Digital Transformation in Pharma Manufacturing

Patent Cliff Deals, Tariff Pressures, and the In Vivo Cell Therapy Bet

Behind the Headlines, Episode 23

ICH Q12 and Post-Approval Changes

Speed, Flexibility Top Large-Molecule Manufacturing Trends

Drug Digest July 2025

Key Insights from Industry Survey Results

Bio/Pharma's Tariff Response

Feliza Mirasol is the science editor for 

FDA’s Center for Drug Evaluation and Research (CDER) issued a 

 to India-based Aspen Biopharma Labs (Aspen) in March 2025 following its inspection of the company’s facility in Malakpet Village, Rangareddy, in Hyderabad, India. The inspection took place Sept. 16–21, 2024. According to the warning letter, FDA inspectors found significant deviations from current good manufacturing practice (CGMP) in the production of APIs (1).

“Because your methods, facilities, or controls for manufacturing, processing, packing, or holding do not conform to CGMP, your API are adulterated within the meaning of section 501(a)(2)(B) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), 21 U.S.C. 351(a)(2)(B),” FDA stated in its March 5, 2025 letter (1).

Deviations found during the inspection include, but are not limited to:

a failure in the design and construction of the buildings and facilities used to manufacture the API in a way that minimizes potential contamination and facilitates appropriate cleaning, maintenance, and operations

failure to demonstrate that the company’s manufacturing process can manufacture an API in a reproducible manner while meeting its predetermined quality attributes and failure to adequately validate written procedures for the cleaning and maintaining equipment

failure to provide records, either originals or copies, that are readily available and promptly retrievable during the retention period

failure to design a documented, ongoing stability testing program that monitors the stability characteristics of API and to use the results for confirming appropriate storage conditions as well as for retest or expiry dates (1).

FDA’s warning to Aspen is the latest in a series of warning letters the agency has issued this year to India-based API manufacturers. In February 2025, the agency separately issued warning letters to 

 (Tyche) regarding deviations from CGMP for APIs (2,3).

In the warning letter to Jagsonpal, FDA based its observations on an inspection conducted on March 20, March 27, and April 3, 2024 at the company’s facility in Bhiwadi Dist., Alwar, in Rajasthan, India. During its inspection, the agency observed a failure of the company’s quality unit to exercise its responsibility to ensure compliance with CGMP in the manufacture of the API produced at the company’s contract facility. In addition, the inspector was denied entry by persons present at the site upon arrival on March 15, 2024, only to be granted entry on March 20, 2024; however, during inspection, access to requested documents was limited. Finally, a (b)(4) drug was manufactured at an unregistered facility and was not listed by the company as required under 

)Title 21, Part 207.41(a) (4), which caused the (b)(4) drug to be misbranded under section 502(o) of the FD&C Act, 21 U.S.C. 352(o).

“[F]ailure to provide current listing information in accordance with 510(j) of the FD&C Act is prohibited under section 301(p) of the FD&C Act, 21 U.S.C. 331(p),” FDA stated in the letter (2).

Meanwhile, the warning letter to Tyche was based on an inspection conducted at this company’s Sarpavaram, Kakinada, India, manufacturing plant on Aug. 12–16, 2024. In this letter, the FDA inspector observed several deviations, including but not limited to:

a failure to record all quality-related activities at the time these are performed

failure to clean equipment and utensils to prevent contamination or carry-over of any material that can alter the quality of the APIs produced beyond the official or other established specifications

failure to test the identity of each batch of incoming production material (3).

Warning Letter: Aspen Biopharma Labs Private Limited

, March 5, 2025 (accessed March 27, 2025).
2. FDA. 

Warning Letter: Jagsonpal Pharmaceuticals Limited

 Title 21, 207.41(a) (Government Printing Office, Washington, DC), 

https://www.ecfr.gov/current/title-21/chapter-I/subchapter-C/part-207/subpart-D/section-207.41

Articles

In the latest of a series of warning letters to India-based API manufacturers, FDA issued a warning letter to Aspen for deviations in CGMP in the production of APIs.

FDA Issues Warning Letter to Aspen Biopharma Labs for API Manufacturing Deviations

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

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The latest tariffs to be announced by President Donald Trump’s administration, on March 27, 2025, are a 25% tax on auto imports, adding to 25% tariffs on Mexico and Canada—with a lower 10% tax on Canadian energy products—a 20% import tax on China, and 25% tariffs on steel and aluminum imports (1).

 the Administration may be targeting pharmaceutical drugs, along with computer chips, lumber, and copper, as one of its next areas where stiff taxes may be imposed.

Just before the new auto import tariffs were announced, the Biotechnology Innovation Organization (BIO), a Washington, DC-based trade association, released on March 26, 2025 results of a membership survey evaluating the health of the global biomedical supply chain as it relates to the potential for higher tariffs (2).

In analyzing the responses to the survey, BIO said that almost 90% of biotech companies based in the United States said they rely on imported components for at least half of their products that are FDA approved (2). Given that statistic, a press release from BIO outlined three key tariff concerns expressed in the survey:

 This was an across-the-board concern in the survey, as 94% of biotech firms said they anticipated “surging” manufacturing costs if tariffs are imposed on imports out of the European Union (EU).

 Proposed tariffs on the EU could also, according to the survey, force approximately half of companies to find new research and/or manufacturing partners—with half of survey respondents saying they would either have to redraw or possibly delay their regulatory filings.

 Four out of five (80%) biotech firms surveyed say they would need at least a year to find alternate suppliers, and nearly half (44%) report that they would theoretically need two years or more (2).

"This survey demonstrates the far-reaching and potentially damaging impacts of the proposed tariffs on our biotechnology industry, on biomedical research and on patients,” BIO President and CEO John F. Crowley said in the release. “We fully support strong policies and programs that incentivize the manufacture of medicines here in America. Re-onshoring key parts of the biotechnology supply chain to the US and our allies and strengthening the American manufacturing base should be a high priority for both national and economic security. It will take years, though, for this shift, and we need to be mindful of the negative consequences of these proposed tariffs. We look forward to working with the Administration and Congress to develop incentives and policies that drive private sector dollars to spur a renaissance of US biotech manufacturing."

® took a look at how the industry might respond to tariffs 

 of the new Trump Administration (3). Since then, more experts have weighed in.

 Joseph Principe, chief commercial officer at Abzena, discussed the impact of the geopolitical climate on the bio/pharma industry (4). That interview can be viewed in full at 

 series, from March 20, takes a look at these impacts (5). 

1. Boak, J. Trump Places 25% Tariff on Imported Autos, Expecting to Raise $100 billion in Tax Revenues. 

, March 27, 2025.
2. Biotechnology Innovation Organization. New Survey: U.S. Biotechs Warn Tariffs Could Impede Access to Cures, Stifle Innovation. Press Release. March 26, 2025.
3. Lavery, P. 

Pharmaceutical Industry Responds to Proposed Trump Administration Tariffs.

DCAT Week 2025: Impact of Geopolitical Climate on Biopharma.

Behind the Headlines: Episode 13; Budget Cuts, Reshoring, and Biotech Buyouts.

The Biotechnology Innovation Organization’s new membership survey said that 90% of US biotech companies rely on imported components for at least half of their FDA-approved products.

BIO Survey: Tariffs May Reduce Access, Delay Innovation, Create Red Tape

Novartis has strengthened its presence in Slovenia following the official opening in February 2025 of a new viral vector production facility (VIFA One) in Mengeš. Novartis invested €40 million (US$43 million) into the facility, which is the company’s first specialized viral vector manufacturing facility in Europe (1). With this investment, Novartis significantly expands its research and production capabilities in the biopharmaceutical sector.

The facility will support the production of viral vectors for breakthrough cell and gene therapies (CGTs) developed for treating certain autoimmune and genetic diseases and cancers. The facility features a fully automated production environment, including advanced robotics, designed to ensure precision and efficiency at all stages of the production process under one roof. The facility also introduces a state-of-the-art methodology aimed at setting a new industry standard but also significantly increasing Novartis’ capabilities to deliver breakthrough therapies under the highest quality standards (1).

Construction on the new facility began in 2023 and represents a significant boost to both the Slovenian economy and the country’s scientific community. "Novartis investments, which are among the leading foreign direct investments in the country, have made a significant contribution to the development of Slovenian science over the past two decades,” said Igor Papič, PhD, Minister of Higher Education, Science, and Innovation of the Republic of Slovenia, during the facility’s opening ceremony in February, where he was the keynote speaker (1). “I am pleased that Novartis is successfully cooperating with universities and scientific research institutions, and that it places great emphasis on developing young talent and transferring knowledge into practice. Cooperation between business and science and a stimulating scientific research and innovation environment, which is also supported by the State with generous funding, are key to the successful development of Slovenian society. They also create an environment that will be even more attractive to foreign investment."

The opening of the Mengeš facility follows a cycle of intense investment by Novartis in Slovenia. In 2024, Novartis’s investments in the country had a total value of €257 million (US$277 million) and mainly focused on the construction of new facilities and the introduction of new technologies (2).

A combination of the new investments and growth in business activity resulted in the need for new employees, leading to a growth in workforce in Slovenia in 2024. In that year, Novartis hired more than 560 new staff in Slovenia, bringing its total workforce in the country to more than 3900 employees by year’s end. Novartis also signed a strategic partnership with the University of Ljubljana, which further strengthened the company’s cooperation with academia and contributes to the joint development of knowledge and innovation (2).

“Novartis globally invested US$9.3 billion in research and development in 2024,” said Aymeric Royere, Novartis Slovenia country president, in a Jan. 31, 2025 company press release (2). “This shows our commitment to continue bringing new innovative medicines to patients worldwide in therapeutic areas where there are still significant unmet treatment needs. At Novartis in Slovenia, we also ensured that all key Novartis medicines, including the newly launched innovative medicines, have been available to patients in Slovenia.”

Slovenia’s Ljubljana and Mengeš locations support more than half of Novartis priority medicines in various stages of development, production, or services, Novartis noted in its Feb. 5, 2025 press release (1). "Slovenian experts significantly contribute to technological advancement and innovation in the research, development, and production of Novartis innovative medicines, addressing the greatest health challenges in society,” said Steffen Lang, PhD, member of the executive committee, and president, Operations, Novartis, in the press release (1). “Their knowledge, expertise, and achievements have been recognized and valued by Novartis from the very beginning and represent the fundamental reason for continuous further investments in Slovenia. One of these is the new viral vector production facility, which, as the only such Novartis production facility in Europe, has significant strategic value.”

Since entering the Slovenian market, Novartis has invested approximately €3.5 billion (US$3.8 billion) in the country, aimed primarily at research, development, and production capacities as well as sustainable environmental technologies. Investments totaling approximately €500 million (US$541 million) are currently underway at the Ljubljana and Mengeš locations, furthering the company’s growth in Slovenia.

In Slovenia, Novartis Opens Its First Specialized Viral Vector Production Facility in Europe

. Press Release. Feb. 5, 2025.
2. Novartis. Novartis in Slovenia Sees a Growth in Workforce and an Enhanced Role in the Global Novartis Network in 2024. Press Release. Jan. 31, 2025.

With a completed €40 million (US$43 million) investment in Slovenia, Novartis has opened its first specialized viral vector production facility in Europe, following earlier significant investments in R&D that has led to the growth of Slovenia’s workforce.

Novartis’ Steady Investments in Slovenia Result in New Viral Vector Manufacturing Facility and a Growing Work Force

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The US Department of Health and Human Services (HHS) announced on March 27, 2025 that it is restructuring the organization in response to a Trump Administration Executive Order. HHS states the restructuring will include the reduction of its workforce by 10,000 full-time employees; however, the total amount of workforce reduction will be closer to 20,000 when combined with other agency efforts such as early retirement and Fork in the Road (1).

The restructure plans are to streamline functions and remove redundant units. These areas will be consolidated into 15 new divisions, such as a new Administration for a Healthy America (AHA), and core functions will be centralized. HHS will reduce regional offices in half from 10 to 5. “The restructuring will improve Americans’ experience with HHS by making the agency more responsive and efficient, while ensuring that Medicare, Medicaid, and other essential health services remain intact,” the agency stated in a press release.

As part of the restructuring, FDA’s staff will be reduced by approximately 3500 full-time employees, with a HHS fact sheet stating the reduction will not impact drug, device, or food reviewers or inspectors (2). Instead, HHS states the changes will streamline operations and centralize administration functions. In addition, the staff of the National Institutes of Health will be reduced by approximately 1200 employees.

The new AHA will combine the Office of the Assistant Secretary for Health (OASH), Health Resources and Services Administration (HRSA), Substance Abuse and Mental Health Services Administration (SAMHSA), Agency for Toxic Substances and Disease Registry (ATSDR), and National Institute for Occupational Safety and Health (NIOSH). “Transferring SAMHSA to AHA will increase operational efficiency and assure programs are carried out because it will break down artificial divisions between similar programs,” the agency stated in the release.

TheAdministration for Strategic Preparedness and Response (ASPR), which is responsible for national disaster and public health emergency response, will transfer to the Centers for Disease Control and Prevention (CDC), and the Assistant Secretary for Planning and Evaluation (ASPE) will merge with the Agency for Healthcare Research and Quality (AHRQ) to create the Office of Strategy.

“We aren't just reducing bureaucratic sprawl. We are realigning the organization with its core mission and our new priorities in reversing the chronic disease epidemic,” HHS Secretary Robert F. Kennedy, Jr., stated in the press release (1). “This Department will do more—a lot more—at a lower cost to the taxpayer. Over time, bureaucracies like HHS become wasteful and inefficient even when most of their staff are dedicated and competent civil servants. This overhaul will be a win-win for taxpayers and for those that HHS serves. That’s the entire American public, because our goal is to Make America Healthy Again.”

HHS. HHS Announces Transformation to Make America Healthy Again. Press Release. March 27, 2025.

HHS. Fact Sheet: HHS’ Transformation to Make America Healthy Again. HHS.gov (accessed March 27, 2025).

 https://www.hhs.gov/about/news/hhs-restructuring-doge-fact-sheet.html

In response to a presidential Executive Order, the Department of Health and Human Services is planning to reduce its workforce, including cutting FDA staff by 3500 full-time employees, and reorganize some key departments.

HHS Announces Restructuring

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Pharmaceutical process development and API manufacturing company CARBOGEN AMCIS has successfully completed a first inspection by the French regulatory authority Agence nationale de sécurité du médicament et des produits de santé (ANSM) at its state-of-the-art sterile drug product manufacturing site in Saint-Beauzire, France, the company announced on March 24, 2025 (1). The inspection was part of a routine process conducted by ANSM to gauge good manufacturing practice (GMP) compliance, and as such, CARBOGEN AMCIS has been granted a GMP certificate for the French facility.

“This achievement reflects our team's dedication and hard work,” said Angie Stevens, vice president of CARBOGEN AMCIS’ Drug Product Business Unit, in a company press release (1). “With this certification, we look forward to actively supporting our customers in bringing new drugs to market and shaping a bright future together.”

The certificate permits the Saint-Beauzire facility to manufacture and release clinical and commercial sterile drug products; ANSM had granted the site authorization to manufacture, test, and release drug products upon its February 2023 opening, with the first clinical batch released in January 2024 (1).

“The successful ANSM inspection reaffirms CARBOGEN AMCIS as a reliable partner in the drug product manufacturing market,” said Helen Caddy-Leach, head of Business Development, Drug Product, in the release (1). “With our extensive expertise and a state-of-the-art GMP-certified facility, we are well-positioned to support customers’ drug product projects with excellence.”

“Having a cutting-edge facility dedicated to the development and manufacture sterile drug products, including to a commercial scale, strengthens our ability to offer comprehensive end-to-end services, from drug substance to drug product,” said Stephan Fritschi, CEO of CARBOGEN AMCIS, also in the release (1). “This integrated approach is a significant advantage in today’s competitive pharmaceutical landscape and provides added value for our clients.”

The Saint-Beauzire site traces its roots back to August 2020, when CARBOGEN AMCIS 

 (now US$113 million) investment to open two new manufacturing facilities, one in its home country of Switzerland focusing on APIs, and the other, the company said at the time, located in France and concentrating on custom development and manufacturing of parenteral drug products (2).

In September 2024, Caroline Ailhas, Pharmaceutical Development director for CARBOGEN AMCIS, 

 eBook, on the topic of prioritization of formulation strategies for temperature-sensitive biotherapeutics (3).

Ailhas explained that maintaining the structure and functionality of a molecule and defining the required duration of stabilization are the main factors challenging formulation development for temperature-sensitive molecules. The latter can vary from a few days to months, or even years. “The definition of this duration significantly impacts the formulation strategy,” Ailhas said (3).

Elsewhere in France in the first quarter of 2025, 

 a €50 million (US$52 million) investment in its site in Mourenx, with the intention of making the site a center of excellence within the company’s network for contract manufacturing of APIs and drug substances for biologics that have been FDA approved (4). That followed a previous €7.3 million (now US$7.6 million) investment, made in the Mourenx site in August 2022, to create a pilot workshop supporting a growing demand for APIs, particularly highly potent APIs to treat cancer.

CARBOGEN AMCIS said the Saint-Beauzire site completes its group portfolio, which includes seven other sites across France, Switzerland, the United Kingdom, China, and The Netherlands offering current GMP (CGMP)-compliant technologies designed to help bio/pharmaceutical companies bring next-generation medicines to market (1).

1. CARBOGEN AMCIS. CARBOGEN AMCIS Secures GMP Certification After Successful ANSM Inspection for its Aseptic Drug Product Manufacturing Site. Press Release. March 24, 2025.
2. CARBOGEN AMCIS. CARBOGEN AMCIS Announces Major Investments of More Than CHF 100 million in Switzerland and France. Press Release. Aug. 17, 2020.
3. Mirasol, F. Prioritizing Formulation Strategies for Temperature-Sensitive Biotherapeutics. 

Pharmaceutical Technology Trends in Formulation

 eBook September 2024.
4. Axplora. Axplora Announces €50 Million Investment in GLP-1 Manufacturing at Mourenx (France) Site. Press Release. Feb. 12, 2025.

The company’s state-of-the-art sterile drug product manufacturing site in Saint-Beauzire, France, opened in February 2023.

CARBOGEN AMCIS Receives GMP Certification Following Successful ANSM Inspection

The Texas-based contract development and manufacturing organization Benuvia Operations has been awarded a five-year contract by the National Institutes of Health (NIH)’s National Institute on Drug Abuse (NIDA) for good manufacturing practice (GMP) synthesis of the naturally occurring psychedelic compound psilocybin, which comes from certain types of mushrooms, as well as other bulk drug substances (1).

Benuvia said in a press release on March 25, 2025 that the contract supports R&D of high-quality APIs for substance use disorders and mental health conditions, initially focusing on synthesis and scale-up of psilocybin, which is a Schedule I substance under the Controlled Substances Act (1). Psilocybin was granted breakthrough therapy designation by FDA in 2018 for treatment-resistant depression, and 2019 for major depressive disorder.

“This contract highlights our leadership in the development and manufacturing of controlled substances under GMP conditions,” Terry Novak, Benuvia CEO, said in the press release (1). “We are honored to support NIH’s efforts in advancing groundbreaking research into innovative therapies for substance use disorders and mental health conditions. Our state-of-the-art capabilities enable us to supply the highest-quality pharmaceutical compounds that meet the most rigorous regulatory standards.”

The contract stipulates that Benuvia Operations will manufacture and deliver GMP-grade psilocybin for NIDA preclinical toxicity studies as well as clinical trials (1). Clinical studies are currently evaluating psilocybin as a potential treatment for depression, anxiety, and substance use disorders.

“Our mission at Benuvia is to provide high-purity, federally approved substances for scientific research and pharmaceutical development,” Novak said. “We look forward to working with the NIH to help evaluate psilocybin’s therapeutic potential for the millions of patients suffering from these conditions.”

Previously, on March 11, 2025, Benuvia Operations announced that it 

 (Certificado de Boas Práticas de Fabricação [CBPF]) from Brazil’s National Health Surveillance Agency (ANVISA), such certification being essential, according to the company, as a regulatory prerequisite for pharmaceutical companies to manufacture and distribute products in Brazil (2).

“Achieving ANVISA’s CBPF certification underscores Benuvia’s strategic commitment to providing high-quality, compliant pharmaceutical manufacturing specifically tailored to Brazil’s rapidly growing healthcare market,” Novak said at the time (2). “ANVISA CBPF certification allows us to be the preferred provider for companies seeking a partner with quicker market entry, reduced regulatory hurdles, and stronger credibility among regulatory bodies, healthcare providers, and pharmaceutical customers.”

“Psychedelic Drugs: Considerations for Clinical Investigations,”

 concerning the use of such drugs—psilocybin among them—for treatment of various medical conditions, including psychiatric and substance use disorders (3).

“Psychedelic drugs show initial promise as potential treatments for mood, anxiety and substance use disorders. However, these are still investigational products,” Tiffany Farchione, director, Division of Psychiatry, Center for Drug Evaluation and Research, FDA, said in a press release announcing the draft guidance (3). “The goal is to help researchers design studies that will yield interpretable results that will be capable of supporting future drug applications.”

The other bulk drug substances beside psilocybin that will be future areas of focus for Benuvia under the contract with NIDA were not disclosed by name in the Benuvia press release (1).

1. Benuvia Operations. Benuvia Operations Awarded NIH Contract for GMP Synthesis of Psilocybin and Other Bulk Drug Substances. Press Release. March 25, 2025.
2. Benuvia Operations. Benuvia Operations Granted GMP Certification from Brazil National Health Surveillance Agency (ANVISA). Press Release. March 11, 2025.
3. FDA, 

Draft Guidance for Industry, Psychedelic Drugs: Considerations for Clinical Investigations

The contract will support R&D of high-quality APIs for substance use disorders and mental health conditions, starting with synthesis and scale-up of psilocybin, which is derived from mushrooms.

Benuvia Awarded NIH Contract for GMP Synthesis of Psilocybin, Other Drug Substances

AstraZeneca has entered into an exclusive license agreement with Alteogen, a Korea-based bio venture company, in which AstraZeneca will gain access to Alteogen’s novel hyaluronidase-utilizing Hybrozyme platform technology, ALT-B4. Under the agreement, AstraZeneca will acquire worldwide rights to use ALT-B4 for the development and commercialization of subcutaneous formulations of several oncology assets in AstraZeneca’s portfolio (1).

Through the agreement, Alteogen will be responsible for clinical and commercial supply of ALT-B4 to AstraZeneca, and AstraZeneca will make an upfront payment to Alteogen as well as additional payments should specific development, regulatory, and sales-related milestones be achieved. Alteogen will also receive royalties on the sales of commercialized products.

“We are dedicated to advancing new medicines for people with cancer and that includes new methods of delivery which are more convenient for patients, physicians and healthcare systems,” said Cristian Massacesi, chief medical officer and Oncology chief development officer, AstraZeneca, in a March 17, 2025 company press release (1). “We look forward to collaborating with Alteogen on several assets in our portfolio with the goal of bringing new subcutaneous options to patients that can transform the way cancer care is delivered.”

Alteogen’s ALT-B4 is a proprietary human recombinant hyaluronidase enzyme that was developed using Hybrozyme technology. ALT-B4 is designed to enable the large-volume subcutaneous administration of parenteral drugs typically administered as an intravenous infusion. ALT-B4 works by temporarily hydrolyzing hyaluronan within the extracellular matrix. Hyaluronan is a major component of the extracellular matrix that plays a key role in regulating inflammation (2). Developing subcutaneous formulations for oncology therapies offers potential advantages, including time savings for patients, clinical staff, and health systems as a result of shorter administration times (1).

“We are excited to expand our Hybrozyme [t]echnology by collaborating with AstraZeneca in their development of novel subcutaneous cancer medicines to meet the needs of patients,” said Soon Jae Park, PhD, CEO, Alteogen, in the press release. 

 in China to boost life sciences R&D and manufacturing. On March 21, 2025, the company said it plans to invest $2.5 billion in Beijing over the next five years under which it will establish a new global strategic R&D center. The investment will support both major research and manufacturing agreements that will further advance life sciences in China (3).

The investment is part of a strategic partnership that AstraZeneca has with the Beijing Municipal Government and the Beijing Economic-Technological Development Area Administrative Office. The investment includes agreements with the biotech companies Harbour BioMed, Syneron Bio, and BioKangtai.

AstraZeneca Enters License Agreement with Alteogen for Subcutaneous Formulations of Multiple Oncology Assets

. Press Release. March 17, 2025.
2. Petrey, A. C.; de la Motte, C. A. Hyaluronan, a Crucial Regulator of Inflammation. 

,101. DOI: 10.3389/fimmu.2014.00101
3. AstraZeneca. AstraZeneca to Invest $2.5 Billion in New Global Strategic R&D Centre, Biotech Agreements and Manufacturing in Beijing. Press Release. March 21, 2025.

Under the agreement, AstraZeneca will use Alteogen’s proprietary hyaluronidase platform technology to develop and commercialize subcutaneous formulations of multiple oncology assets in its portfolio.

AstraZeneca and Alteogen Sign License Agreement for Developing Subcutaneous Formulations of Oncology Assets

Oral Fabhalta (iptacopan), developed by Novartis, has been approved by FDA for treatment of adults with C3 glomerulopathy (C3G) to reduce proteinuria in those patients, the first approved treatment for that condition (1). C3G is a progressive, ultra-rare kidney disease with an average age at diagnosis of approximately 23 years old; approximately half of those diagnosed progress to kidney failure within 10 years, requiring lifelong dialysis and/or a kidney transplant.

C3G is further described by Novartis as an overactivation of a part of the immune system, the alternative complement pathway, that causes deposits of C3 protein to build up in kidney glomeruli, the blood vessel network that filters waste and removes extra fluid from blood (1). This can result in inflammation and/or glomerular damage causing proteinuria, or protein in urine, hematuria (blood in urine), and reduced function of the kidneys. The disease has previously been typically treated with a combination of supportive care, broad immunosuppression, and symptom management.

FDA has approved Novartis’ oral iptacopan treatment twice before; in December 2023, for treatment of adults with paroxysmal nocturnal hemoglobinuria, and August 2024, for reduction of proteinuria in adults with primary immunoglobulin A nephropathy who are at risk of rapid disease progression (1).

A Phase III trial (APPEAR-C3G), in which iptacopan was taken orally twice daily, showed clinically meaningful proteinuria reduction as early as 14 days, and sustained at 12 months (1).

“C3G is a debilitating disease often affecting young people, impacting many aspects of their physical and emotional health, and our previous treatment options came with significant challenges,” Carla Nester, MD, professor of Pediatrics-Nephrology at the University of Iowa, and APPEAR-C3G study co-investigator, said in a Novartis press release (1). “This approval of Fabhalta is historic for the entire C3G community as now, for the first time, we have a therapy that is believed to treat the underlying cause of the disease, providing the potential for a new standard of care for patients.”

The most common adverse reactions in the Phase III study were nasopharyngitis and viral infections; meanwhile, the symptoms not specifically tied to kidney function that patients with C3G may face include high levels of fatigue, certain mobility issues that can affect day-to-day activities, and mental health effects including depression and anxiety (1).

“As someone whose family has suffered from C3G across multiple generations, it is difficult to fully express the physical and emotional challenges of living with this unrelenting disease,” said Lindsey Fuller, C3G patient and co-leader of C3G Warriors. “To finally have an approved treatment—and one that can be taken orally—is something people with C3G have been waiting for. Today’s approval brings new hope for me, my family, and so many others.”

Prior to FDA’s decision, announced in the March 21, 2025 Novartis press release, the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) 

 extended the indication for iptacopan to include treatment of adult patients with C3G (2).

“We extend our deepest gratitude to the patients and investigators who participated in our clinical trials, without whom this first FDA approval in C3G wouldn’t have been possible,” said Victor Bultó, president US, Novartis, in the company’s press release. “With this additional approval for Fabhalta—the second in kidney disease—we will leverage our established capabilities and expertise to bring this innovative treatment to patients in need as we work to help transform care for people living with kidney diseases.”

1. Novartis. Novartis Receives Third FDA Approval for Oral Fabhalta (iptacopan)—The First and Only Treatment Approved in C3 Glomerulopathy (C3G). Press Release. March 21, 2025.
2. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 24-27 February 2025. Press Release. Feb. 28, 2025.

Fabhalta (iptacopan) received a positive opinion for treatment of C3G from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) and has been approved twice before by FDA.

FDA Approves Novartis Oral Treatment for Adults with C3 Glomerulopathy

, the discussion revolves around current and anticipated new trends in equipment and technologies for optimizing the manufacture of new biotherapeutic modalities, such as cell and gene therapies. Automation technologies and continuous manufacturing emerges as significant drivers for the future biomanufacturing landscape. Innovative and progressive technologies will also play a role in establishing and maintain sustainability practices in the industry.

Kerry Love, PhD, Co-Founder and CEO, Sunflower Therapeutics

Kerry Love, PhD, is the co-founder and CEO of Sunflower Therapeutics, a women-owned and led biotechnology company delivering next-generation protein manufacturing solutions that anyone can use to create innovative new medicines, vaccines, foods, and other bio-produced materials. Love is an organic chemist by training, performing her doctoral studies at MIT, and a biotech entrepreneur at heart, having founded two companies and contributed to the starting of many more over the past twenty years. Love has authored more than 40 scientific papers and patents.

 presents the Drug Solutions podcast, where the editors will chat with industry experts from across the pharmaceutical and biopharmaceutical supply chain. Join us as experts share insights into your biggest questions—from the technologies, to strategies, to regulations related to the development and manufacture of drug products.

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The growing development of new biotherapeutic modalities will rely heavily on automation and progressive technologies. 

Drug Solutions Podcast: Equipment Trends for Addressing Production of New Modalities

The European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA), the network of heads of regulatory authorities that are responsible for regulating human and veterinary medicines in the European Economic Area, have published their joint European Union (EU) medicines agencies’ network strategy (EMANS) to 2028, “Seizing Opportunities in a Changing Medicines Landscape,” which EMA said both its management board and HMA have adopted (1).

“I am delighted to present our joint EMA/HMA network strategy to 2028. With new legislation on the horizon, we are preparing for the most significant regulatory reform in decades,” Emer Cooke, EMA executive director, said in a press release (1). “In these uncertain times, when our environment is evolving constantly, we need to be agile and to be able to anticipate transformative changes, as well as to better address supply chain security. Innovations in the development of medicines, including the use of artificial intelligence (AI), require a significant update to our network strategy so that we can seize the opportunities that they present.”

EMANS 2028 is described by EMA as a comprehensive update of EMANS 2025, which was developed to cover the years 2021 to 2025 and has six focus areas, introducing what EMA and HMA call the “One Health Approach”:

accessibility (facilitating pathways to medicines access through the EU’s healthcare systems)

leveraging data, digitalization, and AI (with the end goals of improved decision-making, optimized processes, and increased efficiency)

regulatory science, innovation, and competitiveness (to create a research environment that accelerates the translation of innovation)

antimicrobial resistance and other health threats

availability and supply (for both humans and animals)

sustainability of the network, taking advantage of technological advances (1).

“With the strategy to 2028, our aim is to ensure a transparent, forward-looking and science-driven roadmap to managing the network's public health priorities,” Maria Lamas, chair of HMA Management Group, said in the release (1). “In the face of a new global framework, it is important that the European medicines regulatory agencies contribute to EU competitiveness. We are committed to catalyze the innovation of medicines and their manufacture by leveraging every opportunity to promote public and animal health.”

 that EMANS 2028 was open for public consultation in October 2024, a period which ended Nov. 30, 2024 (2). At the time, EMA said that new regulations (e.g., legislation to address public health emergencies and reform of the EU medicines regulations) and technical advances, such as the use of AI, were some of the main drivers for the changes to the original strategy.

The 2021–2025 EMANS was published in December 2020, and 

 that examined its achievements from January 2021 to June 2023 found that the COVID-19 pandemic, which was still in its first year at the beginning of that timeframe, strengthened the network, allowing it to be on track to meet its goals and objectives regarding drug availability, data management, innovation, antimicrobial resistance, supply chain challenges, and sustainability of the network (3). EMA said that EMANS 2028, prepared in a post-pandemic setting, draws on extensive experience gained in the EU from responding to COVID-19 (1).

The final, adopted version of EMANS 2028 is available via PDF download at 

1. EMA. Joint Strategy Sets Direction of EMA and EU Medicines Regulatory Agencies to 2028. Press Release. March 18, 2025.
2. EMA. Seizing Opportunities in a Changing Medicines Landscape. Press Release. Oct. 9, 2024.
3. EMA. Midterm Report on 2025 Network Strategy Highlights Good Progress During Critical Period of the Pandemic. Press Release. Dec. 20, 2023.

The EU medicines agencies’ network strategy, EMANS, is an update of the five-year strategy previously developed to cover the period 2021 to 2025.

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