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A summary of the key impacts new law, California Consumer Privacy Act (CCPA), will have on business practices related to data flow in specialty pharma, and more.




California Law Requires Legal, Compliance Scrutiny to Maintain Pharma Data Sharing

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

AstraZeneca's Shubh Goel, head of immuno-oncology, gastrointestinal tumors, US oncology business unit, AstraZeneca, discusses:

Results of the NIAGARA trial in bladder cancer

The significance of the five-year overall survival data from the HIMALAYA trial, specifically the long-term efficacy of the STRIDE regimen for unresectable liver cancer

In this episode of the Pharmaceutical Executive podcast, Shubh Goel, head of immuno-oncology, gastrointestinal tumors, US oncology business unit, AstraZeneca, discusses the findings of the NIAGARA trial in bladder cancer and the significance of the five-year overall survival data from the HIMALAYA trial, particularly the long-term efficacy of the STRIDE regimen for unresectable liver cancer. 

Key Findings of the NIAGARA and HIMALAYA Trials

Johnson & Johnson (J&J) has submitted regulatory applications to the FDA and EMA for the approval of Darzalex Faspro (daratumumab and hyaluronidase-fihj) in the United States and subcutaneous formulation in the EU as monotherapy for high-risk smoldering multiple myeloma (SMM). According to the company, the submissions are based on data from the Phase III Aquila trial, which is currently evaluating Darzalez Faspro as an early intervention treatment for the disease.

“There remains an unmet need for early interventions and treatments that are both effective and well tolerated in people living with smoldering multiple myeloma at high-risk of progressing to active multiple myeloma,” said Yusri Elsayed, MD, MHSc, PhD, global therapeutic area head, oncology, innovative medicine, Johnson & Johnson, in a press release. “Darzalex has changed the standard of care in multiple myeloma, and with these submissions to the FDA and EMA, this therapy could become the first approved treatment for patients with high-risk smoldering multiple myeloma, potentially shifting the treatment paradigm.”

The randomized, multicenter AQUILA trial enrolled 390 patients with high-risk SMM. The primary endpoint of the study is progression-free survival (PFS), with secondary endpoints that include time to progression, overall response rate, and overall survival. All patients involved with the trial were diagnosed with SMM at least five years before the start of the study and weren’t included if they had prior exposure to approved or investigational treatments for SMM. The data are expected to be presented at the 2024 American Society of Hematology Annual Meeting next month.

, the FDA approved Darzalex Faspro in combination with bortezomib, lenalidomide, and dexamethasone (D-VRd) for patients with newly diagnosed multiple myeloma (NDMM) who are eligible for an autologous stem cell transplant. In this indication, the therapy can be administered right after diagnosis and could potentially lead to significantly improved outcomes for NDMM patients. The approval was based on positive results from the Phase III PERSEUS study, which compared D-VRd induction and consolidation therapy to the traditional VRd regimen in transplant-eligible NDMM patients.

"Multiple myeloma has a highly varied clinical course among patients and in each individual patient, and there is a continued need for innovation and therapies that employ different targets and combinations to provide patients with treatment options at diagnosis and throughout the course of their disease," said Amrita Y. Krishnan, MD, professor, director, Judy and Bernard Briskin Multiple Myeloma Center, City of Hope, in a press release. "The efficacy data supporting this new quadruplet regimen, combined with its established safety and tolerability profile, provide compelling evidence that adding D-VRd upon initial diagnosis as compared to VRd can deepen responses and prolong remissions in the context of autologous stem cell transplantation."

SMM affects about 15% of new multiple myeloma cases, and those with high-risk SMM face a 50% likelihood of progressing to active disease within two years. Currently, high-risk SMM is generally managed through observation until signs of active multiple myeloma appear, but emerging evidence suggests early intervention could benefit patients at high risk.

 According to the Cleveland Clinic, SMM typically affects people over 60 years of age, with a median diagnosis between 62 and 67 years of age.

J&J stated that if the treatment is approved, Darzalex Faspro could become the first treatment indicated for high-risk SMM, potentially transforming the current management approach.

1. Johnson & Johnson submits applications in the U.S. and EU seeking approval of DARZALEX 

® / DARZALEX® as subcutaneous monotherapy for high-risk smoldering multiple myeloma. 

November 8, 2024. Accessed November 8, 2024. https://www.jnj.com/media-center/press-releases/johnson-johnson-submits-applications-in-the-u-s-and-eu-seeking-approval-of-darzalex-faspro-darzalex-as-subcutaneous-monotherapy-for-high-risk-smoldering-multiple-myeloma 

2. FDA Approves Johnson & Johnson’s Darzalex Faspro in Combination with Bortezomib, Lenalidomide, and Dexamethasone for Newly Diagnosed Multiple Myeloma. 

July 31, 2024. Accessed November 8, 2024.

Accessed November 8, 2024. https://my.clevelandclinic.org/health/diseases/23911-smoldering-multiple-myeloma

Submissions to the FDA and EMA for new indication of Darzalex Faspro are based on results of the Phase III Aquila trial in patients with high-risk smoldering multiple myeloma. 

Johnson & Johnson Submits Regulatory Applications for Darzalex Faspro for High-Risk Smoldering Multiple Myeloma

Johnson & Johnson Innovative Medicine developed a guide to bring clarity around expanded access for terminally ill patients who have run out of treatment options and aren’t eligible for clinical trials. The Guide to Expanded Access was created based on requests from patients and patient representatives. Christine MacCracken, executive director of patient strategies & solutions and leader of the team that developed the guide, spoke with 

 about the guide and broader issues surrounding expanded access.

Pharmaceutical Executive: How difficult is it for patients to access expanded access treatment options?

 One of the challenges that patients encounter, which can hinder their access to investigational medicines outside of clinical trials, is a lack of clear and transparent information. Expanded access varies by country, so it can be difficult for patients, caregivers, and physicians to navigate, especially without resources. Every country has a Health Authority with different requirements and processes for its expanded access programs. Understanding if expanded access is available in your country, the eligibility requirements, and the request process are all important when determining if it’s a potential treatment option. A lack of easily available information is a major obstacle for those exploring expanded access, making a resource like the Johnson & Johnson (J&J) Guide to Expanded Access vital.

PE: How many patients are in need of expanded access?

 This is difficult to assess for a variety of factors. However, any patient who is seriously or terminally ill, has a disease which has exhausted all available treatment options, and is not eligible for a clinical trial, is a potential candidate for expanded access. These programs are designed for this subset of patients who are unable to wait for the medicine to be available and are intended to be a bridge to when the medicine is available to them.

A common challenge for patients and their treating physicians is that expanded access is a just-in-time need during an already highly stressful time; therefore, access to readily available and transparent information is critical.

Because expanded access is an important potential option for patients, J&J built our expanded access program upon a bioethical framework that helps ensure we approach each request consistently and equitably. In 2015, we established two significant items which laid internal and external foundations to support those in need. First, we codified a policy and standard operating procedures to support the JNJ Innovative Medicines organization in considering expanded access as part of the development continuum. Second, in collaboration with New York University, we developed a first-of-its-kind committee to support our decision making, known as the Compassionate Use Advisory Committee (CompAC). We continue to evolve this external panel of ethicists, physicians, and patient representatives to support both development of our expanded access strategies as well as single patient requests. Since 2015, J&J has had the privilege of making investigational medicines available to more than 10,000 patients around the world.

PE: What led to the creation of the Guide to Expanded Access?

 In short, patient requests for information drove this effort. Information can be particularly difficult to find and, if available, may not always be in a format friendly to those not familiar with this option.And sometimes, even for those of us who are educated in this area, the requirements may not always be obvious. The Guide to Expanded Access is a comprehensive educational resource that was co-created with patient representatives, providing information in a people-friendly format for not only patients, but also their caregivers and treating physicians.

Anyone with a serious illness who has been through several lines of treatment and/or procedures can face many obstacles throughout the course of their treatment, but a lack of information should never be one of them. The power of having the right resources and information can’t be underestimated – something I learned as a nurse and firmly believe to this day.

As I mentioned earlier, expanded access processes vary by country, which can be very challenging to understand and navigate. The original Guide to Expanded Access, a first-of-its-kind resource released in 2021, was inspired by patient calls for greater clarity and resources and was designed to address this significant information gap. It was also important that this be geographically focused and company agnostic.

Now, three years later, we’ve released a revamped version after receiving positive feedback from advocates and patients alike. Patients not only led to the creation of this guide–their insights and experiences helped to bring it to life.

PE: What are the new elements in the updated version for 2024?

 The revamped Guide to Expanded Access builds upon the original, maintaining key features while adding new ones to further support patients. 

The guide now includes country-specific expanded access information for the United States, South Korea, and Switzerland, increasing the total countries covered to 30. Guided by patient and advocate feedback, we’ve also enhanced the language throughout for greater clarity.

Like the initial version, the updated guide is designed for easy navigation. It includes a table of contents map with all featured countries identified and linked to their respective pages, enabling patients to quickly locate country-specific information. Once on their desired country page, patients can find details spanning expanded access requirements, the request process, and more.

To increase patient understanding of the information presented, key terms are highlighted in red and linked to a glossary at the end of the guide. Links to external resources, such as a country’s regulatory agency site, are also included throughout.

The Guide is available for anyone to view at 

https://www.janssen.com/sites/www_janssen_com/files/guide_to_ea_june_2024.pdf

The executive director of patient strategies & solutions discusses J&J’s new Guide to Expanded Access.

Expanded Access Treatment Options: Q&A with Johnson & Johnson Innovative Medicine’s Christine MacCracken

Traci Miller, Director, Sonexus™ Access and Patient Support, Cardinal Health, discusses the current digital trends in the patient services industry and how the optimal balance of technology and talent can transform manufacturer-sponsored patient support programs. Hear how Cardinal Health combines best-in-class program and pharmacy operations with smart digital tools to ensure product and patient success and reduce operational costs. 

Unlocking value and cost savings in patient services with technology and talent 

The US Food and Drug Administration (FDA) has issued guidance for excipients deemed to be at high risk of ethylene glycol or diethylene glycol contamination. This webinar will provide insights into legal and scientific constraints, as well as how to ensure safety during the production of pharmaceutical products.

https://www.pharmexec.com/pe_w/fda-guidance



The US Food and Drug Administration (FDA) recently issued guidance for excipients deemed at high risk of ethylene glycol or diethylene glycol contamination.


MedicinesComplete provides quick and easy access to a central source of trusted, expert information on medicines, including 

, the authoritative guide to the properties for excipients, their safe use, and their application.


Updated regularly online, this product has been revised to include safety content for all high-risk excipients to support your organization’s compliance with FDA guidance.

through MedicinesComplete, including Martindale: The Complete Drug Reference, 

, Stockley’s Drug Interactions and BNF help industry professionals:

Advise on legal and scientific constraints;

Ensure the production of safe and efficacious treatments for patients, and;

Assess the safety profile of pharmaceutical products.

Learn how to advise on legal and scientific constraints.

Learn how to ensure the production of safe and efficacious treatments for patients.

Know how to assess the safety profile of pharmaceutical products.

Stay ahead of evolving guidelines with regularly updated content.


Senior International Account Manager
Pharmaceutical Press

Webinar Date/Time: Tue, Nov 19, 2024 11:00 AM EST

Jaime L. Barwig

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