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Quiz me this……….
What is a surrogate endpoint, and what the heck do they have to do with a drug obtaining Accelerated Approval?

People working in the life sciences segment must master a catalog of acronyms and complex programs that are difficult to fully understand…… let alone explain simply. The article today, from IQVIA, is a good primer for anyone to peruse to get relatively easy to explain answers to the question above. It is noteworthy that pathways are not limited to oncology….. so read on.

For readers of this Report that are not inclined to read the article, here are couple top line factoids that might help you pass the quiz.

Accelerated approval applications include surrogate endpoints. The dictionary defines ‘surrogate’ as “someone or something that replaces or is used instead of someone or something else; a substitute for another.” That might sound easy-peasy, but not when it comes to accelerated drug approvals.

A reasonable surrogate endpoint is based on strong evidence but lacks enough clinical evidence to be fully validated. Bundling more surrogate endpoint evidence supports accelerated approval applications.

But there are more details….. and you know what they say about what’s in the details.

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Unlocking Accelerated Approval Pathways: The Role of Surrogate Endpoints in Drug Development

CLICK HERE to access the article

Global Pharma industry has been buzzing about the looming tariffs from the US being applied for everything from Hello Kitty chotchkie to, ‘say it ain’t so’, pharmaceuticals.

The angst caused by a big spike in pricing is the easiest layer to understand….. but wait, other layers lurk below the surface. The article below spotlights the hot issues… “Critical components like active pharmaceutical ingredients (APIs), excipients, packaging materials and processing equipment will now be subject to duties, forcing companies to face immediate cost increases, compliance challenges and market uncertainty. With the prospect of escalating rates and retaliatory trade measures, the financial and operational stakes are climbing fast.”

The article provides a great analysis of the repercussions that U.S. tariffs are likely to have on the pharmaceutical industry, emphasizing the challenges and risks that companies must navigate in response to new trade policies.

Key concerns include:
Implications of Tariffs: The applications of tariffs on finished goods are likely to turn marketing forecasts on their heads given current uncertainty.
Supply Chain Risks: The impact of tariffs the direct and indirect manufacturing and production costs .
Importance of Trade Compliance: Accurate classification under the Harmonized Tariff Schedule is essential to avoid severe penalties. Misclassifications can disrupt supply chains and delay shipments.
Financial Strain on Generic Manufacturers: Tariffs disproportionately increase costs for generic drug manufacturers. This could have direct downstream effects on retail pharmacies and healthcare providers.
Challenges of Reshoring: Reshoring operations is a complex and costly endeavor that requires significant time and investment.
Global Regulatory Challenges: U.S. pharmaceutical companies may encounter increased scrutiny and barriers in international markets as other countries respond to U.S. trade measures, further complicating global supply chains.

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How Trump’s pharma tariffs disrupt drug supply chains

CLICK HERE to access the full article

A recent Executive Order directing HHS to develop price targets for imported drugs has really stirred up the hornet’s nest. It is common knowledge that many drugs in other countries cost a fraction of what we pay in the US. The goal of the Executive Order is to obtain ‘most favored nation’ (MFN) pricing for drugs….. in short, the US should pay the same or less than anywhere else. The Executive Order suggests that the prices of prescription drugs will drop “almost immediately, by 30% to 80%.”

The article spotlighted below is a doozie. Talk about a deep dive.
The authors have peeled the plan to its core and details where the Order goes beyond just MFN.

It is noteworthy that no drugs were targeted by name.
Similarly, no conditions for implementation or compliance were specified… yet.

One initiative, however, jumps out as a real jaw dropper.
The Executive Order directs HHS to “facilitate direct-to-consumer purchasing programs”. That sounds like no PBM, no health plan preauthorization, no pharmacy. We are still letting this concept sink in.

The Order was released with little specificity. However, HHS has already started to get gears in motion. Pharma will be watching HHS closely to determine how to respond.

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Unpacking President Trump’s New Executive Order on “Most-Favored-Nation” Prescription Drug Pricing

CLICK HERE to read the full article

The FDA recently approved a new treatment, Qfitlia (fitusiran) from Sanofi, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors (neutralizing antibodies).

Qfitlia uses a different pathway in the coagulation system to rebalance hemostasis. Qfitlia is approved for those with hemophilia A and B, but, unlike most treatments for the disorder, it also can be used by patients regardless of their inhibitor status.

Traditionally, blood factors are IV administered, however, Qfitlia is administered via a subcutaneous injection making it a convenient option. Qfitlia can be administered once every two months. Dosage and frequency can also be adjusted using a companion diagnostic test.

Over the last three years, the FDA has approved six new hemophilia drugs, including three gene therapies. Qfitlia jumps into this crowded treatment landscape but it is the only therapy approved for all types of hemophilia.

The company has not announced distribution details.

The company has set a list price for Qfitlia at $642,000 annually.

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FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors

CLICK HERE to access the FDA press release

Oncology products continue to be the top products driving the pharma marketplace. They are among the most expensive and complex due to the numerous iterations of the disease. As such, the market access challenges are, and will continue to be, extraordinary.

Today’s highlighted article delves into the intricate strategies required for achieving commercial success. It emphasizes that the high-stakes environment of cancer care, combined with rapid advancements in science and growing payer and regulatory pressures, necessitates a specialized approach that goes beyond traditional market access strategies. Seamlessly integrating market access, patient services, and medical affairs is essential to developing impactful and customized solutions.

Key topics include:
The Unique Complexity of Oncology Market Access
Three Pillars of Product Market Access for Oncology
The Impact of Oncology Pathways on Product Access
Building Trust with Healthcare Ecosystem Stakeholders
Financial Value Proposition and Payment Model Considerations
Key Financial Considerations
The Path Forward: Deep Oncology Expertise as a Necessity

This insightful article offers valuable perspectives that can be broadly applied across the pharmaceutical industry, particularly in scenarios where establishing effective market access is crucial.

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Oncology Brands Face Uniquely Complex Market Access Challenges: A Specialized Approach for Commercial Success

CLICK HERE to access the article

The FDA recently approved a therapy originally approved in 1983, Vykat XR (diazoxide choline) from Soleno Therapeutics, based on a breakthrough indication for the treatment of hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndrome (PWS). It is an oral medication in extended-release tablet form. Vykat XR is the first drug approved to treat hyperphagia.

Prader-Willi syndrome is a rare disease which leaves patients with low muscle tone, short stature and intellectual and developmental problems.

The Prader-Willi Syndrome Association USA estimates that the disorder occurs in 1 in every 15,000 live births, with more than 300,000 people worldwide affected. Soleno says that claims data show there are 10,000 PWS patients in the U.S

The company announced that, based on the average weight of patients in its clinical trials, Soleno will charge $466,200 per year for Vykat XR. The company expects it to launch in Q2. Panther Rare Rx has announced that it has been selected by Soleno to distribute Vykat XR

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Soleno breaks through with FDA nod for Prader-Willi syndrome treatment Vykat XR

CLICK HERE to read the full article

Announcements for newly approved specialty drugs often state that the product will be available through specialty pharmacy in limited distribution. However, the early press releases rarely specify the specialty pharmacy(ies) selected as the designated partner(s).

Here are six LD deals that have been publicly confirmed subsequent to their approvals.

PANTHERx Rare to Distribute Vigafyde

PANTHERx Rare was selected by Upsher-Smith for the distribution of Vigafyde (vigabatrin), a ready-to-use, concentrated oral solution approved for the treatment of infantile spasms in pediatric patients one month to two years old.

PANTHERx Rare to Distribute Vykat

PANTHERx Rare was selected by Soleno Therapeutics to distribute Vykat XR (diazoxide choline extended-release tablets), for the treatment of hyperphagia in individuals four years of age and older with Prader-Willi syndrome (PWS).

Orsini to Distribute Encelto

Orsini Specialty Pharmacy has been selected by Neurotech Pharmaceuticals, Inc., as the exclusive specialty pharmacy partner for Encelto (revakinagene taroretcel-lwey), an FDA-approved encapsulated cell therapy implant for the treatment of Macular Telangiectasia type 2 (MacTel).

CareMed to Distribute Vanrafia

CareMed has been selected by Novartis Pharmaceuticals to join the Limited Distribution Network for Vanrafia (atrasentan), indicated for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) and are at risk of rapid disease progression.

(CareMed is an independent Specialty Pharmacy that is an affiliate of Onco360 Specialty Pharmacy)

Soleo Health to Distribute Qfitlia

Soleo Health has been named the exclusive in-network specialty pharmacy for Qfitlia (fitusiran) by Sanofi. Qfitlia is the first antithrombin-lowering therapy for hemophilia. Qfitlia was approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B with or without factor VIII or IX inhibitors.

Onco360 to Distribute Avmapki – Fakzynja CO-PACK

Onco360 has been selected as a national pharmacy partner by Verastem Oncology for Avmapki – Fakzynja CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy. 

The past year has brought rough financial seas to Walgreens. As recently as this January we reported on Walgreens plans to possibly go private after closing underperforming retail stores and whole divisions such as VillageMD. 

We said then that Walgreens (WAGS) is facing a serious reversal of fortunes to the tune of $billions$ of dollars. The numbers are jaw dropping….. about 10 years ago Walgreens’ market cap was more than $100 billion. They ended the year with a valuation of only $8 billion.

Earlier this month WAGS had yet another major problem to resolve…. a federal anti-kickback charge by the US government. In this case they were able to settle for $50 million.

This story is less about Walgreens and more about the fact that the government is still vigilant about anti-kickback schemes such as that conducted by WAGS. While the financial penalty my only sting, the impact to the company reputation and ability to qualify for other government programs can be impacted. Remember, under the federal False Claims Act, whistleblowers are entitled to a portion of whatever money the government is able to recover.

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Walgreens to pay $50M in anti-kickback case

• Company gave illegal discounts, government says
• Walgreens marketed a savings plan without checking if people were covered by Medicare or Medicaid, officials said. 

CLICK HERE to read the full article

Today’s Report spotlights a relatively insignificant event in therapy distribution. We are talking about COSTCO, yes the warehouse retailer. What they did is simply add a new drug to their pharmacy inventory, a little therapy called Steqeyma. In doing so, however, it reinforced a major shift in the specialty pharmacy industry.

Steqeyma (ustekinumab-stba), a biosimilar to Stelara (ustekinumab), has been added to the Costco Member Prescription Program. Still not impressed? Read on….

Adding a biosmilar to a prescription drug discount card program is not hugely surprising.

What is surprising is that COSTCO has been discreetly adding other specialty pharmacy therapies for several years. In fact, they are affirming that there are now “COSTCO Specialty Pharmacies.”

To be fair, a host of other retailers have been playing in the specialty pharmacy sand box for years. That COSTCO appears to be fully committed to be a player says a lot about the SP market and the shifting competitive landscape. 

The company said that the formulary including the specialty products is currently available for self-funded employer plans and Costco members who are uninsured and want to pay cash for their Steqeyma prescription or who have been denied coverage by their insurers.

FYI…. Steqeyma is available in both subcutaneous injection and intravenous infusion and is indicated for the treatment of plaque psoriasis (PsO) and psoriatic arthritis (PsA) in adult and pediatric patients, as well as Crohn’s disease (CD) and ulcerative colitis (UC) in adult patients.

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Celltrion’s Steqeyma (ustekinumab-stba), now added to the Costco Member Prescription Program

CLICK HERE to read the full article

The FDA recently approved one… no wait, two… no wait, is it really one times two denosumab biosimilars…..?? The/these approval/s include Ospomyv (denosumab-dssb) and Xbryk (denosumab-dssb)… it/they are from Samsung Bioepis.

NO WAIT… the FDA approved yet another pair of denosumab biosimilars, Stoboclo (denosumab-bmwo) and Osenvelt (denosumab-bmwo) from Celltrion.

And if that’s not enough, Wyost and Jubbonti were approved as Prolia and Xgeva biosims in 2024.

All of these biosims were approved as interchangeable with the reference products, Prolia and Xgeva from Amgen.

You should be familiar with the big differences between these ‘twin’ injectables. But, one has a twice yearly administration schedule… the other has a much more frequent dosing schedule. While both products are included in the same FDA approved prescribing information document you need to scroll way down (e.g., page 96) to read the details for the ‘twin’s’ therapy detail and the dosing information for completely different indications. 

CLICK HERE to access prescribing information for Ospomyv and Xbryk

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FDA, EMA Approve Second Pair of Denosumab Biosimilars

CLICK HERE to read the full article