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FDA Approves Immune Tx for Esophageal Cancer – Tevimbra

…………….catching up with FDA approvals

The FDA has approved a new infused therapy, Tevimbra (tislelizumab-jsgr) from BiGene, indicated for the treatment of adult patients with unresectable or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic chemotherapy that did not include a PD-(L)1 inhibitor.

Tevimbra belongs to the class of medicines known as checkpoint inhibitors and works by blocking the PD-1 pathway to help prevent cancer cells from being shielded from the immune system, increasing the immune system’s response. Tevimbra is highly specific for PD-1 and is a type of immunotherapy (immune checkpoint inhibitor) and a type of targeted therapy (monoclonal antibody).

Tevimbra faces well established competition in the esophageal cancer indication including Merck & Co.’s Keytruda and Bristol Myers Squibb’s Opdivo.

Tevimbra  is expected to launch in the 2nd half of 2024. 

Pricing for Tevimbra has yet to be confirmed but analysts suggest that it will launch with a discount of ~20% below Keytruda.

The company did not release distribution details.

CLICK HERE to access prescribing information


BeiGene finally snags FDA approval for PD-1 drug Tevimbra after long delay, Novartis breakup

  • BeiGenePD-1/L1FDA approvalsimmuno-oncology
  • eiGene would have enjoyed Big Pharma support for Tevimbra from Novartis, but the two firms parted ways in September. (Hopewell/BeiGene)
  • Twenty months after the FDA’s original target decision date on cancer drug tislelizumab, BeiGene has finally received the U.S. go-ahead.

CLICK HERE to read the full BiGene press release


FDA Approves Biosim to Actemra – Tyenne

……. catching up on FDA approvals

The FDA recently approved another tocilizumab biosimilar, Tyenne (tocilizumab-aazg) from Fresenius Kabi, indicated for Rheumatoid arthritis, Giant cell arteritis, Polyarticular juvenile idiopathic arthritis, and Systemic juvenile idopathic arthritis. Tyenne is a biosimilar to reference product Actemra (tocilizumab).

Being the second biosimilar to be approved is normally not much news. However, this approval is unique as Tyenne was approved for both IV and subcutaneous administration. Actemra and the preceding biosimilar, Tofidence (tocilizumab-bavi), are available only in IV form. 

All the tocilizumab products carry a Black Box Warning. 

Tyenne was not approved as an interchangeable biosimilar.

CLICK HERE to access prescribing information

The US average monthly cost of IV Actemra for rheumatoid arthritis, administered intravenously, is $2,134 – $4,268 depending on dosage. Pricing for IV and/or Sub-q Tyenne were not available.


FDA Green Lights Second Tocilizumab Biosimilar

CLICK HERE to access the press release


Specialty Pharmacy Patient Choice Award Winners 2023

Several weeks ago the winners of the 2023 annual specialty pharmacy Patient Choice Awards beauty pageant were announced. Sadly, the coverage of the event was limited to a small audience and seemingly never earned a mention in the media…. we track stuff like that closely. Oddly, none of the winners even published their own press release celebrating their good fortune…. yep, we track that kind of stuff too. The only source to pop up on our radar screen was from Managed Markets Insight & Technology (MMIT)….. which ran the pageant.

So as not to let the results go unnoticed here are the winners and the finalists in the four categories reviewed in the selection process —–

Hospital & Health System Specialty Pharmacy: 

Qualitas Specialty Pharmacy (winner)

Parkview Health Specialty Pharmacy

Vanderbilt Health

Independent Specialty Pharmacy: 

PANTHERx Rare Pharmacy (winner)

Parkway Specialty Pharmacy

Onco360 Oncology Pharmacy

Payer/PBM Specialty Pharmacy: 

AcariaHealth Specialty Pharmacy (winner)

CHI Health

CenterWell Specialty Pharmacy (formerly Humana Specialty Pharmacy)

Retail Specialty Pharmacy: 

Publix Specialty Pharmacy (winner)

Rite Aid

Sam’s Club

According to MMIT, the winners and finalists are calculated based on the highest average net promoter scores (NPS) from quarterly Zitter Insights Specialty Pharmacy Patient Satisfaction surveys throughout 2023. The Specialty Pharmacy Patient Satisfaction Survey was created to provide independent, objective benchmarking data across U.S. specialty pharmacies. Pharmacies rely on this data to evaluate their services, improve patient satisfaction, and drive scripts.

So, let’s shout out great big congratulations to all these specialty pharmacies as well as all those that work hard to provide superior service to their patients every day.


MMIT Announces Winners of the Eighth-Annual Specialty Pharmacy Patient Choice Awards

Four leading specialty pharmacies honored for their dedication to the highest quality customer service

CLICK HERE to access the press release


FDA OKs New Indications for Cell Therapy – Breyanzi

………. catching up on FDA approvals

The FDA has granted expanded indications for a therapy initially approved in 2021 adding to the rapidly growing toolkit of cell and gene therapies that are remapping the way patients with a broad range of diseases are being treated.

The FDA approved Breyanzi (lisocabtagene maraleucel; liso-cel) from Bristol Myers Squibb, a CD19-directed chimeric antigen receptor (CAR) T cell therapy, for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least two prior lines of therapy, including a Bruton tyrosine kinase (BTK) inhibitor and a B-cell lymphoma 2 (BCL-2) inhibitor. 

Breyanzi’s approval is based on response rate and duration of response confirmatory trial(s).  It carries a Black Box Warning that include  cytokine release syndrome, neurologic toxicities, and secondary hematological malignancies. Patients with CLL or SLL saw a complete response (CR) and an overall response rate (ORR) of 42.9% during trials. CLL and SLL are among the most common types of B-cell lymphoma.

Breyanzi is made from a patient’s own T cells, which are collected and genetically reengineered to become CAR T cells that are then delivered via infusion as a one-time treatment.

Breyanzi’s list price is $410,300 and has faced stiff competition from two competitors, Yescarta and Kymriah.

Is this term in your pharmaceutical vocabulary?…… CAR-T

CAR-T stands for chimeric antigen receptor T cell (CAR-T) therapy in which T-cells are extracted from a patient’s white blood cells and genetically modified in special facilities to target a specific protein that binds to cancer cells. The modified cells are then infused in the patient.


U.S. FDA Approves Bristol Myers Squibb’s Breyanzi as the First and Only CAR T Cell Therapy for Adults with Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL)

CLICK HERE to access the press release


FDA Approves Biosim for Two Rare Diseases – Bkem

Last week the FDA approved yet another biosimilar…… oh wait…. it was a biosimilar with an indication for not one, but TWO rare diseases. That makes yet another announcement of a new biosimilar a lot more interesting. 

The FDA approved Bkemv (eculizumab-aeeb), from Amgen, as the first interchangeable biosimilar to Soliris (eculizumab) to treat certain rare diseases with the same indicates as the reference product—–

  • the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis; and 
  • the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.

Bkemv is an infused therapy.

Bkemv also carries a Black Box warning for serious meningococcal infections.

PNH is an ultra-rare blood disorder that is characterized by the destruction of red blood cells. PNH occurs when the complement system — a part of the body’s immune system — over-responds, leading the body to attack its own red blood cells. Atypical hemolytic uremic syndrome is a rare genetic disease in which tiny blood clots form in blood vessels and block blood flow. It can lead to kidney failure and heart disease.

Bkemv is a monoclonal antibody that binds to the complement C5 protein and inhibits activation of the complement system, a part of the body’s immune system. This binding prevents the breakdown of red blood cells in the bloodstream (intravascular hemolysis) in patients with PNH and aHUS.

Under a 2020 settlement between the companies, Amgen will be free to launch its version March 1, 2025.

Amgen is not expected to release the price of Bekmv till launch time in 2025. By comparison, Soliris generally cost about $678,000 per year for the average patient. It will be interesting to see the competitive price.

Amgen did not disclose plans for distribution at this time.

CLICK HERE to access prescribing information


FDA Approves First Interchangeable Biosimilar for Two Rare Diseases

May 28, 2024

CLICK HERE to access the press release


FDA Approves Oral Solution for Rare Liver Condition – Livmarli

…………catching up on FDA approvals

The FDA recently approved a new oral solution, Livmarli (maralixibat) from Mirum Pharmaceuticals, Inc, indicated for the treatment of cholestatic pruritus in patients 3 months of age and older with Alagille syndrome (ALGS) and also for the treatment of cholestatic pruritus in patients 5 years of age and older with progressive familial intrahepatic cholestasis (PFIC).  Livmarli is an ileal bile acid transporter (IBAT) inhibitor. It was granted Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC.

Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in those affected. Signs and symptoms of PFIC typically include severe itching, jaundice, failure to thrive, and liver failure. The disease is estimated to affect fewer than 4,000 individuals in the US.

Published pricing for Livmarli oral solution (9.5 mg/mL) is about $56,240 for a supply of 30 milliliters.

Mirum did not announce plans for distribution. Given the small patient base, high cost and oral weight based dosing it is likely to be available through exclusive distribution.

CLICK HERE to access prescribing information


Mirum Pharmaceuticals’ Livmarli Receives FDA Approval for Treatment of Cholestatic Pruritus in Patients with Progressive Familial Intrahepatic Cholestasis

CLICK HERE to access the press release


CGT Driving New Programs at WAGS

Our last few Reports focused on approvals and developments in the Cell and Gene Therapy (CGT) categories. A recent press release from a major national pharmacy chain would seem to reinforce the notion that CGT is the newest best thing since sliced bread.

Walgreens is going through a major remake that the company says is reflective of where the market is and, more importantly, where it is going. They thought it was going towards walk-in medical care which is now in ‘rethink’ mode which is a Report for another day.   But the strategists at WAGS now see CGT as a better bet….. one that is more aligned with their legacy pharmacy model and where the rare/orphan/ultra-orphan market appears to be heating up fast….. and where you need to be a serious player to maintain its ‘creds’ in the marketplace.

According to the press release, “Effective August 1, 2024, AllianceRx Walgreens Pharmacy will become Walgreens Specialty Pharmacy.” While this is a clean-up on its fractionated branding, WAGS is also planning on investing in CGT in a tangible way by opening a “Gene and Cell Services Pharmacy and Innovation Center – a dedicated 18,000-square-foot center in Pittsburgh, PA, with services and capabilities for these emerging therapies, including innovative solutions for managing the complexity of the supply chain, logistics and financing as well as clinical and social needs management to ensure success for patients and partners.” 


Walgreens Launches Gene and Cell Services as Part of Newly Integrated Walgreens Specialty Pharmacy Business

Walgreens is expanding its specialty pharmacy services and investing in its capabilities as the company further grows its core pharmacy business to improve patient outcomes and provide greater value to payers and partners.

CLICK HERE to read the full press release


CGT…… What’s in Your Future?

Today we are pleased to offer a review of an article from which just about anyone working in the specialty pharmacy segment might learn something…. or even a lot.

The topic is cell and gene therapy (CGT)….. which is getting increasingly greater attention given recent approvals and a jaw dropping pipeline.

Can you define cell therapy vs. gene therapy? 

Even now, many would struggle to clearly differentiate between them.

Here’s what the article says……

  • Gene therapy is defined as the repair, replacement, or deactivation of dysfunctional genes to reestablish normal function. 
  • Cell therapy is defined as the transfer of autologous (from oneself) or allogeneic (donor) cellular material into a patient for medical purposes.
  • To devlop a CGT product a target gene or specific type of cell is identified to determine whether that gene or cell can be modulated directly within a patient’s own body (known as in vivo treatment), or if genetic or cellular material must be extracted from the body, modified, grown externally, and then transferred back into the patient (known as ex vivo treatment).
  • Over the past decade+ the FDA has approved more than 30 cell, cord blood, and gene therapy products (see list in the article).
  • Most investigational CGT products will continue to target oncologic conditions and rare diseases.
  • More than 2,000 CGT products globally are in various stages of clinical development. and…. as we have all surely noticed by now,
  • CGT therapies are among the most expensive medications in the world…….5 of the 10 most expensive drugs of 2023 were gene or cell therapies….. keeping health insurance companies awake at night worrying how to pay for them.

A reading of the full article will fill in much more detail of the current and, most importantly, give insight into the CGT ‘cures’ that one day are likely to reshape the legacy SP market. 


Cell and Gene Therapies: A Review of Current and Future Treatments

CLICK HERE to read the full article


FDA Approves Another Co$tly Gene Tx – Lenmeldy

The rate of FDA approvals of gene therapies is picking up….. at a pace not too dissimilar to the bevy of biosimilars we’ve witnessed in recent years. The newest gene therapy is Lenmeldy (atidarsagene autotemcel) from Orchard Therapeutics. Lenmeldy is the first FDA-approved gene therapy that is indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). 

MLD is caused by a mutation on the ARSA gene, resulting in a deficiency of the enzyme arylsulfatase A (ARSA). This deficiency causes sulfatides to build up in cells and damage the central and peripheral nervous systems. As a result, patients experience a loss of motor and cognitive function and early death. MLD, which has no cure, is estimated to affect one in 40,000 individuals in the United States.

Lenmeldy is a one-time, individualized single-dose infusion made from the patient’s own hematopoietic (blood) stem cells (HSCs), which have been genetically modified to include functional copies of the ARSA gene. The stem cells are collected from the patient and modified by adding a functional copy of the ARSA gene. The modified stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow.   The appropriate dose is calculated based on the child’s weight at time of Lenmeldy infusion using the instructions provided by the manufacturer. The processed dose is shipped to the treatment center in liquid nitrogen at less than -130°C (-202°F).

Lenmeldy now holds the austere position of being the most expensive therapy sold in the US. Orchard set the wholesale acquisition cost for Lenmeldy at $4.25 million.

CLICK HERE for prescribing information


Gene Therapy for MLD Gets FDA Approval

CLICK HERE to read the full article


FDA Approves Yet Another Humira Biosim – Simlandi

……. continuing to catch up on FDA approvals

The FDA recently approved a biosimilar, Simlandi (adalimumab-ryvk) from Alvotech and Teva Pharmaceuticals, as a biosimilar to Humira (adalimumab). Since there are already nine….. yep count ‘em….. other adalimumab biosims already approved, is there anything noteworthy to say about Simlandi…. YES!

The noteworthy thing about this approval is that Simlandi was approved with the coveted interchangeable designation. So, the ability for pharmacist substitution without physician approval in many states gives Simlandi a tangible marketing edge. However, the FDA had already approved Pfizer’s Abrilada (adalimumab-afzb) and Boehringer Ingelheim’s Cyltezo (adalimumab-adbm). The more the merrier?

But wait!…. Simlandi is high-concentration as well as citrate-free giving it a slight marketing edge over its interchangeable competitors….. as well as the rest of the pack.


FDA Approves Simlandi, Third Interchangeable Humira Biosimilar

CLICK HERE to read the full press release

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