NORTH CHICAGO, IL — AbbVie announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the conditional marketing authorization of epcoritamab (TEPKINLY), the first and only T-cell engaging bispecific antibody administered subcutaneously (under the skin), as a monotherapy for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) after two or more prior therapies. The European Commission decision on this indication for epcoritamab is anticipated later this year.

"Patients with follicular lymphoma are likely to face disease recurrence and shorter durability of response with each subsequent line of treatment. This positive opinion recognizes the unmet need in the European Union for individuals with relapsed or refractory follicular lymphoma, following failure of other therapies," said Mariana Cota Stirner, M.D., Ph.D., vice president, therapeutic area head for hematology, AbbVie.

FL is typically an indolent (or slow-growing) form of non-Hodgkin's lymphoma (NHL) that arises from B-lymphocytes and is the second most common form of NHL accounting for 20-30% of all cases. In 2023, there were an estimated 13,000 cases of FL in Western Europe. FL is considered incurable with current standard of care therapies.

The CHMP opinion is supported by overall and complete response data from the Phase 1/2 EPCORE NHL-1 clinical trial in 128 patients with R/R FL treated with epcoritamab after two or more lines of prior therapy. The study included patients who were refractory to both anti-CD20 monoclonal antibody therapy and an alkylating agent, patients who were refractory to last prior treatment, and patients whose disease progressed within two years of first systemic therapy. The safety profile of epcoritamab in the pivotal cohort was similar to reports of epcoritamab monotherapy in the pivotal EPCORE NHL-1 diffuse large B-cell lymphoma (DLBCL) cohort.

An additional cohort of 86 patients evaluated a 3-step-up dosing (SUD) schedule to reduce the incidence and severity of cytokine release syndrome (CRS), which is an associated adverse effect from immune-engaging cancer treatments. For the first full dose of this 3-step regimen, mandatory hospitalization was not required. In this cohort, the incidence of CRS was 49% (42 of 86 patients; 9% were grade 2). There were no grade 3 or higher CRS events. The data from this optimization cohort of the EPCORE NHL-1 study were recently published in the Lancet Haematology.

"Each year, thousands of people in Europe are diagnosed with follicular lymphoma, and it's an upsetting reality that many of them will experience relapse and refractory disease," said Catherine Thieblemont, M.D., Ph.D., head of the hemato-oncology department, Paris University, Hôpital Saint-Louis Assistance-Publique-Hopitaux de Paris (APHP) in Paris. "Patients deserve new treatment options, and this positive opinion is the first step to bringing epcoritamab to more patients who need it."

Epcoritamab is being co-developed by AbbVie and Genmab as part of the companies' oncology collaboration. The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization. Both companies will pursue additional international regulatory approvals for the investigational R/R FL indication and additional approvals for the R/R DLBCL indication.

About the Phase 1/2 EPCORE NHL-1 Trial
EPCORE NHL-1 is an open-label, multi-center safety and preliminary efficacy trial of epcoritamab that consists of three parts: a dose escalation part; an expansion part; and an optimization part. The trial was designed to evaluate subcutaneous epcoritamab in patients with relapsed, progressive or refractory CD20+ mature B-cell non-Hodgkin's lymphoma (B-NHL), including FL. In the expansion part, additional patients were enrolled to further explore the safety and efficacy of epcoritamab in three cohorts of patients with different types of relapsed/refractory B-NHLs who have limited therapeutic options. The optimization part evaluates the potential for alternative step-up dosing regimens to help further minimize Grade 2 cytokine release syndrome (CRS) and mitigate Grade ≥3 CRS. The primary endpoint of the expansion part was ORR as assessed by an IRC. Secondary efficacy endpoints included duration of response, complete response rate, duration of complete response, progression-free survival, and time to response as determined by the Lugano criteria. Overall survival, time to next therapy, and rate of minimal residual disease negativity were also evaluated as secondary efficacy endpoints. The primary endpoint of the optimization part was the rate of ≥ Grade 2 CRS events and all grade CRS events from first dose of epcoritamab through 7 days following administration of the second full dose of epcoritamab.

Topline results of the study were shared in December 2023. More information can be found on www.clinicaltrials.gov (NCT03625037).

About Follicular Lymphoma (FL)
FL is typically an indolent (or slow-growing) form of non-Hodgkin's lymphoma (NHL) that arises from B-lymphocytes and is the second most common form of NHL accounting for 20-30% of all cases. FL is considered incurable with current standard of care therapies. Patients often relapse and with each relapse, the remission and time to next treatment is shorter. Over time, transformation to DLBCL, an aggressive form of NHL associated with poor survival outcomes, can occur in more than 25% of FL patients.

About Epcoritamab
Epcoritamab is an investigational IgG1-bispecific antibody created using Genmab's proprietary DuoBody technology and administered subcutaneously. Genmab's DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T-cell-mediated killing of CD20+ cells.

Epcoritamab (approved under the brand name EPKINLY in the United States and TEPKINLY in the European Union) has received regulatory approval in certain lymphoma indications in several countries.

AbbVie will continue to pursue regulatory submissions for epcoritamab across international markets. Both Genmab and AbbVie continue to evaluate the use of epcoritamab as a monotherapy and in combination across lines of therapy in a range of hematologic malignancies.

ROSEMONT, IL — Long Grove Pharmaceuticals, LLC, a supplier of differentiated pharmaceuticals, will bring new supply of Sodium Bicarbonate Injection, USP to the United States. Sodium Bicarbonate Injection, USP is commonly used in critical care settings yet routinely found on the drug shortages list.

"As we look at the launch of our next pharmaceutical products, our focus is always on the area of greatest need. Sodium Bicarbonate Injection supply is historically unstable, creating a challenging environment for hospitals and the patients they serve," said Peter Karas, Chief Commercial Officer at Long Grove Pharmaceuticals. "We’re excited by the opportunity to help alleviate drug shortages for this product and continue our commitment to ensuring patients have access to the medicine they need.”

Long Grove’s Sodium Bicarbonate Injection, USP is Food and Drug Administration (FDA) approved and AP rated. The company will launch its 8.4% Sodium Bicarbonate Injection, USP 50 mEq/ 50 mL vials in the second half of 2024.

Sodium Bicarbonate Injection, USP is indicated in the treatment of metabolic acidosis from severe renal disease, uncontrolled diabetes and cardiac arrest. It is most frequently used in critical care settings.

SAN DIEGO, CA — Halozyme Therapeutics, Inc. announced that argenx received U.S. Food and Drug Administration (FDA) approval for VYVGART® Hytrulo co-formulated with ENHANZE®, Halozyme's proprietary recombinant human hyaluronidase enzyme, rHuPH20, for the treatment of adults with chronic inflammatory demyelinating polyneuropathy (CIDP).

VYVGART® Hytrulo for CIDP is FDA-approved as a once weekly 30-to-90 second subcutaneous (SC) injection. This approval also represents the second FDA-approved indication for VYVGART® Hytrulo with ENHANZE®.

"With this approval, CIDP patients in the U.S. will have access to the first novel mechanism of action to treat CIDP in 30 years, which lessens the burden of treatment as a 30 to 90 second weekly SC injection," said Dr. Helen Torley, president and chief executive officer of Halozyme. "We look forward to continuing to support argenx with our ENHANZE technology that has enabled improved treatment options that provide meaningful benefits for patients and healthcare providers."

The FDA approval is based on the ADHERE study, the largest clinical trial to date studying CIDP. In the ADHERE study, 69% (221/322) of patients treated with VYVGART® Hytrulo, regardless of prior treatment, demonstrated evidence of clinical improvement, including improvements in mobility, function and strength. ADHERE met its primary endpoint (p<0.0001) demonstrating a 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in the risk of relapse versus placebo. 99% of trial participants elected to participate in the ADHERE open-label extension. The safety results were generally consistent with the known safety profile of VYVGART® in previous clinical studies and real-world use.

VYVGART® Hytrulo is also approved in the U.S. for the treatment of generalized myasthenia gravis in adult patients who are anti-acetylcholine receptor antibody positive.

The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled Considerations for Demonstrating Interchangeability with a Reference Product: Update. This draft guidance describes considerations regarding a switching study or studies intended to support a demonstration that a biological product is interchangeable with a reference product.

"Both biosimilars and interchangeable biosimilars meet the same high standard of biosimilarity for FDA approval and both are as safe and effective as the reference product," said Sarah Yim, director of the Office of Therapeutic Biologics and Biosimilars at the FDA.

FDA issued the guidance for industry Considerations in Demonstrating Interchangeability With a Reference Product (May 2019) (Interchangeability Guidance) before receiving and reviewing any biologics license applications (BLAs) submitted under section 351(k) of the Public Health Service Act (PHS Act) for a proposed interchangeable biosimilar.  Since publication of the Interchangeability Guidance, experience has shown that for the products approved as biosimilars to date, the risk in terms of safety or diminished efficacy is insignificant following single or multiple switches between a reference product and a biosimilar product.  Accordingly, FDA’s scientific approach to when a switching study or studies may be needed to support a demonstration of interchangeability has evolved.  As described in this draft guidance, applicants for proposed interchangeable products may choose to provide an assessment of why the comparative analytical and clinical data provided in the application or supplement support a showing that the switching standard set forth in section 351(k)(4)(B) of the PHS Act has been met.

Per the FDA, this guidance is being distributed for comment purposes only. Submit Comments by 08/20/2024, the agency requests, to ensure that your comment can be considered on a draft guidance before it begins work on the final version of the guidance, submit either online or written comments on the draft guidance before the close date.

“Programs like RxPass help reduce cost, while increasing convenience for caregivers, and customers of all ages, which is shown to improve medication adherence and support better health outcomes,” said John Love, Vice President of Amazon Pharmacy. “Many people don’t realize that even if you have great insurance, you can still save on select medications by using programs like RxPass. The overall cost of medications can be lower, and that’s not even factoring in the time and effort saved from not having to drive to a pharmacy or stand in line.”

PRINCETON, NJ — Bristol Myers Squibb announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Augtyro™ (repotrectinib) for the treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy. The approval is based on results from the Phase 1/2 TRIDENT-1 study, which evaluated Augtyro in adult patients with NTRK-positive solid tumors. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

“NTRK fusion-positive tumors can present challenges in the clinical setting, which is why it is important that we have additional treatment options for these patients,” said Alexander Drilon, MD, TRIDENT-1 global trial lead and Chief of the Early Drug Development Service at Memorial Sloan Kettering Cancer Center. “The FDA approval of repotrectinib adds an important tool to our toolbox, offering oncologists a next-generation TKI that can be used across a broad range of NTRK fusion-positive solid tumors for both TKI-naïve and TKI-pretreated patients.”

The TRIDENT-1 trial included both TKI-naïve (n=40) and TKI-pretreated (n=48) patients with NTRK-positive locally advanced/metastatic solid tumors collectively representing 15 different types of cancer. In TKI-naïve patients, with a median follow up of 17.8 months, 58% (95% CI: 41 to 73) had a confirmed objective response rate (cORR); of those, 43% experienced partial responses (PR) and 15% had complete responses (CR). Of the TKI-naïve responding patients, 83% were still in response at one year with Augtyro. The median duration of response (mDOR) was not yet reached. In TKI-pretreated patients, with a median follow up of 20.1 months, the cORR was 50% (95% CI: 35 to 65); of those, 50% experienced PR and no patients achieved CR. Additionally, 42% of TKI-pretreated responding patients were still in response at one year with Augtyro. The mDOR was 9.9 months (95% CI: 7.4 to 13.0). Among those who had measurable central nervous system (CNS) metastases at baseline, intracranial response was observed in 2 out of 2 TKI-naïve patients and in 3 out of 3 TKI-pretreated patients.

Augtyro is associated with the following Warnings & Precautions: central nervous system (CNS) effects, interstitial lung disease (ILD)/pneumonitis, hepatotoxicity, myalgia with creatine phosphokinase elevation, hyperuricemia, skeletal fractures, and embryo-fetal toxicity.

“FDA approval of Augtyro for patients with NTRK-positive tumors adds to its indication in ROS1-positive NSCLC, showing its clinical value for more people across multiple genetic markers,” said Nick Botwood, senior vice president of Medical Oncology at Bristol Myers Squibb. “Previously, there was not an FDA approved treatment option for NTRK-positive cancers that was studied in both TKI-naïve and TKI-pretreated patients across solid tumors. This milestone helps address this area of unmet need and builds on Bristol Myers Squibb’s longstanding legacy of bringing innovations to individuals who are facing cancer and urgently seeking new treatment options.”

“Cancer can be frightening regardless of the type, but having a rare gene fusion driving it can be especially stressful and isolating,” said Susan Spinosa, president and patient co-founder of NTRKers, a patient advocacy group. “It’s exciting to know that there’s a new targeted therapy option for patients with NTRK-positive gene fusions, as this may offer hope to patients and their loved ones navigating this difficult journey.”

Based on clinical and pharmacokinetic data, the recommended dose for Augtyro for pediatric patients aged 12 years and older is the same as for adults, 160 mg orally once daily for 14 days followed by 160 mg twice daily until disease progression or unacceptable toxicity. The safety and effectiveness of Augtyro have not been established in pediatric patients younger than 12 years of age with solid tumors who have an NTRK gene fusion. This is the second indication for Augtyro in the U.S., following its full approval for the treatment of adult patients with locally advanced or metastatic ROS1-positive NSCLC in November 2023.

Disclosure: Dr. Drilon has provided advisory and speaking services to Bristol Myers Squibb.

ALEXANDRIA, VA — The National Community Pharmacists Association (NCPA) today launched a push for payment reform that educates patients on how pharmacy benefit managers are increasing prescription drug prices. The national campaign, featuring television, digital and print ads, aims to push Congress to enact PBM payment reform that has broad bipartisan support in both houses.

As part of the launch, NCPA also released the results of a new poll revealing widespread public concern over prescription drug costs and PBMs:

• 84% of respondents agree that prescription drug costs are too high.
• 73% are concerned about PBMs' impact on drug pricing.
• 68% agree that PBMs are driving up drug costs.
• 60% would be more likely to vote for a candidate who supported legislation addressing PBM abuses.
• 62% support Congress having some oversight into the practices of PBMs to ensure fair pricing and transparency.

The campaign will target patients across the country with educational material.

"Eighty percent of all prescriptions in the U.S. are controlled by just three pharmacy benefit managers, and they are all owned by or affiliated with the largest insurance companies in the country," said NCPA CEO B. Douglas Hoey, pharmacist, MBA. "These are Fortune 15 corporations that behave like monopolies, and they are hurting patients with higher prescription costs and killing off small businesses at the average rate of one every day. There is broad bipartisan support in Congress for reform, and we are determined to get it passed before the end of the year.

"As the national poll shows, most patients don't know very much about PBMs, despite that they have so much control over which prescription patients take and how much they pay for those drugs. So, while we want pharmacy teams to continue their advocacy, this campaign is aimed at patients. We want them to let their members of Congress know that PBM reforms must be enacted this year."

ROSEMONT, IL — It is a New Day for Norepinephrine! Long Grove Pharmaceuticals now offers ready-to-use Norepinephrine in 0.9% Sodium Chloride in the 16 mg/250mL (64 mcg/mL) strength and anticipates the availability of the 4 mg/250 mL (16 mcg/mL) and 8 mg/250 mL (32 mcg/mL) strengths in early summer.

This ready-to-use premix has a 24-month shelf life at room temperature and is safe to use up to seven days after removal from the foil overwrap. A new source for Norepinephrine is a much needed addition to the fragile critical care supply chain. The convenience and safety of this manufactured premix formulation will help hospital pharmacies focus their limited resources on addressing unique and critical patient needs.

“We’re very happy to help play a role in relieving some of the stresses that our hospital pharmacy colleagues face every day. Over the last six months, we have met with many customers who have shared their ongoing challenges and have been very supportive of our efforts to bring a safe, convenient premix Norepinephrine to market.” said Peter Karas, Chief Commercial Officer of Long Grove Pharmaceuticals.

Norepinephrine in 0.9% Sodium Chloride Injection is indicated to raise blood pressure in adult patients with severe, acute hypotension, and it is one of the most commonly used injectable medications in the critical care setting. 

NEW YORK, NY — The FDA Peripheral and Central Nervous System Drugs Advisory Committee unanimously voted that donanemab shows clinical benefit for the treatment of early Alzheimer's disease. If approved, donanemab, developed by Eli Lilly, would become the second disease-modifying drug for Alzheimer's to receive full approval, expanding the arsenal of available drugs needed to treat Alzheimer's with combination therapy.

"Today's vote offers hope that donanemab will be approved in the coming months, but it's important to look at this milestone in the larger treatment landscape for Alzheimer's, which will entail a combination therapy and precision medicine approach," says Dr. Howard Fillit, Co-Founder and Chief Science Officer of the Alzheimer's Drug Discovery Foundation (ADDF). "If approved, donanemab will expand the first class of disease-modifying drugs, serving as the building blocks for future generations of drugs. Anti-amyloids are not a silver bullet, but they offer opportunities for patients to modify the course of the disease while the field works towards developing more novel therapies that target the underlying biology."

The donanemab trials demonstrate the field's ability to conduct innovative and rigorous biomarker-powered trials that provide a definitive answer on a drug's effectiveness. By using a "goldilocks strategy," the TRAILBLAZER-ALZ 2 trial used both amyloid and tau imaging to identify patients who were in the early stages of Alzheimer's and most likely to benefit from treatment.

"It's encouraging to see that some patients essentially enter remission, where they achieve full amyloid clearance with donanemab, with no resurgence in substantial plaque buildup for nearly four years," notes Dr. Fillit. "These findings are a direct result of biomarker tests that can detect, quantify, and monitor plaque buildup in the brain. Biomarkers will continue to revolutionize clinical trial design as we move towards developing drugs that target novel pathways guided by the biology of aging."

This milestone comes at a critical time when drug development is now largely driven by the biology of aging approach. In the current pipeline, nearly 75% of Alzheimer's drugs in development are exploring novel targets related to the various aging pathways like inflammation, metabolic disturbances, and vascular dysfunctions. Some of the promising drugs are from ADDF-funded companies such as Coya Therapeutics, which is developing a combination therapy targeting neuroinflammation, as well as Therini Bio, which is developing a drug to target vascular dysfunction, and PharmatrophiX, which recently completed a phase 2a trial for a neuroprotective drug now ready for phase 2b trials.

Diversity in the drug pipeline coupled with recent advances in biomarkers—notably the advent of accessible and scalable blood-based biomarkers—sets the stage for a new frontier in Alzheimer's care where early detection, diagnosis, and intervention is feasible. Biomarkers will also be integral to the prevention of the disease by identifying patients in the preclinical phase before symptoms present and reducing Alzheimer's risk based on their individual biomarker profiles.

"Today's advisory committee endorsement is a milestone achievement for the researchers, patients, families, and caregivers who have dedicated years to advancing new treatment options, but our work does not end here. We must continue fostering innovation and progress to move closer to the day where Alzheimer's is treated on an individual basis with the help of precision medicine, ultimately halting the progression or preventing the onset of the disease altogether," closes Dr. Fillit.

WASHINGTON, PA – Washington Health System is pleased to announce it has officially joined UPMC, becoming UPMC Washington and UPMC Greene.

In June of 2023, the Boards of Directors of Washington Health System (WHS) and UPMC announced an affiliation agreement between the two entities that would integrate WHS into the UPMC system. One year later, on June 1, 2024, that affiliation became official, ensuring a future of excellent patient care for residents of Washington, Greene and surrounding counties.

“We are elated that the affiliation is complete and look forward to starting a new chapter of collaboration with UPMC,” said Brook Ward, president of UPMC Washington and UPMC Greene. “This affiliation protects the vitality of an essential community asset and solidifies a healthy future for Washington and Greene counties for generations to come.”

UPMC has committed to invest at least $300 million over 10 years to enhance clinical services and upgrade facilities at UPMC Washington and UPMC Greene. These investments by UPMC, combined with contributions the WHS Foundation has made and will continue to make, ensures UPMC Washington and UPMC Greene can provide state-of-the-art health care for local residents for years to come and maintain its position as one of the largest employers in Washington and Greene counties.

“Our focus is on ensuring residents have access to life-saving services and advanced care that is sustainable into the future and preserving jobs of our talented health care workers,” said Ward. “The community is gaining close-to-home access to the nationally recognized, high-specialty care of UPMC. Our local capabilities will expand, creating a destination for world-class care in southwestern Pennsylvania.”

Clinical collaborations between WHS and UPMC for oncology (UPMC Hillman Cancer Center joint venture), pediatric specialties (UPMC Children’s Hospital of Pittsburgh), women’s health (UPMC Magee-Womens) and heart and vascular care (UPMC Heart and Vascular Institute), have been in place for more than a decade, providing care for more than 10,000 patients annually. UPMC will continue to invest in and advance key services locally, including inpatient and emergency care, women’s health, cardiology, surgical services, diagnostics, primary care and specialty and outpatient services.

“UPMC has a long, successful track record of affiliations with like-minded organizations. We know how essential these hospitals are to this region to preserve needed health care services and livelihoods of thousands touched by them, and we are thrilled to welcome UPMC Washington and UPMC Greene to UPMC,” said Leslie C. Davis, president and CEO of UPMC.

UPMC Washington and UPMC Greene will maintain a local Board of Directors consisting of 11 legacy WHS board members and five newly appointed members from UPMC.

The affiliation does not affect community members’ in-network access to WHS hospitals or doctors. UPMC Washington, UPMC Greene and their affiliated outpatient facilities will continue to honor the contracts that are in place with regional and national insurers.

A formal celebration of the affiliation and a press conference will be announced in the coming days.