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Senior director, biopharmaceutical CMC and regulatory affairs, Cencora

What biopharma companies need to know about this evolving regulatory pathway.

FDA’s New Accelerated Approval Draft Guidance 

AbbVie Pedro Valencia ADCs

PE: How is AbbVie using novel cancer biomarkers to develop potential first-in-class ADCs for the treatment of advanced solid tumors?

We're constantly looking for biomarkers that can help us to do two key things. The first is to tackle cancer cells. and spare normal cells. We’re looking for specific antigens that are over-expressing tumor cells, and you see limited expression in normal cells. We want to make sure that target that’s over-expressed is critical for the survival of the cancer cell. Once you block it, you can actually drive a cancer cell death. That’s what we're looking for. In that perspective, c-Met is one of those biomarkers that is over expressing multiple tumor types such as lung, colorectal, gastric, and other GA tumors. We as an industry have tried to tackle c-Met in the past but have been unsuccessful. It would actually be successful to tackle what we call met, which is a gene inside the cell.

There’s this thing called met amplification, where the cancer cell produces multiple copies of the gene, and around 30% of patients have it. We can actually tackle that with small molecules. But once the gene becomes a protein and gets over-expressed in the cell, that over-expression is something we haven’t been able to target. For example, we know that on lung cancer, c-Met over-expression is about 25% to 50% in colorectal cancer, and it could be anywhere from 30% to 70% depending how you measure it, so it’s quite prevalent.

With ADCs, we’re trying to tackle c-Met over-expression on the surface of cancer cells. That was novel, and we can do it through ADCs. 706 has a target called SEZ6, and it’s a target that is over expressed in neuroendocrine cells or neuroendocrine neoplasms. When you think about it, multiple tumor types kind of have a subset of cells that are neuroendocrine driven. Small cell lung cancer is one of the few that has everything driven by neuroendocrine cells. The beauty of SEZ6 is over-expressing those cells and limited expression in normal cells. So, we can tackle it with an ADC and drive the results that we're seeing today.

PE: ADCs have redefined the treatment of cancer in recent years. Can you please discuss the promise of this emerging class and what the future may hold?

At the end of the day, ADCs work like a trojan horse. You have a good antibody that cancer cells are happy to receive, but once you hook into the cancer cell receptor, you deliver the payload, which is pretty much chemotherapy. It’s actually a highly potent version of the regular chemotherapy cancer patients are given on a regular basis. So, from that perspective, our goal is to replace chemotherapy. Our grand goal with ADCs is to replace good old, decades old chemotherapy that is really toxic for some patients. That’s how we see the redefinition of cancer treatment in the future with ADCs. So, what do we see in the future? Well, if you think about it, chemotherapy is being given in combination with other agents. In the future, we see ADCs being given in combination with chemotherapy. There will be one with other agents such as immuno-oncology agents. So, if you combine the ADC and the PD1s, you can start seeing that synergy come together and kind of start really dreaming about cures for the patients. All in all, we're super excited about ADCs replacing chemotherapy. 

In an interview with Pharm Exec Associate Editor Don Tracy, Pedro Valencia, VP, Solid Tumor Pipeline Strategy & Execution, AbbVie, discusses using novel cancer biomarkers to develop potential first-in-class antibody drug conjugates (ADCs) and the promise that they offer. 

Utilizing Biomarkers to Develop First-in-Class Antibody Drug Conjugates to Treat Advanced Solid Tumors

After five years of continued treatment with UCB’s Bimzelx (bimekizumab-bkzx), Phase III clinical trial data revealed that a majority of patients with moderate-to-severe plaque psoriasis (PSO) achieved sustained complete skin clearance, as measured by Psoriasis Area and Severity Index (PASI) 100 scores.

"A primary treatment goal for people living with psoriasis is durable, high rates of complete skin clearance. These five-year results for bimekizumab-bkzx provide valuable evidence for clinical decision-making," said Andrew Blauvelt, MD, MBA, chair, medical board, National Psoriasis Foundation, in a press release. "The sustained complete skin clearance offers important insights into the potential of bimekizumab-bkzx's dual inhibition to provide long-term management of this chronic inflammatory condition."

Among 153 individuals treated with Bimzelx who completed a five-year open-label extension study, 75.2% achieved PASI100 at one year and 67.7% achieved PASI100 at five years, while 92.8% achieved PASI90 at one year and 84.9% achieved PASI90 at five years.

Among patients with PSO at baseline, 68.7%–71.6% achieved PASI100 at three years, aligning with the broader study population. Among 420 patients weighing more than 198 lbs., 88.5% achieved PASI90 and 67.4% achieved PASI100 at four years, while in 351 patients under 198 lbs., 83% achieved PASI90 and 61.6% achieved PASI100 at four years.

The data also included results from 771 PSO patients with cardiometabolic comorbidities. Among 375 patients with baseline hypertension, 82.8% achieved PASI90 and 59.3% achieved PASI100 at four years. In 344 patients with baseline elevated BMI, 82.5% achieved PASI90 and 60.7% achieved PASI100 at four years, while in 62 patients with baseline hyperglycemia, 80.4% achieved PASI90 and 56.9% achieved PASI100 at four years.

According to UCB, patients who were at risk of progressing to psoriatic arthritis (PsA) achieved high rates of PASI100 after three years of treatment. Similar outcomes were observed in patients who had only psoriasis at baseline.

According to the National Psoriasis Foundation, around eight million people in the United States are currently living with some form of psoriasis and 125 million people have psoriasis globally. Around 30% of patients with psoriasis will eventually develop PsA. Caucasians have a 3.6% prevalence rate compared to 1.5% for African Americans; however, the condition is highly likely to be underdiagnosed in African American patients as well as other patients of color due to differences in clinical presentation, according to the National Psoriasis Foundation.

Onset of psoriasis is most common between 20 and 30 years of age and between 50 and 60 years of age, however, the condition can develop at any age. Annual health costs have been found to be greater than the general health population, possibly accounting for approximately $135 billion annually. Sixty percent of people with psoriasis have reported that it impacts their daily living.

"Psoriasis is a chronic condition that increases the risk of developing other serious health issues," said Fiona du Monceau, EVP, head, patient evidence, UCB, in the press release. "These five-year results highlight the robust potential of bimekizumab-bkzx in transforming patient outcomes by offering the possibility of lasting, complete skin clearance. Bimekizumab-bkzx is aiming to set a new standard for treatment success, and our belief in its innovative dual inhibition approach is reflected in our dedication to head-to-head trials, including the BE BOLD Phase III trial in psoriatic arthritis."

These findings will be showcased in six posters at the 2025 American Academy of Dermatology Annual Meeting, alongside additional data for hidradenitis suppurativa, psoriatic arthritis, ankylosing spondylitis, and non-radiographic axial spondyloarthritis.

1. BIMZELX® (bimekizumab-bkzx) Five-Year Data at AAD 2025 Showed Sustained Skin Clearance and Long-Term Efficacy in Moderate-to-Severe Plaque Psoriasis. 

March 7, 2025. Accessed March 7, 2025. https://prnmedia.prnewswire.com/news-releases/bimzelx-bimekizumab-bkzx-five-year-data-at-aad-2025-showed-sustained-skin-clearance-and-long-term-efficacy-in-moderate-to-severe-plaque-psoriasis-302395620.html

Accessed March 7, 2025. https://www.psoriasis.org/psoriasis-statistics

Five-year data from Phase III trials demonstrated that 67.7% of patients moderate-to-severe plaque psoriasis treated with UCB’s Bimzelx achieved complete skin clearance. 

Bimzelx Demonstrates Sustained Skin Clearance in Moderate-to-Severe Plaque Psoriasis

DB-OTO and the Evolving Approach to Genetic Deafness in Children

Last month, Regeneron announced updated results from the Phase I/II CHORD trial in children with otoferlin-related hearing loss. Out of 11 children who received DB-OTO, 10 showed notable hearing improvements at different decibel levels, with some achieving nearly normal or normal hearing thresholds. Pharmaceutical Executive spoke with Jonathon Witton, AuD, PhD, VP, auditory global program head, in more detail about the trial results and the performative potential of DB-OTO.

Given the promising speech and hearing improvements observed in the CHORD trial, how does Regeneron envision DB-OTO reshaping the standard of care for patients with otoferlin-related hearing loss?

I'm a pediatric audiologist by training, and I’m also a neuroscientist. If we go back even just a couple of decades when I was still in the clinic, when we first identified a child who was born with deafness, they would go see their pediatrician. Ultimately, their family would come to the clinic, and by that point, the child was usually a few months old. We could run different clinical assessments, most of them physiologic at that time, to try to understand whether or not the child had any hearing ability. At that point, we might be able to explain to the family what we were seeing based on those results, but usually, we couldn’t tell them why.

The story of DB-OTO goes back to the late '90s when this specific gene, responsible for deafness in the condition we are treating, was first identified. As we started testing more and more children who were coming to the clinic, we realized that more than half of them had a genetic protein deficiency that was actually causing their hearing loss, and we could use a blood test or saliva test to make the molecular diagnosis for the exact cause of the hearing loss. That was the first step toward changing the standard of care.

Building off of that, DB-OTO has been in the works for around eight years now. Over the last 10 years, we’ve been able to design a more elegant solution for how we might provide hearing to children. Right now, we have prostheses. We fit children with devices such as hearing aids and cochlear implants. As I said, we previously didn’t know the underlying cause and were just trying to manage their symptoms.

The approach with DB-OTO is based on the idea that if there is a specific cell type in the inner ear responsible for producing a critical protein for hearing, we could develop a therapeutic to deliver DNA to the cell lacking that functional protein. Potentially, you could then go from having no hearing in the case of these children at their baseline to potentially having good hearing. That can be the therapeutic concept for other protein deficiencies that are causing deafness in these kids.

More than half of these children have a genetic cause for their hearing loss. I think when it comes to the big change, there's going to be an appreciation by clinicians in taking a further step in diagnosis for children who are born with deafness. Describing that they have deafness is not enough. We need to dig deeper and try to understand the underlying cause of their deafness. If no therapeutics are available, the patient may ultimately be managed in the same way, relying on a prosthesis to address symptoms. But if you start implementing therapeutics, the onus for the clinician is to try and figure out what exactly is going on. I think this will result in more molecular diagnoses of hearing loss in clinics around the world.

Next, there will be a change in our expectations for what could be possible for the patients. In the core trial of the first 12 patients, 10 out of the 11 patients with available efficacy data have shown notable improvement in their hearing ability. We have several who have hearing results that are normal or near normal in terms of sensitivity. This kind of change is a breakthrough. It changes your expectations for what could be possible.

Regeneron envisions DB-OTO reshaping the standard of care for hearing loss by shifting from symptomatic management with prostheses, such as hearing aids and cochlear implants, to a molecularly targeted therapeutic approach. Historically, clinicians diagnosed hearing loss without understanding its genetic cause, but advances in genetic testing have revealed that over half of childhood hearing loss cases stem from genetic protein deficiencies. DB-OTO aims to correct this by delivering functional DNA to specific inner ear cells, enabling them to produce the missing protein, potentially restoring hearing to near-normal levels.

The transformative potential of DB-OTO is evident in early clinical trials, where 10 out of 11 patients demonstrated hearing improvements, with some achieving normal or near-normal sensitivity. This represents a paradigm shift—clinicians will now need to incorporate molecular diagnostics into routine care, ensuring that patients receive precise, targeted treatments rather than generalized symptom management.

Regeneron has secured multiple regulatory designations, including the FDA’s RMAT designation, which facilitates early and frequent discussions with regulators. These interactions are crucial in navigating the complexities of gene therapy development, accelerating timelines for approval and commercialization.

Challenges remain, particularly in clinical development and manufacturing. Ensuring scalable, high-quality production of gene therapies is as critical as generating robust clinical data. Regeneron has invested heavily in manufacturing capabilities to meet these demands.

Beyond clinical development, Regeneron actively engages with the hearing loss community, including advocacy groups, clinicians, and families. Insights from these discussions have highlighted practical concerns, such as ensuring continuous hearing for safety. This patient-centric approach shapes the development of DB-OTO and future gene therapies, with plans to expand treatments to broader populations, including adults with age-related and noise-induced hearing loss. Regeneron aims to pioneer gene therapy solutions for a currently untreatable public health challenge.

Jonathon Whitton, AuD, PhD, VP, auditory global program head, Regeneron, discusses the promising results of DB-OTO in the CHORD trial for children with otoferlin-related hearing loss.

Video Series

FDA recently announced new safety requirements for DPD screening in cancer patients. Kristine Ashcraft, founder and CEO of YouScript, spoke with 

 about these updates and the impact it will have on oncology.

Pharmaceutical Executive: How significant an issue is DPD deficiency?

Patients with DPYD variations are 25.6X more likely to experience treatment related death on standard‐dose fluoropyrimidine chemotherapy (5‐fluorouracil or capecitabine).

 Although complete loss of activity is rare, reduced activity impacts up to 8% of patients

 and simple dose reductions can reduce risk without negatively impacting long-term or progression free survival.

 CPIC, the guidelines body for gene-drug interactions, has provided evidence-based guidance on medication adjustments for Fluoropyrimidines based on DPYD genotyping since 2017.

 In 2024, a joint consensus recommendation from leading genetic, lab and pharmacogenomic expert groups on must test and optional variants for DPYD genotyping was published.

PE: What prompted FDA to issue the safety announcement?

 The safety announcement highlighted labeling changes for Capecitabine and fluorouracil issued by the FDA Oncology Center of Excellence under Project Renewal, an FDA initiative that aims to update prescribing information for certain older oncology drugs. Many years prior to this change, representatives of Advocates for Universal DPD/DPYD testing petitioned the FDA to update fluoropyrimidine chemotherapy product inserts to recommend testing. A testing recommendation wouldalign with European guidance in place since 2020. While the FDA made several updates to point out the risks in March of 2024, it stopped shy of a recommendation. In January of 2025, the FDA and AACR convened a workshop to discuss whether to test or not, where all participants seemed to agree that testing for DPD variations should be discussed with all patients.

Translating the momentum from the FDA safety alert into an FDA requirement would strengthen adoption.Furthermore, DPYD testing recommendations in the NCCN guidelines are a critical next step. Although NCCN acknowledges CPIC guideline existence, they do not recommend that providers consider testing in alignment with the recent FDA safety communication. An FDA requirement would also be useful, but the NCCN guidelines are often cited by oncologists as a reference in determining standard of care, and will help to mitigate real and avoidable patient harm for patients.

PE: What are the benefits of performing comprehensive PGx panel tests?

 In an assessment of over 30,000 patients that received panel testing and take at least one PGx impacted medication, almost 60% were taking medications with evidence-based drug or dose change guidance in more than one clinical area.

 A large randomized study of almost 7000 patients across different care settings in several European countries showed a 30% reduction in clinically significant adverse drug reactions in just 12 weeks with PGx panel testing.

 Testing provides immediate benefit, but can also provide ongoing drug and dose optimization. An assessment of almost 500,000 patients with PGx panel testing in the UK Biobank showed that patients would have an atypical response to over 10 medications on average.

Think of PGx panel testing like a seatbelt for medication safety and efficacy that should be used every time medication changes are considered. One model suggested a 10.6% reduction in mortality with the use of PGx testing.

 PGx panel testing and a pharmacist-provided comprehensive medication review would incur a low lifetime cost of $450 averting 106 deaths per 1000. Comparatively, other preventative measures–such as mammograms for breast cancer, low-dose CT scans for lung cancer in high-risk patients, and colonoscopies for colon cancer–all cost more to prevent fewer deaths. So why are they considered the standard of care, while PGx panel testing is not, when all are worthwhile preventative interventions that have likely saved the lives of someone you care about?

Pathogenic DPYD Variants and Treatment-Related Mortality in Patients Receiving Fluoropyrimidine Chemotherapy: A Systematic Review and Meta-Analysis. 

. December 26, 2021. https://pubmed.ncbi.nlm.nih.gov/34506675/

Population-scale predictions of DPD and TPMT phenotypes using a quantitative pharmacogene-specific ensemble classifier. 

. December, 2020. https://pubmed.ncbi.nlm.nih.gov/32973300/

Survival of Patients With Cancer With DPYD Variant Alleles and Dose-Individualized Fluoropyrimidine Therapy-A Matched-Pair Analysis. 

. August 28, 2023. https://pubmed.ncbi.nlm.nih.gov/37639651/

CPIC® Guideline for Fluoropyrimidines and DPYD. 

. https://cpicpgx.org/guidelines/guideline-for-fluoropyrimidines-and-dpyd/

. https://www.pharmgkb.org/literature/15161962

Validation of Pharmacogenomic Interaction Probability (PIP) Scores in Predicting Drug–Gene, Drug–Drug–Gene, and Drug–Gene–Gene Interaction Risks in a Large Patient Population. 

. November 29, 2022. https://www.mdpi.com/2075-4426/12/12/1972

A 12-gene pharmacogenetic panel to prevent adverse drug reactions: an open-label, multicentre, controlled, cluster-randomised crossover implementation study. 

. February 4, 2023. https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(22)01841-4/abstract

Pharmacogenetics at Scale: An Analysis of the UK Biobank. 

. June, 2021. https://pubmed.ncbi.nlm.nih.gov/33237584/

Cost-Effectiveness of Pharmacogenomics-Guided Prescribing to Prevent Gene-Drug-Related Deaths: A Decision-Analytic Model. 

. June, 2022. https://pubmed.ncbi.nlm.nih.gov/36120299/

The YouScript CEO discusses new FDA guidance.

Screening for DPYD: Q&A with Kristine Ashcraft

NIH Funding Cuts Legal Challenges

PE: What should these stakeholders have top of mind as they find new ways to effectively navigate and continue medical research?

Things have definitely changed. I think that funding is now the biggest issue regarding the future of research. People need to get used to the idea of shifting from relying on federal grants for funding to exploring other possible sources.

Currently, there is a policy in place that caps indirect costs at 15%. When people ask what I mean by indirect costs, I’m referring to administrative costs, overhead, and regulatory compliance—factors that are included when determining the necessary funding. It used to be 50%. A company would calculate total costs, and then the government would provide 50 cents on the dollar for these indirect costs. Now, that’s been reduced to 15%, and unsurprisingly, lawsuits have been filed. As of now, the courts have halted it, but the agency has found other creative ways to push funding freezes. One particularly interesting approach was using the Federal Register. The Federal Register issued a notice freeze, which essentially halted the review of all grant proposals submitted to the NIH. This is significant because, for future applications to be considered, notices about upcoming meetings and their impact on research must be published in the Federal Register.

Stakeholders now have to seek new funding sources outside of the NIH, which will be incredibly challenging. They’ll need to turn to the private sector. But if everyone is turning to the private sector for additional funds, there could either be a chilling effect—where people stop planning certain projects—or so much competition that initiatives the NIH was working on won’t receive as much attention. Right now, we’re all just waiting to see how this unfolds.

PE: In addition to the NIH funding cuts, how do you see the clinical trials industry navigating other political challenges, such as the diversity bans?

Researchers will have to add an extra layer of consideration to something they haven’t really done before. They now have to view their trials through a more conservative lens. When conducting research to solve a problem, one would expect the process to be objective, but unfortunately, that’s the reality we’re in now.

This entire situation is disheartening. It could halt many clinical trials focusing on minority groups and stifle progress on certain vaccines. This could lead to entire sectors of science receiving neither funding nor attention. I don’t think we fully understand the magnitude of this yet—it won’t be clear until it actually happens. It’s ironic in a way, but an important question must be asked: What happens when others refuse to innovate? I believe those innovators will leave for places where they’re welcome. Unfortunately, if this continues, we may see a significant exodus of talented scientists from the U.S. over the next few years. I wouldn’t be surprised if many relocate to the EU, Asia, or other regions where scientists are encouraged to be more creative and address critical needs. Regardless of funding, these scientific advancements remain necessary and demand attention.

PE: With some judges indicating that the NIH funding freeze may be illegal, are there grounds for the NIH to continue conducting research, and what options do they have?

NIH has been ordered to continue its current research funding, which is good. The available options will depend on several factors, and those factors will take time to unfold. The only way NIH could move forward without interference from the administration is if Congress steps in and drafts laws that explicitly prevent research funding from being influenced by political reasons. However, given today’s hyper-partisan environment, I don’t believe such legislation will pass.

PE: How could the funding freeze potentially play out legally if the administration defies the courts' ruling, and what are the potential legal implications of such an action?

It’s a nightmare scenario—not just for lawyers like me, but for anyone invested in this issue. It never used to be, but I think it’s the number one concern right now. If the administration defies court rulings, that’s a constitutional crisis—it would fundamentally disrupt how the federal government operates.

I remember back in law school, we discussed this possibility, and it seemed so theoretical, so far-fetched. But now, we have to seriously consider it. The legal implications are too numerous to outline entirely because they depend on how it happens, which cases are affected, and the specific facts of those cases. Then, we’d have to extrapolate to understand the broader consequences. Ultimately, it would mean a collapse of our checks and balances system—the foundation of our democracy. I don’t mean to sound alarmist, but that’s exactly what it is. It’s like a Jenga tower—you pull one piece from the center, and the whole structure collapses. It would throw everything into chaos. Hopefully, this scenario won’t play out, but based on recent events, we have to consider these possibilities.

PE: What are the legal implications of the latest tariffs—including the "reciprocal" variety—and what challenges do they present to the pharma supply chain?

: Reciprocal tariffs could create significant disruptions in the supply chain and many commercial contracts. I don’t want to get overly technical, but many contracts include clauses that activate certain procedures or nullify agreements when events beyond a party’s control occur—such as embargoes or excessive tariffs. These clauses will become increasingly relevant as they may need to address the impact of excessive tariffs. Supply chain disruptions could arise at any moment, particularly with raw materials, which are frequently sourced from overseas.

Some people may have different opinions, but in my view, these tariffs will negatively affect the supply chain and, ultimately, our economy at every level. Prescription drug costs will likely rise significantly as a result.

Unfortunately, Congress has granted broad authority to the executive branch through legislation. Specific laws that come to mind include the Trade Expansion Act of 1962 and the International Emergency Economic Powers Act of 1977. These laws delegate power to the president to adjust tariffs in response to specific circumstances, such as national security concerns or trade negotiations, without requiring new congressional approval. Because of this, the president holds significant control over tariffs, and unless Congress enacts new legislation to curtail this power, the courts have limited options to intervene.

At this point, I’m not entirely sure how the power dynamic will unfold. It used to be that Congress and the president shared these responsibilities, but now, we have to wait and see. Tariffs could be an experiment. That seemed to be the case during this administration’s previous term—some tariffs led to negative consequences, while others remained in place.

Ron Lanton, Partner, Lanton Law, discusses the legal and industry challenges posed by recent National Institute of Health budget cuts,  diversity bans in clinical trials, and pharmaceutical supply chain disruptions due to tariffs.

Navigating NIH Funding Cuts, Diversity Bans, and Tariff Challenges: Legal and Industry Implications for Medical Research

The FDA has approved Samsung Bioepis’ Ospomyv (denosumab-dssb; SB16) and Xbryk (denosumab-dssb; SB16), biosimilars referencing Amgen’s Prolia and Xgeva, respectively. Ospomyv is indicated for osteoporosis treatment in postmenopausal women and men at high risk of fracture, as well as for patients undergoing androgen deprivation or aromatase inhibitor therapy. Additionally, Xbryk is approved for preventing skeletal-related events in patients with multiple myeloma and solid tumor bone metastases, treating unresectable giant-cell tumors of bone, and addressing bisphosphonate-refractory hypercalcemia of malignancy.

“The FDA approval of Ospomyv and Xbryk marks a key step in improving patient access and alleviating treatment cost for patients with osteoporosis and cancer-related bone loss in the US. By providing quality-proven biosimilars, we are helping to address a critical healthcare need and reduce the burden of skeletal fractures that impact patients’ quality of life,” said Byoungin Jung, VP, regulatory affairs team leader, Samsung Bioepis, in a press release. “This achievement underscores our commitment to healthcare innovation through biosimilars and our mission to meet the growing needs in critical therapeutic areas.”

Approval was based on results from Phase I and Phase III clinical trials, including analytical data, non-clinical data, and clinical data. This included a Phase I randomized, double-blind, three-arm, parallel group, single-dose trial, which demonstrated the pharmacokinetic (PK) equivalence between SB16, EU-sourced denosumab, and US-sourced denosumab in healthy male participants. Results of the study met all primary PK endpoints, including area under the concentration-time curve (AUC) from time zero to infinity, AUC from time zero to the last quantifiable concentration, and maximum serum concentration.

Additionally, a Phase III randomized, double-blind, multi-center study demonstrated equivalent efficacy and comparable safety, immunogenicity, PK, and pharmacodynamics. The primary endpoint was the percent change from baseline in lumbar spine bone mineral density at one year and a follow up of 18 months.

Samsung Bioepis warns that Ospomyv and Xbryk are contraindicated in patients with known hypersensitivity to denosumab products and hypocalcemia. Ospomyv is also contraindicated in patients who are pregnant. Adverse events (AEs) of Ospomyv include dermatologic reactions, severe joint, bone, and muscle pain, and serious skin infections. AEs of Xbryk include embryo-fetal toxicity, hypercalcemia, osteonecrosis of the jaw, and fractures to the vertebrae and femoral bone.

According to the International Osteoporosis Foundation, osteoporosis affects one in three women and one in five men over 50 years of age globally. In Europe, the United States, and Japan, it is estimated that a total of 75 million people are currently living with osteoporosis. For the United States, this accounts for around 10.2 million people, with 54 million currently at risk. Additionally, 80% of people with osteoporosis in the United States are women.

According to the Johns Hopkins Arthritis Center, 30% of Caucasian postmenopausal women in the United States have osteoporosis. Additionally, the prevalence of osteoporosis in Caucasian postmenopausal women is around 14% between 50 and 59 years of age, 22% in women between 60 and 69 years of age, 39% in women between 70 and 79 years of age, and 70% in women over 80 years of age.3 In men, the numbers decrease significantly, with 3.3% of men between 50 and 64 years of age developing osteoporosis and 5.7% in men over 65 years of age.

According to Samgsung Bioepsis, the approvals mark the company’s first FDA approval for endocrinology biosimilars.

1. FDA Approves Samsung Bioepis’ OSPOMYV™, XBRYK™ (denosumab-dssb), a Biosimilar to Prolia and Xgeva. 

February 15, 2025. Accessed February 18, 2025. https://www.businesswire.com/news/home/20250215814020/en/FDA-Approves-Samsung-Bioepis%E2%80%99-OSPOMYV%E2%84%A2-XBRYK%E2%84%A2-denosumab-dssb-a-Biosimilar-to-Prolia-and-Xgeva

Accessed February 18, 2025. https://www.osteoporosis.foundation/health-professionals/about-osteoporosis/epidemiology

Accessed February 18, 2025. https://www.hopkinsarthritis.org/arthritis-info/osteoporosis-info/

Accessed February 18, 2025. https://www.healthline.com/health/osteoporosis/prevalence-of-osteoporosis

Ospomyv, a biosimilar to Prolia, is indicated for postmenopausal women and men at high risk of fracture, while Xbryk, a biosimilar to Xgeva, is indicated for preventing skeletal-related events in multiple myeloma and solid tumor bone metastases. 

Cori Gorman, PhD

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