…………………….. catching up on FDA approvals

The FDA recently approved a new therapy, Hympavzi (marstacimab-hncq) from Pfizer Inc., indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors.

This approval is yet another, cutting edge development in the hemophilia category in the past of year+. Hympavzi is the first of its kind and works by targeting a protein in the blood clotting process.

For decades, clotting factor products merely replaced clotting factor. Hympavzi works by reducing the amount of tissue factor pathway inhibitor, a naturally occurring anticoagulation protein. As such, it increases the amount of thrombin, an enzyme that is critical in blood clotting. Ultimately this is approach is expected to reduce the frequency of bleeding episodes.  

Hympavzi is a once-weekly subcutaneous injection and is supplied in pre-filled auto-injector pens.

CLICK HERE to access prescribing information

Pfizer set a wholesale acquisition cost (WAC) at $795,600 for Hympavzi. 

By comparison to other recent hemophilia breakthrough therapies, Pfizer charges $3.5 million for its Beqvez product and matches the price for Hemgenix , a hemophilia B gene therapy.

The company announced that Hympavzi would launch through Orsini Specialty Pharmacy limited distribution.

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FDA Approves New Treatment for Hemophilia A or B

Product is First Non-Factor and Once-Weekly Treatment for Hemophilia B

CLICK HERE to access the press release 

If you work in specialty pharmacy it is a safe bet that you are familiar with pharmacy accreditation. If you are familiar with accreditation you understand that accreditation is a comprehensive approach to measure specialty pharmacy performance against specific standards. And, if you are that familiar with accreditation, you almost assuredly are familiar with URAC, one of the leading independent accrediting organizations in the US.

URAC recently released a report that summarizes performance among its numerous participating specialty pharmacies with a five year lookback. The report focuses on key metrics that are valuable for specialty pharmacies to gauge their own performance against competing specialty pharmacies in the marketplace. Payers and even patients are increasingly attuned to such performance metrics when seeking to do business with an organization that has the health, safety, and financial fortunes in their hands.

Some of the leading metrics cited in the new report include;

• Quality monitoring enhancement
• Fostering industry standards
• Improvement in call center performance
• Dispensing accuracy enhancements
• Challenges in distribution accuracy
• Turnaround time trends
• ….. and more.

The report, therefore, should be mandatory reading for every specialty pharmacy.

CLICK HERE to access the URAC Five-Year Specialty Pharmacy Report: Making a Difference With Measurement

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URAC Releases Specialty Pharmacy Report

• Over the five-year period, URAC-accredited pharmacies responded to patient calls within 30 seconds 75% of the time, improving to 86% by 2023.

CLICK HERE to read the article

…………………………catching up on FDA approvals

The FDA recently approved an ORAL therapy, Itovebi (inavolisib) from Genentech, Inc. Itovebi is indicated in combination with palbociclib and fulvestrant for the treatment locally advanced or metastatic breast cancer in adults with endocrine-resistant, PIK3CA-mutated, hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, as detected by an FDA-approved test, following recurrence on or after completing adjuvant endocrine therapy.

Itovebi is a type of targeted therapy medicine called a PI3K inhibitor. Previously, Piqray (alpelisib) was the only PI3K inhibitor approved to treat breast cancer with a PIK3CA mutation.  Approximately 70% of all breast cancers are hormone-receptor positive, HER2-negative.  PIK3CA is the most commonly mutated gene in hormone-receptor positive, HER2-negative breast cancer, with approximately 40% of patients harboring this mutation. 

Genentech said that Itovebi will launch at a price of $22,867 for a 28-day cycle.

Distribution details were not announced. Although it comes with a relatively high cost, Good Rx shows current availability at a large number of retail pharmacies including CVS, Walgreens, Walmart, etc. 

CLICK HERE to access prescribing information

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FDA approves inavolisib with palbociclib and fulvestrant for endocrine-resistant, PIK3CA-mutated, HR-positive, HER2-negative, advanced breast cancer

CLICK HERE to read the full press release

Quiz me this……………

Would you prefer to have your medication administered intravenously or in pill form?

Did it take more than a nano second to answer that question?

Probably not.

The article below may have some good news for the vast number of patients that deal with the burdens of an infused course of therapy.  As the article below points out, infusions are inconvenient or even inaccessible to patients….. and, complications often arise not from the active drug, but the infusion itself. “Turning these drugs into pills could be transformative.”

But is it doable?  

Researchers say ‘YES’. They are actively working to solve a big hurdle….. ‘bioavailability’….. or how to make oral medications more readily dissolvable in the bloodstream with maximum absorption. (Ever try to mix a vinegrette dressing? Ya got to make the oil combine with the vinegar or water.) The article explains how new technology is being developed to achieve exactly that….. think routinely breaking the oil vs. water barrier.

Are you familiar with the term ‘sol-moiety’? Existing drugs are now being tested in the lab to achieve bioavailability using this technique….. and results have been spectacular with bioavailability increases from nearly zero to 100% being observed. Proof-of-concept is no longer a hurdle.

Read the full article for more details and perhaps a glimpse of the future.

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New strategy could turn intravenous medicines into pills

CLICK HERE to access the full article

Among the many earth-moving changes on the payer / PBM battlefield was last year’s announcement that Blue Shield of California was breaking away from its relationship with CVS’ Caremark and creating a very different model that would include multiple partners. According to the company, partners include Amazon Pharmacy, Abarca, Mark Cuban Cost Plus Drug Company, and Prime Therapeutics, with Caremark still hanging onto specialty pharmacies.

But wait….. BSofCA just announced another big change as part of its ‘Pharmacy Care Reimagined’ model that will further shake up the marketplace opening the door for other payers to emulate.

In short, BSofCA is dethroning Humira from what has been a sacrosanct position at the pinnacle of its specialty drug formulary. Instead, “Blue Shield is teaming up with drugmaker Fresenius Kabi and Evio Pharmacy Solutions to purchase a biosimilar for Humira at a transparent net price of $525 per monthly dose.” That price is significantly below the market reported net price of $2,100.

Moreover, Blue Shield confirmed that members will pay a $0 copay for the biosimilar. This makes the shift a big win financially for both the health plan….. and members. It is also believed that the no cost to member option will measurably boost persistence and improve overall health outcomes.

Details on how exactly Blue Shield will actually adjudicate and ‘pay’ for the meds were not announced. That, in itself, will be noteworthy. This should be fun to follow.

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Blue Shield of California inks deal to buy Humira biosimilar directly from manufacturer

CLICK HERE to read the article

……………………. catching up on FDA Approvals

The FDA recently approved a new infused therapy, Niktimvo (axatilimab-csfr) from Incyte Corporation and Syndax Pharmaceuticals, indicated for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. 

Graft-versus-host disease is a leading cause of death from bone marrow transplants, which are used to treat certain blood cancers, solid tumors and immune disorders. It is estimated that approximately a third of those who receive bone marrow transplants experience GVHD.

Niktimvo doesn’t work like its predecessors which include Incyte’s Jakafi and Sanofi’s Rezurock. Niktimvo is a colony stimulating factor-1 receptor (CSF-1R)-blocking antibody that targets and blocks a protein involved in the development and survival of certain kinds of white blood cells.

Analysts suggest that Niktimvo will launch with a list price of $21,500 / month.

The company did not announce details for distribution.

CLICK HERE to access prescribing information

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FDA approves axatilimab-csfr for chronic graft-versus-host disease

CLICK HERE to access the FDA press release

The FDA recently approved an ORAL therapy, Livdelzi (seladelpar) from Gilead Sciences, Inc., for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.

PBC is a rare, chronic, autoimmune disease of the bile ducts that affects approximately 130,000 Americans, primarily women, and is known to cause liver damage and, ultimately, liver failure if untreated. The disease currently has no cure.

Common symptoms include incessant itching or skin-crawling sensations, as well as debilitating fatigue that is made worse by the itching at night.

Gilead confirmed a list price of $12,606 for 30 tablets (10-mg) which works out to $151,272 per year.

Gilead also confirmed that oral Livdelzi will be distributed through specialty pharmacy.

CLICK HERE to access prescribing information

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Gilead’s Livdelzi (Seladelpar) Granted Accelerated Approval for Primary Biliary Cholangitis by U.S. FDA

First and Only Treatment to Demonstrate Statistically Significant Reductions Across Key Biomarkers, ALP Normalization and Pruritus Versus Placebo –

CLICK HERE to read the full Gilead press release

PBMs are increasingly under the gun to remedy a variety of practices that are not considered consumer and provider network friendly. It is not surprising, therefore, that a PBM might want to tout some of the things that they have done to move the collective patient journey in a positive direction. But, it is in the short list of things that they are promising in the future that we find of greater interest. 

A short video interview with a senior leader at one PBM, OptumRx, recaps some of their successes over recent years but also opens the window on some noteworthy strategies in development.

The pipeline includes a more aggressive approach to cost containment especially through value-based strategies that go beyond pharmacy, in short, a ‘whole of government approach’….. ummm, a ‘whole of medicine approach’. This means embracing a big tent model of all providers that support specialty patients with complex needs…. often with multiple diagnoses. The evolving model must also develop a true cross-benefits model — Medical and Pharmacy to eliminate existing disparities. There’s more……

If you have 10 minutes, the video interview will add detail and other morsels of concepts upon which to chew. (link below)

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Optum Rx’s Vision for the Future of Specialty Care

OptumRx Healthcare Costs Value-Based Care Patient experience

Rising costs, technological advancements, the growing complexity of patient needs – these are all factors transforming the specialty pharma landscape at present.

CLICK HERE to access the video

…………catching up on FDA approvals

The FDA has approved a new therapy, Imuldosa (ustekinumab-srlf) from Accord BioPharma Inc., as a biosimilar to Janssen Biotech’s Stelara. Imuldosa is available in a subcutaneous form as well as an infusible.  

The approval of Imuldosa marks the fifth ustekinumab approval for the US. 

Imuldosa is slated to launch in May 2025 according to sources.

Imuldosa is indicated for—– 

Adult patients with:

• moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy;
• active psoriatic arthritis;
• moderately to severely active Crohn’s disease; and
• moderately to severely active ulcerative colitis.
• Pediatric patients 6 years of age and older with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy; and active psoriatic arthritis.

It is noted that prescribing information specifies subcutaneous dosing and administration for Psoriasis and Psoriatic Arthritis while specifying intravenous dosing instructions for Crohn’s Disease and Ulcerative Colitis.

CLICK HERE for prescribing information

While Imuldosa did not receive a Black Box warning there are a number of warnings and precautions consistent with the other previously approved Stelara biosimilars.

Pricing and distribution plans were not announced.

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FDA Approves Imuldosa, a Biosimilar to Stelara

CLICK HERE to read the article

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.

DirectRx to Distribute Ohtuvayre

DirectRx is now part of the U.S. pharmacy network distributing Ohtuvayre (ensifentrine), a newly approved therapy for chronic obstructive pulmonary disease (COPD). The Verona Pharma therapy was cleared by the U.S. Food and Drug Administration as an inhaled maintenance therapy for adults with COPD.

Amber Specialty Pharmacy to Distribute Ohtuvayre

Amber Specialty Pharmacy has announced that it has been named by Galderma to distribute Nemluvio (nemolizumab-ilto) for the treatment of Prurigo Nodularis in adults. The condition is a chronic, debilitating skin condition.

Orsini Specialty Pharmacy Exclusive Distributor for Miplyffa

Orsini Specialty Pharmacy has been chosen as the exclusive provider for Miplyffa (arimoclomol), the First FDA-Approved Treatment for Niemann-Pick Disease Type C, an ultra-rare and progressive neurodegenerative disease. MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older.

Biologics by McKesson to Distribute Voranigo

Biologics by McKesson has been selected by Servier Pharmaceutical as a specialty pharmacy partner for Voranigo (vorasidenib). Voranigo is approved for the treatment of adults and pediatric patients 12 years of age and older with grade 2 astrocytoma or oligodendroglioma and a susceptible isocitrate dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation following surgery including biopsy, sub-total resection, or gross total resection.

ONCO360 to Distribute Voranigo

Onco360 has also been selected by Servier Pharmaceutical as a specialty pharmacy partner for Voranigo (vorasidenib).