Author: Randy Chase

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 several LD deals that have been recently publicly confirmed subsequent to the approvals.

Rezlidhia HUB to be managed by AscellaHealth

December 13, 2022–(BUSINESS WIRE)–Optime Care, a member of the AscellaHealth Family of Companies, announced a contractual partnership with Rigel Pharmaceuticals, Inc., bringing its full suite patient support/HUB service capabilities to support Rezlidhia (olutasidenib), a recent FDA-approved treatment for adult patients with relapse or refractory acute myeloid leukemia (AML). Optime Care’s services for life sciences manufacturers include pre-commercialization and market access expertise, exclusive distribution partnerships, national medication fulfillment and high-touch patient support and HUB services for enhanced patient outcomes.

Rezlidhia now available at Biologics by McKesson

December 21, 2022 — McKesson Corporation recently announced that its independent specialty pharmacy, Biologics by McKesson, has been selected by Rigel Pharmaceuticals as one of two specialty pharmacies in a limited distribution network for Rezlidhia (olutasidenib). Rezlidhia is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation as detected by an FDA-approved test. 

KRAZATI now available at Biologics by McKesson

Dec. 14, 2022 — Biologics by McKesson has been selected by Mirati Therapeutics as one of two specialty pharmacies in a limited distribution network for KRAZATI (adagrasib), indicated for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with KRAS G12C mutation.

KRAZATI now available from Onco360 

December 15, 2022–(BUSINESS WIRE)–Onco360 has been selected by Mirati Therapeutics to be a specialty pharmacy partner for KRAZATI (adagrasib) indicated for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with KRAS G12C mutation.

HEMGENIX: Orsini Specialty Pharmacy Selected as a Limited Distribution Partner

Dec. 20, 2022 /PRNewswire/ — Orsini Specialty Pharmacy was selected by CSL Behring as a limited distribution partner for Hemgenix (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy option for hemophilia B. Hemgenix is indicated for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes.

The FDA recently approved a biologics license application (BLA) for a new infused therapy, Briumvi from TG Therapeutics, Inc., indicated for the treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease, in adults. Briumvi is a novel monoclonal antibody that targets a unique epitope on CD20-expressing B-cells. CD20 using monoclonal antibodies have proven effective in managing autoimmune disorders such as RMS.

Multiple sclerosis is a neurological disease in which the immune system attacks the brain cells causing physical disabilities.  The National Institutes of Health says that MS affects about 400,000 people in the United States.

The label for Briumvi indicates that there is the potential for serious, life-threatening or fatal, bacterial and viral infections in some Briumvi-treated patients. In MS clinical trials, the overall rate of infections was 56% compared to 54% in teriflunomide-treated patients. The rate of serious infections was 5% compared to 3% respectively. There were 3 infection-related deaths in Briumvi-treated patients.

TG Therapeutics did not announce plans for distribution.

Neither did TG Therapeutics confirm pricing at the time of the announcement. Industry analysts differ in their estimates for cost at launch with pricing ranging from a low of $30,000 to as much as $60,000 per patient per year. By comparison, Roche’s Ocrevus, also for MS, has a current list price of about $68,000 annually.

CLICK HERE for prescribing information

Earlier this month the FDA approved a new infused therapy, Leqembi (lecanemab-irmb) from Eisai R&D Management Co., Ltd., indicated for the treatment of Alzheimer’s disease. Therapy was approved for patients with mild cognitive impairment or mild dementia stage of disease. 

Alzheimer’s is an irreversible, progressive brain disorder affecting more than 6.5 million Americans that slowly destroys memory and thinking skills and, eventually, the ability to carry out simple tasks. Neither Leqembi nor Biogen’s Aduhelm are cures for Alzheimer’s. Neither medication reverses disease progression for those who have already developed symptoms of Alzheimer’s disease or to show clinically significant slowing of cognitive decline, memory loss, or personality and behavior changes.  

It is expected that Leqembi will need to comply with the same barriers as Aduhelm. Currently, Medicare eligible patients must be enrolled in a clinical trial to obtain coverage. Eisai said it doesn’t expect the requirements to be revised or removed in the near term.

Eisai confirmed a launch price of $26,500 year. Biogen cut Aduhelm’s price in the U.S. by about half, effective Jan. 1, 2022. For a 74 kg (163 pounds) patient, the annual cost of Aduhelm dropped to $28,200….. a difference of about $1700 annually vs. Leqembi.

Leqembi is infused every 2 weeks vs. every 4 week infusion schedule for Aduhelm. That burden may prove to be a drag on uptake for Leqembi especially with a requirement for frequent MRIs / PET scans to confirm therapy continuation.

Eisai did not disclose plans for distribution of Leqembi at the time of the announcement. However, a press release dated January 11 confirmed that Soleo Health Specialty Pharmacy has been named as the sole distributor of Leqembi.

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FDA Grants Accelerated Approval for Alzheimer’s Disease 

January 06, 2023 — Today, the U.S. Food and Drug Administration approved Leqembi (lecanemab-irmb) via the Accelerated Approval pathway for the treatment of Alzheimer’s disease. Leqembi is the second of a new category of medications approved for Alzheimer’s disease that target the fundamental pathophysiology of the disease. These medications represent an important advancement in the ongoing fight to effectively treat Alzheimer’s disease.

“Alzheimer’s disease immeasurably incapacitates the lives of those who suffer from it and has devastating effects on their loved ones,” said Billy Dunn, M.D., director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research. “This treatment option is the latest therapy to target and affect the underlying disease process of Alzheimer’s, instead of only treating the symptoms of the disease.”

Leqembi was approved using the Accelerated Approval pathway, under which the FDA may approve drugs for serious conditions where there is an unmet medical need and a drug is shown to have an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. The results of a Phase 3 randomized, controlled clinical trial to confirm the drug’s clinical benefit have recently been reported and the agency anticipates receiving the data soon.

Researchers evaluated Leqembi’s efficacy in a double-blind, placebo-controlled, parallel-group, dose-finding study of 856 patients with Alzheimer’s disease. Treatment was initiated in patients with mild cognitive impairment or mild dementia stage of disease and confirmed presence of amyloid beta pathology. Patients receiving the treatment had significant dose- and time-dependent reduction of amyloid beta plaque, with patients receiving the approved dose of lecanemab, 10 milligram/kilogram every two weeks, having a statistically significant reduction in brain amyloid plaque from baseline to Week 79 compared to the placebo arm, which had no reduction of amyloid beta plaque. 

These results support the accelerated approval of Leqembi, which is based on the observed reduction of amyloid beta plaque, a marker of Alzheimer’s disease. Amyloid beta plaque was quantified using positron emission tomography (PET) imaging to estimate the brain levels of amyloid beta plaque in a composite of brain regions expected to be widely affected by Alzheimer’s disease pathology compared to a brain region expected to be spared of such pathology. 

The prescribing information for Leqembi includes a warning for amyloid-related imaging abnormalities (ARIA), which are known to occur with antibodies of this class. ARIA usually does not have symptoms, although serious and life-threatening events rarely may occur. ARIA most commonly presents as temporary swelling in areas of the brain that usually resolves over time and may be accompanied by small spots of bleeding in or on the surface of the brain, though some people may have symptoms such as headache, confusion, dizziness, vision changes, nausea and seizure. Another warning for Leqembi is for a risk of infusion-related reactions, with symptoms such as flu-like symptoms, nausea, vomiting and changes in blood pressure. The most common side effects of Leqembi were infusion-related reactions, headache and ARIA.

As specified in the prescribing information, Leqembi is indicated for the treatment of Alzheimer’s disease. The labeling states that treatment with Leqembi should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was studied in clinical trials. The labeling also states that there are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied.

The FDA granted this application Fast Track, Priority Review and Breakthrough Therapy designations. 

The approval of Leqembi was granted to Eisai R&D Management Co., Ltd.

The FDA recently approved a novel gene therapy, Adstiladrin (nadofaragene firadenovec-vncg) from Ferring Pharmaceuticals A/S, indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. 

According to the Centers for Disease Control and Prevention, about 57,000 men and 18,000 women are diagnosed with bladder cancer annually, and roughly 12,000 men and 4,700 women die from the disease each year in the United States.

Adstiladrin is administered three times annually via intravesical catheter.

Ferring did confirm that Adstiladrin will be commercially available in the US in the second half of 2023. Ferring did not release pricing information at the time of approval. Industry sources expect the therapy to be priced in the $160k-$260k price range. Given cost and need to track outcomes and patient experience, Ferring will likely distribute this product through SP distribution.

CLICK HERE for prescribing information

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FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer

December 16, 2022 — Today, the U.S. Food and Drug Administration approved Adstiladrin (nadofaragene firadenovec-vncg), a non-replicating (cannot multiply in human cells) adenoviral vector based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

“This approval provides healthcare professionals with an innovative treatment option for patients with high-risk non-muscle invasive bladder cancer that is unresponsive to BCG therapy,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “Today’s action addresses an area of critical need. The FDA remains committed to facilitating the development and approval of safe and effective cancer treatments.”

Bladder cancer, one of the more common forms of cancer, is a disease in which malignant (cancer) cells form a tumor in the tissues of the bladder. These abnormal cells can invade and destroy normal body tissue. Over time, the abnormal cells can also metastasize (spread) through the body. Most newly diagnosed bladder cancers (75% to 80%) are classified as NMIBC – a type of cancer that has grown through the lining of the bladder but hasn’t yet invaded the muscle layer. This type of cancer is associated with high rates of recurrence (between 30 to 80%) and the risk of progression to invasive and metastatic cancer. 

Treatment and care of patients with high-risk NMIBC, including those with carcinoma in situ, or CIS (abnormal cancer cells found in the place where they first formed and that have not spread to nearby tissue), often involves removing the tumor and the use of BCG to reduce the risk that the cancer will recur. Few effective treatment options exist for patients who develop BCG-unresponsive disease. The failure to achieve a complete response, or the disappearance of all signs of cancer as seen on cystoscopy, biopsied tissue, and urine, is associated with an increased risk of death or a disease-worsening event. Without treatment, the cancer can invade, damage tissues and organs, and spread through the body. 

The safety and effectiveness of Adstiladrin was evaluated in a multicenter clinical study that included 157 patients with high-risk BCG-unresponsive NMIBC, 98 of whom had BCG-unresponsive CIS with or without papillary tumors and could be evaluated for response. Patients received Adstiladrin once every three months for up to 12 months, or until unacceptable toxicity to therapy or recurrent high-grade NMIBC. Overall, 51% of enrolled patients using Adstiladrin therapy achieved a complete response (the disappearance of all signs of cancer as seen on cystoscopy, biopsied tissue, and urine). The median duration of response was 9.7 months. Forty-six percent of responding patients remained in complete response for at least one year.

Adstiladrin is administered once every three months into the bladder via a urinary catheter. The most common adverse reactions associated with Adstiladrin included bladder discharge, fatigue, bladder spasm, urinary urgency, hematuria (presence of blood in urine), chills, fever, and painful urination. Individuals who are immunosuppressed, or immune-deficient should not come into contact with Adstiladrin. 

This application was granted Priority Review, Breakthrough Therapy, and Fast Track designations.

The FDA granted approval of Adstiladrin to Ferring Pharmaceuticals A/S.

The FDA recently approved a new antiviral therapy regimen, Sunlenca (lenacapavir) from Gilead Sciences, indicated for the treatment of HIV-1 infection in heavily treatment-experienced adults with multidrug resistant HIV-1 infection failing their current antiretroviral regimen due to resistance, intolerance, or safety considerations. Sunlenca is given in combination with other antiretroviral(s). 

Sunlenca is the first of a new class of drugs called capsid inhibitors. Sunlenca blocks the HIV-1 virus’ protein shell (the capsid)interrupting multiple essential steps of the viral lifecycle. 

As noted, Sunlenca is supplied as both an oral and subcutaneous injectable regimen. It comes with two induction schedule options (see below). Startup doses and then followed by maintenance injections every six months.

Recommended dosage:

Initiation with one of two options followed by once every 6-months maintenance dosing.

• Initiation Option 1

   Day 1                927 mg by subcutaneous injection (2 x 1.5 mL injections)

600 mg orally (2 x 300 mg tablets)

   Day 2                600 mg orally (2 x 300 mg tablets)

• Initiation Option 2

   Day 1                600 mg orally (2 x 300 mg tablets)

   Day 2                600 mg orally (2 x 300 mg tablets) 

   Day 8                300 mg orally (1 x 300 mg tablet)

   Day 15              927 mg by subcutaneous injection (2 x 1.5 mL injections)

• Maintenance

927 mg by subcutaneous injection (2 x 1.5 mL injections) every 6

months (26 weeks) from the date of the last injection +/-2 weeks.

Gilead confirmed that Sunlenca injection and tablets are expected to cost $42,250 in the first year of therapy and $39,000 annually after that (maintenance injections only).

Gilead did not provide detail on how Sunlenca would be distributed. Given its benign safety profile, it could be expected that the therapy regimen (oral and sub-q injectable) will be provided by specialty pharmacy in limited distribution. Compliance and persistence are central to therapy success.

CLICK HERE to access prescribing information

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FDA Approves Sunlenca – New HIV Drug for Adults with Limited Treatment Options

December 22, 2022 The U.S. Food and Drug Administration approved Sunlenca (lenacapavir), a new type of antiretroviral medication for adult patients living with human immunodeficiency virus type 1 (HIV-1), whose HIV infections cannot be successfully treated with other available treatments due to resistance, intolerance, or safety considerations. After the starting dose is completed, Sunlenca is administered…………………

CLICK HERE to access the full FDA press release

So, can you easily explain the differences between a chemically synthesized drug and a biologic? 

You might be surprised how difficult that has become.

The article below should be thought provoking for those in the specialty pharmacy industry as it goes to the core of what exactly SPs actually sell….. chemically synthesized drugs and biologics. But…. if you were asked to list which column your drugs should be listed you would likely be stumped.

Here’s why….. the Consolidated Appropriations Act of 2020 included new guidance on how drugs can be classified. Specifically, the change states that the purpose of the amendment is to allow for the possibility that a manufacturer may chemically synthesize a biologic product. Huh?? 

Here’s an example…. Vondys 53, a biologic analog made through synthetic chemistry – not by any biologic process – is considered a biologic. The author of the (somewhat ‘tongue in cheek) article below polled pharma colleagues for their opinion as to whether similar drugs – are or aren’t biologic drugs. 

The results: 

41% agreed that these new drugs were biologics, 39% disagreed, 20% responded “Confused” 

….. so am I. 

Non-pharmacists may be a bit intimated by the technical references in the article…. just skip over them as that’s not the point of this Alert. The real purpose is to alert those working in the specialty pharmacy industry that today’s crop of new biologics ‘ain’t your grandfather’s biologics’. If you are going to be selling both chemically synthesized drugs and biologics you should have, at a minimum, a basic understanding of these developments.

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What is a Biologic Drug Anyway?

Or, How I Learned to Stop Worrying and Love My Own Definition

January 19, 2020 — Recently I posted on “biologic drugs” which were approved in 2019. All was well in my little scientific universe, until I received this in an email: 

“You recently reported on “14 novel biologic drugs,” referring to ‘novel’ biopharmaceuticals approved through CDER. But, … You also include several synthetic drugs, which are clearly not biopharmaceutical/biologics.”

Wait, what? Synthetic drugs are clearly not biopharmaceutical/biologics? Sure, the siRNA drug, Givlaari, and the antisense oligonucleotide, Vyondis 53, are both technically made by chemical synthesis. But, not considering these RNA-based drugs “biologic drugs” just felt… wrong.

It got me thinking – what is the definition of a biologic drug, anyway? I immediately started my research the way any good, card-carrying scientist does — I punched it into the Google:

GOOGLE:  A biopharmaceutical, also known as a biologic medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesized from biological sources. [source] Wikipedia

Though Wikipedia delivers good answers to probably 90% of my questions, I knew this definition was incomplete – nobody considers natural products like penicillin “biologic drugs.” Not wanting to spend the rest of my Sunday night reading FDA guidance documents, I did instead what any good millennial does – I asked social media.

As one comment pointed out, the term “biologic” itself is anachronistic, coming from a time when chemistry and biology were considered distinct (i.e. before chemists started getting upset about Nobel Prizes in Chemistry going to “biologists”). The Biologics Control Act, which first gave the US government control over the processes to make biological products, was passed in 1902 – long before it was clearly established that proteins are polymers of amino acids and that genetic information is stored chemically in nucleobases.

Digging Deeper

Based on the lack of strong consensus, I decided this was a topic worth diving further into.

Readers likely remember that “biologic” became a charged term in the 2000’s, due to expensive biologic products like filgrastim and epoetin alfa, approved under Biologic License Applications (BLAs) rather than New Drug Applications (NDAs). Since the abbreviated generic drug approval process (ANDA) only applied to originals filed under NDAs, there was no way to create a “generic” biological product without repeating expensive and lengthy clinical trials. This loophole led to the creation of the abbreviated follow-on biologicals approval pathway (aBLA) through the 2009 Biologics Price Competition and Innovation Act (BPCI).

So how are the adjectives, “biologic” or “biological,” legally defined in the US Code of Laws after BPCI in 2009? Turns out, they’re not. The term “biological product,” is legally defined only with examples, not properties:

“The term “biological product” means a virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, protein (except any chemically synthesized polypeptide), or analogous product, or arsphenamine or derivative of arsphenamine (or any other trivalent organic arsenic compound), applicable to the prevention, treatment, or cure of a disease or condition of human beings.”

42 U.S. Code § 262. Regulation of biological products.

With no basis in the text for determining what an “analogous product” is, no definition of what exactly makes a product “biologic” or “biological,” and with, of all things, arsphenamine (or any other trivalent organic arsenic compound) being the only exemplified exception, no wonder everyone is confused. The FDA and the industry spend an enormous amount of resources deciding new registrants on a case-by-case basis, which was part of the justification for a recent change in the definition. 

A Legal Update on Synthetic Processes

It gets better. The Further Consolidated Appropriations Act, 2020, signed into law in December 2019, amended this definition by removing the phrase “except any chemically synthesized polypeptide”: 

“The term “biological product” means a virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, protein (deleted: except any chemically synthesized polypeptide), or analogous product, or arsphenamine or derivative of arsphenamine (or any other trivalent organic arsenic compound), applicable to the prevention, treatment, or cure of a disease or condition of human beings.” 

New legal definition of “biological product” after the Further Consolidated Appropriations Act.

The recent FDA press release on the change states that the purpose of the amendment is to allow for the possibility that a manufacturer may chemically synthesize a biologic product. Under the old definition, if an original biologic product was licensed under a BLA, a new chemically synthesized biologic follow-on could not be licensed through an abbreviated aBLA pathway, since it was made by a synthetic process, but also could not be licensed through an ANDA, since the original was not filed under an NDA.

In the end, what this demonstrates is that there are at least two simultaneous uses of the term “biologic” in the industry – an arbitrary regulatory definition to decide what drugs are licensed under BLAs vs. NDAs, and a scientific colloquialism which most of us use on a day-to-day basis. Neither definition is clear and unambiguous, and no single criterion (e.g. chemical process) universally captures all cases. 

Biolognas, Anybody?

Having both uses at the same time is confusing enough to make you want to give “synthetic biologics” a different name without “biologic” in it. But what would we call them? Biologishes? Biolognas? I guess I have a year to think about it before 2020’s Biologic Drug Approvals post. 

But back to my original question– since oligonucleotide drugs are all filed under NDAs, they’re regulated more like most small molecules than most biologics. But just because they’re filed under NDAs, doesn’t mean we can’t still call them biologics (e.g. insulins are all filed under NDAs). Anyway, all this makes me very glad that we have a lot of brilliant professionals working in our very important Regulatory Affairs departments, though I’m glad it doesn’t include me!

We know that there are a limited number of specialty pharmacies in the US. A small number of customers makes it hard for any new product or service to have enough customers to enable a product to break even….. let alone be profitable. So, we are curious when we see new products coming to market that target SPs. Today we are talking about software programs that manage the upstream / downstream patient journey that SPs have managed for decades (often using Excel spreadsheets and even index cards back in the day.)

The new ‘state-of-the-art’ platforms are fairly sophisticated. They capture loads of patient data that can include patient electronic medical records (with permission), physician ordering portals, prescribing protocols that go down to the specific medication profile, patient financial assistance, obtaining prior-authorizations, refills, patient reported side effects / adverse reactions / dosing schedules / dose reductions, and on-and-on-and-on.

As noted in the article below, Henry Ford Health sees new opportunity in this small segment. Why? Because the segment is suddenly much bigger. Hospitals and health systems are increasingly opening owned and operated specialty pharmacies enabling them to  rapidly catch up with well-established specialty pharmacies by installing one of these software platforms. There are a lot more hospitals than stand alone SPs. 

The Henry Ford platform was first developed a decade ago and it sounds like its newest mousetrap is on par with other leading providers of comparable platforms.

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Henry Ford Health’s Specialty Pharmacy Software Goes National

Platform developed by Henry Ford Health now serves systems in seven states, with plans for expansion

DETROIT/PRNewswire/ — Henry Ford Innovations (HFI), one of the nation’s leading healthcare innovation programs, announced today that its specialty pharmacy software platform, DromosPTM™, now serves pharmacies and patients across the country. Developed and implemented by Henry Ford Health in 2013 to address an internal need, the platform quickly grew beyond Detroit and is now used by seven leading specialty pharmacies and healthcare systems from Massachusetts to Utah. Additionally, more systems are planning to adopt the platform in the coming months.

Signifying its progression in technology- and consumer-impacted healthcare, DromosPTM™ fills a long-existing gap in the specialty pharmacy industry by offering efficient patient-focused care and service. Specialty pharmacies differ from traditional pharmacies by coordinating aspects of patient care and disease management. They deliver medications that demand special handling, storage and distribution for often chronic and rare conditions.

These partnerships allow increased functionality and provide patients across the country a better experience and care.

This first-of-its-kind application solves several problems regularly seen in specialty pharmacy cases such as finding financial assistance for costly prescriptions, enhanced monitoring and emphasizing best practices. For example, organizations are leveraging the platform by expanding their pharmacy capabilities within patient portals, allowing patients to easily request often-complicated prescription refills and transfers.

“We are proud to have leading healthcare organizations joining our DromosPTM™ network to transform lives through advanced patient therapy management,” said Lisa Prasad, Henry Ford Health’s Chief Innovation Officer and leader of Henry Ford Innovations. “These partnerships allow increased functionality and provide patients across the country a better experience and care.” 

DromosPTM™ was designed and developed at Henry Ford Health’s specialty pharmacy, Pharmacy Advantage, which is supported by CarepathRx LLC. At the time, Pharmacy Advantage team members recognized an opportunity to improve care administration and outcomes, resulting in the development of the platform and partnership with HFI for licensing and distribution.

“DromosPTM™ is helping the specialty pharmacy industry scale their businesses and improve both operational and clinical outcomes as their organizations continues to grow” said David Shepherd, President and CEO of Community Care Services. “By helping remove barriers from patients, like cost and prior authorizations, DromosPTM™ has created positive patient outcomes already.”

Established in 2011 to develop and commercialize its intellectual assets, HFI has enacted more than 30 licensing agreements, representing over $100 million in future revenue. HFI remains committed to creating best-of-class partnering programs with early-stage and corporate collaborators and serving as a gateway to international healthcare companies.

To learn more about DromosPTM™ please visit https://dromosrx.com or call (888) 376-6670.

SOURCE Henry Ford Health

The FDA recently approved a novel, ORAL monotherapy treatment, Rezlidhia (olutasidenib) from Rigel Pharmaceuticals, Inc., to treat adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test. However, the approval included a boxed warning for potentially fatal Differentiation Syndrome.

Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that in the United States alone, there will be about 20,050 new cases, most in adults, in 2022.

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.  Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.

Rigel did not announce pricing for the new therapy, nor did they provide details on distribution. Given the segment and relatively small patient base it is likely that Rezlidhia will launch through limited distribution.

CLICK HERE for prescribing information

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FDA approves olutasidenib for relapsed or refractory acute myeloid leukemia with a susceptible IDH1 mutation

The Food and Drug Administration (FDA) has approved olutasidenib (Rezlidhia) capsules for adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible IDH1 mutation as detected by an FDA-approved test. The FDA also approved the Abbott RealTime IDH1 Assay to select patients for olutasidenib.

Approval was based on Study 2102-HEM-101 (NCT02719574), an open-label, single-arm, multicenter clinical trial that included 147 adult patients with relapsed or refractory AML with an IDH1 mutation confirmed using the above assay. Olutasidenib was given orally,150 mg twice daily, until disease progression, unacceptable toxicity, or hematopoietic stem cell transplantation. The median treatment duration was 4.7 months (range: 0.1 – 26 months). Sixteen (11%) patients underwent hematopoietic stem cell transplantation following olutasidenib.

Efficacy was established on the rate of complete remission (CR) plus complete remission with partial hematologic recovery (CRh), the duration of CR+CRh, and the rate of conversion from transfusion dependence to independence. The CR+CRh rate was 35% (95% confidence interval [CI]: 27%, 43%), including 32% CR and 2.7% CRh. The median time to CR+CRh was 1.9 months (range: 0.9 – 5.6 months), and the median duration of CR+CRh was 25.9 months (95% CI: 13.5 months, not reached).

Among the 86 patients who were dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 29 (34%) became independent of RBC and platelet transfusions during any 56-day post-baseline period. Of the 61 patients who were independent of both RBC and platelet transfusions at baseline, 39 (64%) remained transfusion independent during any 56-day post-baseline period.

The most common adverse reactions (≥20%) were nausea, fatigue/malaise, arthralgia, constipation, leukocytosis, dyspnea, fever, rash, mucositis, diarrhea, and transaminitis. The prescribing information contains a Boxed Warning alerting health care professionals and patients about the risk of differentiation syndrome which can be fatal.

The recommended olutasidenib dose is 150 mg taken orally twice daily on an empty stomach (at least 1 hour before or 2 hours after a meal) until disease progression or unacceptable toxicity. For patients without disease progression or unacceptable toxicity, treatment is recommended for a minimum of 6 months allowing for clinical response.

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 designated partner(s).

Here are three LD deals that have been recently publicly confirmed subsequent to the approvals.

Amber Specialty Pharmacy Selected to Dispense TZIELD (teplizumab), a Monoclonal Antibody Treatment to Delay Onset of Stage 3 Type 1 Diabetes in Patients with Stage 2 Type 1 Diabetes

OMAHA, Neb., — Amber Specialty Pharmacy announces that it will begin dispensing TZIELD (teplizumab), an anti-CD3 monoclonal antibody, to delay Stage 3 Type 1 Diabetes (T1D) in patients ages 8 years and older with Stage 2 T1D. Indication for individuals ages 8 years and older with Stage 2 T1D is categorized by the presence of two or more T1D-related autoantibodies and abnormal blood sugars. TZIELD™ is manufactured by Provention Bio, Inc., a New Jersey-based biopharma company.

Orsini Specialty Pharmacy Selected by Provention Bio as a Limited Distribution Partner for TZIELD

ELK GROVE VILLAGE, Ill., /PRNewswire/ — Orsini Specialty Pharmacy, a leading independent specialty pharmacy focused on rare diseases, and gene therapies, has been selected by Provention Bio, Inc. https://www.gilead.com/as a specialty pharmacy for TZIELD® (teplizumab). TZIELD is the first and only treatment indicated to delay the onset of Stage 3 type 1 diabetes (T1D) in adult and pediatric patients aged 8 years and older with Stage 2 T1D approved by the FDA.

ELAHERE Available at Biologics by McKesson

Biologics by McKesson, an independent specialty pharmacy specializing in oncology and rare disease areas, has been selected by ImmunoGen, Inc. as the sole specialty pharmacy provider for ELAHERE (mirvetuximab soravtansine-gynx) for the treatment of adult patients with folate receptor alpha (FRα)-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens.

Rigel Pharmaceuticals recently received the green light from the FDA for their oral IDH1 inhibitor, REZLIDHIA™ (olutasidenib).  REZLIDHIA™ is indicated for treatment of acute myeloid leukemia (AML) in a difficult to treat, relapsed or refractory (R/R) patient population who have a mutation in isocitrate dehydrogenase-1 (IDH1).  Mutations of IDH1 are found in 7-14% of patients with AML and there have been few treatment options for these patients, particularly in the second line.  The FDA approval was supported by data from the open-label Phase 2 registrational study evaluating REZLIDHIA™ monotherapy at a dose of 150 mg twice daily in 153 mIDH1 R/R AML patients. The efficacy-evaluable population was 147 patients who initiated REZLIDHIA™ at least six months prior to the interim analysis cutoff date of June 18, 2021, and who had a centrally confirmed IDH1 mutation.  Acute myeloid leukemia (AML) is a rapidly progressing cancer of the blood and bone marrow that affects myeloid cells, which normally develop into various types of mature blood cells. AML occurs primarily in adults and accounts for about 1 percent of all adult cancers. The American Cancer Society estimates that in the United States alone, there will be about 20,050 new cases, most in adults, in 2022.¹ 

Relapsed AML affects about half of all patients who, following treatment and remission, experience a return of leukemia cells in the bone marrow.² Refractory AML, which affects between 10 and 40 percent of newly diagnosed patients, occurs when a patient fails to achieve remission even after intensive treatment.³ Quality of life declines for patients with each successive line of treatment for AML, and well-tolerated treatments in relapsed or refractory disease remain an unmet need. 

REZLIDHIA™ provided a complete remission (CR) combined with partial hematological recovery (CRh) within less than two months (mean time to response 1.9 months) lasting for over two years (mean duration 25.9 months) and demonstrated a CR+CRh rate of 35% in patients with over 90% of those responders in complete remission.

“The 25.9 months median duration of CR+CRh is a clinically meaningful improvement for AML patients and appears to be longer than currently available treatment options,” said Jorge E. Cortes, M.D., Director, Georgia Cancer Center, Cecil F. Whitaker Jr., GRA Eminent Scholar Chair in Cancer, and Phase 2 trial investigator. “Given the limited treatment options for adult patients with mIDH1 R/R AML, who typically have a poor prognosis, REZLIDHIA™ may provide an effective, new treatment option with a well characterized safety profile.”

Complete remission was achieved for over 28 months (mean 28.1 months).  Over 1/3 of patients (34%) treated with REZLIDHIA™ were able to avoid platelet and/or red blood cell transfusions at 56 days and unlike other treatments in the relapsed/refractory AML space, there is no need for cardiac monitoring. REZLIDHIA™ has a black box warning for differentiation syndrome which can be fatal; if suspected, differentiation syndrome should be treated with corticosteroids and hemodynamic monitoring.  

Hats off to Rigel Pharmaceuticals for getting the FDA nod just four months after acquiring the asset.  REZLIDHIA™ has the potential to address an unmet need in a niche patient population typically subject to a poor prognosis.  It is motivating to see companies like Rigel invest in bringing to market therapeutic options for small, tough to treat patient populations.

Click here for the full press release from Rigel Pharmaceuticals: https://www.rigel.com/investors/news-events/press-releases/detail/349/rigel-announces-u-s-fda-approval-of-rezlidhia

Click here for full prescribing information: https://www.rezlidhia.com/downloads/pdf/REZLIDHIA-Full-Prescribing-Information.pdf