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.

CAMBRIDGE, MA and RAHWAY, NJ — Moderna, Inc. and Merck, known as MSD outside of the United States and Canada, announced the first presentation of results from a planned analysis from the Phase 2b randomized KEYNOTE-942/mRNA-4157-P201 study, a clinical trial evaluating mRNA-4157 (V940), an investigational individualized neoantigen therapy (INT), in combination with KEYTRUDA, Merck's anti-PD-1 therapy, in patients with resected high-risk melanoma (stage III/IV) following complete resection (n=157). With a median follow-up of approximately three years (34.9 months), adjuvant treatment with mRNA-4157 (V940) in combination with KEYTRUDA continued to demonstrate a clinically meaningful and durable improvement in recurrence-free survival (RFS), the primary endpoint of the study, reducing the risk of recurrence or death by 49% (HR [95% CI], 0.510 [0.288-0.906]; two-sided nominal p-value 0.019) compared with KEYTRUDA alone. mRNA-4157 (V940) in combination with KEYTRUDA also continued to demonstrate a meaningful improvement in distant metastasis-free survival (DMFS), a key secondary endpoint of the study, compared with KEYTRUDA alone, reducing the risk of developing distant metastasis or death by 62% (HR [95% CI], 0.384 [0.172-0.858], two-sided nominal p-value 0.015).

These data are being presented during a rapid oral abstract session at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting (abstract #LBA9512). With an additional year of planned follow-up, these data build on the earlier analysis of the primary and key secondary endpoints of the study, presented in 2023. The 2.5-year recurrence-free survival rate of mRNA-4157 (V940) in combination with KEYTRUDA was 74.8%, as compared to 55.6% for KEYTRUDA alone, with the benefit observed across exploratory subgroups.

"We are encouraged by the latest results from the KEYNOTE-942/mRNA-4157-P201 study. These data highlight the sustained benefit in RFS and DMFS of mRNA-4157 (V940) as adjuvant treatment in combination with KEYTRUDA in people with resected high-risk melanoma. Importantly, this benefit was observed across various patient exploratory subgroups, reflecting the potential of mRNA-4157 (V940) for a broad range of these patients," said Kyle Holen, M.D., Moderna's Senior Vice President and Head of Development, Therapeutics and Oncology. "These findings reinforce our commitment to advancing this innovative treatment in collaboration with Merck, and we are dedicated to harnessing mRNA technology to potentially transform cancer therapy and improve patient outcomes."

"The sustained improvements in recurrence-free survival and distant metastasis-free survival observed at approximately three years in the KEYNOTE-942/mRNA-4157-P201 study provide further support of the potential of mRNA-4157 (V940) in combination with KEYTRUDA to help patients with resected high-risk melanoma," said Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories. "We look forward to building on our legacy of turning breakthrough science into medicines that may have a meaningful impact on patients' lives as we continue advancing our broad clinical development program evaluating this novel approach with Moderna."

Additional efficacy and subgroup data

Data from an exploratory subgroup analysis of the Phase 2b KEYNOTE-942/mRNA-4157-P201 study in patients with resected high-risk melanoma (stage III/IV) following complete resection showed that improvement in RFS was observed with mRNA-4157 (V940) in combination with KEYTRUDA compared to KEYTRUDA alone regardless of tumor mutational burden (TMB) or programmed death-ligand 1 (PD-L1) status.

The RFS benefit of mRNA-4157 (V940) in combination with KEYTRUDA compared to KEYTRUDA alone was maintained across both TMB high (HR [95% CI], 0.564 [0.253-1.258]), TMB non-high (0.571 [0.245-1.331]), PD-L1 positive (0.471 [0.226-0.979]), PD-L1 negative (0.147 [0.034-0.630]), and circulating tumor DNA (ctDNA) negative (0.207 [0.091-0.470]) subpopulations. ctDNA positive HR was not estimable due to the small sample size. There were no significant associations between individual human leukocyte antigen (HLA) alleles and RFS observed for mRNA-4157 (V940) in combination with KEYTRUDA.

The exploratory endpoint of overall survival (OS) favored mRNA-4157 (V940) in combination with KEYTRUDA compared to KEYTRUDA alone, with a 2.5-year OS rate of 96.0% vs. 90.2%, respectively (HR [95% CI], 0.425 [0.114-1.584]).

The safety profile with mRNA-4157 (V940) in combination with KEYTRUDA in KEYNOTE-942/mRNA-4157-P201 remains consistent with the primary analysis. The most common adverse events attributed to mRNA-4157 (V940) in combination with KEYTRUDA were fatigue (60.6%), injection site pain (56.7%), and chills (49.0%). The majority of the adverse events attributed to mRNA-4157 (V940) were Grade 1-2, with fatigue being the most common Grade 3 event and no Grade 4-5 events. Immune-related adverse events occurred in 37.5% of patients receiving the combination and 36% receiving KEYTRUDA alone.

Ongoing clinical development programs

Merck and Moderna have initiated Phase 3 randomized clinical trials evaluating mRNA-4157 (V940) in combination with KEYTRUDA as an adjuvant treatment in patients with resected high-risk (Stage IIB-IV) melanoma (INTerpath-001, NCT05933577) and non-small cell lung cancer (INTerpath-002, NCT06077760). Both trials are actively enrolling.

In 2024, Merck and Moderna also initiated a two-part Phase 2/3 randomized clinical trial evaluating mRNA-4157 (V940)in combination with KEYTRUDA as neoadjuvant and adjuvant treatment in patients with resectable locally advanced Stage II-IV (M0) cutaneous squamous cell carcinoma (INTerpath-007, NCT06295809), a Phase 2 randomized clinical trial evaluating mRNA-4157 (V940) in combination with KEYTRUDA as adjuvant treatment in patients with intermediate-high-risk, high-risk, or M1 no evidence of disease renal cell carcinoma (INTerpath-004, NCT06307431), and a Phase 2 randomized clinical trial evaluating mRNA-4157 (V940) in combination with KEYTRUDA as adjuvant treatment in patients with high-risk muscle-invasive urothelial carcinoma post-radical resection (INTerpath-005, NCT06305767).