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FDA Approves Sub-Q Tx for LDL Control – Leqvio

Last week the FDA approved Leqvio, from Novartis Pharmaceuticals. Leqvio is a first-in-class, RNAi sub-Q therapy indicated as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD), who require additional lowering of low density lipoprotein cholesterol (LDL-C).

The startup dose is given via subcutaneous injection by a healthcare professional. The next dose is given after 3 months with subsequent doses every 6 months. Before starting Leqvio the patient should be on a diet to lower cholesterol and a statin.

Novartis priced Leqvio at $3,250 per dose in the U.S., meaning the first year of treatment will cost $9,750 and subsequent years $6,500. That range falls in line with the current list prices of two existing PCSK9 therapies—around $5,400 to $5,850 for Amgen’s Repatha and Regeneron’s Praluent.

As an in-office procedure, Leqvio injections will be covered under the Medicare Part B. But, coverage through Part B could be an advantage for Leqvio over Repatha and Praluent. Physicians will be reimbursed for Leqvio ASP plus 6%. The other two drugs are covered by Part D under the pharmacy benefit.

Given the relatively large patient population and relatively low cost for a specialty drug, it is unlikely that Leqvio will be channeled through a limited distribution specialty pharmacy program. Leqvio qualifies as a specialty pharmacy therapy mainly because it ‘fits’ the specialty class of therapy carved out by Repatha and Praluent.

FDA approves add-on therapy to lower cholesterol among certain high-risk adults

FDA has approved Leqvio (inclisiran) injection as a treatment to be used along with diet and maximally tolerated statin therapy for adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD) who require additional lowering of low-density lipoprotein cholesterol (LDL-C). Leqvio works to reduce circulating levels of LDL-C, commonly known as “bad cholesterol.”

Leqvio is approved at a 284 mg dose administered as an initial under-the skin injection, a second dose at three months, and continued treatment once every six months after that point.

Diseases or Conditions
HeFH is a life-threatening condition in which patients have a mutation in a small group of genes that controls the way the body clears cholesterol. As a result, patients have extremely high levels of LDL-C. People with HeFH generally have cholesterol levels two to three times higher than normal. These individuals are at increased risk of cardiovascular events, such as heart attack, stroke, and coronary artery disease. HeFH occurs in approximately 1 in 250 individuals.

ASCVD involves the buildup of cholesterol plaque in arteries. Approximately 18.3 million American adults (8%) have ASCVD. Clinical ASCVD is an umbrella term and includes conditions such as acute coronary syndromes (sudden, reduced blood flow to the heart), peripheral arterial disease, heart attack and stroke.

The effectiveness of Leqvio was studied in three randomized, double-blind, placebo-controlled trials that enrolled 3,457 adults with HeFH or clinical ASCVD. Enrolled participants were taking maximally tolerated statin therapy but required additional LDL-C lowering based on their risk for cardiovascular events. In all three studies, the main effectiveness outcome measure was the percent change in LDL-C from the beginning of the trial to day 510 (month 17). In each trial, participants received under-the-skin injections of either 284 mg Leqvio or a placebo on four separate days: day 1, day 90 (month 3), day 270 (month 9), and day 450 (month 15).

Study 1 enrolled 1,561 adults with ASCVD. At day 510, the Leqvio group had an average LDL-C decrease of 51% whereas the placebo group had an average LDL-C increase of 1%. Study 2 enrolled 1,414 adults with ASCVD. At day 510, the Leqvio group had an average LDL-C decrease of 46% whereas the placebo group had an average LDL-C increase of 4%. Study 3 enrolled 482 adults with HeFH. At day 510, the Leqvio group had an average LDL-C decrease of 40% whereas the placebo group had an average LDL-C increase of 8%.


The White Bagging War Heats Up Reprise

The public relations battle surrounding White Bagging is heating up. Who’s on each side?

Depends on who you ask….!

  • On one side, hospitals are claiming that their very existence is being threatened due to White Bagging policies.
  • On the other side are the PBMs and the payers.

But, is there yet a third side? We will get to that. 

First, what is White Bagging?

As defined by the hospitals in the article link below….. White Bagging consists of “policies that require hospitals administering certain high-cost medications in an outpatient setting [actually inpatient as well] to receive those medications from third parties contracted with the health plan, instead of providing those medications directly from the hospital pharmacy inventories.” Increasingly, those third parties are specialty pharmacies acting as specialty distributors, under a distributor’s license (no prescription required) or as the pharmacy (with a prescription). 

What’s at stake? Money and control.

The number of rare therapies approved over the past two years has mushroomed. Most of the therapies have been $HUGE$ dollar meds that are hospital administered, often at leading national medical centers. These providers are very concerned about money and control. If they can buy directly and bill under often outdated, traditional contract terms (which never envisioned meds costing in the hundreds of thousands of dollars for a one-time infusion and may still pay them at U&C rates) then chances are they will realize a larger Net profit. If a specialty pharmacy is inserted into that scenario, they may not be able to realize any profit if the SP directly bills the PBM as a pharmacy claim or payer via a medical claim. 

It is no surprise that the hospitals are now upping the ante and turning to the courts and even state legislatures, where they may have significant influence, to seek relief and regain control. 

But…. who is the third party to this battle? Manufacturers.The pharmaceutical manufacturers proliferated the “White Bagging” war by selecting one or a handful of specialty pharmacies to be their preferred distributor(s) for these new infused therapies. Traditional distributors can’t perform all the functions of a specialty pharmacy as they are prohibited from making direct patient contact. Manufactures realized that SPs can help them out with managing patient care, ensuring compliance, tracking outcomes for FDA reporting, in addition to all the traditional SP patient stuff such as clearing prior authorizations and financial assistance. When payers and PBMs realized what was happening they got creative and saw a way to insert utilization review and tighter benefits management into the mix, things that SPs are also adept at juggling. 

It is also noteworthy to mention that SPs are also inventorying their meds for distribution. Traditional distributors charge manufacturers a percentage for their limited services…. better to pay an SP for the full-service package vs. just “park, pack, and ship.” 

So, it is surprising that the hospital lobby is focusing its ire only on the payers and PBMs, allowing the manufacturers off the hook. Then again, there are a lot of big research dollars that flow from manufacturers to the same big medical centers in new drug trials….. just sayin’. 

So, read the article below for a good update on what the leading payers are doing related to “White Bagging”.

Payers Attempt to Impose “White-Bagging” Policies on Hospitals



FDA Approves Infused Tx for Myasthenia Gravis – Vyvgart

The FDA has approved a new INFUSED biologic, Vyvgart (efgartigimod alfa-fcab, Argenx), for treatment of generalized Myasthenia Gravis (gMG) in adults who are acetylcholine receptor antibody positive. gMG is a rare, chronic autoimmune, neuromuscular disease.

Myasthenia gravis is characterized by debilitating and potentially life-threatening muscle weakness and rapid fatigue of any of the muscles under voluntary control. The disease can affect people of any age and is more common in women younger than 40 and in men older than 60. Roughly 3 percent of people who develop gMG will die from it.

There is no cure for myasthenia gravis, but treatment can help relieve other signs and symptoms, such as double vision, drooping eyelids, and difficulties with speech, chewing, swallowing and breathing.

Prior to the approval of Vyvgart, therapies to control gMG include corticosteroids such as prednisone (Rayos), immunosuppressants (e.g., Azasan, Imuran), mycophenolate mofetil (Cellcept), cyclosporine (Sandimmune, Gengraf, others), methotrexate (Trexall) or tacrolimus (Astrograf XL, Prograf, others). Intravenous therapies incude plasmapheresis, intravenous immunoglobulin (IVIg), and monoclonal antibodies such as Rituximab (Rituxan) and eculizumab (Soliris).

The prevalence of gMG in the United States is estimated at 14 to 20 per 100,000 population, which translates into 36,000 to 60,000 cases in the United States. gMG has a low incidence rate of 2.1 to 5.0 per million people per year in the US.

Pricing for Vyvgart was not released upon approval. By reference, Soliris is currently priced at $675,000 per year. Distribution details were not released; however, it is presumed that Vyvgart will launch into limited distribution similar to Soliris.

FDA approves Vyvgart for treatment of myasthenia gravis

The FDA announced the approval of Vygart for treatment of the chronic autoimmune, neuromusucal disease myasthenia gravis in adults who are positive for acetylcholine receptor antibody positive adults.

The approval of the investigational antibody fragment designed to target the neonatal Fc receptor is the first for the new drug class, according to a press release.

“There are significant unmet medical needs for people living with myasthenia gravis, as with many other rare diseases,” Billy Dunn, MD, director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, said in the press release. “Today’s approval is an important step in providing a novel therapy option for patients and underscores the agency’s commitment to help make new treatment options available for people living with rare diseases.”

In a randomized, double-blind, placebo controlled, multicenter Phase 3 trial completed in 2020, patients with myasthenia gravis with acetylcholine receptor antibodies who received Vyvgart (efgartigimod, Argenx) responded to treatment at a higher rate as compared with placebo.

Since the therapy reportedly decreases levels of immunoglobulin, experts warn of an increased risk of infection. Hypersensitivity reactions are also possible, including eyelid swelling, shortness of breath, and rash. In the cases of hypersensitivity or existing infection, treatment should be either discontinued or delayed, according to the press release.


FDA Approves SQ Biologic for Severe Asthma – Tezspire

The FDA approved a new, subcutaneous biologic last week, Tezspire (tezepelumab-ekko) from Amgen and AstraZeneca, an add-on maintenance treatment for adult and pediatric patients (12+) with severe asthma. Tezspire is a first-in-class biologic for this complex condition.

Severe asthma impacts many of the 34 million people living with the disease worldwide. It affects their breathing and can severely limit day-to-day activity. The treatment has proven effective across a spectrum of causes of inflammation (but not indicated for acute bronchospasm or status asthmaticus).

Tezspire is the result of a collaboration agreement by Amgen and AstraZeneca that started in 2012. The companies will share costs and profits equally. AstraZeneca will lead development and Amgen will lead manufacturing. Amgen and AstraZeneca will jointly commercialize Tezspire in the US. Tezspire will enter the segment now led by Xolair (omalizaub) for patients with allergic asthma and Dupixent (dupilumab) for patients with eosinophilic asthma. Analysts forecast Teszpire to break the $$billion mark within three years of launch.

Tezspire will enter the market at an anticipated price of $28,000 per year. Distribution details were not released. Given that both Xolair and Dupixent are only available through limited distribution it is likely that Tezspire will also launch through LD.

FDA Approves Tezspire (tezepelumab-ekko) in the U.S. for Severe Asthma

First and Only Biologic to Consistently and Significantly Reduce Exacerbations in a Broad Population of Severe Asthma Patients
Only Biologic for Severe Asthma Approved With no Phenotype or Biomarker Limitations

THOUSAND OAKS, Calif., Dec. 17, 2021 /PRNewswire/ — Amgen today announced that the U.S. Food and Drug Administration (FDA) has approved Amgen and AstraZeneca’s Tezspire™ (tezepelumab-ekko) for the add-on maintenance treatment of adult and pediatric patients aged 12 years and older with severe asthma.1

Tezspire was approved following a Priority Review by the FDA and based on results from the PATHFINDER clinical trial program. The application included results from the pivotal NAVIGATOR Phase 3 trial in which Tezspire demonstrated superiority across every primary and key secondary endpoint in patients with severe asthma, compared to placebo, when added to standard therapy.2

“Today’s approval by the FDA marks the first time that patients and their physicians will have a biologic option for severe asthma without phenotypic limitations and irrespective of biomarker levels,” said David M. Reese, M.D., executive vice president of Research and Development at Amgen. “Asthma is a complex and chronic inflammatory disease that affects everyone differently. By working at the top of the inflammation cascade, Tezspire helps stop the inflammation that causes asthma attacks at the source and has the potential to treat a broad population of people with severe asthma, including those who have historically lacked effective treatment options.”

Tezspire acts at the top of the inflammatory cascade by targeting thymic stromal lymphopoietin (TSLP), an epithelial cytokine.3 It is the first and only biologic to consistently and significantly reduce asthma exacerbations across Phase 2 and 3 clinical trials, which included a broad population of severe asthma patients irrespective of key biomarkers, including blood eosinophil counts, allergic status and fractional exhaled nitric oxide (FeNO).2,3 Tezspire is the first and only biologic for severe asthma that does not have a phenotype—eosinophilic or allergic—or biomarker limitation within its approved label.4-11

“Due to the complex and heterogeneous nature of severe asthma and despite recent advances, many patients continue to experience frequent exacerbations, an increased risk of hospitalization and a significantly reduced quality of life,” said Professor Andrew Menzies-Gow, director of the Lung Division, Royal Brompton Hospital, London, UK, and the principal investigator of the NAVIGATOR trial. “Tezspire represents a much-needed new treatment for the many patients who remain underserved and continue to struggle with severe, uncontrolled asthma.”

Results from the NAVIGATOR Phase 3 trial were published in The New England Journal of Medicine in May 2021.2 In clinical studies of Tezspire, the most common adverse reactions were nasopharyngitis, upper respiratory tract infection and headache.


PSG Trend Report Can Help Fine-tune 2022 Forecasting

Specialty pharmacies should be well into forecasting for the upcoming year. Cash in fuels cash out….. and you know that more of the former is critical for growth. So, good forecasting provides a roadmap for marketing, sales, purchasing, HR, etc., to ramp up for the new year — or, heaven forbid, plan to trim expenses. Remember, Finance takes an approved forecast and translates it into a company budget.

All good forecasts rely on published reports from pharmacy market analytic resources. Successful forecasts aggregate as much data as possible from multiple sources. So, before you submit your 2022 forecast check out the 2020 Specialty Trend Report from Pharmaceutical Strategies Group (PSG). 

CLICK HERE to download the PSG Report

By now you should know the highlights. 2020 was a sour year for the economy and its impact to specialty pharmacy should not be overlooked. But, there was some palpable market rebound in 2021…. but we won’t see that data till about April 2022. So, the trends through the downturn are harbingers for the upturn. And, the 2-5-year estimates in the report are credible metrics to moderate your optimism (or pessimism) for 2022. 


Omnicell Acquires Certain Assets of ReCept Specialty Pharmacy

The press release below raised eyebrows. At first, we thought that Omnicell had acquired ReCept Specialty Pharmacy. ReCept is a four decades long running specialty provider and has grown to eleven locations mostly in the southern states. But our quick scan of the release proved first impressions wrong.

ReCept has sold off only the assets of a relatively small division, one that provides specialty pharmacy management services for health systems, clinics, and physician groups. Services include operational expertise, HR, technology / integration, workflow management, payer access assistance, and other aspects of managing a specialty pharmacy.

What is noteworthy is that these services are being offered to help health system owned (HOSP) pharmacies compete with independent specialty pharmacies. Is this a case of ‘If ya can’t beat em’… join em’? ReCept would not be the first SP to make this tack. Walgreens, for example, has been active in the HOSP space for several years.

Perhaps the most eye-popping aspect of the announcement was that this division only generated $24 million in the prior year period….. but was acquired by Omnicell for $100 million. That’s not 5X EBIDTA…. It’s 5X Revenue. Well played ReCept….. well played!

ReCept will continue to operate its specialty pharmacies and retail locations. 

Omnicell to Acquire ReCept

Strategic acquisition of leading specialty pharmacy management services provider supports the Autonomous Pharmacy vision and expands Omnicell’s Advanced Services portfolio

Addresses significant need for access to and management of complex medications, expected to improve profitability and patient care

December 02, 2021  — Mountain View, Calif.–(BUSINESS WIRE)–Omnicell, Inc., a leading provider of medication management solutions and adherence tools for health systems and pharmacies, today announced it has entered into a definitive agreement with ReCept Holdings, Inc., a leading provider of specialty pharmacy management services for health systems, clinics, and physician groups, to acquire its business for total aggregate cash consideration of $100 million, subject to customary adjustments. The acquisition will broaden Omnicell’s portfolio of capabilities and services to address the significant need to improve access to and care coordination for complex medications.

“As medical innovations drive new, more complex drugs, pharmacy requires more specialized tools and expertise to help manage the patient’s treatment journey”

Studies have shown that specialty medications represent over 50 percent of the country’s total spending on retail, mail-order, and provider-administered drugs.1 Used for treatment of complex conditions, these medications often require intensive patient management and specialized workflows for dispensing and care coordination. Specialty pharmacies serve as the connection between patients, prescribing physicians, and payors to ensure streamlined access and adherence to these specialty drugs, helping to maintain continuity of care throughout the process, and are expected to improve margin and profitability. As an Advanced Services organization, ReCept provides implementation and managed services for health systems and other provider organizations to optimize their specialty pharmacy programs and the related pharmaceutical aspects of patient care.

The ReCept acquisition will add solutions for specialty pharmacy management services, including specialty pharmacy expertise and operational capabilities, human resources, technology and integration, workflow management, payor access assistance, and other aspects of managing a specialty pharmacy, to Omnicell’s broader medication management infrastructure. This total solution for provider groups, federally qualified health centers (FQHCs), and health systems supports on-site management of specialty pharmacy services, including payor contracting, staffing, licensing, quality assurance, 340B administration, and preferred pricing agreements designed to improve margin and profitability, while keeping the patient at the center of care.

“As medical innovations drive new, more complex drugs, pharmacy requires more specialized tools and expertise to help manage the patient’s treatment journey,” said Randall Lipps, chairman, president, CEO, and founder of Omnicell. “The ReCept acquisition is a strategic investment that expands our portfolio to address the significant market need for specialty pharmacy solutions. Our entry into this rapidly expanding area of practice is a natural progression on the path to the Autonomous Pharmacy and the goal to enhance care, improve efficiency, and drive profitability. We are excited to add these critical solutions to the Omnicell portfolio for our health system clients and prospects.”

“Specialty pharmacy is a key component of medication management. By joining with Omnicell we look forward to maximizing the benefits of our managed services capabilities as part of a broader, intelligent infrastructure that will deliver value for our pharmacy partners,” said Carmine DeNardo, president and chief executive officer, ReCept. “The synergies with Omnicell’s advanced pharmacy technology solutions create a comprehensive strategic partner that will help give health systems broader and deeper expertise across the full spectrum of medication management.”

Compelling Strategic BenefitsDifferentiated Portfolio of Solutions: ReCept provides a full suite of turnkey specialty pharmacy management services, including specialty pharmacy expertise and operational capabilities, technology and integration solutions, assistance in increasing access to payor networks and limited distribution drugs, and performance analytics and clinical outcomes assessment tools. These capabilities are complementary to Omnicell 340B, EnlivenHealth™, and IV Compounding Services, and can also support health systems moving to a consolidated service center approach to pharmacy operations.

Extends the Autonomous Pharmacy Vision: The ReCept acquisition furthers Omnicell’s commitment to deliver the hardware, software, and services to support the Autonomous Pharmacy vision, while advancing the evolution of the pharmacy care delivery model.

Complementary to Existing Solutions: The combination of ReCept’s specialty pharmacy expertise and operational capabilities and Omnicell’s commercial capabilities and client relationships, as well as its 340B program administration solutions and EnlivenHealth’s patient and member management tools, is expected to provide meaningful differentiation in the large and growing specialty pharmacy managed services organizations.

Transaction HighlightsUnder the terms of the agreement, Omnicell will pay total aggregate cash consideration of $100 million, subject to customary adjustments, at closing as provided for in the agreement and plan of merger. The transaction, which is expected to close by the end of the year, is subject to Hart-Scott-Rodino clearance and the satisfaction of other customary closing conditions. The ReCept business that is being acquired recorded approximately $24 million of total revenue (unaudited) for the 12 months ended September 30, 2021.

Omnicell will use cash available on its balance sheet to fund the transaction. Upon closing, the transaction is expected to be accretive to Omnicell’s non-GAAP EBITDA beginning in the first quarter of 2023.

About ReCeptReCept is the national leader in pharmacy and specialty pharmacy management solutions for health systems, federally qualified health centers, Ryan White HIV/AIDS programs and large physician provider groups. With over 40 years of experience in retail and specialty pharmacy management, ReCept has developed a proprietary and turnkey implementation and management platform, leveraging industry best practices to help pharmacies achieve their full potential. ReCept leads its customers through the entire pharmacy lifecycle, from store buildout to licensing to supporting ongoing operations to ensure future success. As a respected partner in the industry focused on quality patient care since 1978, ReCept helps its customers tackle the barriers associated with specialty medications, increasing patient access, improving patient adherence, and driving better outcomes for patients and prescribers. ReCept is a portfolio company of Generation Partners. To learn more about ReCept, visit:

About OmnicellSince 1992, Omnicell has been committed to transforming the pharmacy care delivery model to dramatically improve outcomes and lower costs. Through the vision of the autonomous pharmacy, a combination of automation, intelligence, and technology-enabled services, powered by a cloud data platform, Omnicell supports more efficient ways to manage medications across all care settings. Over 7,000 facilities worldwide use Omnicell automation and analytics solutions to help increase operational efficiency, reduce medication errors, deliver actionable intelligence, and improve patient safety. More than 60,000 institutional and retail pharmacies across North America and the United Kingdom leverage Omnicell’s innovative medication adherence and population health solutions to improve patient engagement and adherence to prescriptions, helping to reduce costly hospital readmissions. To learn more, visit


FDA Approves Oral Tx for CMV Infections – Livtencity

Just before Thanksgiving the FDA approved a new ORAL therapy, Livtencity (maribavir) from Takeda Pharmaceuticals, with an indication for the treatment of adults and pediatric patients (12 years of age and older and weighing at least 35 kg) with post-transplant cytomegalovirus (CMV) infection/disease that is refractory to treatment (with or without genotypic resistance) with ganciclovir, valganciclovir, cidofovir or foscarnet.

Post-transplant cytomegalovirus (CMV) is a rare disease and is one of the most common infections experienced by transplant recipients. The incidence rate varies by specific diagnosis with 16-56 % in Solid Organ Transplant (SOT) recipients and 30-70 % in Hematopoietic Stem Cell Transplant (HSCT) patients.

Takeda has not yet released the cost of Livtencity.

Takeda subsequently announced that the therapy will be available only through a select network of specialty pharmacies and distributors.

FDA Approves First Treatment for Common Type of Post-Transplant Infection that is Resistant to Other Drugs

November 23, 2021 — Today, the U.S. Food and Drug Administration approved Livtencity (maribavir) as the first drug for treating adults and pediatric patients (12 years of age and older and weighing at least 35 kilograms) with post-transplant cytomegalovirus (CMV) infection/disease that does not respond (with or without genetic mutations that cause resistance) to available antiviral treatment for CMV. Livtencity works by preventing the activity of human cytomegalovirus enzyme pUL97, thus blocking virus replication.

“Transplant recipients are at a much greater risk for complications and death when faced with a cytomegalovirus infection,” said John Farley, M.D., M.P.H., director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research. “Cytomegalovirus infections that are resistant or do not respond to available drugs are of even greater concern. Today’s approval helps meet a significant unmet medical need by providing a treatment option for this patient population.”

CMV is a type of herpes virus that commonly causes infection in patients after a stem cell or organ transplant. CMV infection can lead to CMV disease and have a major negative impact on transplant recipients, including loss of the transplanted organ and death.

Livtencity’s safety and efficacy were evaluated in a Phase 3, multicenter, open-label, active-controlled trial that compared Livtencity with a treatment assigned by a researcher running the study, which could include one or two of the following antivirals used to treat CMV: ganciclovir, valganciclovir, foscarnet or cidofovir. In the study, 352 transplant recipients with CMV infections who did not respond (with or without resistance) to treatment randomly received Livtencity or treatment assigned by a researcher for up to eight weeks.

The study compared the two groups’ plasma CMV DNA concentration levels at the end of the study’s eighth week, with efficacy defined as having a level below what is measurable. Of the 235 patients who received Livtencity, 56% had levels of CMV DNA below what was measurable versus 24% of the 117 patients who received an investigator-assigned treatment.

The most common side effects of Livtencity include taste disturbance, nausea, diarrhea, vomiting and fatigue. Livtencity may reduce the antiviral activity of ganciclovir and valganciclovir, so coadministration with these drugs is not recommended. Virologic failure due to resistance can occur during and after treatment with Livtencity, therefore CMV DNA levels should be monitored and Livtencity resistance should be checked if the patient is not responding to treatment or relapses.

Livtencity received Breakthrough Therapy and Priority Review designations for this indication. Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). Priority Review designation directs overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications.

The FDA granted the approval of Livtencity to Takeda Pharmaceuticals Company Limited.


FDA Approves New Infused Tx for Rare PEComa Cancers – Fyarro

Last week the FDA approved a new infused therapy, Fyarro (sirolimus protein-bound particles for injectable suspension / albumin-bound) from Aadi Bioscience, for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa).

Fyarro is the first and only approved therapy for adults with this condition, an ultra-rare and aggressive form of sarcoma with a strong female predominance. Malignant PEComas may arise in almost any body site (typically the uterus, retroperitoneum, lung, kidney, liver, genitourinary, and gastrointestinal tract.

It is estimated that there are only about 100-300 new patients per year in the United States.

Fyarro’s wholesale acquisition cost was announced at $6,785.00 for a single-use 100 milligram vial. Utilization is dependent on the patient’s body mass and any dosing modification resulting in a cost of approximately $468,000 per year. Aadi said that “Discounts, such as Medicaid rebates, sales to 340B covered entities and VA facilities among others, will reduce the net price by approximately 15% to 20%.”

Given the small patient population in the US it is expected that Fyarro will be launched through a specialty pharmacy distribution (direct to physician) program.

Aadi Bioscience Announces FDA Approval of its First Product FYARRO for Patients with Locally Advanced Unresectable or Metastatic Malignant Perivascular Epithelioid Cell Tumor (PEComa)

LOS ANGELES, Nov. 23, 2021 — Aadi Bioscience, Inc., a biopharmaceutical company focusing on precision therapies for genetically-defined cancers with alterations in mTOR pathway genes, today announced that the U.S. Food and Drug Administration (FDA) has approved FYARRO™ (sirolimus protein-bound particles for injectable suspension) (albumin-bound) for intravenous use for the treatment of adult patients with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumor (PEComa). FYARRO is the first and only FDA-approved treatment for advanced malignant PEComa in adults.

Neil Desai, Ph.D., Founder, Chief Executive Officer and President of Aadi, stated, “We are thrilled to have received full FDA-approval of FYARRO. The approval of FYARRO is a momentous event not just for Aadi but, importantly for advanced malignant PEComa patients. We reiterate that all of us at Aadi are incredibly grateful to all of the people with advanced malignant PEComa, their families and caregivers, as well as the healthcare professionals who made the FYARRO clinical studies possible.”

“The approval of FYARRO, the first approved drug for advanced malignant PEComa, an aggressive sarcoma with a poor prognosis and few treatment options, will provide physicians with a new weapon for treating patients with this rare disease,” added Andrew Wagner, M.D., Ph.D., a senior oncologist at Dana-Farber Cancer Institute and the Principal Investigator in the pivotal AMPECT registrational trial. “In our AMPECT trial, FYARRO demonstrated durable responses in mTOR inhibitor-naïve patients with locally advanced unresectable or metastatic PEComa, with an acceptable and manageable safety profile. This is a drug that will be welcomed by the physician community as the only approved therapeutic option for patients with advanced malignant PEComa.”

In the Phase 2 registrational AMPECT trial the overall response rate as assessed by independent review was 39% (12/31) with 2 patients achieving a Complete Response after prolonged follow up. The median duration of response has not been reached with a median follow-up of 36 months, and a range of 5.6 to 55.5+ months and ongoing. Among responders, 92% had a response lasting greater than or equal to 6 months; 67% had a response lasting greater than or equal to 12 months; and 58% had a response lasting greater than or equal to 2 years. As is the case with other therapeutics of the mTOR class, the FYARRO prescribing information includes warnings and precautions related to stomatitis, myelosuppression, infections, hypokalemia, hyperglycemia, interstitial lung disease, hemorrhage, and hypersensitivity reactions. Grade 3 non-hematologic events occurring in more than 10% of patients included stomatitis, rash, fatigue and infections. Grade 3 laboratory abnormalities occurring in more than 10% of patients that worsened from baseline included lymphocytopenia, increased glucose, and decreased potassium. For detailed important safety information, please see below.

Brendan Delaney, Chief Operating Officer of Aadi, added, “We have built a strong commercial team and devised a thoughtful strategy in preparation for FYARRO’s launch. With FYARRO’s demonstrated clinical profile we believe it will become a standard of care for advanced malignant PEComa. We look forward to engaging with physicians to educate the market about this new treatment.”

About Malignant PEComa
Advanced malignant PEComa, defined by the World Health Organization as ‘mesenchymal tumors composed of distinctive cells that show a focal association with blood-vessel walls and usually express both melanocytic and smooth muscle markers,’ are a rare subset of soft-tissue sarcomas, with an undefined cell of origin. While there is no formal epidemiology for malignant PEComa, with a female predominance) and can have an aggressive clinical course including distant metastases and ultimately death. The estimated prognosis based on retrospective reports is 12-16 months. Cytotoxic chemotherapies typically used for sarcoma show minimal benefit and there are currently no drugs approved for this disease. Malignant PEComas have been shown to frequently harbor mutations in the TSC1 and/or TSC2 genes that result in the activation of mTOR pathway making it a rational therapeutic target for this disease.

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