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Ambulatory Infusion Centers Seeing a Resurgence?

Ambulatory Infusion Centers (AICs) are once again gaining traction….. but not for the first time. AICs have actually been around for a very long time. They were popular back at the end of the last century (1980s-90s) but came under fire by the same entities who are now looking at them to slay the cost dragon related to site of service. Back then payers accused AICs of creating derived utilization…. i.e., self referral. In response AICs encountered a wall of contracting resistance with payers…. but, the unintended consequence was to see that spend shift to the hospital outpatient department…. Ouch!

There was actually a resurgence in AICs in the early 2000s, but payers had yet to decide on a contracting strategy to contain infusion delivery costs. So, that brings us to the 2020s and AICs are now regaining their lost luster as payers have finally gotten serious about moving patients to lower cost sites of care.

The article below offers a good recap for the market dynamics pushing the AIC resurgence. Many specialty pharmacies have integrated home infusion into their service models and an AIC presents both a threat and opportunity for them. They might lose home infusion patients to an AIC (especially if the AIC facility is accessible and comfy) or they can offer their own AIC solutions and ingratiate themselves with payers to provide turnkey access to specialty medications and infusion delivery solutions. Given the number of new, very high cost infused specialty meds should also get SPs thinking. Just sayin’

It’s Prime Time For Ambulatory Infusion Centers

AUGUST 25, 2021 — The time is ripe for ambulatory infusion centers (AICs) to gain a stronger foothold in U.S. health care, a panel of experts said during the MHA 2021 Business Summit, held virtually.

“There’s a lot of tailwinds in this industry,” said Reece Norris, JD, a co-founder and the CEO of WeInfuse, an infusion software and consulting company. Payors and health plans are looking for sites of care (SOC) for infusion therapies that are more cost-effective than hospitals without compromising care, Mr. Norris said. With many autoimmune biologic specialty drugs having a limited distribution, the logistics of getting this in the home can be so burdensome that “it makes sense” to administer them in AICs, he noted.

“We are seeing payors push patients out of the hospital into more cost-effective sites of care,” Mr. Norris said. “We see this space continuing to expand, especially as the formulary of drugs gets larger.”

The SOC optimization trend has been building for several years, said Logan Davis, PharmD, MBA, the director of franchise development for Vital Care Infusion Services, in Meridian, Miss. The COVID-19 pandemic further demonstrated to the industry that being dependent on hospital-based infusion centers isn’t ideal, he said.

“There just isn’t enough capacity in the country at the moment” for stand-alone infusion centers, added Bryan Johnson, a co-founder and the CEO of WeInfuse, echoing the push by insurers to administer infused medications outside the hospital setting. “Wherever we can get a chair, an IV pole and a nurse, let’s try to make that site accessible to a patient, whether that’s in a home or through a management company. You’ll continue to see that—there’s just a lot of pressure coming down from the top.”

Adding to the market opportunity for expansion is a robust pipeline of specialty infusion drugs, including aducanumab (Aduhelm, Biogen), approved in June for the treatment of Alzheimer’s disease (see page 20 for more coverage), and several others still in trials for Parkinson’s disease, the panelists said. The boundaries are blurring among specialties, medications and diseases, Mr. Johnson noted. For example, infliximab (Remicade, Janssen) is being used for both gastrointestinal and rheumatic conditions. Medical specialists accustomed to having few infusion medications available aren’t likely to have office-based infusion suites, he added, and as specialty infusion medications cross into more common diseases covering more people, the demand for services will continue to rise.

Payors are reaching out to urgent care center chains, home infusion pharmacies and other regional service providers to open capacity to treat specialty medication patients in AICs, Mr. Norris said. In addition, some home infusion pharmacies are building infusion suites to capture the medical benefit market they could not traditionally access through the pharmacy benefit. Some home infusion pharmacies are adding infusion management services to their list of offerings. The infusion space is experiencing significant investment from both private equity and industry, Mr. Johnson added.

Why Launch?
There are numerous reasons to launch an infusion clinic, Dr. Davis said. Some of his franchisees wanted to be able to accept more referrals in their markets, access Medicare Part B benefits for referred patients, access drugs and better pricing limited to the infusion clinic class of trade, and access commercial payor contracts available to infusion clinics. But a sharp marketing and sales strategy is critical to success when starting out, the panelists said.

“This is not a ‘build it and they will come’ space,” Mr. Norris said. “You can have the best nurses, the nicest building and the finest equipment, but you have to earn the trust in the community from referring providers.” This can involve the hard work of cold calling, creating medication-specific order forms to ease work for referring physicians and treating referrals as gold. Communicate back to referring physicians how the infusion went, he advised, even if it’s just a quick treatment note.

When thinking about the physical space and build-out, most operators focus on factors such as infection control and meeting codes. But it’s equally important to consider patient amenities such as great parking, expanded hours, free Wi-Fi and coffee, Mr. Johnson said.

“Think more Starbucks and less stainless steel,” he said. “These patients have chronic conditions they will visit you with every few weeks, maybe for the rest of their lives. They want to be comfortable. You want them to look forward to coming to your facility.”

Mr. Johnson suggested these additional operational strategies:
Consult resources such as the National Infusion Center Association or the Infusion Nurses Society to note best practices and standards.
Hire a licensed professional (e.g., a physician, physician assistant or nurse practitioner) for clinical leadership. Their expertise will be needed in updating and maintaining protocols as well as dealing with any adverse events and communicating with other health care professionals.
Write and review emergency protocols and post them clearly for your clinical team.

Although the infusion itself is the same whether it’s administered in a hospital or another setting, AICs are different in terms of their regulation, reimbursements and class of trade, commented David Franklin, the president of Advanced Care Consulting Services, in Ray, Mich.

“There’s a really interesting new hybrid where independent infusion centers are partnering with physicians,” he said, “where physicians hold stock in the company, but they partner with independent infusion centers to operate the services through management contracts. That’s what’s going on now more than anything.”

Still, some of these independent infusion centers are facing reimbursement challenges because they may not be recognized by Medicare and Medicaid, Mr. Franklin noted.


New PBM Tackles Rx Claims Under the Medical Benefit

Most insurance programs still place provider administered medications under the medical benefit requiring that the meds be billed to the payer in the traditional claims submission process, not via the PBM. The efficiency of PBM adjudication is not at question. However, moving meds from the medical benefit to the pharmacy benefit is easier said than done. At least two big issues need to be overcome. First, most states require that a substantive change in the contract be approved by the insurance commission and that can take a lot of time. Secondly, the technology needs to be adapted to adjudicate using different reimbursement methodology that can vary from provider contract to contract.

The article below details a possible solution, one which PBMs have been talking about for more than a decade….. but have not yet delivered. TransactRx is launching a program to tackle the challenges related to claim adjudication for medical provider administered medications.

TransactRx cites an example where they have “moved 100% of specialty medications for a payer’s commercial lives from the medical benefit to the pharmacy benefit while retaining the providers’ buy-and-bill drug acquisition model and maintaining all sites of care for their patients.” That is no small feat…. if it is as simple as stated. It also requires that all providers in the ‘network’ submit standard pharmacy (NCPDP) claims, which would present major operational and technical changes for the practices.

It will be interesting to see if TransactRx is able to overcome the challenges and finally offer a sustainable alternative to traditional billing under the medical benefit.

TransactRx Launches SpecialtyXB, a better way to manage buy-and-bill medical benefit specialty drugs

MIAMI, Aug. 11, 2021 /PRNewswire/ — TransactRx announces the launch of SpecialtyXB an innovative solution for payers to move the coverage of medical provider administered specialty drugs from the medical benefit to the pharmacy benefit. TransactRx provides an integrated set of consulting services, provider network management and technology to enable medical providers across all places of service to submit claims and receive payments for provider purchased specialty drugs administered to patients. SpecialtyXB is the newest of the TransactRx cross benefit solutions that manage drug spend across medical and pharmacy benefits.

With SpecialtyXB everyone wins: Payers, Providers and Patients
Specialty medications represent nearly half of the drug spend in the US and are expected to increase by more than 11% this year. Just under 50% of the specialty drugs are administered by hospital outpatient clinics, infusion centers and medical providers that purchase the drugs and are billed under the medical benefit (a model called buy-and-bill). Over the past 10 years the PMPM dollars spent on commercial lives for these medical benefit specialty drugs is up almost 90%. The increases in medical drug spend create several challenges for payers as the utilization data is captured from batch medical claims and adjudicated on platforms that don’t have the same utilization management tools available on PBM platforms used to adjudicate pharmacy claims.

“To respond to these rising costs and management challenges some payers have started to move medical specialty drugs to a white-bagging model or a home infusion model which has decreased costs but has introduced issues with providers and with patients”, said Jack Guinan, CEO, TransactRx. “Our SpecialtyXB service provides an alternative solution that has proven to reduce costs, provide transparency to data, maintains provider relationships and supports the highest level of patient quality of care.”

SpecialtyXB is implemented on the TransactRx Cross-Benefit platform which seamlessly integrates medical and pharmacy systems to enable provider administered specialty drugs to be billed and reimbursed as standard pharmacy (NCPDP) claims. The solution includes the implementation of a medical provider pharmacy network managed by TransactRx which contracts with the payer’s PBM. The experienced TransactRx team, with over a decade of managing medical provider administered drug reimbursement, works with the payer, providers and pharmacy benefit manager to implement and operate the solution.

TransactRx successfully implemented SpecialtyXB with a regional Blue Cross and Blue Shield payer contracting with over 2700 medical providers that support their 640K commercial lives. The implementation resulted in moving 100% of specialty medications for the payer’s commercial lives from the medical benefit to the pharmacy benefit while retaining the providers’ buy-and-bill drug acquisition model and maintaining all sites of care for their patients. TransactRx is in discussions to launch SpecialtyXB with several other payers

For more information how TransactRx can help manage medical provider administered specialty drugs please visit our website

About TransactRx
TransactRx provides Cross Benefit Drug Reimbursement Solutions where everyone wins; payers, providers and patients. TransactRx has developed and operates its patented Cross Benefit Clearinghouse platform, operates national scale provider networks and provides consulting services that enable medical providers to be reimbursed for products and services covered under pharmacy benefits and enables pharmacy providers to be reimbursed for products and services covered under medical benefits.


HHOSPs Spice Up Their Specialty Pharmacy Differentiation

Hospital / Health System Owned Specialty Pharmacies (HHOSPs) have been saying that they are better qualified than independent SPs because they are integrated, essentially bolted to the hip of the hospital clinical team. The reality is that the hospital pharmacy is still the primary source of meds for inpatients, not the HHOSP. So, the claim about better integration leading to better outcomes may be a bit of editorial license.

We’ve been alert to claims in the HHOSP segment that validate enhanced differentiation. The article below details a program offered by Excelera that actually does show differentiation. They are sponsoring a “new specialty-pharmacy-focused educational offering in partnership with Aptitude Health, which provides critical insights for life sciences companies and healthcare professionals to improve cancer care and outcomes.” As described, the program seems to offer tangible content in hematology and oncology, arguably the most challenging area in specialty pharmacy and where specialty needs to be at the peak of its game.

It is also noteworthy that Excelera has partnered with Aptitude Health, which is closely aligned with pharmaceutical companies. Aptitude Health claims to be “…the global market leader in evidence-based solutions for life science companies seeking to identify, develop, and commercialize oncology and hematology innovations.” Independent SPs might do well to take a cue from the competition.

Excelera and Aptitude Health Offer New Specialty Pharmacy Focused Educational Resources for Cancer Care

August 05, 2021 — MINNEAPOLIS–(BUSINESS WIRE)–Excelera, a wholly-owned subsidiary of Shields Health Solutions and the company behind the Excelera Network, announced today a new specialty pharmacy-focused educational offering in partnership with Aptitude Health, which provides critical insights for life sciences companies and healthcare professionals to improve cancer care and outcomes. Together, the companies will publish key takeaways and potential implications for pharmacy professionals from Aptitude’s Evolving Paradigms in Care (EPICS) programs, which include congresses and global perspectives focused on major solid tumor and hematologic malignancies and the incorporation of new clinical data in disease management and treatment approaches. The new initiative underscores the importance of extending disease state-specific hematology and oncology insights in support of the integrated health system model and its multi-disciplinary approach to patient care.

“This collaboration with Excelera to create EPICS program content specifically for specialty pharmacy providers is exciting because it allows the pharmacy audience to benefit from insights into the emerging trends in the landscape and potential changes in prescribing patterns that might result from paradigm-changing studies in oncology and supportive care,” said Kerry Bradley, Vice President of Payor and Market Access, Aptitude Health.

Each EPICS report is based on the outcomes from closed-door roundtable discussions featuring clinical experts engaged in disease-specific discussions on translational and therapeutic advances and their application to clinical decision-making. Aptitude Health produces a high-level summary of the program discussion, as well as an Insights Report, which includes updates on drug development, patient management, strategic recommendations and the current and future disease landscape. The collaborations between Aptitude Health and Excelera will be triggered when information from the syndicated portions of these reports have potential importance for the pharmacy community.

“Excelera established its partnership with Aptitude Health in 2020 to leverage and share the expertise of healthcare providers specializing in cancer care with the specialty pharmacies in the Excelera Network,” said Diane Wolfe, VP Network Strategy at Excelera. “We are pleased to enhance that partnership with content developed from the perspective of specialty pharmacy experts in hematology and oncology who are aligned with academic medical centers, health systems and IDNs.”

About Aptitude Health
Aptitude Health is the global market leader in evidence-based solutions for life science companies seeking to identify, develop, and commercialize oncology and hematology innovations. With a proven track record of success, we transform the world’s growing scientific and clinical knowledge into better patient outcomes. The expertise of our team is backed by unparalleled access to global disease-state experts and healthcare professionals, making Aptitude Health the strategic partner of choice for the world’s most successful life science companies.


FDA Approves New ORAL Cancer Tx – Welireg

Last week the FDA approved a new ORAL therapy, Welireg (belzutifan) from Merck, for adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery. Welireg is the first systemic treatment for cancers linked to VHL which affects about 10,000 Americans annually.

Welireg works by blocking a protein known as HIF-2 alpha which limits the expression of certain genes associated with cellular proliferation and tumor growth. Welireg is administered once daily until disease progression or unacceptable toxicity. There is a serious risk of embryo-fetal harm which led the FDA to require a black box warning.

Merck confirmed that it will launch Welireg at a wholesale price of $26,400 monthly. Details relating to access were not released. However, it is highly likely to be placed in limited distribution due to the relatively small patient population and need to monitor patients for serious side effects and adverse events.

FDA approves belzutifan for cancers associated with von Hippel-Lindau disease

On August 13, 2021, the Food and Drug Administration approved belzutifan (Welireg, Merck), a hypoxia-inducible factor inhibitor for adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery.

Belzutifan was investigated in the ongoing Study 004 (NCT03401788), an open-label clinical trial in 61 patients with VHL-associated RCC (VHL-RCC) diagnosed based on a VHL germline alteration and with at least one measurable solid tumor localized to the kidney. Enrolled patients had other VHL-associated tumors, including CNS hemangioblastomas and pNET.

The primary efficacy endpoint was overall response rate (ORR) measured by radiology assessment, as assessed by an independent review committee using RECIST v1.1. Additional efficacy endpoints included duration of response (DoR), and time- to- response (TTR). An ORR of 49% (95% CI:36, 62) was reported in patients with VHL-associated RCC. All patients with VHL-RCC with a response were followed for a minimum of 18 months from the start of treatment. The median DoR was not reached; 56% of responders had DoR ≥ 12 months and a median TTR of 8 months. In patients with other VHL-associated non-RCC tumors, 24 patients with measurable CNS hemangioblastomas had an ORR of 63% and 12 patients with measurable pNET had an ORR of 83%. Median DoR was not reached, with 73% and 50% of patients having response durations ≥ 12 months for CNS hemangioblastomas and pNET, respectively.

The most common adverse reactions, including laboratory abnormalities, reported in ≥ 20% of patients who received belzutifan were decreased hemoglobin, anemia, fatigue, increased creatinine, headache, dizziness, increased glucose, and nausea. Anemia and hypoxia from belzutifan use can be severe. In Study 004, anemia occurred in 90% of patients and 7% had Grade 3 anemia. Patients should be transfused as clinically indicated. The use of erythropoiesis stimulating agents for treatment of anemia is not recommended in patients treated with belzutifan. In Study 004, hypoxia occurred in 1.6% of patients. Belzutifan can render some hormonal contraceptives ineffective, and belzutifan exposure during pregnancy can cause embryo-fetal harm.

The recommended belzutifan dosage is 120 mg administered orally once daily with or without food.


NCQA to Offer Specialty Pharmacy Accreditations

If you have made healthcare a career then you are likely to be familiar with NCQA. They’ve been around since the early 1990s measuring and then accrediting health plans. The NCQA designation continues to do for healthcare what the Good Housekeeping Seal of Approval does (since 1885) for home products…. spotlighting those organizations / manufacturers that meet exceptional quality standards. NCQA uses measurements, transparency and accountability to not only recognize top health plans, medical providers, and practices but to drive ongoing improvement.

It is with some surprise that NCQA has announced that it will start to offer an accreditation in specialty pharmacy as detailed in the press release below. First, there are a number of highly reputable accrediting organizations that health plans and manufacturers are familiar with and, more importantly, trust. Secondly, there has been a decline in the number of pharmacies breaking into specialty, so, attracting new signups may prove to be a challenge (refer to last week’s Report on URAC accreditations and discounts through ABC).

When a new competitor enters the market, they usually seek to differentiate themselves. But the key differentiation highlighted in the press release relates to value-based contracting (VBC). To be sure, VBC is an admirable quality measure…. but it is still a somewhat minor piece in the specialty pharmacy model.

It will be interesting to see if NCQA SP accreditation is embraced by the marketplace. Since most SPs already have several accreditations the big question will be “Is there room for one more?”

New NCQA Program Highlights Specialty Pharmacies for Commitment to Quality

WASHINGTON, Aug. 10, 2021 /PRNewswire/ — Today NCQA launched Specialty Pharmacy Accreditation, a service to help distinguish quality leaders and support value-based contracting in the rapidly growing field of specialty pharmacy.

“Specialty Pharmacy Accreditation addresses intersecting needs,” said Brad Ryan, MD, Chief Product Officer of NCQA. “Specialty pharmacies want to showcase their quality to stand out from peers so they can gain access to networks and pharmaceutical products. Drug manufacturers want to trust a specialty pharmacy can manage their products appropriately. And payers want to trust a specialty pharmacy can provide quality care to their members.”

In addition to helping pharmacies demonstrate their commitment to quality improvement and show they can be strong partners for value-based contracts, the new program helps pharmacies identify barriers to therapy initiation and adherence.

Organizations interested in NCQA Specialty Pharmacy Accreditation also stand to make meaningful improvements and contributions to quality by participating in a first-of-its-kind Measurement Lab.

The NCQA Specialty Pharmacy Measurement Lab is a collaborative working group composed of quality leaders from specialty pharmacies, health plans, pharmacy benefit managers, manufacturers and other industry stakeholders. Measurement Lab members learn best practices, pilot performance measures and influence the creation of digital measures as benchmarking and performance-based accreditation evolve.

Diverse organizations like those comprising the Measurement Lab helped design the new accreditation program. Organizations NCQA consulted include 11 specialty pharmacies, 9 health plans, 9 drug manufacturers, 6 pharmacy benefit managers and 3 patient advocacy groups. A 14-member Advisory Committee representing those industries—plus academia and CMS—developed the accreditation standards.

Pharmacies meet standards in four areas to earn Specialty Pharmacy Accreditation:
Patient Programs standards evaluate how well pharmacies support patients through the script journey—how patient-centered a pharmacy is and how it ensures patients experience their medications’ intended benefits.
Organization, Administration and Compliance standards evaluate how pharmacies train their staff and align with regulators and other stakeholders.
Specialty Pharmacy Operations standards consider how well pharmacies procure, secure, dispense and ship products to ensure product integrity.
Quality and Performance Improvement standards unify and build on the previous three standards to assess how pharmacies organize and measure continuous improvement.

Eligible organizations are licensed pharmacies that contract with drug manufacturers, health plans or pharmaceutical benefits managers to dispense specialty medications and provide supporting services. Accreditation surveys, which may be virtual or in-person, will begin in early 2022. NCQA determines accreditation status within 34 days after a survey. Specialty Pharmacy Accreditation lasts three years.

About NCQA
NCQA is a private, nonprofit organization dedicated to improving health care quality. NCQA Accredits and Certifies a wide range of health care organizations. It also recognizes clinicians and practices in key areas of performance. NCQA’s Healthcare Effectiveness Data and Information Set (HEDIS®) is the most widely used performance measurement tool in health care. NCQA’s website ( contains information to help consumers, employers and others make informed health care choices. NCQA can also be found on Twitter @ncqa, on Facebook at and on LinkedIn at


URAC Offers Discount on SP Accreditations

Now here’s something ya don’t see every day….. a leading accreditation body cutting a deal to offer a discount on specialty pharmacy accreditation.

Yes, URAC has hitched up with AmerisourceBergen (ASB) to offer a special rate for specialty pharmacy accreditation. It would appear that the discount is available to those pharmacies that use ASB as their wholesaler of record, which gives that pharmacy access to the ASB PSAO portfolio. It is not clear whether this is an offer for new (first time) URAC accreditations or if renewals are also included. Since the cost of a new and renewal accreditation can be pricey any savings could be welcome.

So why offer discounts at all?
The value of a specialty pharmacy accreditation is now well established. In fact, most SPs now obtain two or more accreditations….. often from competing agencies. In prior years we received many inquiries from pharmacies seeking to enter the SP segment….. and accreditation was always a key consideration. But, in the last 24 months, the number of pharmacies making the leap into SP has significantly dried up. That would have an impact on the number of new SP accreditations. As with any business, when market dynamics change marketing tactics respond to those changes. Just sayin’.

URAC and AmerisourceBergen Announce Strategic Relationship

Washington, DC, (GLOBE NEWSWIRE) — URAC, the nation’s largest independent health care accreditation organization and recognized leader in pharmacy accreditation, announced today that is has entered into an agreement with global health care company, AmerisourceBergen to provide discounted pricing on URAC accreditation and designation programs, as well as value-added services to AmerisourceBergen’s dispensing provider customers seeking new accreditation.

This relationship exemplifies URAC’s continuing goal of improving the quality of health care across the country, by making its meaningful accreditation programs available to even more organizations.

“Providers, hospitals and health systems are increasingly building specialty pharmacy and telehealth capabilities, and it’s vitally important they are accredited, so as to ensure the best outcomes for their patients,” said Shawn Griffin, MD, URAC’s President and CEO. “We are really excited about how this partnership will allow us to accredit more provider and hospitals pharmacies and telehealth practices.”

“URAC is glad to work with a world-class organization like AmerisourceBergen that shares the same goal of improving health care for individuals,” concluded Griffin.

“Improving health and wellness of patients through quality care is the cornerstone of our partnership with URAC,” said John Ryan, Vice President of Service Solutions and Operations at AmerisourceBergen. “We understand the critical role accreditation plays in enhancing patient outcomes, and we’re excited to offer this opportunity to our customers and assist them in the accreditation process.”

“We’re hopeful that this relationship will continue to enable pharmacies to provide accessible community-based care and create new and improved opportunities to access the medications their patients need.”

About URAC
Founded in 1990 as a non-profit organization, URAC is the independent leader in promoting health care quality and patient safety through renowned accreditation programs. We develop our evidence-based standards in collaboration with a wide array of stakeholders and industry experts. Our portfolio of accreditation and certification programs span the health care industry, addressing pharmacies, health care management and operations, telehealth, health plans, medical practices and more. URAC accreditation is a symbol of excellence for organizations to showcase their validated commitment to quality and accountability.


FDA Approves New IV Tx for Pompe Disease – Nexviazyme

Last week the FDA approved a new, INFUSED enzyme replacement therapy, Nexviazyme (avalglucosidase alfa-ngpt) from Genzyme, indicated to treat patients 1 year of age and older with late-onset Pompe disease.

Pompe disease is a progressive, multisystemic, debilitating, and often fatal neuromuscular disorder. Enzyme replacement therapy (ERT) is the only approved treatment for all patients with Pompe disease. The first enzyme treatment, Lumizyme (also from Genzyme), was approved in 2006.

There are three types of Pompe disease:

  • Classic infantile onset appears within a few months of birth.
  • Non-classic infantile onset appears at about 1 year of age.
  • Late-onset appears later in a child’s life, or even into the teen years or adulthood.

Pompe disease is estimated to affect 1 in 40,000 in the U.S. with a genetic carrier frequency estimated at 1 in 100. In Pompe disease, mutations in the GAA gene reduce or completely eliminate an essential enzyme needed to prevent excessive lysosomal glycogen accumulation throughout the body. Patients with Pompe disease experience heart enlargement, generalized skeletal muscle weakness, and a life expectancy of less than 2 years if untreated (classic infantile and non-Classic infantile onset).

Genzyme has announced pricing for Nexviazyme similar to that of its sister drug Lumizyme. An annual course of therapy for a pediatric patient will run $330,000 and $758,000 per year for an adult patient. Details on access were not announced but it is almost certain that Nexviazyme, as with Lumizyme, will launch through specialty pharmacy distribution – direct to provider.

FDA Approves New Treatment for Pompe Disease

August 06, 2021 — Today, the U.S. Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease.

Patients with Pompe disease have an enzyme deficiency that leads to the accumulation of a complex sugar, called glycogen, in skeletal and heart muscles, which cause muscle weakness and premature death from respiratory or heart failure. Normally, glycogen—the stored form of glucose—breaks down to release glucose into the bloodstream to be used as fuel for the cells.

“Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, M.D., deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research. “Today’s approval brings patients with Pompe disease another enzyme replacement therapy option for this rare disease. The FDA will continue to work with stakeholders to advance the development of additional new, effective and safe therapies for rare diseases, including Pompe disease.”

Nexviazyme, an enzyme replacement therapy, is an intravenous medication that helps reduce glycogen accumulation. The effectiveness of Nexviazyme for the treatment of Pompe disease was demonstrated in a study of 100 patients who were randomized to take Nexviazyme or another FDA-approved enzyme replacement therapy for Pompe disease. Treatment with Nexviazyme improved lung function similar to the improvement seen with the other therapy.

The most common side effects included headache, fatigue, diarrhea, nausea, joint pain (arthralgia), dizziness, muscle pain (myalgia), itching (pruritus), vomiting, difficulty breathing (dyspnea), skin redness (erythema), feeling of “pins and needles” (paresthesia) and skin welts (urticaria). Serious reactions included hypersensitivity reactions like anaphylaxis and infusion-associated reactions, including respiratory distress, chills and raised body temperature (pyrexia). Patients susceptible to fluid volume overload or with compromised cardiac or respiratory function may be at risk for serious acute cardiorespiratory failure.

The FDA granted this application Fast Track, Priority Review and Breakthrough Therapy designations. Nexviazyme also received an orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted the approval of Nexviazyme to Genzyme Corporation.


FDA Approves First Interchangeable Biosimilar – Semglee

It was way back in March of 2015 when the first biosimilar was approved. There was much consternation over the impact that biosimilars would have on the ‘land of plenty’ world of brand name drugs. As we all know, those concerns never realized as the biggest impediment to triggering a shift to the ‘copycat drugs’ was the lack of interchangeability.

The interchangeability ceiling was finally broken last week with the approval of the first interchangeable biosimilar insulin product, Semglee (insulin glargine-yfgn) from Mylan, indicated to improve glycemic control in adults and pediatric patients with Type 1 diabetes mellitus and in adults with Type 2 diabetes mellitus. Semglee is both biosimilar to and interchangeable with its reference product, Lantus (insulin glargine), a long-acting insulin analog.

It is ironic that the first interchangeable biosimilar is for a non-specialty therapy when much of the original concern over biosimilars related to specialty biologics. As we know, the biologics that have been approved and are commercially available are for therapies not generally dispensed by specialty pharmacies. The remainder are NOT available commercially as they have have been sidelined due to massive legal challenges usually related to patent protections.

Patients have long awaited relief from brand prices and have been disappointed. The approval of Semglee finally delivers measurable relief. The GoodRx price for Semglee with a coupon is only $104 compared to the reference product, Lantus, at more than $400.

Side notes
First, now that we have proof of interchangeability will the FDA be more inclined to grant that designation to other biosimilars dispensed by specialty pharmacies. Probably. However, having an interchangeable designation won’t remove the legal roadblocks can keep FDA approved biosimilars off the commercial market for years.

Second, Walmart announced that it will start selling its own private brand of analog insulin at prices up to a 75% discount to competing products. The private-brand insulin will cost $73 per vial and $86 per FlexPen, prices that are 58% to 75% lower than other insulin products. Details as to source for the ‘private brand’ were not released.

FDA Approves First Interchangeable Biosimilar Insulin Product for Treatment of Diabetes

July 28, 2021 — Today, the U.S. Food and Drug Administration approved the first interchangeable biosimilar insulin product, indicated to improve glycemic control in adults and pediatric patients with Type 1 diabetes mellitus and in adults with Type 2 diabetes mellitus. Semglee (insulin glargine-yfgn) is both biosimilar to, and interchangeable with (can be substituted for), its reference product Lantus (insulin glargine), a long-acting insulin analog. Semglee (insulin glargine-yfgn) is the first interchangeable biosimilar product approved in the U.S. for the treatment of diabetes. Approval of these insulin products can provide patients with additional safe, high-quality and potentially cost-effective options for treating diabetes.

“This is a momentous day for people who rely daily on insulin for treatment of diabetes, as biosimilar and interchangeable biosimilar products have the potential to greatly reduce health care costs,” said Acting FDA Commissioner Janet Woodcock, M.D. “Today’s approval of the first interchangeable biosimilar product furthers FDA’s longstanding commitment to support a competitive marketplace for biological products and ultimately empowers patients by helping to increase access to safe, effective and high-quality medications at potentially lower cost.”

Biological products include medications for treating many serious illnesses and chronic health conditions, including diabetes. A biosimilar is a biological product that is highly similar to, and has no clinically meaningful differences from, a biological product already approved by the FDA (also called the reference product). This means you can expect the same safety and effectiveness from the biosimilar as you would the reference product.

An interchangeable biosimilar product may be substituted for the reference product without the intervention of the prescriber. The substitution may occur at the pharmacy, a practice commonly called “pharmacy-level substitution”—much like how generic drugs are substituted for brand name drugs, subject to state pharmacy laws, which vary by state. Biosimilar and interchangeable biosimilar products have the potential to reduce health care costs, similar to how generic drugs have reduced costs. Biosimilars marketed in the U.S. typically have launched with initial list prices 15% to 35% lower than comparative list prices of the reference products.

More than 34 million people in the U.S. today have been diagnosed with diabetes, which is a chronic (long-lasting) health condition that affects how the body stores and uses sugars and other nutrients for energy. Most food is broken down into sugar (also called glucose) and released into the bloodstream. When blood sugar levels increase, it signals the pancreas to release insulin, which acts like a key to allow blood sugar to enter the body’s cells for use as energy. With diabetes, the body doesn’t make enough insulin to keep sugar levels regulated in the normal range.

“Access to affordable insulin is critical and long-acting insulin products, like insulin glargine, play an important role in the treatment of Types 1 and 2 diabetes mellitus,” said Peter Stein, M.D., director of the Office of New Drugs in the FDA’s Center for Drug Evaluation and Research. “The FDA’s high standards for approval mean health care professionals and patients can be confident in the safety and effectiveness of an interchangeable biosimilar product, just as they would for the reference product.”

All biological products are approved only after they meet the FDA’s rigorous approval standards. The approval of Semglee (insulin glargine-yfgn) as biosimilar to, and interchangeable with Lantus (insulin glargine), is based on evidence that showed the products are highly similar and that there are no clinically meaningful differences between Semglee (insulin glargine-yfgn) and Lantus (insulin glargine) in terms of safety, purity and potency (safety and effectiveness). It also showed that Semglee (insulin glargine-yfgn) can be expected to produce the same clinical result as Lantus (insulin glargine) in any given patient and that the risks in terms of safety or diminished efficacy of switching between Semglee (insulin glargine-yfgn) and Lantus (insulin glargine) is not greater than the risk of using Lantus (insulin glargine) without such switching.

Semglee (insulin glargine-yfgn), offered in 10 mL vials and 3 mL prefilled pens, is administered subcutaneously once daily. Dosing of Semglee (insulin glargine-yfgn), like Lantus, should be individualized based on the patient’s needs and should not be used during episodes of hypoglycemia (low blood sugar) or in patients with hypersensitivity to insulin glargine products. Also, like Lantus, Semglee (insulin glargine-yfgn) is not recommended for treating diabetic ketoacidosis. Semglee (insulin glargine-yfgn) may cause serious side effects, including hypoglycemia (low blood sugar), severe allergic reactions, hypokalemia (low potassium in blood) and heart failure. The most common side effects associated with insulin glargine products other than hypoglycemia include edema (fluid retention), lipodystrophy (pitting at the injection site), weight gain and allergic reactions, such as injection site reactions, rash, redness, pain and severe itching.

The FDA released new materials for health care providers to enhance understanding about biosimilar and interchangeable biosimilar products, including a fact sheet about interchangeable biosimilar products.

The FDA granted approval of Semglee (insulin glargine-yfgn) to Mylan Pharmaceuticals Inc.


FDA Approves New IV Tx for SLE – Saphnelo

The FDA recently approved a new therapy, Saphnelo (anifrolumab-fnia) from AstraZeneca, as a treatment for adults with systemic lupus erythematosus (SLE) who are receiving standard therapy. It is only the third FDA approval of a lupus therapy since 2011. Saphnelo is a first-in-class, type I interferon inhibitor administered by intravenous (IV) infusion.

SLE is the most common form of lupus affecting up to 300,000 people in the US. SLE disproportionately affects women of African-American, Hispanic or Asian lineage. It is a complex autoimmune condition that can affect any organ, and people often experience debilitating symptoms, long-term organ damage and poor health-related quality of life. There is no cure for SLE.

Saphnelo offers a HCP-administered option with a novel mechanism of action. Both Saphnelo and Benlysta are administered IV every 4 weeks in adults. Benlysta IV is also approved for SLE in pediatrics ≥ 5 years of age and a SC formulation is also approved for use in adults. Several other products with varying mechanisms of action are also in phase 3 trials for SLE. A SC formulation of Saphnelo for SLE is in development.

At this time pricing for Saphnelo has not been confirmed by AZ. As a point of reference the two leading therapies for Lupus, Benlysta (SLE, Infused-SC) and Lupkynis (LN, Oral) , are available with a GoodRx coupon as follows: Benlysta for +-$4,000 monthly and Lupkynis at +-$12,000 monthly. Benlysta IV is available through limited distribution, direct-to-office. As such, it is expected that Saphnelo will also be distributed, direct-to-office, via a traditional specialty distributor &/or a SP Distributor.

Lupus Foundation of America Celebrates FDA Approval of Saphnelo as a New Treatment for Lupus

This type I interferon receptor antagonist demonstrated early and sustained benefits for people with moderate to severe systemic lupus erythematosus.

The U.S. Food and Drug Administration (FDA) has approved Saphnelo (anifrolumab-fnia) as a treatment for adults with systemic lupus erythematosus (SLE) who are receiving standard therapy.

“After having only one therapy approved for lupus during the past 60 years, it is a cause for celebration to have two new treatment options approved in 2021 alone for this life-threatening autoimmune disease that affects an estimated 1.5 million Americans,” said Stevan W. Gibson, president and CEO, Lupus Foundation of America. “The pipeline of potential new treatments for lupus remains vibrant, and the Lupus Foundation of America continues its work to bring down barriers to lupus drug development and ensure that people with lupus will have access to the medications they need to improve their quality of life.”

Dr. George Tsokos, a member of the Lupus Foundation of America Medical-Scientific Advisory Council, Professor of Medicine, Harvard Medical School, Chief, Division of Rheumatology and Clinical Immunology, Beth Israel Deaconess Medical Center, noted that Saphnelo is the first lupus therapy designed to inhibit type I interferons. “We have known since the 1970’s that interferons were involved with lupus. With the approval of Saphnelo, we now have one more drug that allows us to translate valuable research knowledge into clinical practice with multiple benefits for our patients with lupus.”

Saphnelo inhibits a key protein in the immune system called the IFNAR receptor that acts as a transmitter, amplifying signals from tiny messengers called type I interferons. This process activates many parts of the immune system and can trigger major inflammation. Saphnelo dampens the excessive type I interferon signature found in up to 80% of adults with lupus and as many as 90% of children with the disease.

Combined data from two large-scale phase III clinical trials showed that more patients who received Saphnelo, in addition to standard therapy, had improvement than those who received placebo in addition to standard therapy. Saphnelo showed benefits on overall lupus disease activity, skin lupus and joints and the ability to taper down steroid doses.

Shannon Lee, a Lupus Foundation of America ambassador diagnosed with lupus 11 years ago, welcomed the news of a new treatment for lupus that can cut down on the use of oral corticosteroids, which can damage the body over time. “Steroids can cause many serious and life-threatening side effects, some of which I experienced while taking them before having to stop due to the issues they caused me,” said Lee. “Having another potential treatment option like Saphnelo is extremely exciting.”

“The approval of Saphnelo is the culmination of years of clinical development,” said Susan M. Manzi, MD, MPH, Lupus Foundation of America Board Chair and Medical Director and Chair, Allegheny Health Network Medicine Institute and Director, Lupus Center of Excellence. “Arriving at this important treatment milestone required the involvement of hundreds of people with lupus from around the world, who volunteered to participate in multiple clinical trials. We are grateful for their unselfish efforts, as well as those of basic and clinical physician-scientists who work tirelessly to support lupus drug development. Without them, we could not bring new life-changing therapies to those who need them.”

The Lupus Foundation of America’s research during the 1980s contributed to the development of Saphnelo through its support of research on interferons. In more recent years, the Foundation helped by educating people with lupus about the importance of participating in clinical trials for this new therapy. As part of its efforts to continue engaging people with lupus in ongoing research, the Foundation created an online data platform, RAY™ (Research Accelerated by You). This platform enables people with lupus and caregivers to share their lupus experiences to help researchers accelerate new treatments and improve disease outcomes.

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