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FDA Approves New CAR T Tx for Multiple Myeloma – Abecma

Specialty pharmacies won’t be dispensing Abecma (idecabtagene vicleucel), approved by the FDA last week. Abecma is an INFUSED cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to, or whose disease has returned after, at least four different types of therapy. Abecma is the first cell-based gene therapy for Adult Patients with Multiple Myeloma.

As you should know by now, these treatments are personalized, made up of a patient’s own immune cells extracted and shipped to processing facility. There, the cells are genetically modified to target a particular protein that acts as a flag for the cancer. The modified cells are manipulated to create a 1-time treatment dose that is shipped back to the facility for infusion.

Celgene Corporation, a Bristol Myers Squibb company, set a price of $419,500 for Abecma. Analysts believe that Abecma will be one of the top-10 sales leaders by 2025. Top 10 by 2025.

We ask this question each time a CAR T therapy is approved…..
So why should specialty pharmacies want to know about a therapy that they can’t dispense?

Simple….. more than 500 gene-based therapies are in development. That should raise a flag for specialty pharmacies. We may start seeing even the most recent generation of SP dispensed ‘wonder drugs’ replaced by 1 dose gene therapies.

SPs that work the Oncology segment should know about ALL specialty therapies in the toolbox. With more approvals of these ‘space age’ therapies such expertise is important to build credibility with patients and Oncologists.

Is this term in your pharmaceutical vocabulary?…… CAR-T.
CAR-T stands for chimeric antigen receptor T cell (CAR-T) therapy in which T-cells are extracted from a patient’s white blood cells and genetically modified in special facilities to target a specific protein that binds to cancer cells. The modified cells are then infused in the patient.

FDA Approves First Cell-Based Gene Therapy for Adult Patients with Multiple Myeloma

March 27, 2021 — The U.S. Food and Drug Administration approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to, or whose disease has returned after, at least four prior lines (different types) of therapy. Abecma is the first cell-based gene therapy approved by the FDA for the treatment of multiple myeloma.

“The FDA remains committed to advancing novel treatment options for areas of unmet patient need,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “While there is no cure for multiple myeloma, the long-term outlook can vary based on the individual’s age and the stage of the condition at the time of diagnosis. Today’s approval provides a new treatment option for patients who have this uncommon type of cancer.”

Multiple myeloma is an uncommon type of blood cancer in which abnormal plasma cells build up in the bone marrow and form tumors in many bones of the body. This disease keeps the bone marrow from making enough healthy blood cells, which can result in low blood counts. Myeloma can also damage the bones and the kidneys and weaken the immune system. The exact cause of multiple myeloma is unknown. According to the National Cancer Institute, myeloma accounted for approximately 1.8% (32,000) of all new cancer cases in the United States in 2020.

Abecma is a B-cell maturation antigen (BCMA)-directed genetically modified autologous chimeric antigen receptor (CAR) T-cell therapy. Each dose of Abecma is a customized treatment created by using a patient’s own T-cells, which are a type of white blood cell, to help fight the myeloma. The patient’s T-cells are collected and genetically modified to include a new gene that facilitates targeting and killing myeloma cells. Once the cells are modified, they are infused back into the patient.

The safety and efficacy of Abecma were established in a multicenter study of 127 patients with relapsed myeloma (myeloma that returns after completion of treatment) and refractory myeloma (myeloma that does not respond to treatment), who received at least three prior antimyeloma lines of therapy. About 88% of patients in the study group had received four or more prior lines of antimyeloma therapy. Overall, 72% of patients partially or completely responded to the treatment. Of those studied, 28% of patients showed complete response—or disappearance of all signs of multiple myeloma—to Abecma, and 65% of this group remained in complete response to the treatment for at least 12 months.

Treatment with Abecma has the potential to cause severe side effects. The label carries a boxed warning for, cytokine release syndrome (CRS), hemophagocytic lymphohistiocytosis/ macrophage activation syndrome (HLH/MAS), neurologic toxicity, and prolonged cytopenia, all of which can be fatal or life-threatening. CRS and HLH/MAS are systemic responses to the activation and proliferation of CAR-T cells causing high fever and flu-like symptoms, and prolonged cytopenia is a drop in the number of a certain blood cell type for an extended period of time. The most common side effects of Abecma include CRS, infections, fatigue, musculoskeletal pain, and a weakened immune system. Side effects from treatment usually appear within the first one to two weeks after treatment, but some side effects may occur later. Patients with multiple myeloma should consult with their health care professionals to determine whether Abecma is an appropriate treatment for them.

Because of the risk of CRS and neurologic toxicities, Abecma is being approved with a risk evaluation and mitigation strategy which includes elements to assure safe use. The FDA is requiring that hospitals and their associated clinics that dispense Abecma be specially certified and staff involved in the prescribing, dispensing or administering of Abecma are trained to recognize and manage CRS and nervous system toxicities and other side effects of Abecma. Also, patients must be informed of the potential serious side effects and of the importance of promptly returning to the treatment site if side effects develop after receiving Abecma.

To further evaluate the long-term safety, the FDA is also requiring the manufacturer to conduct a post-marketing observational study involving patients treated with Abecma.

Abecma was granted Orphan Drug and Breakthrough Therapy designations by the FDA. Orphan Drug designation provides incentives to assist and encourage the development of drugs for rare diseases. 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). Breakthrough Therapy designation was granted based on sustained responses observed in patients with relapsed and refractory myeloma.

Drugs approved under expedited programs, such as Breakthrough Therapy designation, are held to the same approval standards as all other FDA approvals.

The FDA granted approval of Abecma to Celgene Corporation, a Bristol Myers Squibb company.


FDA Approves Yet Another ORAL Tx for MS – Ponvory

Last week the FDA approved a new ORAL therapy, Ponvory (ponesimod) from Janssen Pharmaceutical, to treat relapsing forms of Multiple Sclerosis.

There is no shortage of therapy options that cover the full spectrum of routes of administration….. oral – subQ – infused…. from a host of manufacturers. Ponvory will face stiff market competition now dominated by big names like Roche’s Ocrevus, Novartis’ Kesimpta and Gilenya, and Biogen’s Tecfidera.

Janssen did not disclose pricing but did say that the cost of Ponvory will be comparable to other market leader products which are now priced in the $7,000 – $10,000 per month range.

Janssen is promoting superior efficacy as the reason that prescribers and patients should turn to Ponvory. But, Ponvory will be slotted on payer formularies along side other MS therapies. Those patients paying copays for their MS treatment may see no financial benefit in making a switch. Similarly, a patient paying a coinsurance for a comparably priced therapy may see little out of pocket difference. However, it will be interesting to see if payers include Ponvory in their ‘preferred brand drug’ tier. Usually that is driven by the size of the rebate to the payer.

Janssen did not disclose whether Ponvory will launch through limited distribution. Our data indicates that virtually all leading MS therapies are currently only available through limited distribution.

FDA approves J&J’s multiple sclerosis treatment

March 19 (Reuters) – Johnson & Johnson will launch its newly approved drug for adults with relapsing multiple sclerosis (MS) in the United States in early April 2021 at a similar price point to rival treatments, the company’s unit said on Friday.

The U.S. Food and Drug Administration on Friday approved J&J’s treatment, Ponvory, treat relapsing forms of MS, Janssen Pharmaceutical Co said.

Multiple sclerosis is a debilitating neurological condition in which the immune system eats away at the protective covering of nerves.

J&J is pushing Ponvory as a once-daily oral treatment, as opposed to Kesimpta, which is injected by patients at home, or Ocrevus that is administered as an infusion in a clinic or hospital.

The approval will be cause of concern for Biogen, as it is facing Tecfidera patent expiration and increasing competition in the MS landscape.

The FDA approval was based on data from a two-year late-stage study where Ponvory demonstrated superior efficacy in significantly reducing annual relapses by about 30% compared to Sanofi’s approved MS drug Aubagio, the company said. The drug has also outdone Aubagio at reducing fatigue among patients.

J&J acquired Ponvory as part of its $30 billion buyout of Swiss biotech company Actelion in 2017, to diversify its drug portfolio as its biggest product, Remicade for arthritis, faced cheaper competition.

Ponvory is also under review by the European Medicines Agency (EMA)


No Shortage of Help for Health System Owned Specialty Pharmacies

Integrated specialty pharmacy services sound so upbeat it is hard to, well, beat. The integration being promoted by health system specialty pharmacies launched in recent years use integration of the whole health record as their key differentiator. Frankly, they consciously seek to make standalone specialty pharmacies look like a shabby second choices.

The article below outlines a new program being launched jointly by McKesson Corp and Vanderbilt Health Rx Solutions with the specific purpose of helping hospitals and health systems to open their own ‘owned and operated’ specialty pharmacies. They join a growing number of organizations pushing hospitals to invest in similar deals as we’ve previously reported.

Here’s the noteworthy excerpt from the press release……
“The new service will offer customized specialty pharmacy services and growth strategies to hospitals and health systems in every phase of their maturity. VHRxS’s portfolio of robust specialty pharmacy consulting services will help health systems improve patient and provider satisfaction while creating new revenue streams by fully integrating pharmacy services into specialty clinics.

In fairness, McKesson is not the first wholesaler to help shift specialty pharmacy volume to provider owned operations….. such as assisting Oncology practices to open their own in-office pharmacies. Like other wholesalers, McKesson’s specialty pharmacy division will eventually compete with the same hospital/provider owned specialty pharmacies they help create for the same prescriptions.

McKesson Expands Integrated Pharmacy Services for Specialty Clinics

McKesson, Vanderbilt Health Rx Solutions to leverage collective expertise to advance the mission of health system specialty pharmacies.

March 16, 2021 — IRVING, Texas–(BUSINESS WIRE)–McKesson Corporation, a global healthcare company, announces a new collaboration with Vanderbilt Health Rx Solutions (VHRxS), a national leader in specialty pharmacy strategy and implementation, to offer VHRxS’s full suite of specialty pharmacy consulting services to McKesson’s health system clients. This new, specialized offering from VHRxS will complement the experience of McKesson, which has offered industry-leading pharmacy services for more than 40 years, by bringing the expertise of VHRxS, which was founded by Vanderbilt University Medical Center to advance the mission of health systems specialty pharmacies nationwide.

“By collaborating with McKesson, we can better extend our expertise as a leading health system-based specialty pharmacy provider to help our peers. We know from experience that investing in your specialty pharmacy operations boosts patient and provider satisfaction, which reaps seen and unseen benefits many times over.”

The new service will offer customized specialty pharmacy services and growth strategies to hospitals and health systems in every phase of their maturity. VHRxS’s portfolio of robust specialty pharmacy consulting services will help health systems improve patient and provider satisfaction while creating new revenue streams by fully integrating pharmacy services into specialty clinics.

Scott Miller, president of McKesson Health Systems, shares, “Our teams work closely with customers to understand their challenges and this was a clear need for health systems addressing their specialty pharmacy capabilities. Along with our existing portfolio of services and solutions, this is one more way customers can achieve more for their hospitals and patients by creating new or strengthening existing revenue streams.”

McKesson’s pharmacy leaders and the VHRxS team of experienced, hands-on advisors understand the needs and challenges of health system specialty pharmacies. The teams have deep experience working with health systems in all stages of growth, from developing and implementing initial specialty pharmacy growth strategies to optimizing existing operations to reach the next level.

Jim Hayman, president of VHRxS and Chief Pharmacy Officer at Vanderbilt Health, states, “By collaborating with McKesson, we can better extend our expertise as a leading health system-based specialty pharmacy provider to help our peers. We know from experience that investing in your specialty pharmacy operations boosts patient and provider satisfaction, which reaps seen and unseen benefits many times over.”

The VHRxS consulting services will be customized to meet the needs of each health system as they address their own unique challenges and look to create sustainable specialty pharmacy programs, including:
– Opportunity assessments
– Strategic planning and guidance
– Operating model design and implementation
– Pharmacy accreditation support
– Facility and workflow design
– Access strategies for limited distribution drugs and payer networks


Which Specialty Pharmacies Were the Big Dogs in 2020?

Everyone in the specialty pharmacy business sits up and pays attention whenever a specialty pharmacy is acquired or announces big news. After all, good competition prompts smart companies to work harder to slog their way up the leaderboard.

Each year Drug Channels publishes its list of the Top 15 Specialty Pharmacies. (Can’t tell you how often that list is used in presentations.) We’ve seen the big box pharmacies retain their positions year after year. What has been more interesting is watching the smaller pharmacies elbow themselves onto the list. Many at the top of the list have acquired SPs at the bottom of the list. The smaller players know that and are eager to preen themselves for these suitors. Many ‘targets’ were on the list only for a year or so before getting gobbled up.

But, we have to wonder whether we’ll be seeing aspiring specialty pharmacies make the list in coming years. Just look at the companies that made this year’s list. (Click the link below for access to the article.)

The big box SPs are still there, albeit some of the names have changed, with a lion-sized market share. It is noteworthy that supermarket-owned specialty pharmacies now command the middle third of the list and half of the bottom third! But what is missing? There is not a single independent specialty pharmacy in stark comparison to the same list only a few years ago.

How will the list change in future years? Here’s our prediction.
First, assuming data can be extracted, the hospital and health system owned specialty pharmacies must start to appear. They have the potential to rise rapidly into the Top 15 club…. and might have even qualified this year. Without including these entities there will be a big hole in the analysis. Second, there has been a big uptick in the number of orphan therapies that are uber expensive. The specialty pharmacies that are winning limited (often exclusive) distribution deals for rare therapies can quickly rack up enough $$$$$s to also earn a spot on the list. Any buy-and-bill revenue from these direct-to-office therapies should also be credited to these organizations to reflect an accurate picture of the broader specialty pharmacy market.



FDA Approves New Oral for RCC – Fotivda

Last week the FDA approved a new ORAL Oncology therapy, Fotivda (tivozanib) from Aveo Oncology, for the treatment of adult patients with relapsed or refractory advanced renal cell carcinoma.

Renal cell carcinoma (RCC) is the most common type of kidney cancer and is among the ten most common cancers in both men and women. Approximately 74,000 new cases of kidney cancer are diagnosed with about 15,000 fatalities annually. The five-year survival rate is only 13%.

Fotivda will launch with a wholesale acquisition price of $24,150 per month. This compares with the $20,760 cost of Nexavar and Cabometyx (cabozantinib) with a WAC of about $21,600. Trial patients were given an average of 12.7 cycles which translates into $306,000 annually.

We have confirmed that Fotivda will launch on March 31st through limited distribution. No detail was provided as to the selected specialty pharmacy(ies).

AVEO Oncology Announces U.S. FDA Approval of Fotivda (tivozanib) for Relapsed or Refractory Advanced Renal Cell Carcinoma

FOTIVDA is the First Therapy Approved for Adult Patients with Relapsed or Refractory Advanced Renal Cell Carcinoma Following Two or More Prior Systemic Therapies

March 10, 2021 – BOSTON–(BUSINESS WIRE)–AVEO Oncology today announced that the U.S. Food and Drug Administration (FDA) has approved FOTIVDA® (tivozanib) for the treatment of adults with relapsed or refractory advanced renal cell carcinoma (RCC) who have received two or more prior systemic therapies. FOTIVDA is an oral, next-generation vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI).

“Today’s approval of FOTIVDA provides a new tool for treating patients with kidney cancer who have relapsed or become refractory to two or more prior systemic therapies,” said Brian Rini, MD, Chief of Clinical Trials at Vanderbilt Ingram Cancer Center and principal investigator of the TIVO-3 trial. “With advances in RCC treatment, patients are living longer, increasing the need for proven, well tolerated treatment options in the relapsed or refractory setting. The TIVO-3 study is the first positive Phase 3 study in RCC patients who received two or more prior systemic therapies, and also the first Phase 3 RCC study to include a predefined population of patients who have received prior immunotherapy, the current standard of care in earlier-line treatment. With this approval, I believe FOTIVDA represents an attractive intervention, and expect it to play a meaningful role in the evolving RCC treatment landscape.”

“We believe in FOTIVDA’s potential to provide a differentiated treatment option for the growing number of individuals in the U.S. with relapsed or refractory RCC, and today marks the culmination of many years of hard work and determination of many individuals to bring this therapy to patients,” said Michael Bailey, president and chief executive officer of AVEO. “With today’s approval, AVEO begins its journey as a commercial-stage company, a noteworthy accomplishment in our industry. On behalf of the entire AVEO team, I would like to thank all the patients, their families, and caregivers whose tireless efforts made this day possible.”

“Relapsed or refractory RCC is a devastating disease for which patient outcomes can be limited due to the tradeoff between tolerability and efficacy,” said Dena Battle, president of KCCure. “The FDA approval of FOTIVDA represents an exciting, meaningful advancement by providing a new treatment option for this patient population.”

The approval of FOTIVDA is based on AVEO’s pivotal Phase 3 study, TIVO-3, comparing FOTIVDA to sorafenib in relapsed or refractory advanced RCC following two or more prior systemic therapies. The application is also supported by three additional trials in RCC and includes safety data from over 1,000 clinical trial subjects.

Patients (n=350) enrolled in the TIVO-3 study were randomized 1:1 to receive either FOTIVDA or sorafenib. The main efficacy outcome measure was progression-free survival (PFS), assessed by a blinded independent radiology review committee. Other efficacy endpoints were overall survival (OS) and objective response rate (ORR).

Median PFS was 5.6 months (95% CI: 4.8, 7.3) in the FOTIVDA arm (n=175) compared with 3.9 months (95% CI: 3.7, 5.6) for those treated with sorafenib (HR 0.73; 95% CI: 0.56, 0.95; p=0.016). Median OS was 16.4 (95% CI: 13.4, 21.9) and 19.2 months (95% CI: 14.9, 24.2), for the FOTIVDA and sorafenib arms, respectively (HR 0.97; 95% CI: 0.75, 1.24). The ORR was 18% (95% CI: 12%, 24%) for the FOTIVDA arm and 8% (95% CI: 4%, 13%) for the sorafenib arm.

The most common (≥20%) adverse reactions were fatigue, hypertension, diarrhea, decreased appetite, nausea, dysphonia, hypothyroidism, cough, and stomatitis. The most common grade 3 or 4 laboratory abnormalities (≥5%) were decreased sodium, increased lipase, and decreased phosphate.

The recommended tivozanib dose is 1.34 mg once daily with or without food for 21 days every 28 days on treatment followed by 7 days off treatment (28 day cycle) until disease progression or unacceptable toxicity.

About FOTIVDA (tivozanib)
FOTIVDA (tivozanib) is an oral, next-generation vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI). It is a potent, selective inhibitor of VEGFRs 1, 2, and 3 with a long half-life designed to improve efficacy and tolerability. AVEO received U.S. Food and Drug Administration (FDA) approval for FOTIVDA on March 10, 2021 for the treatment of adult patients with relapsed or refractory advanced renal cell carcinoma (RCC) following two or more prior systemic therapies. FOTIVDA was approved in August 2017 in the European Union and other countries in the territory of its partner EUSA Pharma (UK) Limited for the treatment of adult patients with advanced RCC. FOTIVDA has been shown to significantly reduce regulatory T-cell production in preclinical models1. FOTIVDA was discovered by Kyowa Kirin.


Do You Need Home Infusion Therapy Accreditation?

Some years ago, payers demanded that specialty pharmacies obtain accreditation to even be considered for inclusion in their specialty pharmacy networks. It was an effective blocking tactic…. for a while. As many specialty pharmacies obtained specialty pharmacy accreditation those wraskley payers upped the game by requiring two accreditations.  

Since then, accreditations have been offered for a host of other niche business segments including Mail Order, Long Term Care, and Infusion Pharmacy. Oh, and throw in ‘Distinctions’ in Oncology, and Rare and Orphan Diseases, and more. Piling on accreditations is a strong argument that the specialty pharmacy meets virtually any requirement a payer could throw at them. 

The article below is timely as it raises awareness of the need for yet another accreditation.   CMS is requiring a special accreditation for delivery of Infusion services resulting in infusion confusion. If your organization hasn’t already moved to obtain the new accreditation it is time to play catch up. You may have accreditation in Infusion Pharmacy….. but, that’s not what CMS is looking for. The new version targets the delivery of nursing services in the home.  Just look at it like chocolate and peanut butter. So, read the article to get a full picture or just call your accrediting company and have a discussion.  


Home Infusion Therapy Accreditation Rolls Out

  • Compliance a Must Under CMS Regulations

 By Gina ShawSpecialty Pharmacy Continuum FEBRUARY 23, 2021 — As of Jan. 1, 2021, home infusion providers are officially required by the Centers for Medicare & Medicaid Services (CMS) to obtain accreditation to bill Medicare Part B for home infusion therapy (HIT) services. To make that transition to compliance a success, providers need to consider a number of important factors, according to three organizations now offering the accreditation. 

The first is to know that Medicare HIT accreditation is not necessarily for everyone, noted Jon Pritchett, PharmD, the program director overseeing pharmacy programs for the Accreditation Commission for Health Care (ACHC). “Some places just focus on pediatric infusions, and Medicare may not be a significant part of what they do,” he explained. “But for organizations that have a heavy Medicare population and a lot of those medications are covered, then there’s a good return on investment for this accreditation.” 

It’s also important to know why HIT accreditation has gained traction. Historically…………CLICK HERE TO READ THE FULL ARTICLE


Regulatory Compliance Looming for Hospital Owned Specialty Pharmacies

Almost ten years ago we started to raise the alarm of the threat that hospital-owned and operated specialty pharmacies pose to the Specialty Pharmacy (SP) industry given their ability to direct prescriptions ‘in house’. That trend has accelerated to the point where a large percentage of such systems are now in the business.

The article below is the first that I’ve seen that raises another red flag. This time it is a concern for the health systems, and it has everything to do with a practice that has gotten many a provider into hot water – self-referral.

The article points out that health systems that haven’t yet fully jumped into the Specialty Pharmacy pool are now contracting with outside entities to run their SP operations for them under contract. That kind of relationship can actually generate additional compliance issues and state and federal scrutiny particularly surrounding anti-kickback statutes, and how each ‘partner’ is reimbursed.

We learned in the recent merger of Shields Health and ExceleraRx, that they are promoting contract SP deals. Over the past decade they have helped most of the larger health systems open in-house SP operations. Smaller hospitals, however, are more likely to favor the contract vendor solution and wash their hands of day to day operational challenges. In that case the contract vendor would likely take on more responsibilities and, logically, get more compensation.

Regulators will focus on a number of factors to ensure that the arrangement between the health system and management company is compliant with key statutes. Read the article for a short list of red flags that could suggest a questionable contracting arrangements and consider the following excerpts.
“You can’t just have a management company coming in and taking over everything and giving the hospital a fee for this contractual arrangement.”
“If you can reduce costs, keep utilization low, improve patient outcomes and not be anti-competitive, then it should be a very good arrangement under the Anti-Kickback Statute.”

So, is the tsunami of hospitals and systems cannibalizing traditional specialty pharmacy ready to subside? Not likely…… just sayin’.

Hospitals Contracting for Specialty Pharmacy Services Face Compliance Issues

By Gina Shaw, Specialty Pharmacy Continuum
As more and more health systems enter the specialty pharmacy space, many are turning to outside third-party entities to manage their on-site specialty pharmacies. Although the arrangements offer many benefits, those involved in the partnerships need to be ready for state and federal scrutiny surrounding anti-kickback statutes, reimbursement and other compliance issues that could derail these arrangements if not managed proactively.

In these partnerships, the pharmacies are still owned by the hospitals, but they’re managed by outside vendors with specific expertise in specialty pharmacy operations—such as Recept, Shields Health Solutions and Trellis Rx—in exchange for fees and, sometimes, a share of the profits.

“There are core competencies that come with running specialty pharmacies, and depending on what expertise a hospital or health system already has on board, the management companies can really help with those, including access to payor networks, access to limited distribution drugs and assistance with accreditation,” said Todd Nova, JD, an attorney with Hall, Render, Killian, Heath & Lyman, an Indianapolis-based firm specializing in health law.

But contracting with a third-party specialty pharmacy management company poses a set of legal and regulatory issues that hospital and health-system compliance departments will have to navigate, said John W. Jones Jr., JD, a partner in the Philadelphia-based firm of Troutman Pepper Hamilton Sanders LLP.

Anti-kickback regulations are a major focus for HHS OIG, he noted. The regulators have highlighted several areas of concern that would raise red flags suggesting a questionable contracting arrangement, he said:
The owner (the hospital or health system) expands into a related line of business, which is dependent on referrals from, or other business generated by, its existing business.
The hospital or health system neither operates the new business itself nor commits substantial financial, capital or human resources to the venture—in this case, a specialty pharmacy. Instead, it substantially contracts out virtually all of the new business.
The third-party contractor is an established provider of the same services as the new line of business and, absent the contract, would be a competitor, providing items and services in its own right, billing insurers and patients in its own name, and collecting reimbursement.
The owner and third-party contractor share in the economic benefit (the profits) of the business, in the economic benefit of the owner’s new business.
Payments to the third-party management company vary by the value or volume of business generated for the specialty pharmacy by the hospital.

Jones explained that the Department of Health and Human Services Office of Inspector General (OIG)…………….



FDA Approves New Infused Tx for Rare Multiple Myeloma Condition – Pepaxto

The FDA approved a new infused specialty therapy last week, Pepaxto (melphalan flufenamide) from Oncopeptides AB, for patients with relapsed or refractory multiple myeloma following four or more lines of therapy. It is the first anticancer peptide-drug conjugate approved by the FDA.

Multiple myeloma is a cancer of plasma cells, a type of white blood cell which produces antibodies to help fight infection. Multiple myeloma causes cancer cells to accumulate in the bone marrow.

Approximately 7 per 100,000 Americans per year are diagnosed with multiple myeloma, making it a rare disease. However, the number of patients diagnosed with multiple myeloma is growing and the number of cases diagnosed annually is expected to almost double in 20 years. The average age for diagnosis is 70 years of age, and there is currently no cure.

Pepaxto is administered intravenously over 30 minutes on Day 1 of each 28-day cycle until disease progression or until unacceptable toxicity. Pepaxto is administered in combination with dexamethasone 40 mg orally or intravenously on Days 1, 8, 15 and 22 of each cycle.

Pricing for Pepaxto has not yet been confirmed.

Oncopeptides will release Pepaxto in the U.S. this month. They also announced that the product will be available through both wholesalers and specialty pharmacies. They did not specify whether they will implement a specialty pharmacy limited distribution program for this infused product. Given the small US patient population an LD program is likely.

FDA approves Oncopeptides’ PEPAXTO® ( melphalan flufenamide) for patients with relapsed or refractory multiple myeloma

STOCKHOLM, Feb. 26, 2021 /PRNewswire/ — Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a global biotech company focused on the development of therapies for difficult-to-treat hematological diseases, today announced that the U.S. Food and Drug Administration, FDA, has approved PEPAXTO® (melphalan flufenamide, also known as melflufen), in combination with dexamethasone, for the treatment of adult patients with relapsed or refractory multiple myeloma, who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody.

The product has been granted accelerated approval based on the phase 2 HORIZON study in relapsed or refractory multiple myeloma.

“The accelerated approval of PEPAXTO in the US is an important……….
Click here to access the full press release


FDA Approves New Infused Tx for DMD – Amondys 45

The FDA approved a new infused therapy last week, Amondys 45 (casimersen)) from Sarepta Therapeutics, with an indication for Duchenne Muscular Dystrophy (DMD). Amondys is further limited to treat the 8% of Duchenne patients with the exon 45 skipping genetic mutation. It is administered weekly by IV infusion in the home or infusion center.

DMD is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. The first symptoms are usually seen between 3 and 5 years of age and worsen over time. The disease often occurs in people without a known family history of the condition and primarily affects boys, but in rare cases it can affect girls. DMD occurs in about one of every 3,600 male infants worldwide. Since there are about 40,000 individuals with DMD in the US that translates into only ~3,200 candidates for the 8% that Amondys targets.

There are other dystrophin-gene-mutation, disease-modifying therapies for DMD on the market– Exondys 51 (skips dystrophin gene #51) and two that target gene #53, Vyondys 53 and Viltepso. All directly promote dystrophin production in the body.

People with DMD progressively lose the ability to perform activities independently and often require use of a wheelchair by their early teens. As the disease progresses, life-threatening heart and respiratory conditions can occur. Patients typically succumb to the disease in their 20s or 30s; however, disease severity and life expectancy vary.

Amondys 45 53’s price will be comparable to Vondys 53 and Exondys 51 (both are Sarepta therapies)….. neither of which is cheap. It is dosed based on weight. For example, a 50 lb. child would run a whopping $350,000 per year with older /larger patients costing upwards of $1 million annually.

All DMD products have been launched through limited distribution (direct-to-office / hospital/home infusion) using a specialty pharmacy. Since dosing is weekly home infusion will be common. We have been able to confirm that Sarepta has selected Orsini Specialty Pharmacy to support their limited distribution – direct-to-office (LD-DTO) program.

FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation

February 25, 2021 — Today, the U.S. Food and Drug Administration granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping The agency approved Amondys 45 based on an increase in dystrophin (a protein that helps keep muscle cells intact) production in skeletal muscle observed in patients treated with the therapy. This is the first FDA-approved targeted treatment for patients with this type of mutation. Approximately 8% of patients with DMD have a mutation that is amenable to exon 45 skipping.

“Developing drugs designed for patients with specific mutations is a critical part of personalized medicine,” said Eric Bastings, M.D., deputy director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research. “Today’s approval of Amondys 45 provides a targeted treatment option for Duchenne muscular dystrophy patients with this confirmed mutation.”

Amondys 45 was evaluated in a double-blind, placebo-controlled study in which 43 patients were randomized 2:1 to receive either intravenous Amondys 45 (30 mg/kg) or placebo. All patients were male, between 7 and 20 years of age, and had a genetically confirmed mutation of the DMD gene that is amenable to exon 45 skipping.

In the study, patients who received Amondys 45 showed a significantly greater increase in dystrophin protein levels from baseline to week 48 of treatment compared to those who received placebo.

The FDA has concluded that the data submitted by the applicant demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 45 skipping. A clinical benefit of the drug, including improved motor function, has not been established. In making this decision, the FDA considered the potential risks associated with the drug, the life-threatening and debilitating nature of the disease, and the lack of available therapy.

The most common side effects observed in DMD patients treated with Amondys 45 were upper respiratory tract infections, cough, fever, headache, joint pain and throat pain.

Although kidney toxicity was not observed in the Amondys 45 clinical studies, kidney toxicity was observed in the nonclinical studies. Kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking Amondys 45.

Amondys 45 was approved using the Accelerated Approval pathway, under which the FDA may approve drugs for serious conditions where there is unmet medical need and a drug is shown to have certain effects that are reasonably likely to predict a clinical benefit to patients. Further study is required to verify and describe anticipated clinical benefits of Amondys 45, and the sponsor is currently conducting an ongoing, double-blind, placebo-controlled, multicenter study designed to evaluate the safety and efficacy of Amondys 45 in ambulatory DMD patients.

The FDA granted this application Fast Track and Priority Review designations. Amondys 45 also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA is granting the approval to Sarepta Therapeutics, Inc.

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