Earlier this week we sent a Report outlining why the term ‘BioBETTER’ should be added to your pharma vocabulary. Today we introduce a new biosimilar that is considered a BioBETTER. If you missed that report a BioBETTER is a product that is a biosimilar that has been altered in a way to improve effectiveness, ease of use, etc. A poor example might be the addition of an additive like STP to your gas tank to improve engine performance.

The FDA recently approved a new biosimilar, Zymfentra (infliximab-dyyb) from Celltrion, Inc.  Zymfentra is a subcutaneous (SC) formulation of Inflectra, a biosimilar to Remicade.  It is the world’s first and only subcutaneous infliximab product. 

Zymfentra is a novel tumor necrosis factor (TNF) blocker indicated in adults for maintenance treatment of moderately to severely active ulcerative colitis following treatment with an infliximab product administered intravenously and moderately to severely active Crohn’s disease following treatment with an infliximab product administered intravenously. The approval, however, included a Black Box Warning citing a long list of warnings and precautions. 

The label specifies that Zymfentra is for subcutaneous use only. Additionally, Zymfentra is indicated as maintenance treatment only, starting at Week 10 and thereafter for patients who must complete an intravenous induction regimen with an infliximab product before starting Zymfentra.

CLICK HERE to access Prescribing Information

We will strive to identify any new biosimilar approval that qualifies for the unique designation as a BioBETTER.

What is a Biobetter?

An article published by the Center for Biosimilars used a new word to the specialty pharmacy lexicon…… BioBetter….. prompting us to say “What’s that?”

Biobetter is not a common term in the United States but, perhaps, the term needs to be added to the US Pharma lexicon!

A good example is a recent FDA approval of sub-q  Zymfentra (infliximab-dyyb) a reference product to Inflectra which was approved as an infused therapy in 2016. We will cover this approval later this week.

So WHAT?

The WHAT is that the FDA determined Zymfentra to be a novel drug because it allows for subcutaneous (vs. IV) administration. It was awarded its own unique NDA.

The article from the Centers for Biosimilars explains why the term BIOBETTER should be in common use in the US…… it is now used routinely in Europe. The idea being that we are very likely to see a big uptick in new drug approvals of therapies resulting from intentionally altering an existing  biologic product to improve clinical outcomes. 

Examples of meaningful differences include….. allowing for more time between doses or enhanced tolerability compared with the reference product.  “Other examples of biobetters now include;

Susvimo (based on Lucentis [ranibizumab), Kadcyla (based on Herceptin [trastuzumab), Gazva (based on Rituxan [rituximab), and Neulasta Onpro (based on Neulasta [pegfilgrastim).”

So, are you ready to include Biobetters into your vocabulary?

Announcements for newly approved specialty drugs often state that the product will be available through specialty pharmacy in limited distribution. However, the 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.

Onco360 to Distribute Fabhalta 

Onco360 has been selected as a pharmacy partner by Novartis for Fabhalta (iptacopan) the first oral monotherapy approved for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). Fabhalta is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing control of red blood cell destruction within and outside the blood vessels.

Biologics to Distribute Fabhalta

Biologics has been selected by Novartis as a specialty pharmacy provider for Fabhalta (iptacopan). Fabhalta is the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH), a chronic and rare blood disorder that involves the destruction of red blood cells.

Walgreens added to Distribute Ycanth

Walgreens joins Verrica’s existing specialty pharmacy, Nufactor (originally announced as the ‘exclusive distributor’) to dispense Ycanth, the first FDA approved treatment of molluscum contagiosum in adult and pediatric patients 2 years of age and older.

Orsini Specialty Pharmacy exclusive Distributor for Wainua 

Orsini was selected as the exclusive specialty pharmacy for Wainua (eplontersen) for adults with hereditary transthyretin-mediated amyloid polyneuropathy. Wainua is a subcutaneous injection that can be self-administered.

PANTHERx Rare to Distribute Zilbrysq

PANTHERx Rare was selected by UCB to dispense Zilbrysq, a new FDA-approved therapy for the treatment of adults with gMG who are anti-AChR antibody positive.

There was a flood of media coverage following the FDA’s announcement that they have approved Florida’s request to import low-cost drugs from Canada. While that may bode well for many  patients, one needs to dig a bit deeper to understand whether this decision will be a golden egg for specialty pharmacies.

It is highly likely that other states will quickly jump on this band wagon….  especially since it reads well in political press releases. But, it does not look all that promising for SPs.

The FDA approval requires a ‘plan’ that includes a host of compliance requirements…. and a few exclusions. The exclusions are what tosses a wet blanket on the opportunity for SPs.

Here is where the blanket gets wet…..

Florida must implement its plan in compliance with the requirements of the Department of Health and Human Services’ Safe Importation Action Plan, which was finalized in 2020.

The plan specifies that the following categories are excluded:

• biological products
• drug with a REMS
• infused drugs / intravenously injected drugs
• controlled substances
• drugs inhaled during surgery, and
• certain parenteral drugs

What appears to be absent in the plan, however, are subcutaneously/IM/etc. administered, non-biologic drugs. Hmmmmm?

It will be interesting to see if this slice of opportunity is worth the effort for SPs to pursue participation.

CLICK HERE to read the FDA Safe Importation Action Plan

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FDA Authorizes Florida’s Drug Importation Program

CLICK HERE to read the FDA press release

………. Catching up on FDA approvals

The FDA approved a new therapy at the tail end of last year (it was November and, yes, we are trying to get caught up with the flood of approvals at the end of 2023)….. anyways…. the new therapy is Bimzelx (bimekizumab-bkzx) from UCB approved for the treatment of adults with moderate to severe plaque psoriasis. 

Bimzelx is the first and only approved psoriasis treatment designed to selectively inhibit two key cytokines driving inflammatory processes – interleukin 17A (IL-17A) and interleukin 17F (IL-17F).

Click Here for prescribing information

UCB is offering Bimzelxr as an autoinjector or pre-filled syringe at a list price of $7,200 per syringe. The recommended dosage is 320 mg (given as two subcutaneous injections of 160 mg each) at weeks 0, 4, 8, 12, and 16, then every 8 weeks thereafter. That translates into 10 cycles per year requiring 2 syringes per cycle or 20 syringes which prices out annually at $144,000.

Unlike almost every other specialty manufacturer, UCB has created an “Enhanced Network of Specialty Pharmacies” that provide additional product-specific patient support.  Even more noteworthy, UCB is making the therapy available through all other specialty pharmacies….. but without all the ‘extra’ bennies available through the ‘enhanced’ SPs. 

CLICK HERE for the ‘Enhanced’ list   

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BIMZELX Approved by the U.S. FDA for the Treatment of Adults with Moderate to Severe Plaque Psoriasis

Click Here to access the full press release

Happy New Year….. or to be said otherwise, What’s Happy About It?

The article below doesn’t trumpet the benefit of the changes implemented by Congress ‘fixing’ the vexing issue of direct and indirect reimbursement (DIR) fees that grew by 107,000% over the past decade. As specialty pharmacies know all too well, these fees have cut deeply into profit margins….. and there was a glimmer of hope that the new regulations would be the ‘fix’ they were hoping for. 

According to the article, PBMs are implementing flanking maneuvers that could be even more onerous than DIR fees.

For example—–

• Express Scripts has sent out contracts announcing that it would pay pharmacies approximately 10% below average wholesale price (AWP), “meaning they could lose money on every prescription they fill.”

• “Specialty pharmacies have been receiving new contracts with even more drastic cuts, as much as AWP minus 20%”

• “Specialty pharmacies have been particularly hard-hit, but I think pharmacies across the board of all types have seen their 2024 contract agreements tank in terms of the rates.”

• “PBMs can continue to claw back fees from pharmacies even with this new rule.”

As the adage goes, winning one battle does not mean that the war has been won.

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PBMs Implement Sweeping Reimbursement Cuts and Ramp Up Audits

CLICK HERE to access the full article

…..catching up on FDA Approvals

The FDA has approved a new subcutaneous therapy, Zilbrysq (zilucoplan) from UCB, for the treatment of adults with generalized myasthenia gravis.  Zilbrysq is the first once-daily subcutaneous, targeted C5 complement inhibitor for gMG. It is the only once-daily gMG-target therapy for self-administration.

gMG is a rare autoimmune disease with a global prevalence of 100–350 cases per every 1 million people. Symptoms of the disease include severe muscular weakness, drooping eyelids, difficulty with swallowing, chewing and talking, as well as life-threatening weakness of the respiratory muscles.

Zilbrysq was approved with a Black Box Warning for serious meningococcal infections.

CLICK HERE to access prescribing information 

UCB has not yet confirmed pricing for Zilbrysq.

Given that Zilbrysq is a self-administered, sub-q therapy with a Black Box Warning, it is expected that it will be channeled through specialty pharmacy limited distribution.

CLICK HERE to access the full UCB press release

Specialty pharmacies produce a lot of reporting…… but, the scope of this reporting may be missing the mark in the evolving competitive marketplace. The real challenge in this new environment will be all about outcomes. The challenge will be not in analyzing and reporting outcomes but, rather, what interventions were used to improve outcomes. Therein lies the rub.

Two articles were recently published that drilled down into the deeper layer of outcomes reporting by forcing health care providers to demonstrate how they are proactively changing outcomes through targeted interventions. Note that we say ’health care providers’, not just the pharmacy. The way to proactively impact outcomes will require participation by multidisciplined, non-siloed teams. Hospital systems are using this mantra to promote their differentiated specialty pharmacy model. 

One article lays out a roadmap to consider to navigate these new waters starting with a three-step process

• Environmental scan (developing a list of all potential measures, include quality measures / guidelines for each disease state).
• Expert engagement (rely on subject matter experts to identify the most important measures.)
• Implementation (include front line and informatics expert members in a dedicated team.)

Develop a strategy to produce better outcomes documentation include;

• Determine the value story
• Identify the outcomes that demonstrate value (not just numbers). 
• Define outcome goals
• Create targeted data elements to document outcomes
• Collect the data to report meaningful outcomes
• Report outcomes to key stakeholders 

The kind of outcomes reporting suggested will be hard…… no shortcuts here.

However, it may be the key differentiator that the market will start to demand.

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CLICK each title to access the full article

Start Tracking Outcomes in Specialty Pharmacy Now

Using Data-Driven Monitoring Tools Boosts SP Outcomes

Another milestone has been passed in the advancement of specialty pharmacy therapies. The FDA has approved not one, but two, gene therapies for a major disease, Sickle Cell Anemia. Most noteworthy is that one of the approvals offers real proof of concept for CRISPR gene-editing technology.

FIRST….. The FDA has approved a unique therapy, Casgevy (exagamglogene autotemcel) from Vertex Pharmaceuticals and CRISPR Therapeutics, indicated for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). Casgevy is an autologous genome edited hematopoietic stem cell-based gene therapy. 

Sickle cell affects an estimated 100,000 people in the United States. The disease can impact any race, however, the black community is the most affected with 90+% of all diagnosed cases. In sickle cell disease, the body makes flawed, sickle-shaped hemoglobin, impairing the ability of red blood cells to properly carry oxygen to the body’s tissues.

The process for Casgevy therapy requires significant preparation. Stem cells harvested from the patient’s bone marrow are sent to manufacturing facilities where they are edited using CRISPR/Cas9 technology. Once the cells are incubated, they are infused back into the patient during a month-long hospital stay.

Second….. The FDA also approved another therapy, Lyfgenia (lovotibeglogene autotemcel) from bluebird bio. This gene therapy is specifically designed to treat the underlying cause of sickle cell disease by adding a functional gene. Lyfgenia is also an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events.

The key difference is that Casgevy introduces a manufacturer, gene-edited therapy vs. Lyfgenia which is specifically designed to treat the underlying cause of sickle cell disease by adding a functional gene that enables production of adult hemoglobin that does not form into the crescent shape associated with the disease.

Refer to Prescribing Information for each therapy for more detail on these complex processes especially around dosage and administration.

CLICK HERE to access Casgevy label 

CLICK HERE to access Lyfgenia label 

Neither company disclosed launch pricing at time of approval.

Neither company disclosed distribution plans at this time.

CLICK HERE to read the full press release

Announcements for newly approved specialty drugs often state that the product will be available through specialty pharmacy in limited distribution. However, the 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.

Orsini Specialty Pharmacy to Distribute Adzynma 

Orsini Specialty Pharmacy has been selected by Takeda Pharmaceuticals as the exclusive specialty pharmacy partner for ADZYNMA (ADAMTS13, recombinant-krhn) for prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP). ADZYNMA is the first and only FDA-approved recombinant ADAMTS13 (rADAMTS13) enzyme replacement therapy for people 

Onco360 to Distribute Fruzaqla

Onco360 has been selected as a pharmacy partner by Takeda for Fruzaqla (fruquintinib an oral targeted therapy for certain adults with metastatic colorectal cancer (mCRC).  Fruzaqla is the first and only selective inhibitor of all three VEGF receptor kinases approved in the U.S. for previously treated mCRC regardless of biomarker status. Fruzaqla is a selective oral inhibitor of VEGFR-1, VEGFR-2, and VEGFR-3, which play a pivotal role in blocking tumor angiogenesis.

Biologics by McKesson to Distribute Truqap

Biologics by McKesson announced that it has been selected by AstraZeneca as a specialty pharmacy provider for Truqap (capivasertib), an orally administered drug, in combination with fulvestrant, for the treatment of adult patients with hormone receptor-positive, human epidermal growth factor receptor 2-negative locally advanced or metastatic breast cancer with one or more biomarker alterations (PIK3CA, AKT1, or PTEN).

Walgreens’ (WBA) AllianceRx Pharmacy to  Distribute XDEMVY

Walgreens Boots Alliance’s WBA wholly-owned subsidiary — AllianceRx Walgreens Pharmacy — will distribute Tarsus Pharmaceuticals’  XDEMVY (lotilaner ophthalmic solution) 0.25%. AllianceRx Walgreens is one of the four specialty pharmacies selected to distribute the FDA-approved treatment for Demodex blepharitis (DB), a highly prevalent eyelid disease.

Biologics by McKesson to Distribute Ogsievo

Biologics by McKesson has been selected by SpringWorks Therapeutics as a limited distribution specialty pharmacy for Ogsiveo (nirogacestat). Ogsiveo is a gamma-secretase inhibitor indicated for adult patients with progressing desmoid tumors who require systemic treatment.