The FDA recently approved yet another gene therapy, Beqvez (fidanacogene elaparvovec-dzkt) from Pfizer, Inc., for the treatment of adults with moderate to severe hemophilia B who currently use factor IX (FIX) prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes, and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var as detected by an FDA-approved test. 

Beqvez is a one-time treatment that is designed to enable people living with hemophilia B to produce factor IX themselves.  The current standard of care requires regular intravenous infusions of factor IX typically administered multiple times a week or month.

A one-time dose of Beqvez has reduced bleeds post-treatment compared to standard of care with a median of zero bleeds (range 0 to 19) after up to three years of follow-up, providing sustained bleed protection and potentially avoiding years of treatment burden with prophylaxis for many patients.

Beqvez follows on the heels of the approval of another gene therapy for hemophilia B, Hemgenix, approved in 2023 . According to the World Federation of Hemophilia, more than 38,000 people worldwide are living with hemophilia B. By comparison, upwards of 20,000 males in the US have hemophilia-A.

Pfizer set the treatment’s list price at $3.5 million. That matches the cost of Hemgenix, the other available gene therapy for hemophilia B. Pfizer will offer insurers a warranty providing “financial protections” if Beqvez doesn’t work or its effects don’t last.

CLICK HERE to access prescribing information

Pfizer did not announce details for logistics and distribution.

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U.S. FDA Approves Pfizer’s BEQVEZ (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B

CLICK HERE to access the full article

We’ve written numerous time about outcomes-based contracting. The concept has been around for well over a decade….. yet the practice has experienced slow uptake…. for one reason…. It’s complicated. (Note— manufacturers have been paying for ‘enhanced services’ for many years for their own use, especially for marketing purposes.)

An article on the topic is timely as the number of such contracts continues to see growth in the marketplace. If it has survived this many years, it has proof of concept.

The article is a good…. and quick read…. that spotlights the challenges. The biggest challenge is determining what services are included in the outcomes program…. and then separating the core (basic) services from any ‘enhanced’ services. The enhanced services are add-ons to what the SP would routinely provide and, therefore, can be deemed as compensable ($$). 

But wait…. there’s more…. now comes the challenge of determining how much compensation is appropriate for each of the enhanced services. 

But wait…. the amount of compensation can’t be so great that it could be construed as a kickback. 

But wait, reporting is critical to document patient outcomes and the scope and frequency of outcomes reporting is also compensable and is the keystone in determining whether the target(s) specified in the contract have…. or have not…. been met. Compensation for reporting also needs to pass the ‘fair-market-value’ test. 

These, and many other nuances, make outcomes-based contracts intimidating for specialty pharmacies, manufacturers…. and even payers that will get refunds if outcomes fail to meet contract thresholds. This is where the lawyers really get involved. 

So, read the article below for a cogent recap of the process.

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The Dos and Don’ts of Enhanced Service Contracts

CLICK HERE to read the full article

…………………….catching up on FDA approvals

The FDA recently approved a new ORAL therapy, Vafseo (vadadustat) from Akebia Therapeutics, for the treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months. Vafseo is a once-daily oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor that activates the physiologic response to hypoxia to stimulate endogenous production of erythropoietin to manage anemia.

Approximately 500,000 adult patients in the U.S. on dialysis suffer from anemia due to CKD1 which contributes to developing an array of other serious conditions. CKD patients are typically treated for anemia with injectable erythropoiesis-stimulating agents during dialysis. The availability of an oral treatment option is meant to increase and better maintain hemoglobin concentrations within recommended guidelines.

Vafseo  was approved with a Black Box warning.

Akembia announced that it is expecting to launch Vafseo in January, 2025. The company also announced that Vafseo will be available for purchase through authorized specialty distributors once the Centers for Medicare & Medicaid Services grants a two-year Transitional Drug Add-on Payment Adjustment (TDAPA).

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Akebia Receives FDA Approval of Vafseo (vadadustat) Tablets for the Treatment of Anemia due to Chronic Kidney Disease in Adult Patients on Dialysis 

CLICK HERE to read the full press release

Cardinal Health recently published a very slick report on the State of Biosimilars. 

We’ve heard so much about biosimilars (even from this esteemed reporting source) that many say….. enough already! 

BUT, biosimilars are gaining traction in the marketplace at a logarithmically faster pace so, dear readers, it is time to give biosims the respect….. or at least the attention…. that they deserve. 

The Cardinal report covers a lot of ground. As one might expect, it details the overall market landscape. However, the report also begins to peel back the dark side of biosimilars with insight into how payers and PBMs – in league with manufacturers (typically the reference product manufacturer) – have leveraged their positions to create a web of pricing tactics that make one’s head spin. Formulary management is gasping for breath given the growing cornucopia of biosims (beyond just a handful of big market indications such as the bucket of Humira indications.)

SPs will need to step up their game in this 3-dimensional chess game being fostered in by biosimilars….. especially as relates to purchasing, inventory management, formulary compliance, and more. The fact that biosimilar are being approved with interchangeable designations only adds further complications.

So,….. we recommend that you download the Cardinal Health report and fill in significant information gaps….. as this dear author has also done.

CLICK HERE to download the Cardinal Health report

………. catching up on FDA approvals

The FDA recently approved a new subcutaneous therapy, Winrevair (sotatercept-csrk) from Merck & Co. Winrevair is an activin signaling inhibitor indicated for the treatment of adults with pulmonary arterial hypertension (PAH) a rare, progressive disease. The treatment seeks to increase exercise capacity, improve WHO functional class (FC) and reduce the risk of clinical worsening events.

Winrevair, a biologic, is the first FDA-approved activin signaling inhibitor therapy for PAH. It represents a new class of therapy that works by improving the balance between pro- and anti-proliferative signaling to regulate vascular cell proliferation underlying PAH. 

Winrevair is given once every three weeks by subcutaneous injection and “may be administered by appropriate patients or caregivers with guidance, training and follow-up from a healthcare provider.”

Winrevair will carry a list price of $14,000 per vial. According to data from the company’s trial, most patients will use a single vial every three weeks, which would translate to $238,000 per year.

Merck announced that the therapy will be distributed through specialty pharmacy but did not specify any particular providers(s).

CLICK HERE for prescribing information

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FDA Approves Merck’s WINREVAIR (sotatercept-csrk), a First-in-Class Treatment for Adults with Pulmonary Arterial Hypertension (PAH, WHO Group 1)

CLICK HERE to access the full Merck press release

…………….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 2^nd 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

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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

……. 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.

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FDA Green Lights Second Tocilizumab Biosimilar

CLICK HERE to access the press release

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.

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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

………. 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.

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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

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

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FDA Approves First Interchangeable Biosimilar for Two Rare Diseases

May 28, 2024

CLICK HERE to access the press release