We recently released several Reports on Cell and Gene Therapy (CGT) as it is considered a significant development in disease management with notable implications for the pharmacy field. As CGT continues to proliferate, incorporating information about the complex manufacturing of customized and personalized therapies into the curriculum should be part of our curriculum.

Today’s article probes into production challenges… especially related to scale. Cell and gene therapy is revolutionizing the treatment of complex diseases, but efficient large-scale production remains a significant hurdle. As demand rises, there is an increasing need for reliable, efficient methods of expanding cells—an essential step in manufacturing these therapies.

This is where Real-Time Monitoring in the laboratory comes into play. “A crucial innovation in cell expansion is the ability to visualize metabolic changes in real time. By continuously tracking key metabolic markers—such as glucose and lactate concentrations, pH, and dissolved oxygen levels—researchers can make informed adjustments to the culture environment, optimizing conditions for cell growth.” In short, precise control is vital for producing consistent, high-quality cells for therapeutic use.

Cells are typically cultivated in bioreactors, where nutrients, oxygen, and temperature are carefully regulated to support optimal growth and maintain quality. Real-time sensors allow for the automatic adjustment of culture conditions by monitoring metabolites like glucose and lactate. Sensors inform decisions such as when to replenish the culture media, helping maintain ideal growth conditions. Maintaining a closed system is especially critical in cell and gene therapy manufacturing, where contamination or disruptions can lead to costly delays or compromise therapy quality. This creates an environment conducive to cultivating healthier and more robust cells.

As CGT therapies are approved for larger patient populations the ability to produce the ‘cure’ goes up exponentially. The article also details how new technology is also being developed to support demand.

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Cracking the code to scalable cell and gene therapy

CLICK HERE to read the full article

The FDA recently approved a new oral therapy, Ibtrozi (taletrectinib) from Nuvation Bio Inc., indicated for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC).

ROS1+ NSCLC is a rare and aggressive form of lung cancer, accounting for approximately 2% of new NSCLC cases annually. Ibtrozi, a kinase inhibitor, will compete in the limited NSCLC subset against three established therapies. Analysts suggest that Ibtrozi offers key efficacy vs. tolerability differentiators that make it a contender to break through the pack. Additionally, the approval did not include any warnings for adverse CNS side effects or a restriction of use to only the second line setting.

Nuvation announced it would launch Ibtrozi at a cost of $140,000 annually.
The company did not announce plan for distribution; however, Onco360 Specialty Pharmacy has confirmed its role as a national distribution partner for Ibtrozi.

CLICK HERE to access prescribing information

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FDA Approves Ibtrozi For Adults With ROS1+ NSCLC

CLICK HERE to read the full article

Announcements for newly approved specialty drugs often state that the product will be available through specialty pharmacy in limited distribution. However, the early press releases rarely specify the specialty pharmacy(ies) selected as the designated partner(s).

Here are six LD deals that have been publicly confirmed subsequent to their FDA approvals.

Onco360 to Distribute Ibtrozi

Onco360 has been selected as a national pharmacy partner by Nuvation Bio Inc. for Ibtrozi (taletrectinib), a kinase inhibitor approved for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC).

Onco360 to Distribute Qinlock

Onco360 has been selected as a pharmacy partner by Deciphera Pharmaceuticals, LLC for Qinlock (ripretinib) approved for the treatment of adult patients with advanced gastrointestinal stromal tumors (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib.

Onco360 to Distribute Romvimza

Onco360 has been selected as a specialty pharmacy partner by Deciphera Pharmaceuticals, LLC for Romvimza (vimseltinib), which has been approved by the U.S. Food and Drug Administration for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity.

Onco360 to Distribute Augtyro

Onco360 has been selected as a specialty pharmacy partner by Bristol Myers Squibb for Augtyro (repotrectinib) for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) and 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.

LeMed Specialty Pharmacy to Distribute Harliku

LeMed Specialty Pharmacy was named by Cycle Pharmaceuticals to be the exclusive specialty pharmacy for specialty pharmacy distribution and services for Harliku (nitisinone) tablets. Harliku is the first and only FDA-approved treatment for use in alkaptonuria (AKU). LeMed will manage commercial and non-commercial dispensing for Harliku across the United States.

Ekterly Specialty Pharmacy Network

KalVista Pharmaceuticals has announced that CVS Specialty, Accredo, Optum, and Orsini have been selected as the specialty pharmacy partners for Ekterly (sebetralstat). Ekterly is an FDA-approved oral therapy for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older.

Today’s article posed a seemingly straightforward question: Is there a marketplace for mail order? Our initial response was an emphatic “YES.” However, after further reading, it became clear that our first impressions regarding mail order pharmacy required more careful consideration.

Despite the convenience it promises, mail order pharmacy has historically seen only gradual acceptance among the public. Research from 2000 to 2021 consistently found only a modest rate of adoption, and even the most recent studies indicate that just 10-15% of people choose mail order as their primary method for obtaining prescriptions. Several factors contribute to this cautious approach, but shifts in the industry are poised to spark greater interest.

Insurers are increasingly steering their members toward network pharmacies, often promoting that prescriptions be filled via mail order—particularly when the mail order service (often a PBM) is owned by the insurer itself. Meanwhile, major national retailers including Walmart, Costco, and Amazon are also reshaping the landscape by actively promoting their own mail order pharmacy services. Patients that use a specialty mail service pharmacy for the first time find that this model can also work well for non-specialty meds. As these diverse forces converge, they are set to transform both the marketplace and public perception of mail order fulfillment.

PS: Will a new category be added to future studies on pharmacy fulfillment — DRONE Delivery?

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Is There a Marketplace for Mail Order?

CLICK HERE to read the full article

States are increasingly targeting PBMs, as media scrutiny of PBM practices intensifies and PBMs respond with legal action. Arkansas, for example, has sought relief from drug pass-through pricing, prompting PBMs to challenge state laws restricting their ownership of retail pharmacies.

The article notes: “Large pharmacy benefit managers like CVS Caremark, Express Scripts and Optum Rx are increasingly being blamed for higher drug prices and the outsize role they play in the pharmaceutical supply chain.” This PBM model intertwines the supply chain and acquisition agreements, making reforms potentially expensive. Another complication relates to how PBMs reimburse pharmacies, particularly when they own those pharmacies.

This article provides a brief look at the evolving pharmacy market, which is expected to face greater changes ahead—especially as public awareness grows leading up to the 2026 mid-terms.

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PBMs fight back against state restrictions

CLICK HERE to read the article

It is common to focus on the exciting approvals of cell and gene therapies. However, there has also been significant progress in the small molecule world, where new technologies are starting to supercharge the segment. As referenced in today’s article, “Every day, millions rely on small molecule medicines — yet few appreciate the sophisticated processes ensuring each pill’s safety and efficacy.”

The article cites well over a dozen notable advancements… each of which contribute to unit profitability. For instance, some companies implementing continuous manufacturing technology have reported up to 40 percent reductions in production time and yield increases of as much as 8 percent.

Organizations are introducing AI-driven control systems and robotics, leading to batch rejection rate reductions by as much as 30 percent and decreased troubleshooting times.

Additionally, 3D printing and on-demand manufacturing are being developed for personalized dosing. Manufacturing tailored pills and tablets for individual patients is now possible without interrupting continuous production lines… or even at the point of care.

Developments such as modular micro-plants, flow reactors, and 3D-printed tablets indicate that emerging hardware and software are contributing to faster, more environmentally friendly, and flexible small molecule drug production.

The article also highlights certain challenges facing companies. Primarily, regulatory compliance will require rigorous validation for all new technologies. Non-stop quality control – including extensive testing for impurities, potency, and dissolution – will remain essential for patient safety at the expense of slowing down operations.

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Small Molecule Pharma Companies Are Employing These Advanced Technologies To Get Ahead

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The FDA recently approved yet another biosimilar to Stelara. Starjemza (ustekinumab-hmny) is from Bio-Thera Solutions and Hikma Pharmaceuticals. The lineup of the previously approved Stelara biosimilars follows… Selarsdi (ustekinumab-aekn), Pyzchiva (ustekinumab-ttwe), Otulfi (ustekinumab-aauz), Imuldosa (ustekinumab-srlf), Wezlana (ustekinumab-auub), Yesintek (ustekinumab-kfce) and Steqeyma (ustekinumab-stba), the last of which was approved earlier this year.

Starjemza is indicated for—–
Adult patients with:
moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy;
active psoriatic arthritis;
moderately to severely active Crohn’s disease; and
moderately to severely active ulcerative colitis.
Pediatric patients 6 years of age and older with:
moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy; and
active psoriatic arthritis.

It is noted that prescribing information specifies subcutaneous dosing and administration for Psoriasis and Psoriatic Arthritis while specifying intravenous dosing instructions for Crohn’s Disease and Ulcerative Colitis.

Bio-Thera and Hikma did not release pricing at the time of approval. By way of reference, the list price of Stelara is $13,836 for one month supply. Earlier Stelara biosimilars have projected pricing to come in at a 20% discount to Stelara.

Bio-Thera and Hikma did not specify details for distribution.

CLICK HERE for prescribing information.

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FDA Approves Ustekinumab Biosimilar Starjemza

CLICK HERE to read the company press release

The FDA recently approved a new therapy, Avmapki Fakzynja Co-Pack (avutometinib capsules; defactinib tablets) from Verastem Oncology, indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. The therapy, a combination of avutometinib and defactinib, each kinase inhibitors, is co-packaged for oral use.

Dosing presents a challenge for patients as one drug is administered twice weekly while the companion drug is dosed twice daily for three weeks then followed by a one-week therapy hold for each therapy.

Low-grade serous ovarian cancer (LGSOC) is a rare, persistent subtype of ovarian cancer that differs markedly from high-grade serous ovarian cancer (HGSOC) in both biological characteristics and therapeutic response. LGSOC demonstrates lower sensitivity to chemotherapy, exhibits a high recurrence rate, and frequently necessitates specialized treatment strategies. This malignancy predominantly affects younger women, with diagnoses most commonly occurring between ages 20–30 and 50–60, and the median survival is approximately ten years. It is estimated that 6,000–8,000 women in the United States are currently living with LGSOC. Most cases are associated with mutations in the RAS signaling pathway, and approximately 30% present with a specific KRAS mutation.

The company announced that it will charge $48,500 for a 28-day regimen of the treatment, which includes two 3.2 mg doses per week of Avmapki and two daily 200 mg doses of Fakzynja.

Also, the company has selected Onco360 to distribute the therapy.

Click Here for prescribing information

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FDA Grants First-Ever Approval for Treatment of KRAS-Mutated Recurrent LGSOC

Click Here to read the press release

As our understanding of cell and gene therapy deepens, the demand for specialized knowledge and expertise grows. This document explores the manufacturing and even shipment processes of these groundbreaking therapies, highlighting their challenges and the impact on the patient experience.

Production Processes and Challenges—
The development of cell and gene therapies introduces unique obstacles in pharmaceutical manufacturing. Efficient production and reliable delivery systems are essential to support patients effectively. Below are key pieces to the therapy puzzle in this evolving field.

Key Topics in Cell and Gene Therapy Production Technologies:
・Manufacturing Platforms: The use of automation and standardization with specialized platforms that enhance both scalability and flexibility in production.
・Plasmid Production: Advancements in purification technologies that address issues related to scale, purity, and efficiency in gene delivery.
・Innovative Assays: Cutting-edge analytical methods are being developed to ensure safety, efficacy, and purity.
・Automation in Analytics: Automated analytical tools are expanding capacity, improving scalability, and meeting increasing quality control demands.
・Therapeutic Containers: The creation of rigid, durable containers that support safer storage and that are compatible with automated systems.
・Sterile Connections: Smaller, more efficient aseptic connectors that simplify sterile processing, including at ultra-low temperatures.
・Ecosystem Optimization: Integrating advanced tools, techniques, and platforms that foster a flexible, continuously improving the ecosystem for the production of cell and gene therapies.

Mastering these subjects may distinguish you among your peers and establish you as a ‘the’ knowledgeable and respected member of your group.

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Improving CGT Production to Speed Delivery and Lower Cost

CLICK HERE to read the full article

The FDA recently approved a new therapy, Emrelis (telisotuzumab vedotin-tllv) from AbbVie, indicated for the treatment of adult patients with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) with high c-Met protein overexpression, as determined by an FDA-approved test, who have received a prior systemic therapy.

Emrelis represents a significant breakthrough in the targeted treatment landscape for lung cancer. This novel therapy functions as an antibody-drug conjugate (ADC) with a c-Met-directed mechanism, combining the precision of antibody targeting with the potency of a microtubule inhibitor. Administered exclusively via infusion, Emrelis stands as the inaugural therapy of its kind specifically approved for this group of patients. Its approval, however, may require future trials that confirm its clinical benefit.

Approximately 85% of lung cancers are classified as NSCLC and, despite advances in treatment, lung cancer remains the leading cause of cancer-related deaths. Emrelis is a first-in-class c-Met-directed antibody-drug conjugate (ADC) that targets c-Met expressing cells.

Emrelis is administered intravenously every 2 weeks until disease progression or unacceptable toxicity.

Emrelis launched at a wholesale cost of $13,980 for a 100-milligram vial and $2,796 for a 20-milligram vial. Including step-down dosing at induction, the annual cost of therapy will run ~$400,000.

The company did not disclose any details related to distribution.

CLICK HERE to access prescribing information

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U.S. FDA Approves EMRELIS™ (telisotuzumab vedotin-tllv) for Adults With Previously Treated Advanced Non-Small Cell Lung Cancer (NSCLC) With High c-Met Protein Overexpression

CLICK HERE to access the press release