Author: Randy Chase

Continuing Biosimilar Week

Recently the FDA approved a new biosimilar, Tyruko (natalizumab-sztn) from Sandoz Inc., with indications for adults with relapsing forms of multiple sclerosis, including clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease, and adults with moderately to severely active Crohn’s disease with evidence of inflammation who have had an inadequate response to, or are unable to tolerate, conventional Crohn’s therapies and tumor necrosis factor (TNF) inhibitors.

Tyruko is the first biosimilar to reference product Tysabri.

What was noteworthy about this approval is that it introduced a totally new disease to the relatively short list of supported diseases targeted by other biosimilars. Nearly half of all biosimilars approved to date were for only three drugs….. Humira, Avastin and Neulasta!

The pipeline for biosimilars is where the market now has a laser focus. The amount of investment in developing new biosimilars is beyond significant….. and big returns on investment are expected.  However, the first obstacle to jump start biosimilar uptake (we addressed that in our last REPORT). But, that is changing with approvals including FDA ‘interchangeable’ designations and increasing payor receptivity.

The last remaining challenge is pricing. Tyruko came to market at about a 20% discount to Tysabri which is ‘expected’ and isn’t considered a ‘Blue Light Special’ deal. Much has been written about the pricing strategies that take place behind the scene in which brand manufacturers cut deals with PBMS and payors to offer rebates that negate the benefit of the lower wholesale prices of the biosimilar. None the less, biosimilars are elbowing their ways onto preferred payor formularies.

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FDA Approves First Biosimilar to Treat Multiple Sclerosis

The U.S. Food and Drug Administration today approved Tyruko (natalizumab-sztn), the first biosimilar to Tysabri (natalizumab) injection for the treatment of adults with relapsing forms of multiple sclerosis (MS). Tyruko, like Tysabri, is also indicated for inducing and maintaining clinical response and remission in adult patients with moderately to severely active Crohn’s Disease (CD) with evidence of inflammation who have had an inadequate response to, or are unable to tolerate, conventional CD therapies and inhibitors of TNF-α (tumor necrosis factor, a substance in your body that causes inflammation).

“Biosimilar medications offer additional effective treatment options that have the potential to increase access for people living with relapsing forms of multiple sclerosis,” said Paul R. Lee, M.D., Ph.D., director of the Division of Neurology 2 in the FDA’s Center for Drug Evaluation and Research. “Today’s approval could have a meaningful impact for patients managing their disease.” 

Tyruko is approved to treat the following relapsing forms of MS:……………….  article continues

CLICK HERE to read the full FDA press release

We are going to hear a lot more about biosimilars in coming days. The leader board now shows that 44 biosimilars have been approved to date…. even including a handful of interchangeable products. After initial slow uptake, biosimilar demand is really starting to percolate as these products elbow their ways onto payor formularies.

One of the biggest drags on biosimilar adoption has been confidence. 

Patients… and even providers…. have been reticent to try a biosimilar….. especially when the reference product could be obtained for the same out-of-pocket cost through a payor.   The trend towards increasing coinsurance (generally based on wholesale price) is a factor that is contributing towards patients and providers looking kindlier at biosimilars.  

But, is out-of-pocket cost enough to move the needle…. pun intended? NO

What is really needed is proof of concept. For many new products confidence can be determined in quick order…. even minutes. Medicines, however, need long timelines to prove their mettle…. even years. Well, the years are passing and, finally, biostatisticians are now cobbling data together to show long term biosimilar efficacy. 

The article below is one of many that we should be seeing as post marketing outcomes data for biosimilars are published. The article below details the efficacy of several Oncology biosimilars…. arguably the most challenging category in which to shine. Strong data will go a long way to bolster that confidence factor we mentioned above.

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Oncological Biosimilars Shown to Be as Safe as Their Reference Products

Study finds patients with cancer feel reassured that biosimilars provide a safe, adequate, and cost-effective alternative to the pricier reference drug.

Biosimilars are commonly used in numerous clinical fields, including oncology. These drugs are different from the generic form of chemical products because it is impossible to develop molecules identical to their reference products due to natural variation and their heterogenic product process. Biosimilars are comparable to the reference product regarding structure, biological activity, efficacy, safety, and immunogenicity; however, there is some skepticism surrounding the safety and efficacy of these products among physicians and patients.

In a study published in Cancers, researchers analyzed post-marketing pharmacovigilance data of biosimilar monoclonal antibodies used in oncology compared with their respective reference products. The study authors evaluated the distribution of medicine-reaction pairs related to biosimilars of 3 antitumor biological products—bevacizumab, rituximab, and trastuzumab—as well as their respective corresponding biosimilar drugs—Avastin, MabThera, and Herceptin. ….. article continues

CLICK HERE to read the full article

Ok….. here’s something innovative in the wacky world of specialty pharmacy!

CVS has launched a new subsidiary called Cordavis. The new entity “will work directly with manufacturers to commercialize and/or co-produce biosimilar products”.  The most noteworthy thing in that line is the word ‘co-produce’. They don’t really explain exactly the extent of CVS’ involvement in co-producing….. but that is where the innovation really pops up. 

The focus of the initiative will target biosimilars. 

The article cites that “…. Cordavis to [will] launch biosimilar Hyrimoz with Sandoz beginning in 2024 at more than 80% lower list price than Humira.” This ‘out of the gate’ move is extra noteworthy.

We’ve seen a huge spike in activity around biosimilars in recent months….. and we expect it to continue with new biosims being approved for more and more reference products beyond the many that have already been approved for the early brand targets.

Our Reports over the next two weeks will be dedicated to even more noteworthy biosimilar developments. 

Yes, we’ve used the word ‘noteworthy’ now 4 times in this Report…. which itself says something noteworthy…. Ok, now its 5 times.

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CVS Health Launches Cordavis

New business will bring high quality biosimilar products to market with goal of reducing drug spend and ensuring access to affordable medications

CLICK HERE to read the full article

The article below was published some weeks ago, but its insight is very timely. It is a simple story….. one in which the Pharma industry is able to pull of a ‘switcherew’ that’s perfectly legal…… and one that could save them billions of dollars.

Got your interest?

It all comes down to reformulating, where possible, big $$ infused therapies and making them available in a subcutaneous injectable form. The article references Keytruda for one.

Why’s that important?

As you likely know, the Inflation Reduction Act (IRA) allows, for the first time, the government Medicare health program to negotiate prices for some of its most costly drugs, starting at a minimum discount of 25% to a drug’s price. BUT….. and here’s the big but….. they government is targeting INFUSED therapies. They released the first list of infused drugs only weeks ago.

What we’ve noticed is a spike in recent months in the number of frequently INFUSED drugs that have introduced the same therapy in subcutaneous form….. with some allowing self-administration.

The explanation from Pharma touts the reduced burden on patients who no longer need to visit their doctor or a clinic for a sometimes-lengthy infusion. And, the preference for self-administration is easily understandable. Patients ‘may benefit, where as infusion clinics may not be happy at all.  Heck…. somebody always gets the short straw!

Our guess is that we will see many more Infused therapies move towards offering a sub-q form for select infused products…… especially if their $$ volume is high which could win them a spot on a future price negotiation list.

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Focus: Drugmakers go under the skin, skirting early US Medicare price negotiations

By Michael Erman

NEW YORK, July 28 (Reuters) – Injectable versions of some widely-used cancer drugs including Johnson & Johnson’s blockbuster multiple myeloma treatment Darzalex are likely to be excluded from new U.S. government price negotiations for years, drugmakers told Reuters, protecting billions in revenue.

Whether the government agrees that adding an ingredient enabling infused drugs to be given by injection will allow them to be considered new medicines and significantly delay eligibility for price negotiations is being closely watched by Wall Street and the drugmakers.

J&J, Merck & Co (MRK.N), which is testing an injectable version of its top-selling immunotherapy Keytruda, and Halozyme Therapeutics (HALO.O), which …….  article continues

CLICK HERE to read the full article

Catching up on recent FDA approvals.

The FDA recently approved a new oral therapy, Sohonos (palovarotene) from Ipsen Biopharmaceuticals, for reduction in the volume of new heterotopic ossification (extra-skeletal bone formation) in adults and children aged 8 years and older for females, and 10 years and older for males with fibrodysplasia ossificans progressiva.  Sohonos is the first drug approved for patients with this condition.

Fibrodysplasia ossificans progressiva is a rare, autosomal dominant disease where connective tissue such as muscle, tendons and ligaments gradually turn into bone tissue, causing limited movement, deformities and severe disability. Sohonos is a retinoic acid receptor gamma agonist that curbs progression of the genetic disease by reducing the volume of new, abnormal bone growth.

US approval followed four years in application queue, two clinical trial holds, a complete response letter, a European Commission rejection, a scrapped trial in another indication and multiple safety flags, resubmissions, and other delays.

Ipsen confirmed that Sohonos will launch at a cost of $624,000 annually for a 5-mg daily dose for an ‘average’ patient….. but dosing will also depend on disease severity and patient weight.

Ipsen did not disclose distribution plans for the launch. Given its ‘rare’ designation, cost, and management challenges, it is expected that distribution will be through limited distribution.

Catching up on recent approvals……..

The FDA recently approved a new oral therapy, Akeega (niraparib and abiraterone acetate) from Janssen Pharmaceutical, the first-and-only dual action tablet combining a PARP inhibitor with abiraterone acetate, given with prednisone, for the treatment of adult patients with deleterious or suspected deleterious BRCA-positive metastatic castration-resistant prostate cancer (mCRPC), as detected by an FDA-approved test.

As noted in the press release prostate cancer is one of the most common cancers in the U.S., with an estimated 288,300 new cases and nearly 35,000 deaths expected in 2023. Upwards of 15 percent of patients with mCRPC have BRCA gene alterations. Patients with BRCA-positive mCRPC are more likely to have aggressive disease and may experience poor outcomes and a shorter survival time.

The recommended starting dose is 200 mg niraparib/1,000 mg abiraterone acetate (two tablets). The 100 mg niraparib/1,000 mg abiraterone acetate dose option (two tablets) is available for dose reduction.

Janssen did not confirm the cost of Akeega at the time of approval, however, its cost is listed at $9,600 with coupon through GoodRx.

Janssen did not announce distribution details at the time of approval but at least one specialty pharmacy, Onco360, has since confirmed that they are at least one provider selected to be a limited distribution partner for Akeega.

CLICK HERE for full prescribing details

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U.S. FDA Approves Akeega (Niraparib and Abiraterone Acetate), the First-And-Only Dual Action Tablet for the Treatment of Patients with BRCA-Positive Metastatic Castration-Resistant Prostate Cancer

Approval is based on the Phase 3 MAGNITUDE study, a prospectively designed precision medicine study including the largest population of BRCA-positive patients in combination trials to date with metastatic castration-resistant prostate cancer (mCRPC)

Akeega plus prednisone significantly improved radiographic progression-free survival compared to abiraterone acetate plus prednisone (AAP) in patients with BRCA-positive mCRPC

HORSHAM, Pa., – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced that the U.S. Food & Drug Administration (FDA) has approved Akeega (niraparib and abiraterone acetate), the first-and-only dual action tablet combining a PARP inhibitor with abiraterone acetate, given with prednisone, for the treatment of adult patients with deleterious or suspected deleterious BRCA-positive mCRPC, as detected by an FDA-approved test.

CLICK HERE to read the full Janssen 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 seven LD deals that have been publicly confirmed subsequent to their approvals.

Biologics to Dispense Vanflyta

Biologics by McKesson was selected by Daiichi Sankyo, Inc., as a specialty pharmacy provider for Vanflyta (quizartinib).  Vanflyta is indicated for use in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3-ITD positive as detected by an FDA-approved test. 

Orsini Specialty Exclusive Distributor for Veopoz

Orsini Specialty Pharmacy announced that Regeneron Pharmaceuticals, Inc. has chosen Orsini to be the exclusive specialty pharmacy partner for Veopoz (pozelimab-bbfg). A monoclonal antibody, Veopoz is the first and only treatment for those living with CHAPLE disease, an ultra-rare and life-threatening hereditary disease.

Orsini Specialty to Dispense Pombiliti / Opfolda

Orsini Specialty Pharmacy has been selected by Amicus Therapeutics to dispense Pombiliti and Opfolda, a two-component treatment approved for certain patients with late-onset Pompe disease. Pombiliti is a hydrolytic lysosomal glycogen-specific enzyme indicated, in combination with Opfolda, an enzyme stabilizer, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency).

Nufactor Exclusive Specialty Pharmacy for Ycanth

Nufactor, Inc., a subsidiary of FFF Enterprises Inc., announced that it has partnered with Verrica Pharmaceuticals to be the exclusive specialty pharmacy to dispense Ycanth, the first FDA approved treatment of molluscum contagiosum in adult and pediatric patients 2 years of age and older.

Onco360 to Dispense Ojjaara

Onco360 has been selected as a pharmacy partner by GlaxoSmithKline for Ojjaara (momelotinib) a kinase inhibitor indicated for the treatment of intermediate or high-risk myelofibrosis (MF), including primary MF or secondary MF [post-polycythemia vera (PV) and post-essential thrombocythemia (ET)], in adults with anemia.

Onco360 to Dispense Akeega

Onco360 has been selected as a pharmacy partner by The Janssen Pharmaceutical Companies of Johnson & Johnson for Akeega (niraparib and abiraterone acetate). Akeega is indicated with prednisone for the treatment of adult patients with deleterious or suspected deleterious BRCA-mutated (BRCAm) metastatic castration-resistant prostate cancer (mCRPC).

CarePartners Pharmacy named exclusive distributor of Yuflyma

CarePartners Pharmacy has been named the exclusive distributor for Yuflyma, a biosimilar to Humira. Yuflyma was approved by the FDA in May, 2023. by Celltrion Healthcare, Celltrion’s distribution unit, said Monday it has signed a deal with US specialty pharmacy chain CarePartners Pharmacy to sell its Humira biosimilar Yuflyma in the US market. Celltrion inked an exclusive contract with CarePartners.

It’s ALIVE!!!  Artificial Intelligence is here.

The article below details how HCSC, which covers about 18 million members in Illinois, Montana, New Mexico, Oklahoma and Texas, has introduced the power of AI to fast-track their very hectic prior auth volume. The company received about 1.5 million prior authorization requests in 2022.

What is most impressive is that they claim prior auths can be processed 1,400 times faster than using the traditional method.  WOW! They say that AI technology is now used for 93% of HCSC members for a limited number of procedure codes while they ramp up AI to handle even more procedure codes. 

HCSC also developed  an algorithm that mines historical prior authorization approvals to make it easier to approve treatment within seconds. If the request for a prior auth meets all criteria it is approved with no human intervention. The requests that fall through the sifter then go into a traditional prior authorization review.

Will AI completely eliminate manual review?

— Unlikely, at least in the foreseeable future.

Specific procedure codes and requests for certain (many?) specialty medications are likely to still be subject to lots of review involving extra documentation such as lab tests, genetic testing, site of service, etc. etc. It is conceivable that even these elements could be automated….. but given the difficulty in linking all these disparate criteria into one platform will be a big challenge in the short term.

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How HCSC is using AI to speed up prior authorization

Health Care Service Corporation says using augmented intelligence allows it to process prior authorization requests about 1,400 times faster than before. 

Jul 17, 2023  – Of the many tools that payers use to control costs, prior authorization ranks high on the list of what providers and patients find particularly vexing. However, Health Care Service Corporation hopes to fine-tune the process using the power of artificial intelligence. —–article continues—–

CLICK HERE to access the complete article

Catching up on a slew of FDA approvals in recent weeks….. whew!

The FDA  recently approved a new therapy, Veopoz (pozelimab-bbfg) from Regeneron, for the treatment of adult and pediatric patients 1 year of age and older with Chaple disease, also known as CD55-deficient protein-losing enteropathy. Veopoz is the first and only treatment indicated specifically for Chaple. Administration is by intravenous or subcutaneous injection.

Chaple is an ultra-rare and life-threatening hereditary immune disease driven by an overactivation of the complement system. In healthy individuals, the complement system is a mechanism for destroying microbes. However, those living with Chaple are unable to regulate complement activity due to mutations in their CD55 gene. Without proper CD55 regulation, the complement system may attack normal cells, causing damage to blood and lymph vessels along the upper digestive tract and leading to the loss of circulating proteins. 

There are fewer than 10 patients with Chaple disease diagnosed in the U.S.

Regeneron confirmed that the cost of Veopoz will be $34,600 per vial. 

Veopoz will launch through specialty distribution. 

CLICK HERE to access prescribing information

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VEOPOZ (POZELIMAB-BBFG) RECEIVES FDA APPROVAL AS THE FIRST TREATMENT FOR CHILDREN AND ADULTS WITH CHAPLE DISEASE

CHAPLE is an ultra-rare hereditary disease that can cause potentially life-threatening gastrointestinal and cardiovascular symptoms

Approval represents 10th FDA-approved medicine invented by Regeneron

With the approval of Veopoz, the pre-approval inspection issues related to the aflibercept 8 mg biologics license application (BLA) have been addressed

FDA action on the aflibercept 8 mg BLA is expected in the next few weeks

TARRYTOWN, N.Y., Aug. 18, 2023  — Regeneron Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA) has approved Veopoz (pozelimab-bbfg) for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE disease, also known as CD55-deficient protein-losing enteropathy. Veopoz is the first and only treatment indicated specifically for CHAPLE. With the approval of Veopoz, the pre-approval inspection issues related to the aflibercept 8 mg BLA have been addressed. FDA action on the aflibercept 8 mg BLA is expected in the next few weeks.

……….article continues…………..

CLICK HERE to access the full press release

Catching up on recent approvals…..

Earlier this week we sent a Report on the approval of Talvey from Janssen Pharmaceuticals indicated for he treatment of adult patients with relapsed or refractory multiple myeloma. Within days the FDA issued approval for another subcutaneous therapy, Elrexfio (elranatamab-bcmm) from Pfizer, Inc. Talvey was a first-in-class bispecific antibody.

Like Talvey, Elrexfio is a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager, for adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

Similarly, approval of Elrexfio included a Black Box Warning for cytokine release syndrome (CRS) and neurologic toxicity including immune effector cell-associated neurotoxicity syndrome (ICANS). The assigned REMS program includes monitoring requirement for oral toxicity, weight loss, infections, cytopenias, skin toxicity, hepatoxicity and embryo-fetal toxicity. Seeing a trend here?

Approval requires that patients be hospitalized for 48 hours after all doses within the step-up dosing schedule. Another coincidence to be sure.

Pfizer did not disclose details for distribution or logistics at the time of approval. 

The company did confirm that Elrexfio will launch at a cost of $41,500 per month (about $330,000 annually). This compares with Talvey’s launch price of $45,000 per month.

Given its cost, complex dosing schedule and multiple risks, Elrexfio will likely be handled through specialty pharmacy distribution.

CLICK HERE for full prescribing information