………….. catching up on FDA Approvals

The FDA recently approved a biosimilar….. meh news…… huh?

But wait….. on the same day the FDA approved two biosimilars….. big news?

The more interesting news is that the approval was for two different biosimilars….. and….. both have the same indication!…. perhaps that’s the biggest news.

The approvals included….. Yesafili (aflibercept-jbvf) from Biocon Biologics and Opuviz (aflibercept-yszy) from Biogen, Samsung Bioepis with an indication for the treatment of patients with neovascular (wet) age-related macular degeneration (AMD); macular edema following retinal vein occlusion (RVO), diabetic macular edema, and diabetic retinopathy.

Both therapies  are the first interchangeable biosimilars to reference product Eylea (aflibercept) from Regeneron.  Yesafili and Opuviz are both vascular endothelial growth factor (VEGF) inhibitors and each is administered by intravitreal injection every 4 weeks for the first 5 injections, followed by 2 mg (0.05 mL of 40 mg/mL solution) once every 8 weeks.

Regeneron recorded U.S. sales of $5.72 billion from Eylea in 2023. Regeneron’s reference product, Eylea (intravitreal solution (40 mg/mL), is around $2,000. 

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FDA Approves First Biosimilars to Eylea

CLICK HERE to read the article

…….. catching up on FDA approvals

The FDA recently approved a new infused therapy, Imdelltra (tarlatamab-dlle) from Amgen, indicated for the treatment of adult patients with extensive stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy. Imdelltra is the first bispecific delta-like ligand 3 (DLL3)-directed CD3 Tcell.  

Amgen said that it has been decades of no real treatment advances for this type of lung cancer. With small cell lung cancer, the disease has typically spread beyond the lung by the time it is diagnosed and most patients live just eight to 13 months after their diagnosis, despite having chemotherapy and immunotherapy.

Imdelltra was approved with a Black Box Warning for cytokine release syndrome and neurologic toxicity including immune effector cell-associated neurotoxicity syndrome.

Amgen said the U.S. price for Imdelltra is $31,500 for the first cycle, and $30,000 for subsequent cycles. The company said trial patients were on the treatment for a median of over five months, which would equate to a commercial price of about $166,500.

CLICK HERE to access prescribing information

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FDA grants accelerated approval to tarlatamab-dlle for extensive stage small cell lung cancer

CLICK HERE to read the FDA press release

The FDA recently approved a trastuzumab biosimilar, Hercessi (trastuzumab-strf; HLX02) from Accord BioPharma, to reference product Herceptin, for the treatment of several forms of HER2-positive breast and gastric cancers. Hercessi is supplied in powder form for reconstitution prior to infusion.

Hercessi is the sixth trastuzumab biosimilar approval overall. 

Hercessi was granted interchangeable designation.

Prior biosimilars to trastuzumab are as follows—-

• Ogivri (trastuzumab-dkst)          December 2017
• Herzuma (trastuzumab-pkrb)    December 2018
• Ontruzant (trastuzumab-dttb)   January 2019
• Trazimera (trastuzumab-qyyp)   March 2019
• Kanjinti (trastuzumab-anns)       June 2019

HER2-positive, is an aggressive form of cancer in which cancer cells grow rapidly. However, HER2-positive breast cancer generally responds well to treatment.

Breast cancer is the most common cancer in women in the U.S., and HER2-positive breast cancer accounts for about 20 percent of all breast cancer diagnoses (in 2023, that’s 297,790 women and 2,800 men who will be diagnosed).

Pricing for Hercessi has not been confirmed. 

The company has not disclosed plans for distribution.

CLICK HERE to access prescribing information

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FDA Approves Hercessi, the Sixth Trastuzumab Biosimilar

CLICK HERE to read the full article

We just ran across an interesting article that spotlights four key takeaways from the Asembia Specialty Pharmacy Summit held earlier this year. 

Well, the article is from the perspective of four lawyers who attended the Summit. 

Hmmmmm!!!

Their perspectives and conclusions bear merit for all specialty pharmacies to factor into their strategic planning as they are perhaps better positioned to look at the forest as well as all them there trees.

Here’s a snapshot of their observations……

• Infusion Continues to Dominate Conversations on Market Activity — infusion will continue to impact the overall market especially influenced by site-of-service.
• IRA Implementation Continues to Raise Questions about Impact on the Specialty Pharmacy Market — at this point it remains unclear as to the impact on specialty pharmacies but it is clear that the Act will begin to shift the reimbursement biosphere.
• Outside of True “Specialty,” Cash is King — manufacturers are focusing on cash-pay programs and pharma service vendors are well poised to provide robust support for these programs. But, further attempts by manufacturers to establish closer relationships with patients and eliminate costly channel participants like wholesalers and commercial pharmacies will proliferate. 
• 340B Continues to Come Up, Particularly in Distribution and Infusion Conversations — drug manufacturer contract pharmacy restrictions will lead to new and “innovative” models popping up.

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Key Takeaways from 2024 Asembia Specialty Pharmacy Summit

CLICK HERE to read the full article and the authors’ conclusions.

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 five LD deals that have been publicly confirmed subsequent to their approvals.

Soleo Health to Distribute Ryplazim

Soleo Health has been named a limited distribution specialty pharmacy partner to dispense Ryplazim (plasminogen, human-tvmh), manufactured by Kedrion Biopharma. Ryplazim is the first and only FDA approved therapy for supporting the approximately 500 people in the U.S. affected by plasminogen deficiency type 1 (PLGD-1).

Orsini Specialty to Distribute Olpruva

Orsini Specialty Pharmacy (Orsini), and Zevra Therapeutics, Inc. is now the pharmacy partner for Olpruva (sodium phenylbutyrate) for oral suspension. Olpruva is used along with certain therapies, including changes in diet, for long-term management of certain adult and pediatric patients with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) or argininosuccinic acid synthetase (AS). 

Biologics by McKesson to Distribute Camcevi

Biologics by McKesson has been selected by Accord BioPharma, Inc. as an exclusive specialty pharmacy provider for Camcevi (leuprolide) 42mg injection emulsion for subcutaneous use. Camcevi is a gonadotropin-releasing hormone (GnRH) agonist indicated for the treatment of adult patients with advanced prostate cancer.

Camcevi was originally approved by the FDA on May 25, 2021.

Optime Care to Distribute Gavreto

Optime Care, an AscellaHealth Company, has been selected to distribute Gavreto by Rigel Pharmaceuticals, Inc. Gavreto, a targeted oral therapy, is used to treat adults with non-small cell lung cancer (NSCLC) that has spread and also adults and children 12 years of age and older with advanced thyroid cancer or thyroid cancer that has spread who require a medicine by mouth or injection (systemic therapy) and who have received radioactive iodine and it did not work or is no longer working caused by abnormal RET genes.

Onco360 to Distribute Rytelo

Onco360 has been selected as a pharmacy partner by Geron Corporation for Rytelo (imetelstat) a first in class oral, oligonucleotide telomerase inhibitor, approved for the treatment of adult patients with low to intermediate risk myelodysplastic syndromes (MDS) with transfusion dependent (TD) anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA).Onco360 Selected as a Limited Distribution Specialty Pharmacy Partner for Rytelo™ (imetelstat).

The FDA recently approved an ORAL Tx, Xolremdi (mavorixafor) from X4 Pharmaceuticals, indicated in patients 12 years of age and older with WHIM (warts,hypogammaglobulinemia, infections and myelokathexis) syndrome to increase the number of circulating mature neutrophils and lymphocytes. WHIM is the first therapy specifically indicated in patients with WHIM syndrome, a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 pathway dysfunction.

People with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia) and experience serious and/or frequent infections. X4 Pharmaceuticals cited that at least 1,000 people are currently diagnosed with WHIM syndrome in the U.S.

The company has set a wholesale acquisition cost for Xolremdi on an annual basis at $496,400 for patients greater than 50 kilograms and $372,300 for patients less than or equal to 50 kilograms.

In a rare event in itself, X4 announced that Xolremdi will be commercially available in the U.S. through specialty pharmacy partner PANTHERx Rare.

CLICK HERE to access prescribing information

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X4 Pharmaceuticals Announces FDA Approval of Xolremdi (mavorixafor) Capsules, First Drug Indicated in Patients with WHIM Syndrome

CLICK HERE to read the full X4 press release

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