Aligning mRNA Progress with Regulatory Streamlining

BioPharm Peer Exchange

Dosage Forms: Roundtable on Oral Solid Dosage Trends

Drug Solutions Podcast: 

Pharmaceutical packaging must advance to adapt to new, complex modalities

Out-of-the-Box Packaging Advancements

From Theory to Reality: Automating Process Development

Drug Digest: 

The Importance of Appropriate Supplier Oversight

Ask the Expert

Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

Rare Disease Day, which is recognized on the final day of February each year—hence, February 29 in leap years, the rarest date on the calendar—shines a light not only on the 

6000 or more diseases that affect some 300 million people

 around the world, but also the medicines and therapies in development to help those impacted (1).

On Feb. 28, 2025, the European Medicines Agency 

 to Vyjuvek (beremagene geperpavec), a topical gene therapy for the ultra-rare genetic skin-blistering condition, dystrophic epidermolysis, which is caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene (2).

Earlier in February, FDA granted breakthrough therapy designation to radiprodil, GRIN Therapeutics’ investigational, potent negative allosteric modulator selectively targeting the N-methyl-D-aspartate receptor subtype 2B (NR2B or GluN2B), for treatment of seizures associated with GRIN gene-related neurodevelopmental disorder with gain-of-function mutations (3).

Meanwhile, FDA’s Center for Drug Evaluation and Research’s Accelerating Rare Disease Cures program, established in 2022, released its Year Two Annual Report in October 2024, and the Learning and Education to Advance and Empower Rare Disease Drug Developers initiative continues to offer insights to those inquisitive about the design and conduct of rare disease clinical trials, according to an FDA update (4).

According to data from Elsevier’s Scopus, the amount of literature on rare diseases experienced a steady climb starting in 2018 and hit a peak in 2021 after the onset of the COVID-19 pandemic, having leveled off since then but still well above pre-2018 levels (5). The United States has been dominating other countries in terms of rare disease research output, although China is a clear second and outpaces the US in most other disease areas. And since 2020, there has been an explosive increase in inquiries as to how artificial intelligence can be used to shape this research.

Yet despite this increased focus, challenges remain when it comes to ensuring effective clinical trials for rare diseases, as outlined by Phesi in a February 2025 report (6). The five main obstacles listed in that report are: limited patient populations or difficulties with recruitment; geographic dispersion and uncertainty of investigator site selectivity; heterogeneity in disease presentation and progression; a lack of historical clinical trial data in many cases; and regulatory and/or ethical concerns.

Growing uncertainty at the federal level may also become a mounting challenge for the rare disease community. Just this week, the FDA and NIH-sponsored Rare Disease Day 2-day event was postponed (7) with little explanation in the wake of sweeping layoffs within the FDA and NIH. The event "seeks to bring together a broad audience including patients, patient advocates, caregivers, health care providers, researchers, trainees, students, industry representatives, and government staff," according to the event website, but the postponement has left many in the community concerned about the future support for rare diseases. 

This adds to growing concern about future federal support for rare disease research and drug development, as the Rare Pediatric Disease Designation and Priority Review Voucher Programs also ended in December 2024 and were not renewed (8), leaving little financial motive for pharmaceutical companies to fund rare disease drug development. 

Rare Disease Day Celebrated on February 28.

, Feb. 27, 2025.
2. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 24–27 February 2025. Press Release. Feb. 28, 2025.
3. GRIN Therapeutics. GRIN Therapeutics Receives FDA Breakthrough Therapy Designation for Radiprodil. Press Release. Feb. 25, 2025.
4. FDA – CDER. FDA Continues to Make Inroads in Rare Disease Drug Development. Press Release. Feb. 28, 2025.
5. Elsevier – Scopus – Rare Disease Research. Received via email correspondence on Feb. 27, 2025.
6. Phesi Rare Disease Showcase, 

5 Challenges of Delivering Effective Clinical Trials for Rare Diseases—And Strategies for Overcoming Them

; Phesi, February 2025.
7. FDA-NIH Rare Disease Day 2025. FDA.gov. Updated February 25, 2025. https://ncats.nih.gov/news-events/events/rdd
8. Rare Pediatric Disease Designation and Priority Review Voucher Programs. FDA.gov. Update September 27, 2024. https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/rare-pediatric-disease-designation-and-priority-review-voucher-programs

Articles

Regulatory clearances and approvals continue as agencies work to disseminate literature, raise awareness, redouble research efforts, and improve the efficacy of clinical trials.

Rare Disease Day 2025: Pharmaceutical Treatments Advancing in the US, EU

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The European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) recommended marketing authorization approval for four medicines in February 2025. The recommended drugs treat a number of types of cancer, immunodeficiencies, and a rare genetic skin blistering disease.

Conditional marketing authorization was given to Lynozyfic (linvoseltamab) for the treatment of the bone marrow cancer, relapsed and refractory multiple myeloma. The generic drug, Trabectedin Accord (trabectedin), was granted approval to treat advanced soft tissue sarcoma and relapsed platinum-sensitive ovarian cancer.

Deqsiga (human normal immunoglobulin) was granted approval as areplacement therapy for primary or secondary immunodeficiencies and immunomodulation in patients with certain autoimmune diseases.

A treatment for the ultra-rare genetic skin blistering condition, dystrophic epidermolysis bullosa, was given a positive opinion. The orphan drug, Vyjuvek (beremagene geperpavec), went through the agency’s PRIority MEdicines scheme, which offers early and enhanced regulatory support. Dystrophic epidermolysis bullosa is caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene.

In addition to the new drug approval recommendations, CHMP extended therapeutic indications for 16 drugs, including Abrysvo, Calquence, Columvi (an orphan drug), Darzalex (an orphan drug), Enhertu, Imfinizi, Jaypirca, Prevymis (an orphan drug), Rinvoq, Stelara, Supemtek Tetra, and Tremfya.

The indications for Kaftrio (ivacaftor/tezacaftor/elexacaftor) and Kalydeco (ivacaftor) were extended to include patients aged two years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “This positive opinion brings disease-modifying treatment to all cystic fibrosis patients with modulator responsive mutations,” the agency said in a press release (1).

Ixchiq’s (chikungunya vaccine (live)) indication was extended to include immunization for adolescents 12 years and older to protect against the Chikungunya virus, a viral disease transmitted to humans by infected mosquitoes. 

The orphan drug, Fabhalta (iptacopan), had its indication extended to include treatment of adult patients with the ultra-rare kidney disease, complement 3 glomerulopathy.

At the meeting, CHMP also provided the results from its re-examination of the initial opinion for the indication extension for Keytruda (pembrolizumab) to treat unresectable non-epithelioid malignant pleural mesothelioma. The committee’s original decision was upheld. CHMP also held up its rejection of marketing authorization for Kizfizo (temozolomide) to treat neuroblastoma.

EMA also noted that the application for Pelgraz Paediatric (pegfilgrastim), for the treatment of neutropenia and prevention of febrile in children with cancer was withdrawn. The application of Rilonacept FGK Representative Service GmbH (rilonacept) to treat idiopathic pericarditis was also withdrawn.

The application to extend the indication of Dupixent to treat moderate-to-severe chronic spontaneous urticaria in adults and adolescents was withdrawn as well. In addition, the authorization holder for the COVID-19 vaccine, Bimervax, withdrew the application to include an adapted version to target the JN.1 strain of SARS CoV-2.

At the February meeting, CHMP also stated that the marketing authorization for Leqembi (lecanemab), which received a positive opinion in November 2024 for the treatment of mild cognitive impairment or dementia due to Alzheimer’s disease, does not need to be updated. “In January 2025, the European Commission (EC) asked the committee to consider information on the safety of Leqembi that became available after the adoption of the positive opinion and whether this would require an update of their recommendation. The CHMP has now concluded that its opinion recommending the marketing authorization does not need to be updated and has provided a response to the EC, which will now resume the decision-making process for this medicine,” EMA stated in the press release.

EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 24-27 February 2025. Press Release. Feb. 28, 2025.

Treatments for autoimmune diseases, multiple cancers, and skin blistering have been recommended for marketing approval.

EMA Recommends Four Medicines for Approval in February

Felicity Thomas is the former Associate Editorial Director of Pharmaceutical Technology, Pharmaceutical Technology Europe, BioPharm International.

Industry Outlook 2025: Interesting Relations and Detailed Discussions on Data

The political landscape in the United States has shifted, and while industry is learning about the various changes and new appointments, the full impact is hard to predict, not least because of the continuation effect from previous governments, points out Preeya Beczek, managing director and co-founder of Beczek.COM Ltd. However, there are three or four areas that she believes will be of potential interest with the new administration in place.

“I'm quite interested to think about China relations with the US and this BIOSECURE Act. I think that's going to perhaps impact the import tariffs, and then this could have a ripple effect on the global pharma industry, pharma economy,” Beczek explains. “The second thing, and this is not a new thing, is around anti-vaccine, anti-pharma sentiments, perhaps with the RFK [Robert F. Kennedy, Jr.] influence.”

Additionally, there is also discussion around the cost of drugs and provision of care and privatization that will continue throughout 2025, Beczek adds. “And then this final point I'm going to make is around digital health, health technology, AI [artificial intelligence], and mix that in the balance with data privacy,” she says.

Looking across the Atlantic, towards Europe, which has also undergone political shifts in 2024, industry is still working to get to grips with recent legislative changes, such as the new pharmaceutical legislation and the European Union clinical trials regulation, Beczek remarks. “So, coming back to that thing around information, data security, and what data [are] out in the public domain, right? There're still decisions that need to be made there at the very detailed level, and companies can expect to have detailed discussions around this,” she says.

Preeya Beczek, Managing Director and Co-Founder of Beczek.COM Ltd

Preeya Beczek is Managing Director and Co-Founder of Beczek.COM Ltd, a specialist in regulatory affairs and compliance. Preeya has worked in the regulatory affairs and compliance space for over 25 years, focusing on regulatory affairs activities at a strategic and operational level. Prior to her current role, Preeya worked in industry performing a lot of infrastructure and process development to support companies with new systems, initiatives with AI automation, regulatory, information management, system software modernization, and so on.

Videos

Preeya Beczek from Beczek.COM gives her thoughts on the areas to watch with the new US administration and how Europe might be finalizing preparations for previous legislative changes in an interview with Pharmaceutical Technology®.

Global pharmaceutical manufacturer Eli Lilly and Company, whose headquarters are in Indianapolis, Indiana, recently announced that it will be building four new domestic manufacturing sites in the United States, less than a month 

after President Donald Trump announced the intention to impose steep tariffs

 on imports from Canada, Mexico, and China (1,2).

The news from Lilly came the same day that Trump also threatened a 25% tariff on goods imported from the European Union, the 

After Trump announced the initial penalties on Canada, Mexico, and China on February 1—against which only the latter country has taken effect so far, pending ongoing negotiations—the Brookings Institution predicted that if the Canada and Mexico tariffs wind up being imposed, pharmaceutical exports from Mexico could fall by 61% (4). Should Canada and Mexico eventually enact retaliatory tariffs, as China has done, Brookings estimates the dropoff to climb to 72%. In addition, The Council on Foreign Relations calculated that US exports of vaccines, blood antisera, toxins, and cultures could collectively be one of the top five export categories most affected by the tariffs to the tune of $5 billion in exports to China, $2 billion to Canada, $994 million to Mexico, and $41 billion to the rest of the world (5).

In Lilly’s announcement on February 26, company chair and CEO David A. Ricks said an incentive passed during the prior Trump Administration was the impetus for the company’s decision to expand nationally—provided that incentive is continued during Trump’s current term.

"The Tax Cuts and Jobs Act legislation passed in 2017 during President Trump's first term in office has been foundational to Lilly's domestic manufacturing investments, and it is essential that these policies are extended this year,” Ricks said (1). “We believe that our investments in America and upskilling our nation's workforce will spark a significant ripple effect. For every job we create, many more will be generated, positively impacting the communities that host our innovative new sites."

Previous total domestic capital expansion commitments, made from 2020 to 2024, included both Research Triangle Park and Concord, in North Carolina; at the LEAP Innovation District in Lebanon, Indiana, the site of a new Lilly Medicine Foundry; Kenosha County, Wisconsin, where a manufacturing site has been acquired and expanded; and the company’s home city of Indianapolis, throughout which expansions and updates were made to multiple manufacturing facilities (1).

Three of the new sites will focus on API manufacturing, particularly bolstering small-molecule chemical synthesis capability and strengthening Lilly’s supply chain (1). The fourth is intended to extend Lilly’s global parenteral manufacturing network for future injectable therapies, according to the press release.

Negotiations on where the sites will be located are currently ongoing, and those locations should be announced later in 2025.

In total, Lilly’s capital expansion commitments in the US since 2020 now exceed $50 billion, with the February 26 announcement representing more than half that figure, as commitments from 2020 through 2024 totaled $23 billion (1).

Lilly said roughly 13,000 high-wage jobs could be created through the new sites: 3000 for highly skilled workers at the sites, when completed, among them engineers, scientists, operations personnel, and laboratory technicians (1). Close to another 10,000 construction jobs are expected to be needed.

"To deliver on our big bets on next-generation modalities like small molecules, biologics, and nucleic acid therapies, Lilly is investing in the state-of-the-art manufacturing infrastructure needed to deliver tomorrow's safe and reliable medicines," said Edgardo Hernandez, executive vice president and president of Lilly Manufacturing Operations, in the press release (1). "We are not just building facilities. We are creating a future where American innovation leads the world in pharmaceutical manufacturing, requiring a highly skilled workforce prepared to shape the future of health care. This is a significant step for our company, our communities, and the patients we serve."

1. Eli Lilly and Company. Lilly Plans to More Than Double US Manufacturing Investment Since 2020 Exceeding $50 Billion. Press Release. Feb. 26, 2025. https://lilly.mediaroom.com/2025-02-26-Lilly-plans-to-more-than-double-U-S-manufacturing-investment-since-2020-exceeding-50-billion
2. The White House. Fact Sheet: President Donald J. Trump Imposes Tariffs on Imports from Canada, Mexico and China. 

, Feb. 1, 2025 (accessed Feb. 4, 2025). https://www.whitehouse.gov/fact-sheets/2025/02/fact-sheet-president-donald-j-trump-imposes-tariffs-on-imports-from-canada-mexico-and-china/
3. Politi, J. and Inagaki, K. Donald Trump Threatens to Impose 25% Tariffs on EU Goods. 

, Feb. 26, 2025. https://www.ft.com/content/2f0288f6-3f6a-4334-b666-3f0122981842
4. Meltzer, J.P. Trump’s 25% Tariffs on Canada and Mexico Will Be a Blow to All Three Economies. 

, Feb. 3, 2025 (accessed Feb. 4, 2025). https://www.brookings.edu/articles/trumps-25-tariffs-on-canada-and-mexico-will-be-a-blow-to-all-3-economies/
5. O’Neil, S. K. and Huesa, J. What Trump’s Trade War Would Mean, in Nine Charts. 

, Feb. 1, 2025 (accessed Feb. 4, 2025). https://www.cfr.org/article/what-trumps-trade-war-would-mean-nine-charts

Previous domestic capital expansion commitments since 2020 had been made in Research Triangle Park and Concord, both in North Carolina; Kenosha County, Wisconsin; Lebanon, Indiana; and Lilly’s home city of Indianapolis.

In Wake of Trump Tariffs, Lilly Plans New US Pharmaceutical Manufacturing Sites

businessman working with sign of the top service Quality assurance, Guarantee, Standards, ISO certification and standardization concept. | Image Credit: ©Looker_Studio– stock.adobe.com

Process development is a key factor in the development and manufacture of biologics. Processes must be developed and validated so that product specifications can be met to create a safe and effective biologic drug. Contract development and manufacturing organizations (CDMOs) offer sponsor companies the expertise to develop these processes, which then must be validated. Process validation is a necessity to be sure the manufacturing process produces the desired, safe product.

For cell culture processes, process validation must be performed before good manufacturing practice (GMP) batches can be produced for commercial production, according to Daniel Giroux, vice-president of Biologics Development at Abzena. Formal process characterization studies must then be performed before the process validation so that one can understand the impact of variations of product parameters from their targets. Small-scale studies are performed on a validated scale-down model, and the information obtained is then applied to large-scale production.

Characterization and Qualification of Processes

Bio/Pharma Outsourcing Innovation eBook 2025

“You need to understand what are allowable ranges of process parameters and what will have an impact on your process or on the product quality, and those [parameters] that won’t [have an impact]. Then you define your process envelope …the combination of all these parameters, such as agitation, temperature, feed timing, feed amounts, and so on. [These are determined with] design-of-experiment approaches, because you have to measure [the variations’] impacts, both individually, as they change, but also when they change in combination,” says Giroux.

After process characterization and studies are performed and acceptable ranges established, batch records can be drafted, and validation protocols can be created, says Giroux. “And then you have to run multiple at-scale batches, so you know if your commercial process is going to be at the 2000-L scale, then your validation protocol will dictate how many batches you want to run. A typical number is three. It doesn’t have to be three. It could be more. Possibly could be less, rarely,” Giroux explains. “And then you execute your production batches, and you analyze heavily the impact on the product that’s coming out. You do more analysis than you normally would, something like this, and then you basically show that you met the acceptance criteria that you set up in your validation protocol.”

An essential part of process characterization is the establishment of critical process parameters (CPPs), according to Bob Schrock, PhD, senior director, Global Head of Process Development, at Lonza. Proposed critical process parameters (PCPPs) are obtained after process characterization studies are carried out.

“Once we do all the process characterization studies—and there can be literally thousands and thousands of samples, and [you have] done 12 or 15 different process characterization studies—the results are discussed with PD (process development) and MSAT (manufacturing, science, and technology) and from these, the actual CPPs can be determined,” says Schrock. “These CPPs are integrated into the process, and the validation of the process is then basically what we call the PPQ [process performance qualification] runs, the PPQ runs that are done just prior to commercialization. For [much of the] processes, the cell culture portion is only part of the process, for example, [with] viral vector manufacturing. There’s, of course, purification steps downstream from the [cell] culture portion.”

According to Schrock, PPQ runs show that a process is validated and robust and the number of runs needed depends on the variability of the process. “For a viral vector program that uses a large and well-characterized cell bank, you can do the minimum of three runs if they run perfectly. But for something [such as] autologous cell therapy, where you are starting with different cells from patients or healthy donors, you may need to do more,” says Schrock. “And then, of course, the question of using healthy donors to validate a process that is intended to start with patient samples and patient leukopaks packs, that’s always a deep and spirited discussion where you need to get creative on how you justify that. But at the end of your PPQ runs, you write a huge report that’s part of your BLA [biologics license application] submission, and it’s a cornerstone of your BLA. [It] proves that your process is validated.”

Pharmaceutical Technology®/Pharmaceutical Technology® Europe/BioPharm International

®
eBook: Bio/Pharma Outsourcing Innovation 2025
February 2025
Pages: 22–24

When referring to this article, please cite it as Haigney, S. Characterization and Qualification of Processes. 

Pharmaceutical Technology Bio/Pharma Outsourcing Innovation

Contract manufacturers have special expertise in the characterization and validation of biopharmaceutical manufacturing processes.

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Cosette Pharmaceuticals, of Bridgewater, NJ, announced on Feb. 20, 2025 that it has an agreement in place to buy all remaining shares of Australia-based Mayne Pharma Group (1). The current price per share is AUD$7.40, which equates to approximately US$4.69; when the transaction is completed, the total consideration will be an approximate US$430 million.

In a press release, Cosette said that the acquisition will strengthen its dermatology and women’s health businesses, furthering its pharmaceutical footprint in the United States while also asserting a presence in international markets (1). Chiefly, Cosette said it wants to expand access to women’s health therapies. The company said that upon the close of the deal, it will be able to market 12 patent-protected products centering on women’s health and dermatology—some well-known brand names Cosette mentioned in the press release include Annovera, Bijuva, Imvexxy, Intrarosa, Nextstellis, Rhofade, and Vyleesi—plus have numerous programs in clinical development.

“This acquisition marks a transformational step for Cosette, adding patented, high-growth products to solidify our leadership in women’s health in the US and expanding our reach globally,” Apurva Saraf, president and CEO of Cosette Pharmaceuticals, said in the press release (1). “By combining Cosette’s strong portfolio with Mayne Pharma’s proven commercial expertise, the combined company will be well positioned to further invest in innovation, portfolio expansion and better serve our patients. We look forward to a timely closing and welcoming Mayne Pharma to the Cosette family.”

Cosette reports a staff of more than 350 employees, while Mayne Pharma employs more than 480, suggesting that the workforce of the combined company will more than double (1). Two state-of-the-art, FDA-approved manufacturing sites will be operational as a result of the merger, in Salisbury, South Australia, and in Lincolnton, NC, in the US.

“Joining the Cosette team amplifies our shared mission to improve lives through innovative and accessible women’s health and dermatology medicines,” said Shawn Patrick O’Brien, Mayne Pharma CEO, in the Cosette press release. “This transaction represents a unique opportunity for our employees, customers, and patients. Together, we will extend the reach of important therapies, drive innovation, and improve patient access.”

 was announced in May 2024, when the United Kingdom-based retail and commercial bank Santander UK said it was providing a £30 million (approximately US$38 million) loan to UK-based Ennogen Healthcare International, which focuses on not only dermatology but also respiratory and pain relief therapies, especially among those with chronic conditions (2).

 Metrics Contract Services, which had been its full-service specialty contract development and manufacturing organization, to Catalent in August 2022 for $475 million (3).

In this latest transaction, the boards of directors of both Cosette and Mayne Pharma have approved Cosette’s share purchase agreement, and Mayne Pharma has directed its shareholders to vote in favor of the merger, according to the press release (1). The acquisition is expected to close in the second quarter of 2025.

1. Cosette Pharmaceuticals. Cosette Pharmaceuticals Announces Acquisition of Mayne Pharma, Transforming Company into a Leader in Women’s Health and Dermatology. Press Release. Feb. 21, 2025.
2. Santander. Santander Provides Pharma Manufacturer Ennogen with £30 Million Credit Facility for Growth. Press Release. May 2, 2024.
3. Catalent. Catalent to Acquire Metrics Contract Services for $475 Million to Expand High-Potent Capabilities and Oral Development and Manufacturing Capacity. Press Release. Aug. 9, 2022.

While the boards of directors of both companies have approved the transaction, it is not expected to close until the second quarter of 2025, as Mayne Pharma’s shareholders must still vote in favor of the acquisition.

Cosette Pharmaceuticals Adds to Dermatology and Women’s Health Portfolio with Mayne Pharma Purchase

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Suppliers of raw materials, lab equipment, manufacturing equipment, and other elements that go into the development and manufacture of bio/pharmaceuticals play an important role in the ecosystem that is sustainable manufacturing. Sponsor companies and contract development and manufacturing companies must ensure that the suppliers they use are following sustainability guidelines to truly produce ‘green’ products.

Stefan Lutz, PhD, SVP of Research at Codexis, a provider of enzymes and enzyme technology platforms, points to organic solvents from petrochemical sources as a “dominating factor” in global warming potential (GWP). “Therefore, technologies that replace organic solvents with aqueous-based processes or enable the design of routes to have fewer organic solvent-using steps are highly desirable in terms of improving this key GWP sustainability metric,” says Lutz. “As an added benefit, enzymatic processes often enable more selective synthesis of target molecules, potentially improving yields and simplifying downstream processing.” For the manufacture of small-molecule drugs, Lutz also points to the use of biocatalysts instead of stoichiometric reagents and rare or precious metal catalysts as potential solutions to for economic or hazardous waste production issues.For large-molecule drugs, such as therapeutic RNA oligonucleotides, Lutz calls out the use of organic solvents as an environmental factor.

“Approximately 3000 kg of acetonitrile is used per kg of RNA produced giving rise to a very large carbon footprint. Enabling higher coupling efficiencies, or fragment-based syntheses, will give higher purity, full-length products that could require less or even no further purification, which is a significant contributor to overall solvent use.”

Codexis has focused on sustainability since the company was founded in 2002, and its enzymatic solutions for API synthesis, asserts Lutz, create higher yields with reduced energy use and less waste. Oligonucleotide manufacturing, for example, uses traditional chemical processes, according to Lutz, that create hazardous reagents and chemical waste and are energy intensive. “By contrast, biocatalytic processes using enzymes generally require milder conditions, utilize renewable resources, and generate less hazardous waste,” he explains.

“Codexis is currently launching its first-in-class Enzyme Catalyzed Oligonucleotide (ECO) Synthesis manufacturing platform to provide a highly versatile and effective tool box for production of therapeutic RNA oligonucleotides. The ECO Synthesis platform addresses critical sustainability challenges of traditional manufacturing methods by eliminating the reliance on hazardous chemicals and significantly decreasing the use of acetonitrile,” Lutz says.

Ecolab—which provides contamination control strategies and solutions, water management programs, and resins for downstream purification—uses greener chemistry principles, material and energy sourcing, and packaging initiatives, according to Christine Yore, vice president, Enterprise Corporate Accounts, Ecolab Global Life Sciences. The sustainable manufacture of biopharmaceuticals is challenged by high water consumption, refrigeration energy demands, and chemical and plastic waste generation, according to Yore. Closed-loops systems and water recycling are key to combat these challenges. The company’s Water for Climate program offers a way for companies to optimize water use across operations, therefore reducing water consumption and minimizing the impact on the environment. “We are also seeing that Digital technology is also playing a key role in sustainability initiatives,” says Yore. “Our Ecolab 3D technology provides key insights to our customers by tracking and monitoring water quality and water flow.” Yore also points to the company’s ion-exchange resin and jetting technologies and its cleaning, disinfection, and decontamination products that help improve operations while reducing environmental impacts. Ecolab has committed to sourcing enough clean electricity via a wind farm to power all of its European operations by 2030. Additionally, in 2023, responsible packaging practices at its facilities avoided 14.5 million pounds of virgin plastic use.”

Verdot, which supplies purification systems for the manufacture of biopharmaceuticals and gene therapies, has been proactively anticipating the sustainability requirements of the companies that use their systems by embedding sustainability into their operations, according to Charles Ruban, president and CEO, Verdot. As part of its sustainability strategy, the company launched carbon compensation initiatives in 2025.

“We have achieved one of the lowest carbon footprints, averaging 100 tons equivalent carbon of equivalent carbon per billion euro in revenue,” says Ruban. “And we’ve done that through eco design, you know, really optimizing the material usage of our products and really selecting appropriately the sustainable supplier that we wanted to work with. … [we are] designing energy efficient equipment that [optimize] material use, and we’re partnering with suppliers committed to systemic sustainability, so our carbon neutral initiative reflects the commitment of overcoming those models while enabling compliance to globally reduce their environmental footprint.”

®
eBook: Bio/Pharma Outsourcing Innovation 2025
February 2025
Pages: 20

When referring to this article, please cite it as Haigney, S. Supplying Sustainably. 

Sponsor companies and contract development and manufacturing companies must ensure that the suppliers they use are following sustainability guidelines to truly produce ‘green’ products.

Supplying Sustainably

Circular economy concept. Energy consumption and CO

 emissions are increasing. Sharing,reusing,repairing,renovating and recycling existing materials and products as much possible. | Image Credit: ©Miha Creative- stock.adobe.com

The manufacture of pharmaceuticals involves active chemicals and other materials that may impact the environment. The latter half of the 20th century saw a surge in a movement to reduce climate change that continued into the 2000s. In 2015, the Paris Agreement put forth principles to keep global warming under 2 oC by 2050 (1). In 2019, the European Union introduced the European Green deal that committed Europe to become climate neutral by 2050 (1). In 2014, according to IQVIA, the healthcare industry contributed to 4.4% of the world’s carbon footprint (1). In response to these and other initiatives, the pharmaceutical industry has been working toward more sustainable manufacturing practices.

Working Together to Improve the Environment

The push toward sustainability in pharmaceutical manufacturing also is seen in the increased requirements put on contract development and manufacturing organizations (CDMOs) by sponsor companies to follow sustainability practices, as contract organizations play a significant role in the development and manufacture of drugs.

“As significant third-party suppliers, CDMOs play a major contributing role in their sponsors’ overall emissions,” says Niall Harkin, executive director of Almac Group. “These requirements are a factor in negotiating partnerships as sponsors increasingly request evidence of sustainable or green manufacturing, and documenting these is now the norm on any new RFPs [request for proposals].”

“When we’re negotiating and contracting with our partners, they’re wanting to work with companies who do have ESG [environmental, social, and governance] sustainability policies in place,” says Andy Burns, vice president, MDI (Metered Dosed Inhaler) Business Development at Kindeva. Pharma companies that are more established have entrenched sustainability procedures, according to Burns, and those companies often require contract manufacturing and development organizations to follow those procedures before signing a contract.

According to Rajendra Kumar Sahu, chief executive officer of Navin Molecular, standards required by sponsor companies may include EcoVadis certification and a minimum requirement of a bronze medal at the CDMO’s last assessment. EcoVadis, founded in 2007, provides business sustainability ratings of more than 150,000 companies (2).

Sponsors may also require science-based targets to reduce carbon emissions and the reporting of scope 1 and 2 carbon emissions. “Such importance is placed on these [criteria]; customers are beginning to refuse to work with suppliers that do not meet at least one of these standards, and there are a number of companies making it clear that suppliers will be expected to be able to meet all three requirements/standards in the near future,” stresses Sahu.

“We used to see only Big Pharma or large generic drug companies having specific sustainability requirements. But now a lot of small to mid-sized biotech companies are talking to us and evaluating us on our sustainability related practices,” says Saharsh Davuluri, vice-chairman and managing director, Neuland Laboratories. “One of our largest generic customers has required us to apply for the SBTi [Science Based Targets initiative] validation, which is something that we are seeing other companies follow as well. On the innovator side, we’re seeing some of the large innovators enquire about our EcoVadis rating, beyond the standard frameworks of assessments that are available.”

®
eBook: Bio/Pharma Outsourcing Innovation 2025
February 2025
Pages: 12–20

When referring to this article, please cite it as Haigney, S. Working Together to Improve the Environment. 

Contractors are working with sponsor companies to establish and maintain sustainable manufacturing practices in pharma.

Feliza Mirasol is the science editor for 

Next-generation biotherapeutics pose challenges to the development of manufacturing processes and analytical characterization because of their complexity and, in some cases, novelty. From cell and gene therapies (CGTs) and new vaccines to antibody-drug conjugates (ADCs), successful development and commercialization of these next-generation therapies may hinge on partnerships with service providers that offer specific focus, expertise, and technological experience. At certain points in the discovery and development process, developers—especially smaller firms—come to realize the unmet needs that prompt them to seek an outsourcing provider and/or partnership.The first order of business would be to bring in a partner with the correct expertise and technology to tackle the challenges of bringing a complex biotherapeutic to market. 

Outsourcing Next-Gen Therapies Relies on Expertise and Technology

Bringing next-generation therapies to market brings up multiple challenges, according to Monika Paule, CEO, Caszyme. These challenges range from scientific and technical hurdles to regulatory and societal barriers. “One of the major obstacles is the social perception and acceptance of novel technologies such as CRISPR [clustered regularly interspaced short palindromic repeats]. Addressing public concerns, providing accurate information, and engaging in meaningful dialogue about the benefits, risks, and ethical considerations of these technologies are crucial,” Paule says.

Ali Pashazadeh, CEO, Treehill Partners, a transaction and drug development advisory firm focused on the biotech industry, points to the fact that the biopharmaceutical industry is currently experiencing its fourth year of financial drought, which has been one of the biggest challenges lately to bringing next-generation therapies to market. “Whilst next-generation technologies are incredibly exciting in their potential, these really require, for the first time, excellence in drug development to be combined with excellence in manufacturing,” Pashazadeh says.

Speaking specifically on nucleic acid-based therapies, Bernard Sagaert, CEO of etherna, emphasizes that the challenges are still big, even after the success of the COVID-19 vaccines. The challenges on the RNA front are tied in with RNA quality, where sometimes an inferior quality of RNA is used, which always leads to project failure in discovery or preclinical stages, cost of goods, or duration of expression, he explains. “On the delivery side, targeted expression and off-target expression reduction, balancing immune expression (regulate up or down, or be immune-neutral), and stability are the main challenges. All of these challenges can be different based on the therapeutic area where the products need to be used, whether it is in prophylactic or therapeutic vaccines, autoimmune diseases, gene editing, oncology, protein replacement, etc.,” Sagaert says.

Meanwhile, other challenges include technical and regulatory complexities, such as optimizing gene-editing precision and efficiency, ensuring scalable and cost-effective manufacturing, navigating complex regulatory requirements, and addressing safety and ethical considerations. “Overcoming these hurdles requires broad scientific expertise, strong analytical capabilities, and strategic collaborations among key ‘players’ to accelerate development while minimizing risks,” says Paule.

For the most part, it is the complexity of manufacturing involved in next-generation biotherapies that drives outsourcing in this sector. “These therapies require specialized expertise in areas such as gene and cell therapy production, advanced analytics, and regulatory compliance—expertise that many biopharma companies do not have in-house. Of course, everyone would love to do everything in-house, but the amount of initial investment needed, the need for speed in development, cost efficiency, and enhanced scalability are the main factors driving outsourcing,” says Paule.

“A certain level of complexity multiplies directly in line with” the scale of manufacturing capacity,” adds Jason Jones, global business development lead, Cellular Origins. Jones explains that if processes are complex, variable, and expensive when manufacturing 1000 doses of a next-generation therapy, then these processes will be 100x more complex, variable, and expensive if they are continued to be produced the same way as production scales up 100x.

When referring to this article, please cite it as Mirasol, F. Outsourcing Next-Gen Therapies Relies on Expertise and Technology. 

The value of outsourcing partnerships for next-gen biotherapeutics rests in the expertise and technologies that service providers bring to the table.

Delegating concept. Wooden figurines and arrows as symbol of delegation. | Image Credit: © Vitalii Vodolazskyi - stock.adobe.com

The market research and consulting firm DelveInsight predicts that a global contract development and manufacturing organization (CDMO) market already valued at more than $100 billion could potentially grow by almost half that amount, to an eventual total value of nearly $144 billion, over the second half of the 2020s.

That expansion across the market, which represents a theoretical compound annual growth rate of 6.5%, not only reflects the rise in some serious diseases, various cancers among them, but also the increasing popularity of outsourcing in the drug development process—which, DelveInsight said, comes with certain benefits in cost savings.

The firm was even retained by an unnamed “large pharma client,” looking to improve its API and finished dosage development, to produce a one-off report with the objective of benchmarking top CDMO candidates for outsourcing its projects, analyzing the CDMOs’ R&D, manufacturing, and commercializing capabilities.

Such attention to and emphasis on this research underlines the importance the bio/pharma industry is currently investing in its outsourcing decisions, and as the CDMO market continues to grow, the details of those decisions figure to get more and more precise.

To that end, when CPHI shared its top outsourcing trends for CDMOs across the globe for 2024, several key themes emerged. Chief among these were more careful consideration of geographic diversification and its implications, end-to-end or “one-stop-shop” organizations being able to improve efficiency and scalability, market shifts increasingly driven by merger and acquisition (M&A) activity, and equal responsibility between the drugmaker and the CDMO/contract research organization (CRO) engaged in a partnership—also, to a certain extent, similarly shared risk management responsibility between CDMO/CRO and customer.

Increasingly, the responsibilities referenced in seeking these potential partnerships are centered on sustainability. 

® conducted a series of interviews in January 2025 that explored this aspect, 

perhaps best encapsulated by Saharsh Davuluri,

 vice chairman and managing director for Neuland Laboratories.

“Sponsors are increasingly requiring sustainable or green manufacturing practices when negotiating for partnerships with Neuland,” Davuluri said in the interview. “I think this trend is also kind of aligned with the broader industry shift toward sustainability … we are seeing that a lot of these companies are having their own approach ... or their own frameworks in what they would like to measure their partners with. This is not just about Europe versus [the United States], but also within a particular geography, every company has a different approach.”

As the 2025 market finds its footing, we present a non-exhaustive list of partnerships announced, developed, or forged so far in the year to date, representing companies around the world offering a wide variety of specializations. With this list summarizing only a fraction of the collaborations that have progressed in just mere weeks’ time so far in 2025, there will be plenty to watch for in the months ahead.

When referring to this article, please cite it as Lavery, P. Savings and Sustainability Sought by Pharma Partners. 

With 2025 now in full swing, this article presents a review of the latest developments in outsourcing services, new facilities, and M&A activity thus far this year.

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