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Patrick Lavery is an Editor for the MJH Life Sciences brands 

. Previously at MJH, he filled the same role for 

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London-based Hikma Pharmaceuticals announced in a press release on Jan. 14, 2025, an exclusive commercial partnership, for a period of six years, with Emergent BioSolutions for the sale of Kloxxado (naloxone) hydrochloride (HCl) nasal spray, at a dosage of 8 mg, in the United States and Canada (1). Kloxxado was approved by FDA in April 2021 for emergency treatment of known or suspected opioid overdose manifested by respiratory and/or central nervous system depression, in both adult and pediatric patients.

According to the press release, Hikma will continue to produce its 8 mg naloxone HCl nasal spray at its manufacturing facility in Columbus, Ohio, and then provide it exclusively to Emergent (1).

The incidence and prevalence of opioid use in seven countries has been studied as part of the DARWIN EU project, short for Data Analysis and Real World Interrogation Network, which began in 2022. In announcing its intention to expand capacity for real-world data studies in 2024, the European Medicines Agency said the completed opioid study, and an additional study on take-home naloxone that was also conducted, helped to determine duration of prescription opioid use, characterization of users, and prescribing and dispensing indications (2).

"We are thrilled to be partnering with Emergent, who have deep experience in getting lifesaving naloxone nasal spray into the hands of those who can use it to help save lives," Hafrun Fridriksdottir, president of Hikma's Generics business, said in the press release. "This partnership combines Hikma's excellent nasal spray manufacturing capabilities with Emergent's well-established naloxone HCl nasal spray commercial expertise and strong stakeholder engagement. Hikma remains committed to ensuring that all forms of naloxone we produce—Kloxxado nasal spray, as well as the injectable vials and prefilled syringes that we will continue to manufacture and market—are widely accessible to all who can benefit from them, including patients, friends, family members and the public health community."

Emergent also distributes Narcan nasal spray, in 4 mg dose, the first FDA-approved, over-the-counter naloxone product for emergency treatment of opioid overdose (1).

Hikma last made headlines in December 2024 when its liraglutide injection was approved by FDA, making it the first generic version of Novo Nordisk’s Victoza, a glucagon-like peptide-1 receptor agonist, in the US, indicated for improving glycemic control in adults and pediatric patients aged 10 years and older with type 2 diabetes (3).

As for Emergent, word came in June 2024 that Bora Pharmaceuticals, of Taiwan, had expanded its North American footprint by making a $30 million purchase of an 87,000-square-foot sterile manufacturing facility, in Baltimore, Md., that had been previously owned by Emergent (4).

1. Hikma Pharmaceuticals. Hikma Announces Exclusive Commercial Partnership with Emergent BioSolutions for Kloxxado (naloxone HCl) Nasal Spray 8 mg. Press Release. Jan. 14, 2025.
2. HMA and EMA. 

Big Data Steering Group (BDSG): 2023 Report.

 Jan. 18, 2024.
3. FDA. FDA Approves First Generic of Once-Daily GLP-1 Injection to Lower Blood Sugar in Patients with Type 2 Diabetes. Press Release. Dec. 23, 2024.
4. Bora Pharmaceuticals. Bora Pharmaceuticals Expands North American Footprint with Agreement to Acquire First Sterile Manufacturing Facility. Press Release. June 20, 2024.

Articles

Kloxxado was approved by FDA in 2021 for emergency treatment of opioid overdose, and Emergent also distributes the FDA-approved over-the-counter treatment Narcan.

Hikma and Emergent Enter Six-Year Partnership to Sell Naloxone Spray in US and Canada

Scribe Therapeutics, a genetic medicines company based in Alameda, Calif., announced on Jan. 13, 2025 that it had achieved a success milestone for one of the targets in a research collaboration with Sanofi (1). That partnership, which centers around Scribe’s focus on clustered regularly interspaced short palindromic repeats (CRISPR) technology for cell therapies, began in 2022 and was expanded upon in 2023 with the intention of advancing 

Scribe’s CRISPR by Design is a data-driven design and engineering approach that was developed to optimize the company’s CRISPR-based platforms and assets (1). It works by engineering bacterial immune systems into a suite of genome and epigenome editing tools, with the end goal of providing safer and more effective genetic therapies by improving activity, specificity, and deliverability, according to a press release from Scribe.

The release did not reveal exact details of the milestone reached, but the company said it is eligible to receive payments of more than $1.2 billion for the achievement of certain research, development, regulatory, and commercial milestones, as well as royalties described in the “high-single-digit to mid-teen” range (1).

“We are excited about the progress we have made towards creating potentially curative in vivo genetic medicines with a partner like Sanofi,” said Benjamin Oakes, PhD, co-founder and chief executive officer of Scribe, in the press release. “The complementary expertise of our teams has enabled the program to move quickly and attain early validation of our CRISPR genome editing technologies. We look forward to further advancement of this program with a direct line of sight towards bringing breakthrough CRISPR-based therapeutics to patients with significant unmet need.”

 program with Sanofi speaks to the versatility and strength of Scribe’s CRISPR-based platforms,” said Svetlana Lucas, PhD, chief business officer at Scribe, in the release. “We are pleased to reach this important milestone and continue progressing our collaboration to accelerate and expand our patient impact globally.”

The 2023 expansion, previously reported, combined Scribe’s proprietary CRISPR XE, or X-Editing, technologies with Sanofi’s targeted, non-viral delivery technologies (2). Scribe received $40 million up front, and it was at that time that the company said the additional $1.2 billion might eventually be available if certain milestones were hit.

At the time, the genomic medicines being worked on through this partnership were primarily concerned with 

 treatment of sickle cell disease, although Scribe said other diseases would also be addressed (2).

“We’re encouraged by what we’ve accomplished to date with Scribe in creating 

 NK [natural killer] cell therapies and now look forward to accelerating our ability to effectively leverage genome editing 

 through Sanofi’s innovative research work in targeted lipid nanoparticles (LNPs), for 

 therapies with the potential to dramatically improve treatment outcomes and ultimately to change patients’ lives,” Christian Mueller, global head of the Genomic Medicine Unit at Sanofi, said in the 2023 release (2).

1. Scribe Therapeutics. Scribe Therapeutics Achieves Milestone for 

 Program in Collaboration with Sanofi. Press Release. Jan. 13, 2025.
2. Scribe Therapeutics. Scribe Therapeutics Expands Collaboration with Sanofi to Advance 

 Genetic Medicines for Sickle Cell and Other Genomic Diseases. Press Release. July 17, 2023.

Although specifics about the milestone were not immediately provided, Scribe is eligible to receive more than $1.2 billion for certain research, development, regulatory, and commercial benchmarks.

Scribe Reports Milestone for In Vivo Program Collaboration with Sanofi

Telix Pharmaceuticals announced on Jan. 13, 2025 that it had entered into an asset purchase agreement with ImaginAb, an antibody engineering company based in Inglewood, Calif. (1). The acquisition gives Telix a pipeline of next-generation therapeutic candidates, a proprietary and novel biologics technology platform, and a protein engineering and discovery research facility, which is located in California.

Included in the transaction are early stage drug candidates against high-value targets, among them namely DLL3 and integrin αvβ6, plus other novel targets that are still in the discovery stage (1).

Telix’s lead imaging product, gallium-68 (marketed under the brand name Illuccix), has been approved by FDA, the Australian Therapeutic Goods Administration, and Health Canada (1).

The purchase price for the transaction is US$45 million (AU$73 million), including a deferred payment of up to US$4 million in equity by the conclusion of a 15-month period of indemnity (1). As certain milestones are achieved, Telix has agreed to pay up to a US$185 million total, with royalties also payable on net sales.

“The combination of a proprietary drug discovery platform, pipeline of promising theranostic assets, and a talented team of subject matter experts will enhance Telix’s research and innovation capability now and into the future,” Richard Valeix, chief executive officer, therapeutics for Telix, said in a company press release (1). “This acquisition will enable Telix to explore new disease areas with state-of-the-art radiotherapeutic technology.”

Intellectual property, meanwhile, utilizing small, engineered antibody formats that enable highly specific cancer targeting, and fast tumor uptake and blood clearance, has also been acquired by Telix from ImaginAb.

ImaginAb, a clinical-stage biotechnology company, develops engineered antibodies which the company said in the release maintain the specificity of full-length antibodies while remaining biologically inert within the body (1). These antibodies are designed to bind specifically to cell surface targets using positron emission tomography and optical imaging technology, according to the release.

“As the radiopharmaceutical sector gains momentum, there is a significant need for targeting agents to be more selective, deliver less off-target radiation, and better match the pharmacology and radiobiology of a given radionuclide,” Anna M. Wu, PhD, co-founder and board member of ImaginAb, said in the release. “The team’s deep expertise in antibody engineering and the resulting development of a valuable, proprietary platform technology has led to clinical proof-of-concept. Telix is the right partner to unlock the future therapeutic potential of this platform.”

Also on January 13, Telix, which is based in North Melbourne, Australia, announced that it had exceeded its previously stated guidance of US$490 million to US$510 million for fiscal year 2024 with a reported total unaudited full-year 2024 revenue of approximately US$517 million. That revenue was bolstered by a figure of US$142 million in the fourth quarter, which the company said represented an increase of 46% over the 2023 fourth quarter (2).

1. Telix Pharmaceuticals. Telix to Acquire Next-Generation Therapeutic Assets and Innovative Biologics Technology Platform. Press Release. Jan. 13, 2025.
2. Telix Pharmaceuticals. Telix Exceeds FY24 Guidance with US$142M Q4 Revenue. Press Release. Jan. 13, 2025.

The transaction adds a pipeline of early-stage drug candidates against high-value targets to Telix’s portfolio, along with other novel targets currently in the discovery stage.

Telix Enters Agreement with ImaginAb to Purchase Next-Gen Therapeutic Candidates

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The contract research, development, and manufacturing organization (CRDMO) BioDuro announced on Jan. 9, 2025 the opening of a fully automated solid-phase peptide synthesis scale-up laboratory at its Zhangjiang High-Tech Park campus in Shanghai, China (1). In the press release detailing the launch, BioDuro said the state-of-the-art, kilogram-scale lab would support peptide synthesis scale up to 800 mmol.

Created in response to what the company said is growing demand from global partners for efficient, scalable peptide manufacturing, the facility’s equipment includes automated solid-phase peptide synthesizers, cleavage systems, and freeze-drying equipment, designed to produce a range of molecules including both linear and cyclic peptides and peptide-drug conjugates (1).

“Demand for peptide-based programs has increased considerably, and the opening of this new laboratory provides BioDuro’s customers with a significant step forward in addressing the capabilities required to scale their candidates to compete in this fast-growing field,” Armin Spura, PhD, CEO of BioDuro, said in the press release. “We remain committed to continuously advancing our CRDMO capabilities and capacity, driving breakthroughs that deliver life-changing therapies to patients worldwide.”

BioDuro said the new laboratory complements its existing peptide discovery synthesis center in China, which is at the CRDMO’s Beijing campus (1). There, the focus is on early-stage research and development. Meanwhile in Shanghai, the new laboratory will enable a “seamless” integration, according to the press release, of projects from the discovery to development stages.

BioDuro’s comprehensive peptide platform integrates biology with drug metabolism and pharmacokinetics services, which the company said has the ultimate effect of accelerating peptide development (1).

Specialists on the chemistry teams at both the Beijing and Shanghai campuses, BioDuro said, are well-versed in both solid- and liquid-phase peptide synthesis, which from a client perspective provides additional levels of flexibility and customization (1).

In October 2024, BioDuro, which is headquartered in Irvine, Calif., announced the launch of a pilot-scale production laboratory enabling scale-up synthesis for drug substances from 500 grams to kilograms, on the same site where earlier that month, the company had said it was opening its new Compound Management Center (2,3). At the time, these facilities were said to be joining a group of BioDuro sites that altogether employ a total workforce of more than 2000.

 spoke with Kevin Li, MBA, about BioDuro-Sundia’s role in integrated services, and the benefits such services can provide to pharma companies.

“One side can handle both drug substance and drug product, and one side can handle the pre-formulation, formulation development, until commercial manufacturing, everything in one side,” Li said in the interview, “so that integrated solution can really help customers to accelerate their innovation through multiple benefits” (4).

1. BioDuro. BioDuro Opens New Solid-Phase Peptide Synthesis Scale-Up Facility. Press Release. Jan. 9, 2025.
2. BioDuro-Sundia. BioDuro-Sundia Launches New Kilogram Laboratory to Speed Development from Discovery to Preclinical and Clinical Phases. Press Release. Oct. 24, 2024.
3. BioDuro-Sundia. BioDuro-Sundia Launches New Compound Management Center, Strengthening Drug Discovery Services. Press Release. Oct. 2, 2024.
4. Thomas, F. 

CPHI Milan 2024: Highlighting the Benefits of Integrated Services.

This is the latest facility launch for BioDuro, following a kilogram laboratory and compound management center, both on the same site, in October 2024.

BioDuro Opens Solid-Phase Peptide Synthesis Scale-Up Facility in Shanghai

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

Pharmaceutical Technology chats about the increasing prevalence of challenging APIs in the development pipeline and the hurdles facing formulators and manufacturers as a result of this trend with Jens Schmidt from Lonza.

Dealing with Challenging APIs

Chronic illnesses and an aging global population are increasing the demand for innovative pharmaceutical products to help alleviate symptoms, prevent illness, or even provide a cure. As a result, pharmaceutical companies are investigating more complex and challenging APIs to seek out the most effective treatments for patients; however, these ingredients pose a number of development hurdles.

According to Jens Schmidt, associate director MSAT at Lonza, there are a few factors driving the trend of increasingly complex and challenging APIs entering the development pipeline. One clear driver has been the industry’s improved understanding of biological pathways, which has led to the investigation of more intricate molecular structures to target such pathways, he explains. Additionally, the desire to achieve very high selectivity while minimizing side effects, the use of multi-target drugs, and overcoming pathogen resistance are also contributing to the rising prevalence of more complex molecules being developed, Schmidt adds.

When formulating more challenging APIs, more advanced techniques and specialized solutions are required to overcome issues such as poor solubility and bioavailability, Schmidt continues. “There can be pure manufacturing challenges like flow properties. [which] are typically not so great compared to standard or smaller molecules,” he says. “So, process development is getting even more important. It's important for a smaller molecule as well, but it's more even more important for the larger molecule [drugs].”

Jens Schmidt is the Associate Director of Manufacturing, Science, and Technology (MSAT) at Lonza.

Jens Schmidt is the Associate Director of Manufacturing, Science, and Technology (MSAT) at Lonza. In this role, he leads a team of MSAT scientists and serves as the technology lead for high-throughput experimentation focused on small molecules.
Before joining Lonza in 2017, Jens gained valuable experience as a research associate at the University of Oxford and the California Institute of Technology (Caltech). He is an organic chemist by training and holds a Master of Science in Chemistry from the University of Leipzig and a PhD from the University of Hamburg.

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Feliza Mirasol is the science editor for 

US contract research organization and contract development and manufacturing organization PackGene Biotech has entered into a collaboration with Genetic Cures for Kids (GC4K), an Australian non-profit group, and the Belfer Gene Therapy Core Facility at Weill Cornell Medicine, to deliver an accessible, custom-tailored gene therapy solution for treating hereditary spastic paraplegia type 56 (SPG56), an ultra-rare neurological disease, PackGene announced on Jan. 8, 2025.

Under the alliance, the team will use adeno-associated virus serotype 9 (AAV9) to deliver the healthy gene directly into patient cells. PackGene specializes in providing comprehensive services, including plasmid DNA, viral vector, and messenger RNA production as well as supporting programs from research stage to commercialization. PackGene has pledged to subsidize costs and fast-track the development of a clinical-grade gene therapy for SPG56. Meanwhile, Weill Cornell Medicine’s Belfer Gene Therapy Core Facility has tailored its AAV manufacturing capabilities to serve small, patient-driven projects.

“Our work at the Belfer Gene Therapy Core Facility is driven by a commitment to making gene therapy accessible to all patients, especially those with rare diseases [such as] SPG56,” said Stephen M. Kaminsky, professor of Research in Genetic Medicine at Weill Cornell Medicine, in a press release (1).

SPG56 is a progressive, degenerative disorder that typically begins in early childhood (2). This global alliance was prompted by the parents of Tallulah Moon, a young child who suffers from the disease. The parents founded GC4K in an effort to find a cure for their daughter. Children lose motor and cognitive abilities as the disease progresses. From the start, diagnosing SPG56 can be an uphill battle, especially considering that genetic testing is limited in its ability to detect the disease due to its ultra-rare nature. According to the press release, fewer than one in a million people worldwide are diagnosed, and most families are left with no treatment options (1).

This collaboration between PackGene, Weill Cornell Medicine, and GC4K is meant to show that ultra-rare diseases can draw international attention, funding, and breakthrough innovations in a pharmaceutical industry where large patient populations and high-return therapies are often prioritized, according to the press release. This project uses “top-tier science” to deliver tailored solutions for such a small population of children and is an approach that can be replicated for thousands of other conditions that are currently “too rare” to draw significant investment (1).

“Together with my colleague Professor Dolan Sondhi, our long-established track record in supporting therapeutic development programs, combined with our expertise in AAV vector manufacturing, is being harnessed to potentially deliver life-changing treatments for Tallulah. This effort also serves as an example for addressing thousands of other rare genetic conditions,” Kaminsky said in the release.

This initiative has potential to go beyond a single disease such as SPG56. GC4K aims to create a replicable framework to address more than 7000 other genetic conditions that currently have no cure.

“Our collaboration with Genetic Cures for Kids and Weill Cornell Medicine’s Belfer Gene Therapy Core Facility is a testament to the power of compassion paired with scientific excellence,” said Xin Swanson, PhD, chief scientific officer, PackGene Biotech, in the release. “We are leveraging our expertise not just to create plasmid DNA and AAV vectors, but to reshape the future landscape of gene therapy—making it affordable, accessible, and deeply human.”

“As the mother of a child living with SPG56, I’ve come to understand that families like ours are not just fighting diseases—we’re fighting for the attention, resources, and urgency that our children’s lives deserve,” said Golden Whitrod, founder of Genetic Cures for Kids and mother to Tallulah Moon, in the release. “Until governments seriously invest in the credible and worthwhile market of personalized medicine, family foundations like ours will continue to lead the charge. But we cannot do it alone. The generosity and philanthropy of companies [such as] PackGene and institutions [such as] Weill Cornell Medicine remind us that compassion and innovation can change the world, one life at a time.”

A Global Alliance to Save Lives: How Two Parents and PackGene Are Redefining What’s Possible in Gene Therapy with Help from Weill Cornell Medicine

. Press Release. Jan. 8, 2025.
2. Wong, S. S-N.; Yuen, L. Y-P.; Kan, E.; et al. CYP2U1: An Emerging Treatable Neurometabolic Disease with Cerebral Folate Deficiency in 2 Chinese Brothers. 

, 101023. DOI: 10.1016/j.ymgmr.2023.101023

Through this global alliance, PackGene, Weill Cornell Medicine, and GC4K, an Australian non-profit, intend to deliver a custom-tailored gene therapy solution to treat hereditary spastic paraplegia type 56, a particularly rare neurological disease.

PackGene and GC4K Team Up Along with Weill Cornell Medicine for Gene Therapy to Treat Rare Neurological Disease

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The European Medicines Agency (EMA) announced on Jan. 10, 2025 that it is prepared for the implementation of the health technology assessment regulation (HTAR) that comes into effect on Jan. 12, 2025 (

) (1, 2). The HTAR accelerates and widens access to new medicines in the European Union and creates a framework for the assessment of certain high-risk medical devices. The allows for cooperation between regulators in the evaluation of the risks and benefits of medicines, expanding the cooperation between EMA and health technology assessment (HTA) bodies.

New regulations will start with new active substances for oncology products and advanced therapy medicinal products (ATMPs). Rules will then be expanded to orphan drug products in January 2028 and then all centrally authorized products in 2030.

The agency has been working with the European Commission (EC), EU Member States, and stakeholders from industry, healthcare, patients, and academia in preparation for its support of the regulation. EMA’s support of the HTAR will include helping to conduct timely joint clinical assessments (JCA) by the HTA Coordination Group (HTACG) by providing relevant information from its own regulatory assessments. The agency will also collaborate with the HTA Coordination Group in joint scientific consultations to provide scientific advice to technology developers and generate evidence to satisfy regulators and HTA bodies. EMA will also exchange information on upcoming applications and technologies for planning purposes.

"EMA welcomes the new HTA regulation and is ready to do what it takes to support the European Commission and the Member States in its successful implementation,” said EMA’s Executive Director, Emer Cooke, in the press release. “While our role is supportive, the new regulation will enable our collaboration across decision-makers reinforcing efforts to improve access to medicines for all patients in the EU. In our network strategy to 2028, facilitating the path to accessibility of new medicines for patients is a priority and the framework for cooperation and the exchange of information between regulators and HTA bodies provided by the new rules will help galvanize our efforts."

EMA has a legal obligation to notify the EC, which is the secretariat to the HTACG, and ensure that procedures are being followed and work is done is a timely and transparent way when marketing authorization applications are received that fall into the scope of JCA. HTACG estimates in 2025 it will need to conduct 17 JCAs for cancer drugs and eight JCAs for ATMPs. EMA also must provide information from ongoing regulatory assessments, details of which will be developed by EMA’s committee for human medicines and the JCA subgroup.

The first request period for JSC will be launched by the HTACG in February 2025, and developers can request parallel JSC with EMA.

“The future processes for parallel JSC have been built based on experience gathered since the initial piloting in 2008 and the iterations of process improvement performed over time. This year, the HTACG is planning to initiate five to seven joint scientific consultations for medicinal products and one to three joint scientific consultations for medical devices,” EMA stated in the press release.

EMA. New EU Rules for Health Technology Assessments Become Effective. Press Release. Jan. 10, 2025. 

https://www.ema.europa.eu/en/news/new-eu-rules-health-technology-assessments-become-effective

EU. Regulation (EU) 2021/2282 of the European Parliament and of the Council of 15 December 2021 on health technology assessment and amending Directive 2011/24/EU (Text with EEA relevance), Dec. 15, 2021. EUR-Lex. 

https://eur-lex.europa.eu/legal-content/EN/TXT/?uri=CELEX:32021R2282

The HTAR, which aims to increase access to new medicines, becomes effective Jan. 12, 2025.

EMA Prepared for Health Technology Assessment Regulation Implementation

This story has been updated to include a comment by Glenn Wright, president and CEO of the Parenteral Drug Association.

An email communication, dated Jan. 10, 2025, released by FDA’s Center for Drug Evaluation and Research (CDER), announced the retirement of CDER Director, Patrizia Cavazzoni, effective January 18.

Cavazzoni joined FDA in 2018 as deputy director of operations in CDER and also served as acting principal deputy commissioner of Food and Drugs briefly in 2019. She was made director of CDER in 2021. Prior to working with FDA, Cavazzoni spent several years working in the pharmaceutical industry in the areas of clinical development, regulatory affairs, and safety risk management (1).

In a letter to her CDER colleagues announcing her retirement, Cavazzoni praised her team for how it handled the COVID-19 pandemic and the growth the center has shown in her years as director (2).

“While I could spend a lot of time enumerating our achievements, I want to highlight our biggest success: our people and our culture,” Cavazzoni said in the letter. “As I leave CDER, I can say with confidence that the Center is at its strongest. We have hired and retained record numbers of the best scientists and professionals. We have built a cohort of world-class managers and executives who are solidly at the helm of our organization and will continue to lead CDER. And most important of all, we created a culture that fosters respect for one another, inclusion, accountability, and diversity of thought and life experience. Our culture is what makes us strong and resilient, and it’s the foundation of what we have accomplished and will accomplish in the future.”

As of the time this article was written, FDA has not yet announced a replacement for Cavazzoni. Her predecessor, Janet Woodcock, was CDER director from 1994 to 2004 and then again from 2007 to 2021 (3).

®, Glenn Wright, president and CEO of the Parenteral Drug Association (PDA), said the following about Cavazzoni's retirement: 

“On behalf of PDA and our many industry members worldwide I thank Dr. Cavazzoni for her work as the CDER Center Director within FDA. Her efforts to move the industry and FDA forward to better serve patients is much appreciated. Communication between FDA and Industry is an essential part of ensuring expectations are well understood and concerns and issues raised. Her openness, and that of the Center she leads, to meet with Industry on topics of importance, speak at conferences and attend stakeholder meetings has been very helpful in this rapidly changing world.”

FDA. Patrizia Cavazzoni, M.D., Director–Center for Drug Evaluation and Research. FDA.gov. April 12, 2021. 

https://www.fda.gov/about-fda/center-drug-evaluation-and-research-cder/patrizia-cavazzoni

CDER Trade Press. CDER Director Patrizia Cavazzoni to Retire from FDA. Email Correspondence. Jan. 10, 2025.

FDA. Janet Woodcock, M.D. FDA.gov. Feb. 1, 2024. 

https://www.fda.gov/about-fda/fda-organization/janet-woodcock

Patrizia Cavazzoni, director of FDA’s Center for Drug Evaluation and Research, has announced her retirement from the agency.

Cavazzoni Retires as CDER Director

The clinical-stage biotechnology company Cour Pharmaceuticals has received an orphan drug designation from FDA for its CNP-104 for treatment of primary biliary cholangitis (PBC). CNP-104 is a biodegradable nanoparticle that encapsulates the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), which is a key autoantigen in PBC, according to Cour in a Jan. 8, 2025 company press release (1).

Orphan drug status is given to treatments for rare diseases, which within the parameters of FDA are characterized as diseases that affect fewer than 200,000 people in the United States (1). The designation allows for various incentives including tax credits for clinical trials, exemption from user fees, and up to seven years of potential market exclusivity following eventual FDA approval.

PBC is an autoimmune liver disease that is a leading cause of liver transplants among women, whom the disease disproportionately impacts (1). It is considered chronic and life-threatening. Typical symptoms include impaired bile flow, or cholestasis, and accumulation of toxic bile acids, which can lead to fibrosis, cirrhosis, and ultimately liver failure, according to Cour.

Other symptoms which Cour said affect everyday quality of life are severe itching, or pruritus, and fatigue (1). CNP-104 was designed to induce tolerance to pathogenic activated PDC-E2 T cells that customarily drive inflammation.

“Receiving [o]rphan [d]rug [d]esignation for CNP-104 underscores its potential to become the first disease-modifying treatment for individuals with PBC,” said Dannielle Appelhans, Cour president and chief executive officer, in the press release (1). “This designation follows our presentation of positive topline data from the Phase IIa clinical trial of CNP-104 in PBC at The Liver Meeting 2024. Notably, in addition to demonstrating favorable T cell responses among treated participants, CNP-104 slowed disease progression, as evidenced by a statistically significant reduction in liver stiffness measured by FibroScan by day 120 of the study period. We are now collaborating with our distinguished clinical advisors and key opinion leaders to advance CNP-104 to the next phase of clinical development.”

In December 2024, the European Medicines Agency’s Committee for Medicinal Products for Human Use recommended an orphan drug approval for another treatment for PBC, Gilead Sciences’ Livdelzi (seladelpar) (2). FDA granted this treatment an accelerated approval in August 2024, and a final European Commission decision is expected in the first quarter of 2025 (3–4).

Cour said that CNP-104 has already received fast track designation from FDA in January 2022, which would make it eligible for accelerated approval and priority review pending the satisfaction of certain criteria (1).

1. Cour Pharmaceuticals. Cour Pharmaceuticals Secures FDA Orphan Drug Designation for CNP-104 in Primary Biliary Cholangitis. Press Release. Jan. 8, 2025.
2. EMA. 

Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 9–12 December 2024.

 Press Release. Dec. 13, 2024.
3. Gilead Sciences. Gilead’s Seladelpar Receives Positive CHMP Opinion for Primary Biliary Cholangitis. Press Release. Dec. 13, 2024.
4. Gilead Sciences. Gilead’s Livdelzi (Seladelpar) Granted Accelerated Approval for Primary Biliary Cholangitis by US FDA. Press Release. Aug. 14, 2024.

CNP-104 is a biodegradable nanoparticle that previously received fast track designation from FDA in January 2022, which would eventually make it eligible for accelerated approval and priority review.

Cour Pharmaceuticals’ PBC Treatment Gets FDA Orphan Drug Designation

FDA has granted fast track designation to Johnson & Johnson (J&J) for posdinemab, an investigational phosphorylated tau-directed monoclonal antibody (mAb) for treating patients with early Alzheimer’s disease (AD), J&J announced on Jan. 8, 2025. Discovered by J&J, posdinemab is in a Phase IIb study (AuTonomy) and has shown potential in targeting disease-associated phosphorylated tau in cerebrospinal fluid (CSF) from AD patients who are being treated. The mAb has also shown an ability to block the development and spread of tau aggregates in non-clinical models of disease.

FDA’s decision on posdinemab marks the second fast track designation granted to J&J in 2024 for its AD portfolio. In July 2024, FDA granted fast track designation to J&J’s anti-tau active immunotherapy, JNJ-2056, which represents the first active immunotherapy that targets tau in a preclinical AD population (1). JNJ-2056 is in a Phase IIb trial (ReTain), which is currently actively enrolling patients. This Phase IIb trial will evaluate the potential of JNJ-2056 to activate the immune system so that it generates antibodies against pathological phosphorylated tau. The goal with this mode of action is to delay or prevent the onset of AD symptoms and, ultimately, progression of the disease.

“As the average age of the global population increases, the number of people impacted by this debilitating progressive disease continues to rise. Alzheimer’s disease places a substantial emotional and financial burden on patients and their loved ones and has a significant economic and societal impact. At J&J, we envision a future where we can help stop the progression of AD the moment it’s detected,” said Bill Martin, PhD, global therapeutic area head, Neuroscience, Johnson & Johnson Innovative Medicine, in a company press release (1).

Posdinemab targets the mid-domain of AD-specific phosphorylated tau. The mAb is designed to bind to pathological phosphorylated tau when released from neurons and to neutralize it before it can seed or spread to another neuron. Tau, which is abundant in neurons, has a notorious reputation, which is due to the fact that it is found deposited in cells as fibrillar lesions in many neurodegenerative diseases, especially AD (2). Posdinemab has shown promise in reducing tau seeding, which is the process by which toxic tau spreads through the brain, in both 

“Posdinemab has the potential to slow the spread of tau pathology in the brain—which may slow cognitive decline. [FDA’s] [f]ast [t]rack designation reflects the urgent unmet need for new treatment options for the millions living with AD,” said Martin in the press release.

Meanwhile, JNJ-2056 (formerly ACI-35.030, in collaboration with AC Immune) is an investigational anti-tau active immunotherapy that targets the c-terminal region of pathological phosphorylated tau. This therapy is designed to inhibit the seeding and spreading of pathological tau as well, also with the goal of delaying or preventing the onset of clinical symptoms in preclinical AD. The Phase IIb in which JNJ-2056 is being evaluated includes participants with preclinical AD who have tau pathology in their brains but have not yet developed cognitive impairment.

Johnson & Johnson’s Posdinemab and Tau Active Immunotherapy Receive US FDA Fast Track Designations for the Treatment of Alzheimer’s Disease

. Press Release. Jan. 8, 2025.
2. Stoothoff, W. H.; Johnson, G. V. W. Tau Phosphorylation: Physiological and Pathological Consequences. 

Biochimica et Biophysica Acta (BBA)—Molecular Basis of Disease

 (2–3), 280-297. DOI: 10.1016/j.bbadis.2004.06.017

Posdinemab is the second tau-directed investigational therapy by J&J that has received fast track designation from FDA this year for Alzheimer’s disease.

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