Author: Randy Chase

How well do you understand cell based therapy?

It’s complicated!

Given the pace of research and the number of already approved cell-based products it is now high on the list of essential knowledge for leaders in the pharma industry and for specialty pharmacies that want to play in that sandbox.

MSC-based cell therapy involves the administration of MSCs as a medicinal product. These cells are characterized by ways in which they can be used as a therapeutic agent in human treatments.

But, the mechanisms of MSC therapy are poorly understood. According to the NIH, MSC have a short survival time after infusion, it is believed that MSC pass on their effects to other cell types. The scientific stuff now goes on in language that is likely foreign for those without a doctorate degree. SO, this Report will increase your knowledge level, but only enough to make you dangerous.  

Here are some bullet points that you may want to consider —–

The Importance of Regulation in Ensuring Safety and Impact on Treatment Cost for Mesenchymal Stem Cell Therapies

CLICK here to access the full article

Regulation is essential to ensure the safety and efficacy of mesenchymal stem cell (MSC) treatments. It upholds high standards in production, application, and monitoring of these therapies. Without it, the risk of suboptimal quality and potential serious adverse effects increases.

Key Aspects of MSC Regulation:

Establishing guidelines for MSC isolation, expansion, and characterization.

Ensuring consistency, viability, and contamination-free cells in therapies.

Developing clinical trial protocols to assess safety and effectiveness in various medical conditions.

Role of Regulatory Agencies:

Agencies like the FDA mandate rigorous testing for new treatments.

Approval is required before widespread use of MSC-based therapies.

Regulatory Challenges and Impact on Treatment Costs:

While regulation is crucial for MSC treatment safety, it also contributes to increased costs. The rigorous testing and validation, adherence to Good Manufacturing Practices (GMP), and the need for sterile, high-quality production facilities add significant expenses.

Factors Contributing to Increased Costs:

Time-consuming and expensive clinical trials with extensive participant involvement.

Maintenance of GMP compliant facilities for MSC production.

Impact on Treatment Availability:

Lengthy regulatory approval processes can delay treatment availability to patients.

Balancing Safety and Cost in MSC Therapies:

Despite the challenges, the regulatory framework is essential for maintaining the integrity of MSC therapies. The increased costs are an investment in the treatment’s long-term success, building trust among patients, healthcare providers, and stakeholders in regenerative medicine.

Benefits of a Strong Regulatory Framework:

Ensures patient safety and high-quality care.

Contributes to the long-term success and trustworthiness of MSC treatments.

Our next Report will be on yet another Cell Based therapy!

Quiz me this…..!

What new technology emerged in the 1970s that truly changed the world?…. Certainly, you immediately thought of ‘the computer’ in your top 5 answers.

So, what new technology is now emerging in the 2020s that will truly change the world of specialty pharmacy?….. tick… tick… tick… time’s up! The answer that many of you may have thought of is…. Cell and Gene Therapy.

Yep, C&G is gonna’ have a huge impact on pharmacy across the board. If you play it out to the extreme, there will be, one day, a cell or gene therapy (or some other magical potion or gizmo) that will cure every ache and ill out there. Bye bye pharmacy!

OK, that may be a pipe dream… but it might be best to start planning for the tidal wave as opposed to a mere changing tide.

The article below is on point with the changing tide in pharmacy. It is worth a read… both intellectually and practically from a business perspective.

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8 Trends to Watch in the Cell and Gene Therapy Market

Click Here to access the article

…………. catching up on recent FDA approvals

The FDA recently approved a new therapy, Crenessity (crinecerfont) from Neurocrine Biosciences, Inc., to be used together with glucocorticoids (steroids) to control androgen levels in adults and pediatric patients 4 years of age and older with classic congenital adrenal hyperplasia (CAH).

Classic congenital adrenal hyperplasia is a rare genetic condition affecting the adrenal glands, which produce hormones such as cortisol and androgens. Patients with classic CAH do not produce enough cortisol and produce too many androgens. These patients require high doses of glucocorticoids because the glucocorticoids also help to reduce the excess levels of androgens. Crenessity works by reducing excessive adrenal androgen production, which helps reduce the amount of glucocorticoid treatment needed.

Crenessity was granted an Orphan Drug designation. The estimated prevalence of CAH is 1/10,000. Annual incidence ranges from 1/5,000 to 1/15,000.

Dosing for adults on therapy are 2 capsules daily. Crenessity is also available as an oral solution for children who are dosed based on weight.

Neurocrine has set the US list price for Crenessity at approximately $38,333 for a 30-day supply for adults ($460,000 annually). For pediatric patients weighing less than 20 kilograms, the 30-day supply is priced at about $19,167 ($230,000annually).

Crenessity is available through limited distribution. PantherRx Rare Specialty Pharmacy has confirmed that they were selected as a limited distribution partner.

CLICK HERE to access prescribing information

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FDA Approves New Treatment for Congenital Adrenal Hyperplasia  

CLICK HERE to read the FDA press release

…………………. catching up on recent FDA approvals

The FDA recently approved a new infused therapy, Bizengri (zenocutuzumab-zbco) from Merus N.V., indicated for adults with pancreatic adenocarcinoma or non–small cell lung cancer (NSCLC) that are advanced unresectable or metastatic and harbor a neuregulin 1 (NRG1) gene fusion who have disease progression on or after prior systemic therapy.

Bizengri is a bispecific antibody that binds to the extracellular domains of HER2 and HER3 expressed on the surface of cells, including tumor cells, inhibiting HER2:HER3 dimerization and preventing NRG1 binding to HER3. The therapy is the first and only treatment approved for these indications. Approval for these indications may be contingent upon verification of clinical benefit in a confirmatory trial(s).

Bizengri was approved with a Black Box warning for Embryo-Fetal Toxicity.  

The company did not announce the price for Bizengri.

Similarly, the company did not announce plans for distribution.

CLICK HERE to access prescribing information

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Merus Announces FDA Approval of Bizengri (zenocutuzumab-zbco) for NRG1+ Pancreatic Adenocarcinoma and NRG1+ Non–Small Cell Lung Cancer (NSCLC) 

Click Here to read the full company press release

The FDA recently approved a biosimilar, Yesintek (Ustekinumab-kfce) from Biocon Biologics Ltd, as a biosimilar to the reference product, Stelara (Ustekinumab).  

This approval is the newest biosimilar approval for a Stelara competitor. 

The currently approved biosimilars include — Selarsdi (ustekinumab-aekn), Pyzchiva (ustekinumab-ttwe), Otulfi (ustekinumab-aauz), Imuldosa (ustekinumab-srlf), Wezlana (ustekinumab-auub), and Yesintek (ustekinumab-kfce).

Yesintek 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.

Prescribing information specifies subcutaneous dosing and administration for Psoriasis and Psoriatic Arthritis while specifying intravenous dosing instructions for Crohn’s Disease and Ulcerative Colitis.

CLICK HERE for prescribing information

The company plans its product launch in the United States later in February following a settlement with J&J. Distribution details were not announced.  

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FDA Approves Biocon Biologics Ustekinumab Biosimilar, Yesintek

Click Here to read the full article

……….. catching upon FDA approvals

The FDA recently approved a new ORAL specialty therapy, Revuforj (revumenib) from Syndax Pharmaceuticals, for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients one year and older.  

Revuforj is the first and only menin inhibitor approved for the condition.  

The therapy was granted Orphan Drug status.

The company priced Revuforj at $39,500 per month, or $474,000 annually.

Revuforj was approved with a Black Box warning for differentiation syndrome, a potentially fatal condition in which leukemia cells rapidly release cytokines. 

CLICK HERE to access prescribing information 

Syndax announced that Revuforj launched through a network of specialty distributors and specialty pharmacies and full availability all doses by Q2-2025. An oral solution will be available through an expanded access program to allow for dosing of patients who weigh less than 40 kg.

As of this date only Onco360 has confirmed its selection to be an authorized dispensing pharmacy for Revuforj.

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Syndax Announces FDA Approval of Revuforj (revumenib), the First and Only Menin Inhibitor to Treat Adult and Pediatric Patients with Relapsed or Refractory Acute Leukemia with a KMT2A Translocation

Click here to access the full company press release

………… catching up on FDA approvals

The FDA recently approved a new infused therapy, Aucatzyl (obecabtagene autoleucel) from Autolus Inc., indicated for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). Aucatzyl is a CD19-directed genetically modified autologous T cell immunotherapy.

Acute lymphoblastic leukemia is a cancer in which B-cell lymphoblasts are found in the bone marrow and blood. ALL accounts for less than 1% of all cancers in the United States. The American Cancer Society estimates that in 2024 there will be about 6,550 new cases of ALL and about 1,330 deaths. Survival rates remain very poor in adult patients with r/r ALL, with median overall survival of eight months.

Aucatzyl was approved with a Black Box warning, however, the FDA did not impose a REMS program which is uncommon.

The company confirmed that the annual wholesale acquisition cost for Aucatzyl is $525,000. It is priced in the same general price bracket as two competing products, Kymriah and Tecartus.  

The company did not announce details on its plans for distribution.

CLICK HERE to access prescribing information

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FDA approves obecabtagene autoleucel for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia  Aucatzyl

CLICK HERE to read the full FDA press release

The NHIA recently released a comprehensive list of therapies that have been routinely dispensed by home infusion providers. This comprehensive grouping, especially by therapeutic category and related prescribing practices, is both insightful and practical.   

Specialty pharmacies that distribute key therapies may benefit from a reliable marketplace point of reference. In a broader sense, the marketplace can benefit from both seeing the large (and growing) array of therapies that can be administered in a lower cost ‘site of service’ outside the hospital. This distinction also goes to the heart of the ‘site of service’ appropriateness debate currently hotly contested around oncology therapies.

CLICK HERE to access the NHIA Home Infusion Drug List

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NHIA Home Infusion Drug List

The National Home Infusion Association (NHIA) Home Infusion Drug List contains the names and therapeutic categories of drugs that were compiled from medication dispensing reports submitted by home infusion providers and reflects current prescribing practices in the United States. The National Home Infusion Association (NHIA) Home Infusion Drug List may serve as a reference for decisions related to the site of care for administering drugs requiring intravenous or subcutaneous infusion.

This reference only lists prescribed drugs and does not endorse home infusion for patients who do not meet the criteria for safe administration in the home or alternate site of care. NHIA members and other practitioners using the list should exercise independent judgment and provide the patient with a choice in their site of care decision based on their individual situation. Additionally, not all home infusion providers offer every medication included on the list. Providers may specialize and only offer certain therapies.

The NHIA Home Infusion Drug List was developed collaboratively by NHIA staff and member volunteers comprised of pharmacists, nurses, and other professionals with extensive experience and expertise in the field of home infusion. The NHIA Quality and Standards Committee reviewed the list by category and drugs included for accuracy. The category selected for each medication was based on the chemical form of the active ingredient, the clinical use for a particular diagnosis, and/or the corresponding per diem where applicable. The committee members made recommendations that were incorporated prior to finalizing and approving the list and communication materials.

The FDA recently approved a new infused therapy, Vyloy (zolbetuximab-clzb) – in combination with fluoropyrimidine and platinum-containing chemotherapy – from Astellas Pharma Inc., for the first-line treatment of adults with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors are claudin (CLDN) 18.2 positive as determined by an FDA-approved test.

Vyloy is the first and only CLDN18.2-targeted therapy approved in the U.S. After a starting dose of 800 mg/m2 Vyloy is infused 600 mg/m2 every 3 weeks or 400 mg/m2 every 2 weeks.

Vyloy has been priced at $1600 per 100mg vial.

Onco360 has been selected as a national pharmacy partner by Astellas for Vyloy.  

CLICK HERE to access prescribing information

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Astellas’ VYLOY (zolbetuximab-clzb) Approved by U.S. FDA for Treatment of Advanced Gastric and GEJ Cancer

VYLOY is the first and only CLDN18.2-targeted treatment approved in the U.S. for adults with advanced gastric and gastroesophageal junction cancer whose tumors are CLDN18.2 positive

CLICK HERE to read the full press release

So, how much do you know about bispecific therapy?

Can you even offer a passing definition?

Well, read on dear reader….. and learn enough to impress your colleagues!

Why is knowing about bispecific therapies even remotely important?

Although they have been around for more than a decade, bispecifics are back and making big news in disease management. Currently, there are over 100 bispecifics in clinical development. AstraZeneca, BioNTech and Summit Therapeutics are just a few pharma with pipeline products. KOLs believe that bispecifics are the next new thing in immunotherapy with promise to even surpass the pioneering performance of monoclonal antibody therapy Keytruda.

Here’s the definition that you need to know to sound well informed……

Bispecific antibodies are engineered to bind to cancer cells and healthy immune system cells (T-cells) at the same time, activating the healthy immune system cells to recognize and destroy cancerous cells. 

The article below does a good job of filling in more detail to assuage your insatiable curiosity.

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With Myriad Recent Approvals in Cancer, The Era of Bispecifics Is Here

 As companies roll out data showing the power and improved safety profile of antibodies that target two antigens, analysts say the class could overtake monoclonal antibody Keytruda as the “immunotherapy backbone” of solid tumor treatment.

CLICK HERE to read the full article