Chronic Disease Priorities, Biomarker Breakthroughs, and CGT-RLT Synergies

Behind the Headlines, Episode 22

ICH Q12 and Post-Approval Changes

The quickly evolving development of biopharmaceuticals is impacting innovation in the industry overall.

Advancements in Biopharma Spur Innovation

What Pharma Manufacturers Need to Know

Every Pharmaceutical Manufacturing Professional Should Know

TOP NEWS on grunge world map | Image Credit: © Sean K - © Sean K - stock.adobe.com

FDA announced on July 21, 2025 that George Francis Tidmarsh, MD, PhD has been appointed the new director of the agency’s Center for Drug Evaluation and Research (CDER), which is tasked with ensuring the safety and efficacy of drugs in the United States (1). Tidmarsh has more than 30 years of experience in biotechnology, clinical medicine, and regulatory science.

Tidmarsh attended Stanford University where he earned a degree in medicine and a PhD in cancer biology, as well as completing a residency in pediatrics. He also completed two subspecialty programs at Stanford in pediatric oncology and neonatology and is the founding co-director of Stanford’s Master of Translational Research and Applied Medicine (M-TRAM) program. M-TRAM trains students and researchers on how to translate scientific research into real-world application.

“Dr. Tidmarsh is an accomplished physician-scientist and leader whose experience spans the full arc of drug development—from bench to bedside,” said FDA Commissioner Marty Makary. “His appointment to lead CDER brings exceptional scientific, regulatory, and operational expertise to the agency. I look forward to working with him to strengthen our drug review programs, foster innovation, and advance cross-agency initiatives that improve health outcomes for the American public.”

A founder and CEO of multiple biopharmaceutical companies over the years, including La Jolla Pharmaceutical Company and Horizon Pharma, Tidmarsh has been involved with the clinical development of seven FDA-approved drugs from discovery through approval (2). Unlike previous CDER directors, Tidmarsh lacks previous experience in government, but has served as an expert on FDA panels (3).

It is speculated that Tidmarsh may play a role in reassessing FDA’s approach to prescription drug advertising (4). According to Bloomberg, Tidmarsh has expressed concerns about inconsistencies by FDA regarding the regulation “long-standing restrictions on off-label communication by manufacturers and questioned some vaccine makers’ ads during the pandemic” (4). Tidmarsh, according to Bloomberg, has also expressed concerns about fraud in academic science and funds a program supporting investigation of scientific fraud (4).

Tidmarsh has served on advisory boards across academia, government, and industry and has authored 143 scientific publications and patents.

“It is good to see a scientist and physician with phenomenal experience like Dr. Tidmarsh at the helm of CDER,” Aloka Srinivasan, a member of 

®’s Editorial Advisory Board, and principal and managing partner of Raaha LLC, said of the appointment. “Dr. Tidmarsh understands how the pharmaceutical industry operates and the roadblocks to better treatment of many diseases. As a former FDA employee and a member of the pharmaceutical industry, I hope that he is able to change the culture of the office he is heading, and incorporate better understanding of their role, which, other than just approving drugs is to see the bigger picture and understand how the approval of a drug may affect the American public."

Former CDER director retired in January 2025

CDER’s previous director, Patrizia Cavazzoni, resigned in January 2025. She started with FDA as deputy director of operations of CDER and served as acting principal deputy commissioner of Food and Drugs briefly in 2019. In 2021, she was made the director of CDER.

“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 a letter to her colleagues at the time of her retirement. “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” (5).

FDA. Stanford Faculty Member George Tidmarsh, MD, PhD Named Director of Center for Drug Evaluation and Research. Press Release. July 21, 2025. 

https://www.fda.gov/news-events/press-announcements/stanford-faculty-member-george-tidmarsh-md-phd-named-director-center-drug-evaluation-and-research

Joseph, A. FDA Taps Biotech Veteran George Tidmarsh to Lead Drug Center. 

https://www.statnews.com/2025/07/21/george-tidmarsh-fda-marty-makary-drug-center-director/

Samorodnitsky, D. FDA’s CDER Gets New Chief in Biopharma Veteran George Tidmarsh. 

Langreth, R. and Hornblower, S. FDA Appoints Biotech Executive as Top Drug Regulator Under RFK Jr. 

Haigney, S. Cavazzoni Retires as CDER Director. 

https://www.pharmtech.com/view/cavazzoni-retires-as-cder-director

Articles

George Francis Tidmarsh, MD, PhD, founding co-director of Stanford’s Master of Translational Research and Applied Medicine program, has been appointed as the new director of the Center for Drug Evaluation and Research.

FDA Appoints New CDER Director, George Tidmarsh

Simona Guidi, Associate Director, Cell and Gene Therapies at ProPharma discusses human contamination considerations during drug manufacturing

Operators Are the Main Source of Contamination in Pharmaceutical Manufacturing


In this short clip from the June episode of our 

 video series, Simona Guidi, Associate Director, Cell and Gene Therapies at ProPharma, explains the importance of considering humans as a top source of contamination in pharmaceutical manufacturing.

Check out the full episode with Guidi on 

 in which she answers questions about options for controlling adventitious contamination for cell processing requiring extensive manipulation and how to protect the product and operating when moving to a closed processing system for cell therapy production.

Videos

Young man having asthma attack or choking suffering from respiration problems | Image Credit: © Feodora - stock.adobe.com

The Medicines and Healthcare products Regulatory Agency (MHRA) has approved EURneffy 2 mg, a needle-free adrenaline nasal spray designed to treat anaphylaxis in adults and children weighing 30 kilograms (66 pounds) or more in the United Kingdom (1). It’s the first product of its kind authorized in the country, and its launch is anticipated in the latter half of 2025, following final market access negotiations.

This milestone approval introduces a fundamentally different route of administration for emergency adrenaline, delivered intranasally rather than via auto-injector (1). The shift comes as manufacturers and developers across the pharmaceutical industry continue to explore more intuitive, less invasive drug delivery formats that can meet real-world demands, particularly in high-stakes use cases like allergic reactions (2–6).

Redesigning emergency treatment with device innovation

·MHRA approval of EURneffy introduces the first nasal adrenaline spray for anaphylaxis, offering a needle-free alternative to traditional auto-injectors.

·EURneffy features 30-month shelf life and high temperature stability, addressing long-standing cold chain and storage challenges in pharma logistics.

·The product’s design reflects growing industry emphasis on human factors engineering to improve real-world usability and adherence in emergency drug delivery.

EURneffy’s design eliminates the needle entirely, a departure from decades of reliance on injection-based delivery (1). For patients and caregivers, this simplifies use during the chaotic moments of anaphylaxis, when speed and confidence are critical. It also opens new considerations for drug-device development: designing for intuitive usability while maintaining pharmacologic equivalence to injectable formats.

Henriette Mersebach, MD, executive vice president of research and development at ALK, emphasized this point in a statement, stating, “The approval of EURneffy 2 mg in the UK introduces an alternative and novel adrenaline treatment option for adult and adolescent patients with life-threatening allergies. EURneffy could improve the lives of people with severe allergic reactions and may facilitate that patients and caregivers continually carry adrenaline wherever they go" (1).

The product is also formulated to be stable for up to 30 months and at a wider range of temperatures than existing auto-injectors (1). This extended stability means fewer constraints in transportation and storage, allowing for broader reach and potentially reducing waste from expired inventory. It also reflects a growing focus within pharma on formulations that ease cold-chain requirements without compromising efficacy (7–11).

Clinical program supports pharmacological performance

MHRA approval followed a review of data from more than 700 participants enrolled in the EURneffy development program (1). Across a range of dosing scenarios, the pharmacokinetic and pharmacodynamic profiles of the nasal spray were consistent with those of traditional auto-injectors. No serious adverse events were reported during clinical studies.

Anaphylaxis affects approximately eight in every 100,000 people annually in Europe, and about one in 300 will experience it at some point in their lives (1). Despite this prevalence, hesitancy and misuse around auto-injectors remain common. EURneffy highlights how thoughtful design and delivery innovation can help improve adherence by minimizing user burden.

The nasal spray was originally developed by ARS Pharmaceuticals, based in the United States (1). In 2024, ALK entered into a licensing agreement with ARS to commercialize the product outside the United States, Australia, New Zealand, Japan, and China. The partnership expanded in 2025 to include co-promotion in the US market.

Although the approval marks a significant product development and regulatory milestone, the company noted it will not impact its financial guidance for 2025 (1).

EURneffy Approved as the First Needle-free Anaphylaxis Treatment of Adults and Children in the UK

Nasal Drug Delivery Technology Market Size - Global Industry, Share, Analysis, Trends and Forecast 2024 – 2032

Nasal Drug Delivery Technology Global Market Report 2025 – By Dosage Form (Nasal Spray, Nasal Drops, Nasal Gels And Ointments, Nasal Powders, Other Dosage Forms), By Container Type (Pressurized Containers, Non-Pressurized Containers), By Therapeutic Application (Allergic And Non-Allergic Rhinitis, Nasal Congestion, Asthma, Vaccination, Other Therapeutic Applications), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, Online Pharmacies), By End User (Home Care Settings, Hospitals) – Market Size, Trends, And Global Forecast 2025-2034

Nasal Drug Delivery Market Size & Share Analysis – Growth Trends & Forecasts (2025 – 2030)

Intranasal Drug and Vaccine Delivery Market Size, Share & Trends Analysis Report By Product, By Dosage (Deep Learning, Machine Learning), By Application, By Distribution Channel, By Region, And Segment Forecasts, 2025 – 2030

Nasal Drug Delivery Market Size, Share and Industry Analysis, By Type (Dry Powder Inhalers (DPIs), Metered Dose Inhalers (MDIs), and Nebulizers), By Application (Asthma, Chronic Obstructive Pulmonary Disease (COPD), and Others), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies) and Regional Forecast, 2019-2032

Improving Sustainability in Pharma Cold Chain

Pharmaceutical Temperature Controlled Packaging Solutions Market Size Growth Acceleration During 2023- 2032

Cold Chain Innovation - Transforming the Pharmaceutical Packaging Industry

Pharmaceutical Temperature Controlled Packaging Solutions Market Investment Opportunities & Competitive Benchmarking

Newly approved needle-free adrenaline spray offers longer shelf life, better stability, and easier use, signaling a shift in emergency drug delivery design.

UK Anaphylaxis Nasal Spray Approval Helps Reshape Emergency Allergy Treatment

3d render, number six glowing in the dark, pink blue neon light | Image Credit: © NeoLeo - stock.adobe.com

Pharmaceutical compounding, particularly in 503A and 503B environments, involves complex formulation and regulatory challenges far beyond academic theory. This summary of 

 on this topic—written by industry professionals Paul Pluta RPh, PhD, Alan Mancini RPh, Nishant Thakar, RPh, PharmD, and Varanya Chaiyaperm, RPh, PharmD—distills the essential technical and operational takeaways from a comprehensive, six-stage model of pharmaceutical compounding. Ideal for professionals in drug development, manufacturing, QA/QC, and regulatory compliance, this summary emphasizes real-world application, risk mitigation, and alignment with quality frameworks like Quality by Design, Good Manufacturing Practices (GMP), and Quality Risk Management (QRM).

Stage 1: design the compounding blueprint

Establish a complete plan including ingredients, packaging, calculations, labeling, and patient-specific factors.

Integrate regulatory standards (US Pharmacopeia, FDA bulk lists, GMP) and proactively identify risks using QRM tools.

Ensure aseptic setup, verified materials, and technician preparedness—especially critical for sterile and high-risk compounds.

Require dedicated or disposable equipment for hazardous or cross-contaminating drugs (e.g., antineoplastics, allergens).

Stage 3: dosage form completion & quality evaluation

Conduct rigorous physical and analytical testing; 503B batches must meet GMP-level standards.

Finalize documentation with full traceability and ensure preparations are quarantined until verified.

Stage 4: packaging & patient-ready delivery

Assemble patient/client information equivalent to commercial products. Ensure all packaging, labeling, and delivery logistics meet regulatory and clinical standards.

Implement safeguards for temperature control and light protection during distribution.

Stage 5: independent verification before release

Engage an independent observer to verify formulation integrity, test results, documentation, and labeling accuracy.

Use checklists to prevent critical errors and maintain consistent approval protocols.

Stage 6: dispensing, distribution & final safety checks

Dispense only after final pharmacist approval, ensuring two-point patient ID verification.

Monitor for common hazards like name mix-ups, transcription errors, and shipping delays.

Management takeaways: quality starts at the top

Leadership must prioritize hiring, SOP adherence, continuous training, and fostering a quality-driven culture.

Management bears ultimate responsibility for SOP execution, staff performance, and patient safety outcomes.

The six-stage compounding model offers a structured, risk-aware approach to compounded drug development. For pharmaceutical manufacturing professionals, it ensures regulatory alignment, enhances process reproducibility, and reinforces a culture of quality and accountability across the compounding lifecycle.



A structured, risk-based framework for pharmaceutical compounding that enhances quality, regulatory compliance, and process consistency.

The 6-Stage Compounding Framework Every Pharmaceutical Manufacturing Professional Should Know

Kyiv, Ukraine - December 22, 2020: Backlit single shot image of GlaxoSmithKline logo on tv screen with a hand holding an Covid-19 vaccine concept | Image Credit: © Олександр Луценко - stock.adobe.com

FDA has approved a prefilled syringe version of GSK’s Shingrix, a recombinant zoster vaccine used to prevent shingles, eliminating the need for reconstitution of separate vials prior to administration (1). The newly approved format aims to ease preparation for healthcare professionals, enhancing efficiency and reducing the risk for handling errors during vaccination.

The previous formulation of the shingles vaccine required clinicians to combine a lyophilized antigen with a liquid adjuvant prior to administration (1). FDA’s approval of the new single-syringe version follows data confirming its technical comparability with the original two-vial presentation.

·FDA approval of the prefilled syringe version of the recombinant zoster vaccine supports manufacturing shifts toward ready-to-use, single-dose formats.

·Technical comparability data enabled regulatory clearance without reformulation, highlighting the value of platform consistency in vaccine development.

·Prefilled syringe design may enhance uptake by reducing administration errors and simplifying logistics across healthcare and immunization programs.

Brigid Groves, vice president of professional affairs at the American Pharmacists Association, emphasized the practical benefits of this update (1): “The prefilled syringe presentation of GSK’s shingles vaccine is good news, providing a convenient method of administration. The FDA approval is a positive step toward driving prevention of this painful disease, and as a practicing pharmacist I welcome the availability of this new presentation.”

Expanded access and continued eligibility guidelines

As with the original version, the prefilled syringe formulation is licensed in the United States for immunization of adults aged 50 and older, as well as individuals aged 18 and older who are or will be immunocompromised due to disease or medical treatment (1). Current guidance from the Centers for Disease Control and Prevention recommends a two-dose series of the vaccine for these groups to help prevent shingles and its complications.

Tony Wood, chief scientific officer at GSK, commented on the public health rationale for the updated presentation, stating “at GSK, we are committed to advancing scientific innovation and delivering practical solutions that address the needs of the healthcare community. This new presentation of Shingrix was developed to streamline the vaccination process, supporting healthcare professionals to provide protection against shingles, a disease that one in three US adults will develop in their lifetime” (1).

In parallel to the US approval, the prefilled syringe presentation is under review by the European Medicines Agency, with the application accepted for evaluation in January 2025 (1). Regulatory submissions are also being considered for additional international markets.

These developments reflect a broader industry focus on increasing adult immunization rates through simplified vaccine formats.

Shingles, also known as herpes zoster, is a painful skin rash caused by reactivation of the varicella-zoster virus (VZV)—the same virus responsible for chickenpox (2). While not all individuals with latent VZV will develop shingles, nearly 99% of US adults over 50 harbor the virus, and about one million cases are diagnosed annually in the US (3).

With age, immune function diminishes, increasing the likelihood that the virus will reactivate (4). This makes older adults more susceptible to shingles and its associated complications, including postherpetic neuralgia, a condition characterized by long-lasting nerve pain.

The recombinant zoster vaccine is a non-live, subunit formulation that combines glycoprotein E with the proprietary AS01B adjuvant system (1). It is designed to trigger a strong immune response even in older adults, whose immune systems may respond less robustly to vaccination. The vaccine is not intended to prevent chickenpox or primary varicella infection.

The newly approved Shingrix format comes just days after GSK announced that FDA had accepted GSK’s application to extend the indication for the company’s respiratory syncytial virus (RSV) vaccine, adjuvanted (Arexvy) to adults aged 18–49 who are at increased risk (5). The vaccine is approved in the US to prevent lower respiratory tract disease (LRTD) caused by RSV in adults older than 60 years. It is also approved to treat people aged 50–59 who have increased risk for LRTD caused by RSV.

US FDA Approves GSK’s Shingrix in a Prefilled Syringe Presentation. Press Release

Harpaz, R.; Ortega-Sanchez, I.; Seward, J.; et al. 

Prevention of Herpes Zoster: Recommendations of the Advisory Committee on Immunization Practices (ACIP)

Varicella Zoster Virus Infection: Clinical Features, Molecular Pathogenesis of Disease, and Latency

GSK Seeks Expanded Use Approval from FDA for RSV Vaccine Arexvy

FDA Approves Shingrix Prefilled Syringe to Simplify Vaccine Administration

Tore Bergsteiner summarizes the promise of AI

AI Boosts Early Disease Detection and Diagnostic Accuracy

 back in March 2025, Tore Bergsteiner, managing partner at MAIN5, a European consulting firm specializing in digitally enabled change for life sciences R&D organizations, discusses how AI is increasingly enhancing diagnostics and pathology by enabling early disease detection through advanced image analysis, improving treatment outcomes and patient safety. He also notes how AI helps address global shortages of pathologists by boosting diagnostic efficiency and accuracy. Both points still hold true 4 months later.

Check out our full interview with Tore 

Tobin bridge, Zakim bridge and Boston skyline panorama at sunset | Image Credit: © mandritoiu - stock.adobe.com

The 15th annual Partnership Opportunities in Drug Delivery (PODD) conference, set for October 27–28, 2025, at the Westin Copley Place in Boston, will spotlight the latest developments in drug delivery technology with a program of expert panels, presentations, and industry-driven sessions (1).

Organized by the Conference Forum, the event brings together pharmaceutical developers, biotech innovators, and drug delivery specialists to examine advances in the formulation, design, and commercial deployment of therapeutics across a range of modalities (1).

Focus on strategic partnering in drug delivery

·Drug delivery partnerships are evolving, with top pharma leaders outlining strategic evaluation frameworks for external technologies and collaboration models.

·Pharma innovation leaders are refining internal scouting, sharing insights into sourcing enabling technologies that accelerate drug development pipelines.

·Health economics of drug delivery are under scrutiny, as payers assess how delivery systems influence value, pricing pressure, and access for emerging therapies.

Partnering to Advance Drug Delivery Innovation

, director of strategic innovation for rare diseases at Chiesi USA (1). The session will explore critical considerations for evaluating external drug delivery platforms, with panelists offering perspectives from both early-stage and mature development environments.

, research & development advisor for Genetic Medicine at Eli Lilly and Company

, head of advanced drug delivery at AstraZeneca

, head of global biopharmaceutics and senior research fellow at Pfizer

, director of portfolio strategy & integration at Bristol-Myers Squibb.

The discussion will focus on partnership pathways, strategic fit assessments, and the evolving landscape of delivery technology collaborations (1).

Pharmaceutical leaders share internal approaches to innovation

Representatives from Novartis and AbbVie will offer insights into their respective organizations' approaches to sourcing and integrating drug delivery and enabling technologies (1). 

, global head of innovation at Novartis, and 

, senior director of landscaping & platform definition at AbbVie, will present as part of the conference’s 

These presentations are expected to provide transparency into current technology scouting efforts and collaborative models used to accelerate therapeutic development (1).

Economic considerations in drug delivery adoption

 will explore the intersection of innovation and reimbursement, with a focus on the pharmacoeconomics of delivery-enabled therapeutics (1). The panel will assess how delivery systems influence value perception, pricing pressures, and market access—especially in the context of drug classes such as incretins and nucleic acid-based therapies.

Dr. Gail Ryan, PharmD, formerly of Point32Health.

Technology tracks cover injectable, non-injectable, and genetic delivery

The 2025 agenda includes a comprehensive lineup of drug delivery technology presentations categorized into several themes (1):

injectable formulations and delivery platforms

self-injection devices, wearables, and connected delivery tools

oral, nasal, pulmonary, and alternative non-injectable systems

cell and gene therapy delivery approaches

innovations in excipients and combination product platforms

primary packaging solutions including syringes and containers.

Emerging topics in advanced therapeutic delivery

Sessions will also explore specialized approaches to delivery challenges in advanced modalities (1). Topics include:

brain delivery via formulation and physical mechanisms

novel vehicles for genetic medicine administration

tissue-specific targeting for nucleic acid therapeutics

manufacturing scale-up strategies for clinical and commercial readiness

sustainability in drug and device development.

Industry participation reflects broad engagement

The conference will feature speakers and presenters from across the global pharmaceutical ecosystem (1). Participating companies include major developers and technology providers working in small molecules, biologics, cell and gene therapies, and RNA-based drugs.

Participating organizations include: Amgen, Astellas Pharma, AstraZeneca, Beam Therapeutics, Biogen, Boehringer Ingelheim, Bristol-Myers Squibb, Chiesi USA, Eli Lilly and Company, Genentech, Gilead Sciences, GSK, Merck, Moderna, Novartis, Novo Nordisk, Pfizer, Regeneron, Roche, Sanofi, Sun Pharma, Takeda Pharmaceuticals, Teva Pharmaceuticals, UCB, and others.

PODD’s role in the drug delivery ecosystem

"PODD connects industry leaders and innovators who advance the design and development of drug delivery systems and devices to improve patient lives," said Andrew Goldstein, senior conference producer (2).

Designed to support scientific exchange and partnership building, PODD offers attendees a structured environment to explore delivery solutions for a wide range of therapeutic challenges (1). The forum supports deal-making, technology scouting, and collaboration-building across traditional boundaries in the pharmaceutical sector.

The full agenda release comes three weeks after keynotes were announced (3). At the time, Goldstein said, “In presenting a strategic-level program for R&D scientists and business development professionals, the PODD Conference is committed to securing keynotes who demonstrate excellence in science.”

Keynotes will feature prominent voices in pharmaceutical science and patient advocacy (3). 

, MD, PhD, co-founder and chief scientific officer of Regeneron, will join Massachusetts Institute of Technology’s 

, to discuss the current landscape and future potential of drug delivery innovation. 

, chief scientific advisor at Novo Nordisk, will present on the development of liraglutide and semaglutide, emphasizing how drug delivery intersected with the scientific and clinical advancement of these obesity treatments. Patient advocate 

 will share his experience living with sickle cell disease and undergoing treatment with a clustered regularly interspaced short palindromic repeats, or CRISPR, -based therapy, as well as his efforts to improve access to genetic medicines. 

, university distinguished professor at Northeastern University, will present on strategies for endogenous targeted delivery, addressing how biological transport mechanisms can be leveraged to improve drug efficacy and safety.



Partnership Opportunities in Drug Delivery 2025

Leading Drug Delivery Conference PODD Announces its 15th Annual Agenda

PODD Drug Delivery Conference Announces 2025 Keynotes. Press Release

The annual Boston conference will explore strategic partnering, device innovation, and delivery solutions for emerging therapeutics.

PODD 2025 Unveils Agenda Highlighting Innovation in Drug Delivery Technologies

ADELAIDE, SOUTH AUSTRALIA. - On November 15, 2018. – The University of South Australia is a public university, it is main campus is located on North Terrace in the Adelaide city centre | Image Credit: © arliftatoz2205 - stock.adobe.com

A novel once-weekly injectable formulation developed by researchers at the University of South Australia (UniSA) introduces a new drug delivery paradigm for Parkinson’s disease and potentially other chronic conditions (1). Designed to administer levodopa and carbidopa over seven days via a biodegradable, subcutaneous 

 forming implant, this system could reduce pill burden for patients while presenting significant implications across pharmaceutical discovery, development, and manufacturing (2).

“Our goal was to create a formulation that simplifies treatment, improves patient compliance, and maintains consistent therapeutic levels of medication,” said 

, professor of Pharmaceutical Science at the University of South Australia (1). “This weekly injection could be a game-changer for Parkinson’s care. Levodopa is the gold-standard therapy for Parkinson’s, but its short life span means it must be taken several times a day.”

 forming biodegradable implants enable sustained levodopa release, requiring novel aseptic processing and polymer-handling techniques.

· Formulation uses FDA-approved PLGA and Eudragit polymers, spotlighting the growing role of excipient science in lifecycle drug innovation.

· Weekly injectable supports 505(b)(2) and EMA hybrid pathways, offering a streamlined regulatory route for reformulated legacy therapeutics.

Extending innovation through delivery science

Although the active ingredients—levodopa and carbidopa—are well-established in Parkinson’s therapy, the innovation lies in the delivery system. This approach reflects a growing emphasis on lifecycle management of existing drugs through formulation science, an area increasingly recognized as critical in modern drug discovery pipelines (3). The use of a pH-responsive polymer blend—PLGA (an FDA-approved biodegradable polymer) and Eudragit L-100—to achieve consistent plasma levels over a full week demonstrates the role of excipient and polymer science in extending the therapeutic impact of legacy molecules (2).

This formulation trend aligns with industry strategies focused on enhancing the therapeutic index and patient adherence of known pharmacophores through novel delivery systems, especially in diseases with long treatment durations and high unmet needs (4).

Patient-centric reformulation and regulatory potential

From a drug development perspective, UniSA’s injectable system offers a reformulation strategy that aligns with patient-centric design principles, increasingly prioritized by regulators and industry sponsors alike (5). Parkinson’s disease currently requires multiple daily doses of oral levodopa/carbidopa to manage motor symptoms due to the drugs’ short plasma half-lives and rapid peripheral metabolism (6). This leads to fluctuations in drug levels and decreased clinical control, a challenge that this once-weekly delivery system aims to address by providing a more stable pharmacokinetic profile (2).

 data from UniSA’s study indicated that more than 90% of levodopa and 81% of carbidopa were released steadily over seven days (1). The biodegradable implant degraded by more than 80% during that period, and cell viability tests showed no significant cytotoxicity (2). These early findings support the potential for human translation.

Importantly, development teams may consider regulatory pathways, such as FDA’s 505(b)(2) route or the European Medicines Agency’s hybrid applications, which allow for the incorporation of published literature and data on approved actives, reducing the burden of full clinical development while still requiring robust evidence of safety and efficacy for the new delivery system (7,8).

 forming injectable implants introduces new challenges and considerations for pharmaceutical manufacturing. UniSA’s system requires formulation techniques that accommodate both the controlled encapsulation of active compounds and the precise modulation of polymer degradation kinetics. Manufacturing such systems typically involves aseptic processing, solvent handling, and quality control of biodegradable polymer blends, all of which differ significantly from those used in oral solid dosage forms (9).

Administering the formulation through a 22-gauge needle also implies the need for tight control over viscosity, injectability, and sterility during scale-up, especially under good manufacturing practice conditions (2). In addition, such polymers as PLGA and pH-sensitive materials like Eudragit may require controlled storage temperatures to maintain stability and prevent premature polymerization or phase separation (10). These requirements may necessitate cold-chain logistics infrastructure or temperature-monitored storage and shipping protocols.

Moreover, the potential for this delivery platform to be customized for release periods ranging from days to weeks presents a modular approach that could appeal to contract development and manufacturing organizations and large-scale manufacturers focused on flexible manufacturing lines for different release profiles and therapeutic indications (2).

Combination therapy and platform applications

UniSA researchers also noted that the injectable platform could be adapted for other chronic conditions requiring sustained drug exposure, including cancer, neurodegenerative diseases, diabetes, chronic pain, and infectious diseases (1). In this context, the platform offers potential for future combination products, in which two or more actives could be delivered together in a single depot, especially relevant in Parkinson’s, for which adjunctive therapies like MAO-B inhibitors or dopamine agonists are frequently prescribed alongside levodopa (6).

Such versatility could support combination regimens that improve therapeutic outcomes and simplify complex dosing schedules, a goal aligned with broader efforts in pharmaceutical development to enhance real-world adherence and reduce polypharmacy burden (11).

The UniSA injectable system for Parkinson’s therapy illustrates how innovative drug delivery mechanisms can drive significant therapeutic and operational advances, even without introducing new molecular entities. For professionals working across the drug development spectrum, this approach exemplifies how novel formulations can improve adherence, reduce dosing frequency, and create new opportunities for market differentiation.

As the researchers move toward human trials, this work may catalyze further investment in long-acting delivery systems that support sustained and controlled drug exposure. Downstream, it may also prompt regulatory and commercial stakeholders to reconsider value frameworks for reformulated legacy therapies with demonstrated real-world impact.


Weekly Injection Could be Life Changing for Parkinson’s Patients. Press Release

Nakmode, D.; Abdella, S.; Song, Y.; Garg, S. 

Development of an In-situ Forming Implant System for Levodopa and Carbidopa for the Treatment of Parkinson’s Disease

Effects of New Formulation Strategy on Life Cycle Management in the US Pharmaceutical Industry

Repurposing and Reformulation: Unlocking New Therapeutic Avenues through Formulation Optimization

Patient-Focused Drug Development: Collecting Comprehensive and Representative Input: Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders

 (CDER, CBER, June 2020) (accessed July 15, 2025).

Olanow, C.; Agid, Y.; Mizuno, Y.; et al. 

Levodopa in the Treatment of Parkinson’s Disease: Current Controversies

Applications Covered Under Section 505(b)(2)

 (CDER, October 1999) (accessed July 15, 2025).

Biodegradable weekly implant delivers stable levodopa levels, aiming to streamline Parkinson’s care while reshaping drug reformulation and delivery strategies.

Featured Content

Behind the Headlines, Episode 22

ICH Q12 and Post-Approval Changes

Advancements in Biopharma Spur Innovation

What Pharma Manufacturers Need to Know

Every Pharmaceutical Manufacturing Professional Should Know