Today’s Report spotlights a relatively insignificant event in therapy distribution. We are talking about COSTCO, yes the warehouse retailer. What they did is simply add a new drug to their pharmacy inventory, a little therapy called Steqeyma. In doing so, however, it reinforced a major shift in the specialty pharmacy industry.

Steqeyma (ustekinumab-stba), a biosimilar to Stelara (ustekinumab), has been added to the Costco Member Prescription Program. Still not impressed? Read on….

Adding a biosmilar to a prescription drug discount card program is not hugely surprising.

What is surprising is that COSTCO has been discreetly adding other specialty pharmacy therapies for several years. In fact, they are affirming that there are now “COSTCO Specialty Pharmacies.”

To be fair, a host of other retailers have been playing in the specialty pharmacy sand box for years. That COSTCO appears to be fully committed to be a player says a lot about the SP market and the shifting competitive landscape. 

The company said that the formulary including the specialty products is currently available for self-funded employer plans and Costco members who are uninsured and want to pay cash for their Steqeyma prescription or who have been denied coverage by their insurers.

FYI…. Steqeyma is available in both subcutaneous injection and intravenous infusion and is indicated for the treatment of plaque psoriasis (PsO) and psoriatic arthritis (PsA) in adult and pediatric patients, as well as Crohn’s disease (CD) and ulcerative colitis (UC) in adult patients.

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Celltrion’s Steqeyma (ustekinumab-stba), now added to the Costco Member Prescription Program

CLICK HERE to read the full article

The FDA recently approved one… no wait, two… no wait, is it really one times two denosumab biosimilars…..?? The/these approval/s include Ospomyv (denosumab-dssb) and Xbryk (denosumab-dssb)… it/they are from Samsung Bioepis.

NO WAIT… the FDA approved yet another pair of denosumab biosimilars, Stoboclo (denosumab-bmwo) and Osenvelt (denosumab-bmwo) from Celltrion.

And if that’s not enough, Wyost and Jubbonti were approved as Prolia and Xgeva biosims in 2024.

All of these biosims were approved as interchangeable with the reference products, Prolia and Xgeva from Amgen.

You should be familiar with the big differences between these ‘twin’ injectables. But, one has a twice yearly administration schedule… the other has a much more frequent dosing schedule. While both products are included in the same FDA approved prescribing information document you need to scroll way down (e.g., page 96) to read the details for the ‘twin’s’ therapy detail and the dosing information for completely different indications. 

CLICK HERE to access prescribing information for Ospomyv and Xbryk

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FDA, EMA Approve Second Pair of Denosumab Biosimilars

CLICK HERE to read the full article 

The FDA recently approved a new ORAL therapy, Romvimza (vimseltinib) from Deciphera Pharmaceuticals, LLC, indicated for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity.

Tenosynovial giant cell tumor (PVNS) or giant cell tumor of the tendon sheath (GCT-TS), is primarily treated through surgical resection. Treatment with TGCT typically involves direct removal of the tumor nodule. Treatment for diffused-type TGCT (DTGCT) with larger tumors affecting major joints, includes total synovectomy, joint replacement, or, in extremely rare cases, or amputation.

The highest prevalence of TGCT accounts for approximately 45% of total cases. Giant cell tumors can occur in the knee, ankle, hip, and other areas. The article cites that diffuse TGCT was most frequently observed in the knee, with an estimated 30,000 cases in the US in 2023.

Pricing details for Romvimza have not yet been announced.

Distribution details have not yet been announced by the company.

CLIC HERE to access prescribing information

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FDA approves vimseltinib for symptomatic tenosynovial giant cell tumor

CLICK HERE to read the full press release

How do you validate value? 

How do you demonstrate the ability to ‘walk the talk’?

The National Association of Specialty Pharmacy (NASP) has taken its years of specialty pharmacy knowledge and suggests it has a model that answers those questions. The validation points include improved patient outcomes, reduced costs, better medication adherence, cutting waste and increased patient safety.

If you think all this sounds familiar, it should. It has been the mantra for the SP industry for decades. What is different is the degree of proactive intervention that should push the standard of care past the performance ‘red line’.

NASP is still at work adding detail to the framework to facilitate implementation. For example, the framework will include a variety of new metrics such as affordability, supply chain impact, and tracking not only the number of interventions as well as the type such as patient, physician, care team, financial, etc., etc. to paint a very broad picture and understanding of each patient’s journey.

The current standard of care is already expensive. And, the expanded vision suggested by the framework would logically be even more costly. Perhaps AI may provide the secret sauce need to make the framework a reality.

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NASP’s 5-Step Procedure Tracks Patient Intervention Effectiveness

CLICK HERE to read the full article

Following what seems to be a new trend, the FDA has approved yet another 20+ year old therapy based on a major remodel… this one was originally approved in 2004. 

The therapy in question is Onapgo (apomorphine hydrochloride) injection from Supernus Pharmaceuticals, now available for subcutaneous use indicated for the treatment of motor fluctuations in adults with advanced Parkinson’s disease. 

Onapgo is the first and only subcutaneous apomorphine infusion device for the treatment of motor fluctuations in adults with advanced Parkinson’s disease.  Onapgo is a wearable subcutaneous infusion device that provides continuous treatment during the waking day for more consistent control of ‘OFF’ time. Onapgo is the second under-the-skin infusion-based therapy approved by the FDA this year for Parkinson’s, the first being Vyalev which delivers a continuous infusion of levodopa and carbidopa. Onapgo is a dopamine agonist that acts like dopamine, the brain chemical that affects movement and decreases in people with Parkinson’s. By dosing continuously sub-q, Onapgo bypasses the gastrointestinal (GI) tract allowing improved dosing direct to the brain.

Supernus expects to launch Onapgo in Q2-2025. As noted in an article, one analyst expects pricing to be in line with Vyalev and launch with a WAC of ~$119,000 for a year of therapy. 

The company did not release any details related to distribution.

CLICK HERE to access prescribing information

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Second Under-the-Skin Infusion for Parkinson’s Earns FDA Approval

CLICK HERE to access the full article

The FDA recently approved a new therapy originally approved in 2006, Susvimo (ranibizumab) from Genentech, an ocular implant providing customized therapy for diabetic macular edema (DME). This therapy has been granted a new indication and approved an updated delivery system.

Susvimo is a refillable eye implant surgically inserted into the eye during a one-time, outpatient procedure. Susvimo continuously delivers a customized formulation of ranibizumab over time. 

Susvimo is the first and only FDA-approved treatment shown to maintain vision for patients with DME. Susvimo is a vascular endothelial growth factor (VEGF) inhibitor designed to bind to and inhibit VEGF-A, a protein that has been shown to play a critical role in the formation of new blood vessels and the leakiness of the vessels.

DME is the leading cause of vision loss for patients with diabetes affecting 12% of type 1 and 28% of type 2 diabetic patients. The lifetime risk of developing DME for people with diabetes is about 10%.

Genentech originally confirmed pricing for the implant and Susvimo at $16,950 the first year, and two refills every six months in year two are priced at $16,000.

Genentech did not announce details for distribution.

CLICK HERE to access prescribing information

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FDA approves Roche’s Susvimo as the first and only continuous delivery treatment for the leading cause of diabetes-related blindness

• Susvimo is the first and only continuous delivery treatment that offers an alternative to regular eye injections to treat diabetic macular edema (DME)
• With as few as two treatments per year, Susvimo may help people with DME maintain their vision
• Approval marks the second indication for Susvimo in addition to neovascular or ‘wet’ age-related macular degeneration (nAMD)

CLICK HERE to read the full press release

The FDA recently approved another biosimilar, Avtozma (tocilizumab-anoh) from Celltrion, as a third biosimilar to Actemra. As with the reference brand, Avtozma is indicated for the treatment of multiple diseases including rheumatoid arthritis (RA), giant cell arteritis (GCA), polyarticular juvenile idiopathic arthritis (pJIA), systemic juvenile idiopathic arthritis (sJIA) and coronavirus disease (COVID-19).

Avtozma is available in both intravenous (IV) and subcutaneous (SC) formulations. Approval for Avtozma specifies infused administration for RA. The other approved indications are eligible for SC administration.

Avtozma was also approved with a Black Box Warning similar to the reference product.

Dosing of Avtozma is weight based and varies widely by disease indication and dosing schedule. Dosing strength varies widely, from as little as 12mg (juvenile) to 162mg (recommended adult dosing).

Genentech announced that the average monthly cost of Actemra administered via IV runs between $2,308 to $4,616, depending on dose a person receives. The average monthly cost of Actemra administered subcutaneously ranges from about $2,478 to $4,956 based indication and frequency.

CLICK HERE to access prescribing information

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U.S. FDA approves Celltrion’s AVTOZMA® (tocilizumab-anoh), a biosimilar to ACTEMRA

CLICK HERE to access the article

The FDA recently approved a very old drug, Spravato (esketamine) from Janssen Pharmaceuticals, for a very new indication. Esketamine was originally approved by the FDA in 1970, a mere 35 years ago!

Spravato is indicated for the treatment of:

• Treatment-resistant depression (TRD) in adults, as monotherapy or in conjunction with an oral antidepressant.

• Depressive symptoms in adults with major depressive disorder (MDD) with acute suicidal ideation or behavior in conjunction with an oral antidepressant.

Spravato (56mg or 84mg) is administered intranasally and only under the supervision of a healthcare provider. It was approved with a Black Box Warning and an associated REMS program. Dosing is twice weekly in weeks 1-4, once weekly in weeks 5-8, and once every two weeks in weeks 9 and after.  

The cost of Spravato ranges between $700 and $1200 per dose. 

Year 1 cost will range between $23,800 (56mg) and $40,800 (84mg).

Janssen also announced specialty pharmacy distribution options as follows;

“The specialty pharmacy pathway is the process of acquiring Spravato through a REMS-certified specialty pharmacy. This pathway allows treatment centers to treat patients who are covered under the pharmacy benefit. The pharmacy pathway does not require your treatment center to purchase Spravato but does require shipment coordination before each treatment session. Using the specialty pharmacy pathway, your treatment center receives patient-assigned product from a REMS-certified pharmacy and bills only for associated healthcare services.”

The following SPs are REMS certified and were included because of their geographic coverage, health plan coverage, and fulfillment capabilities.

• AllianceRX — (Walgreens/Prime)
• CVS Specialty — (CVS Caremark)
• Humana Specialty — (Humana)
• AcariaHealth —  (Centene)

CLICK HERE to access the J&J brochure detailing specialty pharmacy access

CLICK HERE for prescribing information

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

Onco360 to Distribute Vyloy 

Onco360 has been selected as a national pharmacy partner by Astellas for Vyloy (zolbetuximab-clzb), approved in combination with fluoropyrimidine- and platinum-containing chemotherapy for the first-line treatment of adult patients with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction adenocarcinoma whose tumors are claudin (CLDN) positive, as determined by an FDA-approved test.

Onco360 to Distribute Gomekli

Onco360 has been selected as a national pharmacy partner by SpringWorks Therapeutics, Inc. for Gomekli (mirdametinib) for the treatment of adult and pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) not amenable to complete resection.1

Orsini to Distribute Attruby

Orsini has been selected by BridgeBio to distribute Attruby (acoramidis), an orally administered treatment for cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM). ATTRUBY is a near-complete (≥90%) stabilizer of transthyretin for the treatment of adults with ATTR-CM, to reduce cardiovascular death and cardiovascular-related hospitalization. 

Orsini to Distribute Kebilidi

Orsini announced that it has been chosen by PTC Therapeutics as the exclusive specialty pharmacy provider for the adeno-associated virus vector-based gene therapy Kebilidi (eladocagene exuparvovec-tneq). Kebilidi is approved for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency.

Orsini to Distribute Yorvipath

Orsini is partnering with Ascendis Pharma for the distribution of Yorvipath, a FDA approved parathyroid hormone analog (PTH1-34) indicated for the treatment of hypoparathyroidism in adults

PantherRx Rare to Distribute Attruby

PantherRx Rare has been selected by BridgeBio to distribute Attruby (acoramidis), an orally administered treatment for cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM). ATTRUBY is a near-complete (≥90%) stabilizer of transthyretin for the treatment of adults with ATTR-CM, to reduce cardiovascular death and cardiovascular-related hospitalization. 

Panther Rx Rare to Distribute Yorvipath

PantherRx Rare is partnering with Ascendis Pharma for the distribution of Yorvipath, a FDA approved parathyroid hormone analog (PTH1-34) indicated for the treatment of hypoparathyroidism in adults

Panther Rx Rare to Distribute Tryngolza

PantherRx Rare was selected by Ionis Pharmaceuticals, Inc. for the distribution of Tryngolza (olezarsen), the first and only treatment approved by the FDA as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).

Panther Rx Rare to Distribute Crenessity

PantherRx Rare was selected by Neurocrine Biosciences, Inc. for the distribution of Crenessity (crinecerfont), an oral selective corticotropin-releasing factor type 1 (CRF1) receptor antagonist indicated to improve androgen control and enable a reduced glucocorticoid dose in patients 4 years of age and older with classic congenital adrenal hyperplasia (CAH).

AnovoRx Specialty Pharmacy to Distribute Increlex

AnovoRx has been named the exclusive distributor of Increlex (mecasermin injection) from Eton Pharmaceuticals (following the acquisition of Increlex from Ipsen S.A. Increlex is a biologic product used to treat pediatric patients 2 years of age and older who suffer from the severe primary insulin-like growth factor 1 deficiency (SPIGFD) a rare condition.

How well do you understand cell based therapy?

It’s complicated!

Given the pace of research and the number of already approved cell-based products it is now high on the list of essential knowledge for leaders in the pharma industry and for specialty pharmacies that want to play in that sandbox.

MSC-based cell therapy involves the administration of MSCs as a medicinal product. These cells are characterized by ways in which they can be used as a therapeutic agent in human treatments.

But, the mechanisms of MSC therapy are poorly understood. According to the NIH, MSC have a short survival time after infusion, it is believed that MSC pass on their effects to other cell types. The scientific stuff now goes on in language that is likely foreign for those without a doctorate degree. SO, this Report will increase your knowledge level, but only enough to make you dangerous.  

Here are some bullet points that you may want to consider —–

The Importance of Regulation in Ensuring Safety and Impact on Treatment Cost for Mesenchymal Stem Cell Therapies

CLICK here to access the full article

Regulation is essential to ensure the safety and efficacy of mesenchymal stem cell (MSC) treatments. It upholds high standards in production, application, and monitoring of these therapies. Without it, the risk of suboptimal quality and potential serious adverse effects increases.

Key Aspects of MSC Regulation:

Establishing guidelines for MSC isolation, expansion, and characterization.

Ensuring consistency, viability, and contamination-free cells in therapies.

Developing clinical trial protocols to assess safety and effectiveness in various medical conditions.

Role of Regulatory Agencies:

Agencies like the FDA mandate rigorous testing for new treatments.

Approval is required before widespread use of MSC-based therapies.

Regulatory Challenges and Impact on Treatment Costs:

While regulation is crucial for MSC treatment safety, it also contributes to increased costs. The rigorous testing and validation, adherence to Good Manufacturing Practices (GMP), and the need for sterile, high-quality production facilities add significant expenses.

Factors Contributing to Increased Costs:

Time-consuming and expensive clinical trials with extensive participant involvement.

Maintenance of GMP compliant facilities for MSC production.

Impact on Treatment Availability:

Lengthy regulatory approval processes can delay treatment availability to patients.

Balancing Safety and Cost in MSC Therapies:

Despite the challenges, the regulatory framework is essential for maintaining the integrity of MSC therapies. The increased costs are an investment in the treatment’s long-term success, building trust among patients, healthcare providers, and stakeholders in regenerative medicine.

Benefits of a Strong Regulatory Framework:

Ensures patient safety and high-quality care.

Contributes to the long-term success and trustworthiness of MSC treatments.

Our next Report will be on yet another Cell Based therapy!