Pharmaceutical Technology Is Now PharmTech: A Sharper Name for a Smarter Industry

Navigating FDA Shakeups and Obesity Drug Wars

Behind the Headlines Episode 28

Ask the Expert

Closing the Gap in Modern Process Control and Automation for QC/QA

Drug Digest December 2025

How It Helps Avoid Facility and Equipment Problems

A “Plan, Prevent, Prove” Approach

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

Since 2020 and the United Kingdom’s exit from the European Union, Britain has been locked out of the Horizon research programme, despite agreements being in place for associate membership, as arguments over the Northern Ireland protocol persisted. However, in March 2023, after the implementation of the Windsor framework, talks between the UK and EU about the association with the programme resumed, culminating in reports of a draft deal being reached on 5 July (1).

This draft deal is reportedly now under consideration by the UK’s Prime Minister, Rishi Sunak (1). Sunak is being urged to finalise the deal to ensure the scientific community in the UK can regain the advantages of the Horizon programme, such as funding and leading multi-country research consortia.

“Outside Horizon Europe, the UK is in real danger of ceding our hard-won position in the global R&D hierarchy and becoming less attractive as a research partner and less attractive for foreign direct investment,” stressed Andy Slaughter, member of parliament for Hammersmith in a parliamentary debate on the topic of Horizon Europe (2). “As part of Horizon Europe, the UK can influence the future direction of billions of pounds worth of research investment to more closely align with UK strategic priorities.”

Cost has been a factor delaying the draft deal. Although not required to pay participation fees for the two missed years, the UK government has requested further discounts due to the fact that researchers and businesses in the country have been weakened when compared with EU counterparts from missing out on collaborative opportunities. Additionally, the UK government have pushed back on the matter of how much research funding the country will receive in return for its input into the programme (3).

Officially, talks are still ongoing with no confirmed deal struck yet—at the time of writing—so, for now, a return to a closer scientific research relationship between the UK and the EU remains just out of reach on the horizon.

1. Gallardo, C. Britain and EU Agree Draft Horizon Deal. 

, 5 July 2023.
2. UK Parliament. Research and Development Funding and Horizon Europe. Parliamentary Debate, 18 April 2023.
3. Ghosh, P. Horizon Research Deal with EU Awaits Sunak’s Signature. 

Felicity Thomas is the European/senior editor for Pharmaceutical Technology Group.

When referring to this article, please cite it as Thomas, F. The Swiss Appeal. 

Articles

The UK and EU have negotiated a new draft deal for Britain to re-join the Horizon Europe research programme.

New Deal on the Horizon

Jill Wechsler is Pharmaceutical Technology's Washington Editor, jillwechsler7@gmail.com.

US Capital Building. | Image Credit: © W.Scott McGill - W.Scott McGill - stock.adobe.com

FDA recently took the unusual step of approving the import of a widely used cancer drug from a Chinese manufacturer in the face of an outcry from physicians and patients over short supplies of vital oncology therapies. Although the regulators have revved up efforts in recent years to better prevent and address drug shortages, serious problems continue to plague drug production and distribution, particularly for a number of generic injectables.

National drug shortages rose in the first quarter of this year, according to the University of Utah Drug Information Service for the American Society of Health-System Pharmacists (ASHP) (1). Most of the serious shortages involve sterile injectables that are difficult to obtain quickly from alternative sources. FDA officials indicated they were looking for additional temporary suppliers of cisplatin, carboplatin, and methotrexate, all in short supply at cancer centers. FDA posted information on temporary importation of unapproved cisplatin from Chinese manufacturer Qilu Pharmaceuticals, to be distributed in the United States by Canadian-based Apotex Corp., on its drug shortages page on June 6, 2023 (2). FDA Commissioner Robert Califf noted in a social media message that the drug will come from registered facilities and will undergo careful assessment to ensure that the imported injectable chemotherapy is safe for patients.

Another example of FDA exercising regulatory flexibility is its decision to permit India manufacturer Intas Pharmaceuticals to export cisplatin and several other medically necessary injectable medications in short supply in the US, despite citation for multiple manufacturing deficiencies found during recent site inspections (3). FDA has required the company to provide third-party certification to confirm that products meet quality attributes and are not involved in data integrity violations. With the resumption of FDA plant inspections delayed by the COVID-19 pandemic, particularly with foreign facilities, the agency has uncovered more data and production quality issues that have led to import restrictions which have limited supplies.

Manufacturers are required to notify FDA in advance of anticipated production and supply problems, and the agency encourages industry to establish redundant production operations. But plant closures and problems in obtaining needed ingredients and supplies often cause supply reductions that can hit patients hard. Although FDA gained added authority during the COVID-19 pandemic to expand manufacturer notification of supply difficulties, notably for medical devices, some of these authorities are expiring. Legislation to strengthen reporting requirements for drug and medical devices makers have been proposed for inclusion in a measure to reauthorize the Pandemic and All-Hazards Preparedness Act, but dissension has delayed such efforts. Some legislators also want to revise the Controlled Substances Act to ensure patient access to ADHD treatment Adderall by better control of supply and preventing diversion.

FDA also would like to be able to require industry reports on rising demand for certain drugs, as occurred during the recent surge in respiratory infections. The agency currently lacks authority to collect this information and cannot compel any firm to produce more of a certain product. Brand manufacturers of new treatments, such as Novo Nordisk’s new weight-loss drug Wegovy, can better manage supply by taking steps to lessen demand, such as reducing supplies of a lower starting dose to discourage new patient treatment. Ideally, firms could respond to supply problems by shifting production lines to increase production of a needed medicine, but often are hampered by limited staff, resources, and ingredients. FDA recommends that manufacturers stockpile backup supplies of critical medicines, but, again, cannot require such action.

The broader problem is that shortages of cancer drugs and many widely used sterile injectables are produced by generic-drug manufacturers with limited redundancy in production due to very tight margins, as health plans and pharmacy managers push down prices. If a facility inspection uncovers serious quality issues or a company experiences increased costs for materials and operations, that may compel a plant shutdown that can impact the broader supply chain. In remarks at the annual meeting of the Biotechnology Information Organization (BIO) in June 2023, Commissioner Califf ascribed most drug shortages to generic-drug companies going out of business, either because they fail to invest in quality operations or because they can’t make a profit, as hospitals and pharmacies seek lower prices.

Such concerns were cited in a discussion of supply chain resilience at the Food and Drug Law Institute (FDLI) annual conference in May 2023, where a generic-drug maker warned against imposing added costs and burdens on industry, such as requiring production of extra supplies or stockpiles, due to unpredictable revenues from their commodity-like products. He advised against FDA’s proposed quality metrics requirements, which assume that purchasers will pay more for medicines from facilities that earn high quality ratings, a scenario that generics firms consider unlikely.

FDA described the challenges in coping with these issues in its latest report to Congress on drug shortages for 2022 (4). This annual update required by Congress since 2012 cites a decline in new drug shortages last year, but acknowledges still high ongoing shortage situations, as seen in the recent ASHP report. The agency continues to press for early notification of anticipated shortage situations to provide time to work with manufacturers to identify treatment options and to provide regulatory flexibility where appropriate. The report also describes increased collaboration with foreign regulatory authorities to identify supply chain disruptions involving overseas suppliers. FDA would like more information from manufacturers on supply chain components, such as inventory levels, and proposes to expedite the review and approval of generic-drug applications and supplements to address short supplies.

FDA. FDA Drug Shortages. Current and Resolved Drug Shortages and Discontinuations Reported to FDA. 

FDA. Letter to Mr. Nimish Chudgar, Intas Pharmaceuticals Limited. June 2, 2023. 

FDA. Report to Congress. Drug Shortages CY 2022. 

Jill Wechsler is Washington reporter for 

When referring to this article, please cite it as Wechsler, J. Drug Shortages Continue to Plague FDA and Manufacturers. 

Problems continue despite actions by regulators to better prevent and address drug shortages.

Drug Shortages Continue to Plague FDA and Manufacturers

Siegfried Schmitt, PhD, is Vice President Technical at Parexel International, Siegfried.Schmitt@parexel.com.

Question mark background | Image Credit: © Leigh-Prather - Leigh Prather - stock.adobe.com

Q: FDA publishes redacted Warning Letters and Form 483s with inspection observations on their website. What can we learn from these, and where do we need to be cautious?

A: Such information can be considered compliance intelligence (i.e., it forms part of the pool of information a company relies on to build an appropriate quality management system, design its systems and operations in such a way that they adhere to the regulations and deliver a product that meets its predetermined quality attributes, and to build and staff their organization adequately). Regulations and laws are limited in their comprehensiveness, which means that there are many possible ways for companies to achieve compliance. Sometimes, the appropriateness is challenged or found defective in inspections by regulators, and this will lead to observations on FDA’s Form 483. In cases where FDA finds that the shortcomings are not addressed sufficiently and completely, the agency may issue a Warning Letter (WL). These are meant to be a last warning (1,2).

Clearly, any company can learn from mistakes made by others, helping them to prevent making the same mistakes. To understand the benefits and limitations of the learnings from WLs and form 483s, we need to look at what information they contain, or not. An observation on Form 483 may read like this: “Your media fills failed to accurately simulate commercial operations. Our inspection found the aseptic operations simulated during your media fills were not sufficiently representative of commercial aseptic manufacturing operations for (b)(4). For example, the type and frequency of manual interventions was not representative.” What we can learn from this is that media fills must be as similar as possible to normal aseptic operations.

However, there are many things that we do not know, such as:

Did the company apply a risk-based approach that was not acceptable to the inspectors, or was it simply that the company decided to do less during the media fills?

Were the operators following the protocol or did they deviate?

Did the protocol provide sufficient clarity and detail or did the company rely on the experience of the operators?

Is there an appropriate quality culture in that company?

Were operators and managers insufficiently trained or aware of the regulations, or were they knowingly ignorant?

Was there adequate quality oversight or not?

Was this the first time, the company performed media fills?

Does the operation require many different manual interventions or only a few?

Did this happen throughout the media fill or perhaps only during start-up, or perhaps only one of the shifts, or perhaps only with some of the media fills?

It is very unlikely that a reader of the FDA observation will have the answers to these questions. Yet, this is not necessary. It is important to understand the context of the observation and appreciate its seriousness. Then you need to ask the above questions about your operations, about your media fills. That is the real value that you will gain from these regulatory observations.

When referring to this article, please cite it as Schmitt, S. The Inspection Result: Warning Letters and Form 483s. 

FDA Warning Letters and Form 483s can offer a path to better compliance, says Siegfried Schmitt, vice president, Technical, at Parexel.

The Inspection Result: Warning Letters and Form 483s

The topic of “innovation” is an ephemeral, shapeshifting beast. While today the industry envisions concepts such as personalized vaccines or oral biologics, 10 years from now pharmaceutical leaders will have surely moved on to even bigger, brighter, and more compelling technologies. 

However, it is because of this continual evolution that innovation, as a concept, holds a sanctified status for members of the pharmaceutical industry. Consequently, any perceived diminution will be challenged with immediate furor. For instance, the 

, who’s price controls have received significant pushback from venture capitalists (1), has now resulted in Merck, known as MSD outside of the United States and Canada, suing the US federal government (2). Similarly, in the Amgen v. Sanofi Supreme Court case, which tested whether broad genus claims were legally permissible (3), both companies’ arguments centered around stifling innovation—just at different steps of the pharma lifecycle. 

I question to what end “stifling innovation” can serve as an effective argument when it can seemingly be applied anywhere, to any situation, without constraint. If any proposed rule change stifles innovation, then the industry’s structure will remain status quo; however, isn’t maintaining the status quo itself antithetical to the core ethos of innovation, indeed to that of the very concept of progress itself? Innovation is crucial, and strong efforts to protect it should be made. We should not conflate innovation with simple “change.” However, if we view a change opposite of one’s interest as antithetical to innovation, we risk losing sight of our ultimate goal: real progress. 

1. Playter, G. The United States and the Global BioPharma Market. 

2. Merck. The Inflation Reduction Act’s Negative Impact on Patient-Focused Innovation, Value and Access. 

3. Wingrove, P. Amgen-Sanofi Patent Case Divides Makers of Antibody Drugs. 

When referring to this article, please cite it as Playter, G. Innovation Idolotry. 

If we view a change opposite of one’s interest as antithetical to innovation, we risk losing sight of our ultimate goal.

Innovation Idolatry

Mike Hennessy Jr. is the Chairman and CEO of 

Prescription drug patent exclusivity was extended during Hatch-Waxman Act’s convoluted negotiations to increase generic drug access and overall drug affordability. Later, around 2009, part of the prescription price increase allowance and extension argument for biologics was based on what was termed a “looming patent cliff.” Patent protection is now 20 years from date of filing in the United States, but market exclusivity appears to average a little over 13 years in practice (1). Following Hatch–Waxman, the generic products’ share of total prescriptions in the US increased from 36% in 1994 to 90% in 2019 (1). Loss of income due to lower pricing of generics estimated at $185 billion was evenly balanced by price hikes for prescription drugs of $187 billion (2). This helped create the conditions we face today where once again there is a prescription drug price crisis because 10% of prescriptions have an average cost of $20 a day and account for 80% of all prescription drug spending, despite the fact that (largely due to Hatch-Waxman) 90% of prescriptions are generics that cost on average, $1 a day (2). 

To revisit the cost issue, President Biden signed the Inflation Reduction Act (IRA), while pharmaceuticals reportedly face, once again, a looming patent cliff (3). With regard to IRA, biopharma executives describe the Medicare drug price negotiation process “as basically enforced price fixing on certain established medicines and that it will affect how companies allocate capital and force hard decisions on investments in the very costly process of drug testing and production” (4). 

However, at the 2023 BIO conference in Boston, FDA Commissioner Robert Califf stated that drug prices are too high, and that “FDA officials are providing technical assistance to their counterparts at the federal Centers for Medicare & Medicaid Services as they prepare to negotiate drug prices with companies for the first time” (5). On limiting reimbursements for accelerated clinical trials, Califf went on to say, “‘sounds like common sense to me’ for government health insurers to pay less for drugs that haven’t yet been fully approved. ‘If I had a basketball that’s probably going to stay inflated and it looks pretty good in the store but we don’t really know, you wouldn’t really pay the same as you would for a first-rate basketball’ guaranteed to stay firm” (5).

1. Grabowski, H.; Long, G.; Mortimer, R.; and Bilginsoy, M. Continuing Trends in U.S. Brand-name and Generic Drug Competition. 

. August 2021. Taylor and Francis online. 

2. Engelberg, A. Unaffordable Prescription Drugs: The Real Legacy of the Hatch-Waxman Act, 

4. Wechsler, J. Biden Hits Drug Prices for Boosting Health Care Costs. 

5. Weisman, R. FDA Chief’s Unwelcome Message to Biotech Execs: Prices of Drugs Are Too High. 

Mike Hennessy Jr. is the President and CEO of 

When referring to this article, please cite it as Hennessy, M. Drug Pricing’s Static Changes. 

The debate surrounding drug pricing changes rages on.

Drug Pricing’s Static Changes

Ross Engineering custom fabricates tanks, pressure vessels, and reactors up to 100,000 gal or larger that are used throughout the processing industries for various manufacturing and storage functions.

The 9400-gal Condensate Receiver Tank is designed and built according to American Society Of Mechanical Engineers (ASME) standards for up to 5 psi at 200 °F. The tank is constructed from 304 stainless steel and includes multiple nozzles for venting, instrumentation, and discharge as well as a 24-in. American Petroleum Institute manway.

ROSS Engineering also offers hydrotesting, passivation, ASME code stamp, ladders, jacketing, insulation, agitators, and other customization.

Ross Engineering custom fabricates tanks, pressure vessels, and reactors for various manufacturing and storage functions. 

Custom-Fabricated Processing Tools

Bruker’s timsTOF Ultra mass spectrometer is designed for use with the company’s 4D-proteomics timsTOF platform. The device uses a new captive spray ionization ion source that provides a larger capillary relative to past models as well as optimized vortex gas flow.

The device is designed to identify over 55,000 peptides that map into 5000 protein groups at the single-cell level of 0.125 nanogram protein loading at less than 1% false discovery rate. More than 4800 protein groups are quantified at a coefficient of variation of less than 20%.

The parallel accumulation-serial fragmentation duty cycle can go as fast as 300 Hz for tandem mass spectrometry, which is intended to provide performance improvements for processes involving ultra-low sample amounts. This includes unbiased single-cell proteomics and single-cell lipidomics, unbiased spatial proteomics, immunopeptidomics, phosphoproteomics, post-translational modification analysis, and protein–protein interaction studies.

Bruker Corporations timsTOF Ultra mass spectrometer features a captive spray ionization ino sources that provides optimized vortex gas flow. 

Ultra-Low Sample Optimized Mass Spectrometer

TurboFil Packaging Machines’ Mini-Monoblock MDN-50

TurboFil Packaging Machines’ Mini-Monoblock MDN-50 fill/finish machine is designed to accurately and expediently fill, seal, cap, and label spray multi-dose nasal dispensers. The machine is suitable for vials with volumes from 4 to 30 mL and can process containers at a rate of up to 50 pieces per minute. It can be used with containers with tamp, crimp, and screw closures.

The machine uses a scroll conveyor designed to enable seamless vial loading while also allowing for a compact overall footprint. Vials are then transported to a starwheel ceramic piston filler station for clean and accurate filling; a second nozzle can be incorporated in the filler station for faster filling. Following this, the filled and sealed vials are conveyed to an optional wrap-around labeler with a thermal transfer printer that can apply lot code, manufacture/expiry dates, and other necessary information.

The Mini-Monoblock MDN-50 features a 7-in. touchscreen human–machine interface and escalating password levels for tiered personnel access. A three-color stack light with an audible alarm can alert personnel to potential issues, while ethernet capability allows for remote diagnostics.

TurboFil Packaging Machines Mini-Monoblock MDN-50 is designed to accurately and expediently fill, seal, cap, and label spray multi-dose nasal dispensers. 

Nasal Spray  Fill/Finish Machine

Beckman Coulter Life Sciences’ Light Array Module

The Light Array Module (LAM) from Beckman Coulter Life Sciences is designed to be used with the BioLector XT Microbioreactor, which faciliates the ability to work with light-dependent organisms requiring photosynthesis for growth. The device is intended for high-throughput screening and process optimization for various phototrophic organisms, including microalgae and cyanobacteria.

The module provides customizable light settings of 400–700 nanometers within the photosynthetic spectrum, including both day and night-time lighting. The device uses 16 different light-emitting diode (LED) types to achieve this degree of spectral flexibility; each LED can be controlled individually for maximum irradiances and photon flux densities of up to 3500 micromoles reaching one square meter per second.

The LAM uses real-time biomass, chlorophyll concentration, and fluorescence measures to provide real-time data without the need to pipette samples for analysis. The device is also capable of cultivation parameter monitoring and feeding strategy optimization.

Beckman Coulter Life Sciences’ Light Array Module enables high-throughput screening and process optimization for various phototrophic organisms. 

High-Throughput Phototrophic Organism Screening Module

Bianca Piachaud-Moustakis is lead writer at Pharmavision, Pharmavision.co.uk.

Complement Therapeutics (CTx) is a Germany-based preclinical stage biotechnology company focusing on the R&D of novel therapeutic approaches to address unmet needs in disorders that affect the body’s immune system, known as the complement cascade. The complement cascade is a part of the immune system that enhances (or complements) the ability of antibodies and phagocytic cells to clear microbes and damaged cells from an organism, promote inflammation, and attack the pathogen’s cell membrane (1). When activated by one of several triggers, the complement system works in conjunction with other components of the immune system to clear invading pathogens (1).

Based on the research of the company’s founders, Simon Clark, Paul Bishop, and Richard Unwin from the University of Manchester, CTx aims to develop innovative and effective therapeutics to address unmet needs in complement-mediated diseases, particularly in the fields of age-related macular degeneration (AMD), kidney disease, and various haematological conditions. Through an extensive programme of translational research, the scientists have gained powerful new insights into the ways the complement cascade works and how it is dysregulated in AMD.

Founded in 2020, CTx has subsidiaries in the United Kingdom (UK), operating as Complement Therapeutics Ltd, and in the United States (US), trading as Complement Therapeutics Inc., as well as research laboratories in Stevenage, UK.

Research conducted by Tracxn Technologies indicates that CTx ranks fourth among 94 active competitors (2). CTx’s key competitors include: CureVac, a Germany-based biopharmaceutical company that develops messenger RNA (mRNA)-based therapeutics focused on prophylactic vaccines, cancer immunotherapies, and molecular therapies. CureVac was the world’s first company to successfully use mRNA for medical purposes (3); Rezolute Inc. (formerly Antriabio), a US-based biopharmaceutical company specialized in the development of drugs for metabolic and orphan diseases. A leading candidate under development is a plasma kallikrein inhibitor (PKI) designed to treat diabetic macular oedema (DME). This oral therapy, formulated to be administered once a day, is designed to reduce inflammation and vascular leakage caused by DME through targeting the kallikrein-kinin system (4); and Visus Therapeutics Inc., a US-based clinical-stage pharmaceutical company that specializes in developing therapeutic solutions for vision care. One of the company’s flagship products includes VT-1051, a novel, injectable, sustained-release delivery system that delivers a Ciliary neurotrophic factor (CNTF) analogue and a FAS/TNF-α inhibitor which has the potential to preserve photoreceptors, prevent programmed cell death, and improve vision in patients suffering from geographic atrophy (GA) (5).

CTx was spun out of the University of Manchester and has raised a total of €77 million over three rounds of financing according to data derived from Crunchbase (6). In 2021, CTx secured initial seed funding from BioGeneration Ventures (BGV), subsequently receiving a further €5 million in Round 2 seed funding from BGV and Forbion in February 2022. With this funding, CTx advanced its lead investigational product, CTx001 through pre-clinical proof-of-concept, and secured an Innovation Passport from the UK Medicines and Healthcare Products Regulatory Agency (MHRA) (7). CTx001 is a highly innovative adeno-associated virus (AAV) gene therapy designed for the treatment of GA secondary to dry AMD, which is a leading cause of blindness, and for which no licensed treatment currently exists.

Through the financing received from BGV and Forbion, CTx also initiated a one-year, natural history non-interventional i-GAIN (investigating Geographic Atrophy Insights) study in patients who have a confirmed diagnosis of GA in one or both eyes in the UK (8). The study is designed to evaluate the relationship between genetics, blood biomarkers, and phenotypic changes in the eye of people with GA. Data derived from the i-GAIN study is designed to support the clinical development planning for CTx001, particularly in enabling the identification and stratification of patients with complement-driven AMD for future interventional studies (8).

More recently in April 2023, CTx announced that it had successfully secured an additional €72 million in Series A financing. The third round of financing was led by Gimv, a Belgian-based private equity and venture capital fund, co-led by Forbion (as an existing investor), and further joined by BGV, Panakès Partners, Cambridge Innovation Capital (CIC), Hadean Ventures and Seroba Life Sciences (9).With this latest round of financing, CTx will continue the development, as well as complete a Phase Ib clinical proof of concept of its lead product CTx001. The novel therapy is expected to have the potential to offer superior efficacy compared to competitive drugs, as well as reduce the burden of treatment among patients through a “one and done” approach (10).

CTx will also use the funds to expand its laboratory-based activities at its site in Stevenage, UK, as well as evaluate its pipeline assets for non-ocular indications. According to Rafiq Hasan, CEO and managing director at Complement Therapeutics GmbH, “the support of this broad syndicate enables us to generate additional data demonstrating CTx001’s unique and differentiated mechanism of action, with the potential to transform the treatment landscape in [GA]” (9).

The financing will also be used to further develop the company’s novel Complement Precision Medicine (CPM) platform, which enables the quantification of over 30 complement cascade proteins from a single systemically drawn blood sample (11). The CPM platform will also facilitate the stratification of patients with AMD and other conditions for enrolment into future clinical trials, and support the subsequent commercialisation of CTx’s assets. According to Cipriani et al. (2021), the CPM platform has been validated in normal human samples and two distinct disease cohorts (12).

Alongside the financing, Michaël Vlemmix (Gimv), Rob Woodman (Panakès), Anne Horgan (CIC), and Roger Franklin (Hadean Ventures) will join the CTx board as new members.

AMD is a chronic and progressive degenerative disease of the macula, the central part of the retina (or the light-sensitive tissue at the back of the eye) that controls sharp, straight-ahead vision (13). AMD is a common condition that blurs a patient’s central vision and is a leading cause of blindness for older adults. Research conducted by Guymer et al. (2023) estimates that AMD is present in 8.69% of the global population, affecting 196 million people in 2020; and its prevalence is expected to increase to 288 million by 2040 (14).

According to the US National Eye Institute (NEI), dry AMD has three stages: early, intermediate, and late, with the disease usually progressing slowly over several years. Also termed GA, there is currently no treatment for late dry AMD, which is driven by a combination of factors including genetic predisposition, natural ageing changes, and lifestyle factors, such as smoking and nutritional intake. Genetic and molecular studies have identified the complement system as a key driver of AMD onset and progression, and there is increasing evidence that complement inhibition can slow the progression of GA (15).

Over the past few years, R&D efforts in the field of complement-mediated diseases have received the support of the US National Institutes of Health (NIH), which has awarded grants worth US$837 million for research in this domain (16). With enhanced funding and growing recognition of the complement system as a multidimensional innate immune surveillance mechanism, it can be expected that more novel therapeutic targets will be discovered in the future. CTx is therefore well placed to take advantage of potential future opportunities by addressing unmet needs in complement-mediated diseases, particularly AMD, to which it can strive to achieve market dominance and leadership position. With the active involvement of big pharma players as well as new entrants, the drug development landscape of complement therapeutics is likely to expand further with the market poised to witness steady growth over the coming years.

Complement Therapeutics. The Complement System. Complementtx.com (accessed 9 June 2023).

Traxcn Technologies Ltd. Complement Therapeutics Competitors. Tracxn.com (accessed 9 June 2023).

CureVac. About Us. Curevac.com (accessed 9 June 2023).

Rezolute Inc. For Patients: Living with DME. Rezolutebio.com (accessed 9 June 2023).

Visus Therapeutics. Product Pipeline. Visustx.com (accessed 9 June 2023).

Crunchbase. Complement Therapeutics Financials. Crunchbase.com (accessed 9 June 2023).

CISION PR Newswire. Complement Therapeutics Secures €72 Million in Series A Financing to Advance Novel Therapies Targeting Complement-Mediated Diseases. PRnewswire.com, 17 April 2023 .

BIA. Complement Therapeutics Enrol First Patient in Non-interventional i-GAIN Study of People Diagnosed with Geographic Atrophy. Bioindustry.org. 8 Feb. 2023.

Complement Therapeutics.Complement Therapeutics Secures €72 Million in Series A Financing to Advance Novel Therapies Targeting Complement-Mediated Diseases. Press Release, 17 April 2023.

Pharmaceutical Technology. Complement Therapeutics Wins Financing for New Therapies Development. Pharmaceutical-technology.com, 18 April 2023

Complement Therapeutics. Complement Precision Medicine (CPM) Platform. Complementtx.com (accessed 9 June 2023).

Cipriani, V.; Tierney, A.; Griffiths, J.R.; et al. Beyond Factor H: The Impact of Genetic-risk Variants for Age-related Macular Degeneration on Circulating Factor-H-like 1 and Factor-H-related Protein Concentrations. Am. J. Hum. Genet. 2021, 108 (8), 1385–1400.

National Eye Institute (NEI). Age-related Macular Degeneration (AMD). Nei.nih.gov. 22 June 2021.

Guymer, R.H.; Campbell, T.G. Age-Related Macular Degeneration. The Lancet. 2023, 401 (10386), 1459–1472.

Armento, A.; Ueffing, M; Clark, S.J. The Complement System in Age-related Macular Degeneration. Cell. Mol. Life Sci. 2021, 78 (10), 4487–4505.

Globe Newswire. Next Generation Complement Therapeutics Market, 2035. Press Release, 4 April 2022.

Bianca Piachaud-Moustakis is a lead writer at PharmaVision.

When referring to this article, please cite it as Piachaud-Moustakis, B. Frontrunner in Therapies for Complement-Mediated Diseases. 

A novel complement therapeutic CTx001 offers a novel approach to treating geographic atrophy. 

Featured Content

Pharmaceutical Technology Is Now PharmTech: A Sharper Name for a Smarter Industry

Behind the Headlines Episode 28

Ask the Expert

Drug Digest December 2025

A “Plan, Prevent, Prove” Approach