The need to increase efficiency and productivity is driving adoption.

Intensifying Downstream Processes

Effective cleaning and disinfection is imperative when operating and utilizing a cleanroom.

Cleanroom Cleaning: Proper Methodology and Determining Efficacy 

Taking key considerations into account along the cell therapy supply chain can set cell therapy developers up for success.

Establishing Consistency and Scalability of Cell Therapy Material 

Growing antibody demand requires long-term scale-up solutions.

Maximizing Long-Term Scale Up 

mRNA vaccines have led to expanded nucleic acid therapy development.

Nucleic Acid-based Therapeutics Take Spotlight 

Implementation of information gathered from novel data analysis techniques has become a priority.

The Ongoing Transformation of Process Controls

2021 brought innovations in pH sensitive antibody purification, single-use fermentation separators, and more.

What’s New in Downstream Processing?

Patrizia Cavazzoni, MD, director of FDA’s Center for Drug Evaluation and Research (CDER), issued a statement on May 19, 2022 announcing a draft guidance, 

Risk Management Plans to Mitigate the Potential for Drug Shortages

to help drug manufacturers develop, implement, and maintain risk management plans. The new guidance is part of the agency’s effort to address disruptions in manufacturing which cause drug shortages.

The draft guidance provides a framework for the development of risk management plans in alignment with the International Council for Harmonisation’s (ICH’s) Q9 

 guideline. Q9 identifies risk factors when developing risk management plans.

“Today’s draft guidance is an important step in what the nation needs to achieve an increasingly secure and resilient drug supply. For Americans to have access to an uninterrupted supply of safe, effective, and high-quality drugs, all entities involved in the manufacture of a drug should take every step available to reduce risks and threats to the drug supply chain. Risk management plans can serve as a safeguard, helping manufacturers prepare for and respond to hazards that could lead to drug supply disruptions and shortages,” said Cavazzoni in the statement.

Articles

The agency is asking drug manufacturers to ensure a strong supply chain by developing risk management plans.

FDA Stresses Risk Management Plans

Catalent announced on May 19, 2022 that it has begun a $175 million project to expand its manufacturing facility in Winchester, Ky. According to a company press release, the facility, which specializes in large-scale oral dose forms, will have two new buildings and a total of 107,000 ft² added to its manufacturing footprint.

The expansion is slated to broaden the site’s highly potent material handling capabilities and containment vault, provide additional laboratory space, and add further capacity for existing turnkey operations, such as dual fill encapsulation and pan coating. These expansions follow a 2015 doubling of the site’s footprint to 190,000 ft² and a $40 million investment in 2019 that installed new equipment for stick pack dosage manufacturing, integrated packaging lines, and expanded roller compaction and fluid bed capacity.

“We are witnessing a broad increase in demand for manufacturing, and the nature of the development pipeline being strong in both oncology treatments and complex molecules, necessitates the expansion of specific capabilities in areas such as highly potent materials handling,” said Aris Gennadios, president of softgel and oral technologies, Catalent, in the press release. “Our Winchester site has a rich history in the successful commercialization of new products, and this expansion is the latest in a series of strategic investments, totaling hundreds of millions of dollars, to ensure that the current and future needs of our customers can be met.”

Catalent has announced a $175 million project to expand its manufacturing facility for large scale oral dose forms at its Winchester, Ky, site.

Catalent Invests $175 Million to Expand Manufacturing Capabilities and Capacity in US Facility

FDA announced on May 13, 2022 that it has approved Eli Lilly and Company’s Mounjaro (tirzepatide) injections as a once-weekly treatment for adults with type 2 diabetes to help manage blood sugar levels. Tirzepatide is a first-in-class medicine that activates both the glucagon-like peptide-1 and glucose-dependent insulinotropic polypeptide receptors, which ultimately leads to improved blood sugar control.

The approval follows five clinical trials wherein three different doses of tirzepatide (5 mg, 10 mg, and 15 mg) were evaluated as either a stand-alone therapy or as an add-on to other diabetes medicines. On average, the patients randomized to receive the maximum recommended dose of 15 mg had a lowering of their hemoglobin A1c level by 1.6% more than placebo when used as a stand-alone therapy, and by 1.5% when used as an add-on. Trials comparing it to other medicines found that it lowered levels by 0.5% more than semaglutide, 0.9% more than insulin degludec, and 1.0% more than insulin glargine.

Additionally, the study found that participants (largely obese, with an average body mass index measured between 32 and 34 at the time of enrollment) in the tirzepatide groups lost more weight than other treatments. Patients lost 15 pounds more than placebo when neither were used with insulin and 23 pounds more than placebo when both were used with insulin. The average weight loss with the maximum recommended dose of tirzepatide was 12 pounds more than semaglutide, 29 pounds more than insulin degludec, and 27 pounds more than insulin glargine. Patients who received insulin without tirzepatide tended to gain weight during the study.

“Given the challenges many patients experience in achieving their target blood sugar goals, today’s approval of [tirzepatide] is an important advance in the treatment of type 2 diabetes,” said Patrick Archdeacon, associate director of the Division of Diabetes, Lipid Disorders, and Obesity, FDA’s Center for Drug Evaluation and Research, in the agency’s press release.

Tirzepatide causes thyroid C-cell tumors in rats. It is unknown whether tirzepatide causes such tumors, including medullary thyroid cancer, in humans, but FDA states that it should not be used in patients with a personal or family history of medullary thyroid cancer or in patients with multiple endocrine neoplasia syndrome type 2. Reported side effects include nausea, vomiting, diarrhea, decreased appetite, constipation, upper abdominal discomfort and abdominal pain.

FDA has approved Mounjaro (tirzepatide) injections as a treatment to improve blood sugar control in adults with type 2 diabetes.

FDA Approves Lilly’s Novel, Dual-Targeted Treatment for Type 2 Diabetes

Chris Spivey is the editorial director of 

In pharmaceutical science, several divergent paths typically exhibit clear validity. Decisions often boil down to embracing instead of eschewing risk, to accelerate as the only priority, or to trust a partner rather than emphasize self-reliance. Each molecule, each company or research group possessing that molecule, has an assortment of strengths and weakness to guard against or to leverage. Multiple factors weigh in the balance when deciding how best to move forward, and increasingly the route chosen is to outsource research, development, and manufacturing.

Balancing Internal Focus Against External Capabilities

The global contract development and manufacturing organization (CDMO) industry, averaging across several market research firm estimates, is currently valued around $100 billion (1). In part, this growth is shaped through direct regulatory influences suggesting, if not demanding, that a drug have multiple sites of manufacture to spread the risk for supply chain planning. Above regulations, companies also seek to reduce the complexity of their operations internally, and to reallocate internal resources most effectively.

When referring to this article, please cite it as C. Spivey, “Balancing Internal Focus Against External Capabilities,” 

BioPharm International Partnerships for Outsourcing

Demand for outsourced services of technical R&D activities is increasing.

Pfizer and BioNTech announced on May 17, 2022 that FDA has expanded emergency use authorization (EUA) to include a booster dose after completion of the primary series of the companies’ COVID-19 vaccine in children five through 11 years of age. The booster dose should be given at least five months after the second dose of the two-dose primary series. More than 8 million children aged 5–11 years old have completed a primary series of the vaccine in the United States to date.

The EUA is based on data that show children five through 11 years of age had a significant immune response following a booster dose of the vaccine during the time when Omicron was the prevalent variant. More than 4000 children in this age range have participated in the companies’ COVID-19 vaccine clinical trial to date.

The companies have also submitted an application to the European Medicines Agency for a booster dose in this age group and plan to file similarly with other regulatory agencies around the world. The Pfizer-BioNTech COVID-19 vaccine is currently the only COVID-19 vaccine authorized for use in the US for children in this age group. The vaccine is based on BioNTech’s proprietary mRNA technology, and the company is the Marketing Authorization Holder in the US, European Union, the United Kingdom, and Canada. BioNTech and Pfizer jointly hold EUAs or equivalents in the US and other countries.

Pfizer and BioNTech’s COVID-19 vaccine has been granted EUA for booster doses in children five through 11 years of age.

Pfizer and BioNTech Granted EUA for Booster Dose of COVID-19 Vaccine in Young Children

Pfizer and BioNTech announced on May 13, 2022 that they have reached an agreement with the European Commission (EC) to update their COVID-19 vaccine supply agreement. The update includes rephased planned deliveries to help support the EC and Member States’ immunization programs.

Doses that were scheduled for delivery in June through August 2022 will now be delivered in September through fourth quarter 2022. The delivery schedule change does not affect the companies’ full-year 2022 revenue guidance or the full-year commitment of doses to be delivered to the EC Member States in 2022.

The companies are continuing to evaluate potential variant-based vaccines and expect to share data in the coming months. The Pfizer-BioNTech COVID-19 vaccine is currently authorized under Emergency Use Authorization in individuals five years of age and older.

The companies have reached an agreement with the European Commission to update their COVID-19 vaccine supply agreement.

Pfizer and BioNTech Update COVID-19 Vaccine Supply Agreement with EC

Pfizer announced its $11.7 billion acquisition of Biohaven Pharmaceuticals, a clinical-stage biopharmaceutical company, on May 10, 2022. This acquisition will grant Pfizer the rights to rimegepant, a dual-acting migraine therapy approved in the United States for acute treatment and episodic prevention of migraine in adults. It is also approved in the European Union for acute treatment of migraine and prophylaxis of episodic migraine.

According to a company press release, this deal will also grant Pfizer the rights to zavegepant, which is on track for US approval in the second quarter of 2022 as an intranasal spray for the acute treatment of migraine; it is also in development as an oral soft gel for chronic migraine prevention. Additionally, Pfizer will acquire a portfolio of five other preclinical calcitonin gene-related peptide (CGRP) assets.

“Today’s announcement builds on our legacy of delivering breakthroughs for patients living with complex pain disorders and diseases that disproportionately impact women,” said Nick Lagunowich, global president, Pfizer Internal Medicine, in the press release. “[Rimegepant], which is already the [number one] prescribed migraine medicine in its class in the United States, coupled with Biohaven’s CGRP pipeline, offers hope for patients suffering from migraine worldwide.”

“We are excited to announce Pfizer’s proposed acquisition of Biohaven, recognizing the market leadership of [rimegpant], our breakthrough all in one migraine therapy, and the untapped potential of our CGRP franchise,” said Vlad Coric, chairman and CEO, Biohaven, in the press release. “Pfizer’s capabilities will accelerate our mission to deliver our migraine medicines to even more patients, while the new R&D company is well positioned to bring value to patients and shareholders by focusing on our innovative pipeline for neurological and other disorders. We believe this transaction represents significant future value creation for patients and our collective shareholders.”

Pfizer will acquire Biohaven Pharmaceuticals, a clinical-stage biopharmaceutical company specializing in migraine treatments, for $11.7 billion.

Pfizer to Acquire Biohaven Pharmaceuticals for $11.7 Billion

Avacta Group, a clinical-stage oncology drug company, announced that it would be relocating its Therapeutics division on May 11, 2022.The division will now be headquartered at Scale Space in Imperial College’s White City Campus, London, United Kingdom. This move follows Avacta’s recent transition of lead candidate AVA600, a tumor-activated form of doxorubicin, into clinical trials in August 2021.

Scale Space is part of the broader White City Innovation District, which is currently home to over 140 companies across a broad range of industries, including pharmaceutical and biotech. According to a company press release, Avacta’s new HQ will be part of a life science cluster at the west London site.

“This move is the result of a clear vision to bring together the research and development teams into one hub to facilitate the translation of our in-house research from bench to bedside,” said Fiona McLaughlin, chief scientific officer of Avacta, in the press release. “We are now at the heart of the innovative White City community in London, readily accessible to our collaborators around the world and poised to take Avacta to the next level in its evolution as a clinical stage biopharmaceutical company.”

Avacta is relocating its therapeutics headquarters to Scale Space in Imperial College’s White City Campus, London, United Kingdom.

Avacta Establishes New Therapeutics Headquarters

Peter Gaskin, PhD, is the senior director of scientific advisory services for Charles River, an AAPS sustaining partner.

It wasn’t long ago that pharmaceutical companies did their own research and development in-house, from discovery through investigational new drug (IND) submission. Today, it’s hard to find pharmaceutical companies of any size, from large global pharmaceutical entities down to the smallest biotech company, that has not outsourced some aspects of their work to a third-party organization, whether that be academic institutions, biotech startups, or contract research organizations (CROs). These relationships are likely to continue to grow exponentially. A new market report released this year estimates that the global biotechnology and pharmaceutical services outsourcing market will surpass $91.4 billion US by 2028 because of a 5.2% compound annual growth rate (CAGR) (1).

Today, roughly two-thirds of R&D is being outsourced, with CROs growing dramatically to fill the gap. How can service providers and clients assure that the relationship will be mutually advantageous? What are some of the best practices to ensure a high-quality product? This article will look at how to ensure contracts are established and signed quickly so that science moves forward, why quality assurance (QA) is integral to meeting regulatory compliance, how compromise and coordination are necessary for any winning partnership, and whether geography is important given the globalization of today’s economy.

The Essential Ingredient in any Sponsor-CRO Partnership: Trust

BioPharm International’s Partnerships for Outsourcing eBook

1. H. Balfour, “Biotech and Pharma Services Outsourcing Market to Be Worth Over $91bn by 2028,

When referring to this article, please cite it as P. Gaskin, "The Essential Ingredient in any Sponsor-CRO Partnership: Trust," 

To build trust and ensure a high-quality product, companies must be sure their contracts are firm and their lines of communication even firmer.

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

To foster the development of treatments for undertreated and rare conditions, FDA grants orphan drug designation to drugs and biologics that treat, prevent, or diagnose diseases that affect fewer than 200,000 people in the United States or drugs that meet certain cost recovery provisions (1). This designation comes with incentives such as tax credits, user fee exemptions, and market exclusivity. To receive such designation, sponsors must submit requests and present data to support the request.

 asked Judith Jones, FRSC, Director Regulatory Affairs, Global Regulatory Affairs, Catalent, and Matthew Mollan, RPh, PhD, Regional Head of Operations, Early Phase Development North America, Catalent, about the specific ways contract development and manufacturing organizations (CDMOs) can assist sponsors in developing, applying for, and manufacturing orphan drugs.

When referring to this article, please cite it as S. Haigney, “Fostering Orphan Drugs,” 

CDMOs offer expertise and customization options for sponsors of orphan drugs.

Fostering Orphan Drugs

James Park is executive vice-president and managing director of the Global Sales Center at Samsung Biologics.

The COVID-19 pandemic highlighted the potential of an array of modalities within the biopharmaceutical industry. In particular, the success of messenger RNA (mRNA)-based COVID-19 vaccines has encouraged biopharmaceutical companies to explore and utilize these technologies for other diseases and look to rapidly expand their capacity and capabilities in response to an upsurge in demand.

Preparing for the Future: Expanding Capacity and Facilitating Multi-modalities

At the end of 2019, the combined market capitalization of the five publicly listed companies focusing on mRNA platforms was $15 billion, and as of August 2021, that value of capitalization reached more than $300 billion (1). This significant boost in market valuation reflects the optimism that mRNA technology can deliver much more and go beyond the current prophylactic COVID-19 vaccines.

When referring to this article, please cite it as J. Park, “Preparing for the Future: Expanding Capacity and Facilitating Multi-modalities,” 

CDMOs need to consistently demonstrate agility as demand increases for newly developed therapeutic modalities. 

Grant Playter is the Assistant Editor for 

Actions speak louder than words, and in the highly competitive biopharmaceutical market large acquisitions are often the equivalent of shouting one’s intentions from the rooftops. In this vein, 

 has collected the latest acquisition news from major contract, development, and manufacturing companies (CDMOs) in the hopes of providing insight into possible market movements.

Merck KGaA, “Acquisition of Exelead Completed, Plans to Invest More Than € 500 Million in Technology Scale-Up,” Press Release, Feb. 23, 2022.

Catalent, “Catalent Acquires Facility in Oxfordshire to Expand Biologics Capabilities in the UK and Across Europe,” Press Release, April 6, 2022.

Recipharm, “Recipharm acquires GenIbet to bolster Biologics offering,” Press Release, Feb. 1, 2022.

Recipharm, “Recipharm expands its advanced therapy medicinal products (ATMPs) offering into Virotherapies with the acquisition of Vibalogics,” Press Release, Feb. 18, 2022.

Recipharm, “Recipharm Acquires Arranta Bio, a CDMO Leader in Advanced Therapy Medicinal Products (ATMPs), to Expand Its Niologics Offering in the US,” Press Release, Feb. 18, 2022.

Samsung Biologics, “Samsung Biologics Reaches Agreement with Biogen to Acquire Full Ownership of Samsung Bioepis,” Press Release, Jan. 27, 2022.

FDA, “FDA Approves First Interchangeable Biosimilar Insulin Product for Treatment of Diabetes,” Press Release, July 28, 2021.

FDA, “FDA Approves Cyltezo, the First Interchangeable Biosimilar to Humira,” Press Release, Oct. 18, 2021.

FDA, “FDA Approves First Biosimilar to Treat Macular Degeneration Disease and Other Eye Conditions,” Press Release, Sep. 17, 2021.

ten23 health, “Launch of ten23 health – opening a new chapter in contract pharmaceutical development and manufacturing services,” Press Release, Sep. 1, 2021.

ten23 health, “Swissfillon, a Leader in Sterile Filling of Complex Pharmaceuticals, Joins ten23 health and Enhances the Combined Company’s Integrated Offering to Its Customers,” Press Release, Oct. 25, 2021.

Fujifilm Diosynth Biotechnologies, “Fujifilm Expands its Advanced Therapies CDMO Business with the Acquisition of a Dedicated Cell Therapy Manufacturing Facility from Atara Biotherapeutics, Inc.,” Press Release, Jan. 26, 2022.

Fujifilm Diosynth Biotechnologies, “FUJIFILM Diosynth Biotechnologies Doubles its Existing Laboratory Footprint in North Carolina,” Press Release, Jan. 5, 2022.

Grant Playter is the Assistant Editor at 

When referring to this article, please cite it as G. Playter, "The CDMO Carousel," 

Acquisitions in the CDMO space can give us insight into a company's vision for their future.