Author: Randy Chase

……….catching up on FDA approvals

We usually don’t report on expanded indications for a therapy previously approved by the FDA.

This time one such approval is noteworthy.

It is not uncommon that a new therapy comes to market and, within months, then applies for an expanded indication. There is a pattern wherein the initial NDC is often for a rare / orphan condition that is fast-tracked enabling supplemental filings to also be fast-tracked. The new approval of Kevzara is a clear exception to that rule.

Way back in 2017 the FDA approved Kevzara (sarilumab), from Sanofi, indicated for the treatment of adult patients with moderately to severely active rheumatoid arthritis. Clinical trials started post-approval to include juvenile arthritis. 

Like the Energizer Bunny….. Sanofi kept going and going and going  in the ensuing 7 years to expand the 2017 indication….. and succeeded. They finally received approval for the treatment of individuals with active polyarticular juvenile idiopathic arthritis (pJIA). pJIA is a form of arthritis that affects multiple joints at one time.

As Kevzara is a sub-q therapy, specialty pharmacies may be alert to distribution opportunities.

CLICK HERE to access prescribing information

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FDA Approves Sarilumab for Active Polyarticular Juvenile Idiopathic Arthritis

CLICK HERE to read the press release

We’ve written about Value-Based Contracting (VBC) numerous times. 

The logic of VBC is self-evident. 

However, the feasibility and implementation of VBC is a totally different matter….. it is simply not easy. 

Great ideas often pop up but fade away because they are too hard to manage. 

VBC may soon be the exception to the rule.

VBC requires a team effort. As we know, forming a team in most organizations can be a nightmare and healthcare is a poster child for poorly formed team efforts.  None the less, VBC has not withered and is striving to blossom in rocky soil.

The article below is some proof of the prospects for VBC. Key members in VBC are health plan providers who have been skeptical of the entanglements that VBC portends. As you are likely familiar, health plans have a long-standing national association, AHIP (America’s Health Insurance Plans), and, based on their comments, the organization seems to be warming up to VBC. 

There is already proof of concept that VBC works for health plans.  The prospect that manufacturers will be accountable (usually financially) is a lever that clearly can work in health plans’ favor. 

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Providers are Key to Success of Value-Based Specialty Programs | AHIP 2024

CLICK HERE to read the full article

The FDA recently approved a new infused therapy, Rytelo (imetelstat) from Geron Corporation, for the treatment of adult patients with low – to intermediate-1 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).

Rytelo is an oligonucleotide telomerase inhibitor and is the first and only telomerase inhibitor approved by the FDA. It is indicated to be administered as an intravenous infusion over two hours every four weeks.

Rytelo is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In MDS, abnormal bone marrow cells often express an enzyme, telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. 

The company has priced a single dose (47 mg) of Rytelo at $2,471. 

Actual dosage is determined by patient weight.

Distribution will be supported by specialty pharmacies.  

Onco360 has confirmed that it was named as a distribution partner.

CLICK HERE for prescribing information

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After 33 years, Geron finally crosses FDA finish line with blood cancer med Rytelo

CLICK HERE to read the full article

In the third century BCE a wonder of the world, the Colossus of Rhodes, stood as testament to ever lasting power and strength. As history tells, even a colossus will eventually fall. Now, in the 21^stcentury, a colossus in the pharmaceutical industry is rapidly falling away…. and is a harbinger of the demise of other, perhaps smaller, colossi. 

Ok…. We are talking about the colossus called Humira.

Yes, this pharmacy powerhouse therapy has held the #1 position on the US leader board for the past two decades. However, even PBMs are now throwing in the towel and are starting to embrace less costly biosimilars to dethrone the king.

By example, PBM colossi CVS and Accredo / Evernorth are now demoting Humira and adding biosimilars in preferred positions on their formularies.  With that ice broken….. and payers and patients and government pushing for cost relief….. the tea leaves now confirm that biosimilars have finally found their footing in the marketplace…… THE BARBARIANS ARE AT THE GATE!

The article below is the closest thing to a eulogy we have heard in quite a while…. and it cites some sober statistics.

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PBMs Start to Embrace Biosimilars

CLICK HERE to access the full article

………….. 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