Month: September 2025

The FDA recently approved a new infused therapy, Datroway (detumomab deruxtecan-dlnk) from AstraZeneca, indicated for the treatment of adult patients with unresectable or metastatic, hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease.

Datroway is the first and only TROP2-directed therapy approved in the US for the treatment of lung cancer. Advanced EGFR-mutated non-small cell lung cancer (NSCLC) is a subtype of lung cancer defined by the presence of specific mutations in the epidermal growth factor receptor (EGFR) gene. These mutations make the cancer cells grow and divide more rapidly. EGFR mutations are most commonly found in adenocarcinomas.

Datroway is a weight-based therapy requiring 6 mg/kg given as an intravenous infusion once every 3 weeks (21-day cycle) until disease progression or unacceptable toxicity.

AstraZeneca did not set a list price for Datroway at time of approval.
The company did not comment on distribution plans for the product.

CLICK HERE to access prescribing information

---

Datroway approved in the US for patients with previously treated advanced EGFR-mutated non-small cell lung cancer

CLICK HERE to read the company press release

The FDA recently approved a sub-q therapy, Yeztugo (lenacapavir) from Gilead, indicated for pre‑exposure prophylaxis (PrEP) to reduce the risk of sexually acquired HIV-1 in adults and adolescents weighing at least 35 kg who are at risk for HIV-1 acquisition. Individuals must have a negative HIV-1 test prior to initiating Yeztugo.

Yeztugo is the first injectable pre‑exposure prophylaxis (PrEP) given twice yearly via subcutaneous injection in the abdomen for adults and adolescents. Traditionally, HIV prevention relied on daily oral PrEP regimens, which have adherence challenges.

Lenacapavir’s twice‑annual dosing offers a near‑vaccine‑like convenience. Clinical trials showed near‑complete protection, zero infections in one cohort and dramatically fewer in another.

Market analysts suggest that the cost for Yeztugo will be set at approximately $28,000 per year.
Gilead did not comment on distribution details.

CLICK HERE to access prescribing information.

---

Lenacapavir HIV PrEP: Not an AIDS Vaccine, but Vaccine Adjacent

CLICK HERE to read the full article

The FDA recently approved Andembry (garadacimab-gxii) from CSL for the prophylactic treatment of hereditary angioedema (HAE). This approval marks a significant milestone as Garadacimab is the first and only Factor XIIa inhibitor approved for routine prevention of HAE attacks, offering once-monthly dosing for all patients from the start. Additionally, Andembry is approved for subcutaneous, self-injection delivered in 15 seconds or less via an autoinjector with a citrate-free formula.

Hereditary angioedema is a rare, genetic disorder characterized by unpredictable and potentially life-threatening swelling episodes in various parts of the body, including the face, abdomen, and airways. The condition is caused by a deficiency or dysfunction of the C1 esterase inhibitor, leading to uncontrolled activation of the kallikrein-kinin system and excess bradykinin production.

Garadacimab targets activated Factor XII (Factor XIIa), a key driver in the inflammatory cascade of HAE. Its unique mechanism represents a novel therapeutic class, providing a preventive option without requiring titration or loading doses. In clinical trials, Garadacimab demonstrated statistically significant reductions in HAE attacks with a favorable safety profile. Common side effects reported in trials included injection site reactions, upper respiratory tract infections, and headache. No serious treatment-related adverse events were observed.

Specific cost details have not yet been disclosed publicly as of the approval date.
Andembry will be available in the U.S. through specialty pharmacies.

CLICK HERE to access prescribing information

---

U.S. Food and Drug Administration Approves CSL’s ANDEMBRY (garadacimab-gxii), the Only Prophylactic Hereditary Angioedema (HAE) Treatment Targeting Factor XIIa with Once-Monthly Dosing for All Patients From the Start

CLICK HERE to read the full press release

Are you familiar with the term “Virtual Patients”?

As the field of CGT (cell and gene therapy) rapidly evolves, understanding this concept will be increasingly important. The article we showcase today explains in extraordinary depth the role of virtual patients and will deepen your insight into their impact on drug development.

Virtual patients are computer-generated models designed to imitate the clinical traits of real individuals. They provide a new way to develop drugs, allowing researchers to simulate clinical trials without recruiting actual participants. This approach is especially significant for rare diseases, where assembling large trial groups is often unfeasible.

Pharmaceutical development is often hindered by lengthy timelines, frequent failures, and rising demands for strong evidence from regulators. One solution the article promotes is the use of virtual patient cohorts—digital representations of people generated using techniques like digital twins, statistical models, or random parameter settings. These virtual groups can stand in for real participants throughout every stage of drug development, from preclinical research and rare disease studies to traditional clinical trials.

What are the benefits? Biosimulation (there’s another term for you to embrace) may lower development costs and improve odds of success. Virtual patient models can better capture diversity and patient populations that traditional trials often exclude. And, by reducing dependency on large-scale human trials, simulations could streamline timelines and foster innovation.

What are the roadblocks? Outcomes depend heavily on model assumptions and quality of data inputs resulting in computational errors. Developing credible simulations requires significant computational power and expert knowledge. And there are currently no established protocols or guidelines for creating and validating virtual patient cohorts.

While promising, virtual patient techniques should complement, not replace, conventional laboratory and clinical methods. Blending them into a mixed-methods strategy may enhance efficiency, personalize treatments, and ultimately improve patient outcomes.

In summary, virtual patient simulations have the potential to transform drug development by lowering costs, capturing greater patient diversity, and accelerating innovation. However, addressing technical challenges and establishing clear standards will be crucial for their successful adoption.

---

Virtual Patients in Clinical Trials for Drug Development: A Narrative Review

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 thirteen (13) LD deals that have been publicly confirmed subsequent to their approvals.

Orsini Specialty Pharmacy to Distribute Sephience
Orsini has been chosen by PTC Therapeutics as a specialty pharmacy partner for Sephience (sepiapterin), an FDA-approved oral therapy indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU).

Orsini Specialty Pharmacy to Exclusively Distribute Dawnzera
Orsini Specialty Pharmacy announced that it has been named the exclusive specialty pharmacy partner for Ionis Pharmaceuticals’ Dawnzera , an FDA-approved prophylactic medicine to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older, self-administered via subcutaneous auto-injector.

Onco360 Specialty Pharmacy to Distribute Lynozyfic
Onco360 Specialty Pharmacy, has been selected as a national pharmacy partner by Regeneron Pharmaceuticals for Lynozyfic (linvoseltamab-gcpt), which has been has granted accelerated approval by the Food and Drug Administration to treat adult patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

Onco360 Specialty Pharmacy to Distribute Modeyso
Onco360 Specialty Pharmacy has been selected as a pharmacy partner by Jazz Pharmaceuticals for Modeyso (dordaviprone). It is indicated for the treatment of adult and pediatric patients one year of age and older with diffuse midline glioma harboring an H3 K27M-mutation with progressive disease following prior therapy.

Onco360 Specialty Pharmacy to Distribute Hernexeos
Onco360 Specialty Pharmacy has been selected as a pharmacy partner by Boehringer Ingelheim for Hernexeos (zongertinib) approved for the treatment of adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain (TDK) activating mutations, as detected by an FDA-approved test, and who have received prior systemic therapy.

Senderra Specialty Pharmacy to Distribute Ebglyss
Senderra Specialty Pharmacy has been selected as a national pharmacy partner by Lilly to distribute Ebglyss (lebrikizumab-lbkz), Lilly’s first-line biologic treatment for moderate-to-severe atopic dermatitis in adults and pediatric patients 12 years of age or older who weigh at least 40 kg, who are not well controlled with prescription topical therapies, or who cannot use topical therapies.

PANTHERx Rare Pharmacy to Distribute Brinsupri
PANTHERx Rare Pharmacy was selected by Insmed as part of the network of specialty pharmacies to dispense Brinsupri (brensocatib), a once daily oral medication to treat non-cystic fibrosis bronchiectasis (NCFB) in adults and pediatric patients 12 years and older.

Maxor Specialty Pharmacy to Distribute Brinsupri
Maxor Specialty Pharmacy, a VytlOne company, was selected by Insmed to be a limited distribution provider of Brinsupri. Brinsupri is a dipeptidyl peptidase 1 (DPP1) inhibitor indicated for the treatment of non-cystic fibrosis bronchiectasis (NCFB) in adult and pediatric patients 12 years of age and older.

Amber Specialty Pharmacy to Distribute Brinsupri
Amber Specialty Pharmacy was selected by Insmed to be a limited distribution provider of Brinsupri. Brinsupri is a dipeptidyl peptidase 1 (DPP1) inhibitor indicated for the treatment of non-cystic fibrosis bronchiectasis (NCFB) in adult and pediatric patients 12 years of age and older.

Option Care Health to Distribute and Administer dexamethasone sodium phosphate (eDSP)
Quince Therapeutics Inc., selected Option Care Health as lead specialty infusion therapy provider for suites across the U.S. This strategic relationship will support the commercial development and launch of encapsulated dexamethasone sodium phosphate (eDSP) in the U.S.,

Walgreens Specialty Pharmacy to Distribute
Yeztugo (lenacapavir), manufactured by Gilead, is the first and only FDA-approved twice-yearly option for HIV prevention as of August 2025.
Nemluvio (nemolizumab), manufactured by Galderma, is approved for the treatment of patients 12 years and older with moderate-to-severe atopic dermatitis in combination with topical corticosteroids and/or calcineurin inhibitors when the disease is not adequately controlled with topical prescription therapies. It is also approved to treat adults with prurigo nodularis.
Imkeldi (imatinib), manufactured by Shorla Oncology, is approved to treat certain forms of leukemia and other cancers.

Recent Reports have discussed the development of cell and gene therapy (CGT) and its potential to alter medical treatment approaches. Today’s article indicates that CGT is advancing internationally. For instance, the United States has approved 43 CGT products, including CAR T-cell and CRISPR-based therapies, and the United Kingdom is integrating these treatments into the NHS.

However, obstacles remain. In the US, fragmented healthcare coverage and delayed access persist, while budget limitations in the UK affect adoption rates. High therapy costs present challenges to health systems globally, especially in lower-income countries with limited infrastructure.

The article notes that expanding access may require funding innovations, regulatory cooperation, manufacturing coordination, public-private partnerships, and new technology adoption. Developments such as mRNA, artificial intelligence (AI), and scalable production methods are contributing to improved precision, speed, and accessibility of CGT. mRNA technology can support in vivo immune cell reprogramming, potentially reducing costs and complexity in treating solid tumors. AI tools facilitate target discovery, therapy design, and clinical trial efficiency. Regulatory refinement and collaboration among sectors may enhance the implementation of individualized care.

Advancements like in-vivo gene editing and mRNA-based immunotherapies aim to offer broader treatment of chronic diseases, improve results, and decrease long-term costs. To increase impact, priorities include scaling production, aligning regulatory requirements, and considering new payment structures.

In summary, CGT offers an individualized approach with the potential to improve outcomes and reduce ongoing care demands globally, particularly if manufacturing, cost, and access barriers are addressed, thereby extending therapies to underserved groups.

---

Global Cell and Gene Therapy: The Next Wave of Biotechnology Innovation

CLICK HERE to read the full article

The FDA recently approved a new therapy, Enflonsia (clesrovimab-cfor) from Merck & CO., indicated for the prevention of respiratory syncytial virus (RSV) lower respiratory tract disease in neonates and infants who are born during or entering their first RSV season. Enflonsia is a F protein-directed fusion inhibitor.

Enflonsia is currently the only RSV preventive approved at a fixed dose (105-mg) regardless of infant weight, which may simplify administration, and provides protection for 5 months—the length of a typical RSV season.

As noted in the article, “RSV disease is the leading cause of infant hospitalization in the U.S. and can lead to serious respiratory conditions like bronchiolitis and pneumonia.” Further, “Merck reported that Enflonsia reduced hospitalizations due to RSV-associated lower respiratory tract infections by more than 90%.”

RSV is responsible for between 58,000 and 80,000 hospitalizations of young children and between 60,000 and 160,000 hospitalizations of seniors in an average year, the bulk of which happen in the fall and winter. These are estimated to result in between 6,000 and 10,000 deaths among seniors and 100–300 deaths among children younger than 5 annually.

The wholesale price for Enflonsia 105mg has been set at $556 a dose, Merck said, which is the same price charged for its major competitor, Sanofi and AstraZeneca’s Beyfortus (nirsevimab).
The company did not release any details related to distribution.

CLICK HERE to access prescribing information

---

FDA Approves Clesrovimab to Prevent RSV in Infants During First Season

CLICK HERE to access the article

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.

---

Cracking the code to scalable cell and gene therapy

CLICK HERE to read the full article