2025 (accessed May 22, 2025).

Guidance for Industry, Importation of Certain FDA-Approved Human Prescription Drugs, Including Biological Products, and Combination Products under Section 801(d)(1)(B) of the Federal Food, Drug, and Cosmetic Act

FDA announced efforts to streamline its drug importation process to help U.S. states and Native American tribes access lower-cost Canadian drugs while maintaining safety standards.

FDA Refines Regulatory Pathway for Canadian Drug Importation

Genetic engineering concept. Collage with glowing DNA molecule over female hand on blue background | Image Credit: © Prostock-studio

 (Mirai) announced on May 20, 2025 that the company has entered into a strategic collaboration with 

 aimed at advancing the development and manufacturing of nucleic acid therapeutics (1). The agreement brings together Mirai’s technology platform for the design and optimization of genetic medicines with Thermo Fisher’s development and current good manufacturing practice (CGMP) capabilities.

The collaboration will initially focus on leveraging Thermo Fisher’s RNA and advanced formulations facility to support the industrialization of Mirai’s development candidates, according to the release (1). In addition to technical collaboration, the agreement includes a direct investment into Mirai as part of the broader partnership between Thermo Fisher and 

·Mirai Bio and Thermo Fisher are partnering to integrate design, delivery, and CGMP manufacturing for nucleic acid-based therapeutics.

·The collaboration will leverage Thermo Fisher’s RNA and advanced formulations facility to support scalable development of genetic medicines.

·Mirai’s machine intelligence platform will be combined with Thermo Fisher’s technical and manufacturing expertise to streamline drug development workflows.

“This collaboration is an expansion of our long-standing relationship with Thermo Fisher and a reflection of our shared vision for accelerating the development of enabling technologies to develop first-in-class therapies,” said Justine 

, executive partner, origination and corporate development at Flagship, in the release (1). “In this case, the combination of Mirai’s innovation in targeted delivery and cargo design and Thermo Fisher’s deep technical and commercial manufacturing expertise will maximize opportunities for the co-creation of novel medicines and help realize the full potential of both enterprises.”

Mirai offers end-to-end services for partners in the genetic medicine space. The company’s machine intelligence-based platform enables high-throughput design and 

 testing of targeted delivery systems, facilitating rapid and scalable development processes.

“Thermo Fisher is a trusted industry leader with deep technical expertise and proven developmental and GMP services and commercial manufacturing capabilities,” said Travis Wilson, executive chairman of Mirai and growth partner, Flagship Pioneering, in the release from Mirai (1). “Coupling these with Mirai’s unique delivery and genetic medicine optimization technologies will support accelerated program development and commercialization for our life science partners worldwide. We’re thrilled to enter into this collaboration to further strengthen Mirai’s platform and fully realize our vision of co-creating targeted, potentially curative programmable medicines with end-to-end optimization.”

, president, drug substance, pharma services at Thermo Fisher, stated, “Mirai is on a journey to break down the barriers currently limiting the full potential of genetic medicines and offer fully integrated design, delivery, and manufacturing capabilities to innovative partners. We’re excited to combine our life sciences expertise and manufacturing capabilities with Mirai’s machine intelligence-powered platform to help bring the next generation of medicines to patients in need.”

This new collaboration is part of an ongoing effort by both organizations to address key challenges in genetic medicine development and to jointly create integrated solutions that can support the next wave of therapeutic innovation (1).

Thermo Fisher is focused on working sustainably. Adam Goldstein, senior director of R&D Collaborations at Thermo Fisher Scientific, 

 that sustainability-by-design in bioprocess development involves integrating sustainable practices and principles from the very beginning of the development process, which includes considering environmental impacts, resource efficiency, and waste reduction during the design phase, to create processes that are not only efficient but also lower the environmental footprint (2).

Goldstein went on to emphasize the importance of the innovations that result from adopting a sustainability-by-design approach because they can lead to improved product quality and process efficiency. “Driving higher titer in smaller volumes means yielding higher throughput of product in a smaller footprint. The benefits are lower cost of goods in manufacturing while simultaneously delivering the product in a much more sustainable fashion with regards to emissions reductions,” he commented (2).

Mirai Bio Announces Partnership with Thermo Fisher Scientific to Accelerate Development of Genetic Medicines

Sustainability by Design in the Context of Bioprocess Development.

Mirai Bio and Thermo Fisher partner to integrate AI-driven design with CGMP manufacturing, aiming to streamline development of genetic medicines.

Mirai Bio and Thermo Fisher Partner to Advance Genetic Medicine Manufacturing

·Grifols received FDA clearance to initiate a Phase II trial of GRF312, an immunoglobulin (IG)-based eye drop for treating dry eye disease.

·The trial builds on Phase I/II data showing significant symptom reduction with IG drops and no difference in safety profile compared with placebo.

·This marks a potential first use of IG for an ocular surface indication, expanding its therapeutic scope beyond systemic applications.

, a global healthcare company specializing in plasma-derived medicines, announced on May 21, 2025 (1) that it has received clearance from 

 to initiate a Phase II clinical trial of its immunoglobulin-based eye drop, GRF312 Ophthalmic Solution, for the treatment of dry eye disease (DED).

Dry eye disease is a chronic, multifactorial inflammatory condition that affects more than 100 million individuals worldwide, according to a company press release (1). It is characterized by insufficient tear production or excessive tear evaporation, leading to ocular discomfort, pain, and in severe cases, irreversible damage to the ocular surface. Standard treatments—such as artificial tears, corticosteroids, cyclosporine, and lifitegrast—may take weeks or months to deliver meaningful relief and do not work for all patients.

The Phase II trial is expected to begin in the third quarter of 2025 and will evaluate the safety, tolerability, and efficacy of GRF312 in 100 patients with DED. The clinical program builds upon a Phase I/II pilot study led by Grifols’ partner 

University of Illinois College of Medicine, Department of Ophthalmology

, according to Grifols (1). That study demonstrated a significant reduction in both signs and symptoms of DED after eight weeks of treatment with the IG formulation, administered twice daily. The tolerability profile was comparable to that of placebo.

“We believe our immunoglobulin eye drops have the potential to make a qualitative leap in how dry eye disease is treated,” said Dr. 

Jörg Schüttrumpf, Grifols chief scientific innovation officer

 in the May 21 release (1). “Research suggests that the broad-spectrum anti-inflammatory and immunomodulatory properties of an ocular surface IG are superior to currently approved therapies, including artificial tears and anti-inflammatories. We are using our extensive and deep knowledge of immunoglobulins to benefit the millions of patients globally who aren’t receiving sufficient relief.”

Development of GRF312 stems from a 2023 collaboration and licensing agreement between Grifols and Selagine, under which Grifols acquired exclusive global rights to the IG-based ophthalmic treatment (2). The agreement leverages Grifols’ capabilities in developing and manufacturing immunoglobulin therapies and Selagine’s research and clinical experience in ophthalmology.

“Selagine and Grifols joining efforts and complementary expertise successfully led the treatment to a Phase II clinical trial,” said 

, founder and President of Selagine and BA field professor of ophthalmology at the 

University of Illinois College of Medicine

. “The results obtained to date and this promising development reinforce the potential of this novel treatment approach to significantly improve the quality of life of dry eye disease patients.”

The trial marks one of the first formal efforts to investigate immunoglobulin as a therapeutic agent for the ocular surface—a potential first-in-class application. If successful, GRF312 could represent a new mechanism of action for addressing inflammation and immune dysregulation in DED, with implications for both patient outcomes and therapeutic innovation.

 that its subsidiary Biotest had garnered its first FDA approval with the intravenous immunoglobulin therapeutic, Yimmugo (3). Set to be distributed by Kedrion, preparations for an early 2025 launch in the United States was underway as of October 

2024 for Yimmugo (4). No further news of the launch was available at the time of writing.

Grifols Receives FDA Clearance of IND Application for Phase 2 Trial of Immunoglobulin Drops for Dry Eye Disease

Grifols Enters into Global Collaboration and Licensing Agreement with Selagine to Develop Immunoglobulin Eye Drops to Treat Dry Eye Disease. Press Release

FDA Approves Immunoglobulin Therapeutic to Treat Primary Immunodeficiencies

Expanding Treatment Options: Yimmugo® Coming Soon for PID Patients in the US

Grifols received FDA clearance to begin a Phase II trial of GRF312, an immunoglobulin eye drop for dry eye disease, aiming to improve current treatments.