The FDA recently approved Papzimeos (zopapogene imadenovec-drba), a new therapy from Precigen Inc., for the treatment of adults with recurrent respiratory papillomatosis (RRP). This marks the first and only approved therapy available for the rare condition.

RRP is a debilitating and sometimes life-threatening disease caused by chronic infection with HPV types 6 or 11. The infection leads to recurrent benign tumors, or papillomas, within the respiratory tract. While typically noncancerous, papillomas consist of abnormal epithelial cell growths that can obstruct airways and require repeated surgical interventions.

RRP affects an estimated 27,000 adults in the United States, highlighting the unmet need for effective treatment options. Until now, patients have relied primarily on repeated surgical procedures to manage symptoms.

According to Precigen, Papzimeos published a defined dosing schedule: patients receive treatment on day one, followed by another dose at week two, and then once every four weeks thereafter. The therapy is supplied as a frozen suspension for subcutaneous injection. Prescribing information states that the therapy be rapidly thawed and immediately administered. Given the complexity of the disease, need for close monitoring, and preparation & handling, administration will take place in a specialist’s office.

Precigen has announced a list price of $115,000 per vial, totaling approximately $460,000 for the first twelve-week course of treatment.
Distribution and logistics details have not yet been disclosed.

CLICK HERE to access prescribing information

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Precigen Announces Full FDA Approval of Papzimeos (zopapogene imadenovec-drba), the First and Only Approved Therapy for the Treatment of Adults with Recurrent Respiratory Papillomatosis

CLICK HERE to access the press release

In the ever-evolving landscape of specialty pharmaceuticals, affordability and access remain front and center for patients, payers, and pharmacies alike. MedImpact Holdings Inc. is now poised to make a significant stride in this arena with the announcement of direct access to an unbranded biosimilar version of ustekinumab-aekn, a lower-cost alternative to the well-known reference drug, Stelara.

Breaking Down the Announcement
MedImpact’s new biosimilar will be distributed by Anda, affiliated with Teva Pharmaceuticals USA, Inc., and available for purchase from Birdi, Inc., MedImpact’s preferred partner. Most importantly, any licensed specialty pharmacy will have access to this product, potentially opening the door to wider market competition.

It is well known that a drug’s cost rises at each step in the supply chain. By adopting an unbranded strategy, MedImpact aims to bypass traditional markups and inefficiencies that inflate prices along the journey from manufacturer to patient. The program will provide specialty pharmacies with a new sourcing channel… not just MedImpact clients… more choice and control over their pharmacy benefit spending.

The model is also about transparency. Moving away from convoluted rebate structures and towards clear, upfront pricing gives payers and members access to true costs when the medication is dispensed.

Will this program start a sea change in the marketplace? MedImpact’s move is more than just another product launch… it’s a signal of changing tides in specialty pharmacy, where value, choice, and transparency are fast becoming the new standard. The availability date for the unbranded ustekinumab-aekn biosimilar is January 01, 2026.

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MedImpact Offers Low Cost, Unbranded Ustekinumab-aekn Biosimilar to Any Specialty Pharmacy in the US

CLICK HERE to read the full press release

The FDA recently approved a new ORAL therapy, Brinsupri (brensocatib) from Insmed Inc., indicated for the treatment of non-cystic fibrosis bronchiectasis in adult and pediatric patients 12 years of age and older.  Brinsupri is a dipeptidyl peptidase 1 (DPP1) inhibitor and is the first and only treatment for the condition. Non-Cystic Fibrosis Bronchiectasis is a serious, chronic lung disease that can lead to permanent lung damage.

Approximately 500,000 U.S. patients are diagnosed with Non-Cystic Fibrosis Bronchiectasis (NCFB). This approval offers patients effective treatment to defer the frequent exacerbations when symptoms worsen. Symptoms include coughing, increased mucus, shortness of breath and fatigue. Until now, treatment options were limited to antibiotics, airway clearance devices, and in severe cases, surgery.

Insmed will price Brinsupri at $88,000 per year before discounts during a company conference call.

The company also confirmed that Brinsupri will launch via specialty pharmacy limited distribution. Three SPs have confirmed their selection by Insmed as a distributor of the therapy, PantherRx Rare, Maxor Specialty Pharmacy, and Amber Specialty Pharmacy.

CLICK HERE to access prescribing information

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FDA Approves BRINSUPRI (brensocatib) as the First and Only Treatment for Non-Cystic Fibrosis Bronchiectasis, a Serious, Chronic Lung Disease

CLICK HERE to read the company press release

We are all aware that orphan disease drugs are typically very expensive. Yet, health plans tend to avoid risk-sharing arrangements that could significantly mitigate those costs. Reluctance stems from diverse factors such as the differences in dosing schedules (daily vs. weekly, etc.), administration routes (provider- vs. patient-administered), and tolerability issues, etc. Collectively, the abundant variables of each unique course of treatment create significant barriers to implementing and tracking risk contract performance.

But, the landscape is changing. Between 2020 and 2024, roughly 20-25 new orphan drugs gained FDA approval annually, with projections indicating 160-200 more could enter review by 2030. Many of these new treatments include gene and cell therapies, biologics, and high-cost small molecules for the same conditions. This increasing pipeline will likely lead to multiple high-priced options competing for the same orphan indication, intensifying market competition.

As more therapies emerge for the same conditions, will pricing pressures curb future growth, especially if new treatments do not demonstrate significant clinical benefits? To justify premium pricing amid stiff competition, later entrants might consider risk-sharing contracts, especially if they can substantiate clinically meaningful advantages.

Can risk contracting be that key differentiator? Risk contracts are particularly appealing when clinical data support superior outcomes, enabling companies to justify higher prices. Such contracting aligns well with the rising emphasis on value-based care in managed health plans and presents a strategic opportunity for new entrants to position themselves as committed to delivering that value.

With an expanding pipeline, building risk-sharing arrangements into drug launch strategies could be a necessity for orphan drug developers… provided they can minimize operational burdens on payers.

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Risk Contracting When Orphan Disease Space is Crowded

Health plans face challenges in risk arrangements for orphan drugs, but increasing competition may open doors for innovative pricing strategies.

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The FDA recently approved a new ORAL therapy, Hernexeos (zongertinib tablets) from Boehringer Ingelheim, indicated for the treatment of adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) and whose tumors have HER2 (ERBB2) tyrosine kinase domain activating mutations. Hernexeos is a tyrosine kinase inhibitor therapy. Hernexeos is being evaluated in ongoing trials, across a range of advanced solid tumors with HER2 alterations.

Lung cancer remains the leading cause of cancer-related deaths worldwide, with cases projected to surpass 3 million annually by 2040. Non-small cell lung cancer (NSCLC) accounts for the majority of diagnoses. Because early symptoms are often absent or misinterpreted, most patients are not identified until stage III or IV, when the disease has already spread to organs.

The company did not confirm pricing at the time of approval. A market analyst suggests that “Hernexeos will launch with an estimated $100,000 annual treatment cost and 10,000 eligible patients in the U.S., Hernexeos could generate $1 billion in peak sales—a figure that could rise with label expansions into first-line therapy or other HER2-mutant cancers.”

Onco360 Specialty Pharmacy has been selected as LD partner to distribute Hernexeos.

CLICK HERE to access prescribing information

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U.S. FDA grants accelerated approval to Boehringer’s Hernexeos as first orally administered targeted therapy for previously treated patients with HER2-mutant advanced NSCLC

CLICK HERE to read the company press release

Navitus Health Solutions recently announced a significant development in its transparency-driven pharmacy benefit management model. Beginning January 1, 2026, its clients will gain access to Costco Pharmacy’s cost-plus pricing for prescriptions at both retail and mail-order locations.

Under this model, drug costs are calculated based on Costco’s acquisition price, plus a fixed markup and a pharmacy service fee. For Navitus clients, this means direct access to the purchasing power of Costco, with the assurance that all savings are fully passed through under Navitus’ 100% pass-through PBM structure. Additionally, Costco cost-plus model clients will have the same clarity for brand and generic drugs across Costco locations.

The partnership also introduces a consumer-friendly convenience… members living within an hour of a Costco Pharmacy will be eligible for free same-day delivery through Instacart, expanding accessibility while reinforcing Costco’s reputation for efficiency and value.

Navitus emphasized that this heightened level of transparency equips payers with a powerful means to better control drug spend, while also ensuring clients benefit from fully auditable spending.

For the pharmaceutical industry, this partnership underscores the accelerating shift toward cost-plus pricing and transparent PBM models – an evolution that could fundamentally change how drug costs are managed across the healthcare system, particularly as stakeholders push back against longstanding frustrations with the traditional PBM approach.

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Costco Pharmacy partners with Navitus

CLICK HERE to read the press release

The FDA recently approved a new therapy, Zusduri (mitomycin) from UroGen Pharma. Zusduri, an alkylating drug, is indicated for the treatment of adult patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC).

Zusduri is administered only by intravesical instillation once weekly for six weeks delivered directly into the bladder thereby eliminating the need for surgery. The medication targets low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). More than 59,000 U.S. patients yearly face this recurrent disease.

The company did not disclose pricing for Zusduri at the time of approval.
Similarly, no details as to distribution were released.

CLICK HERE to access prescribing information

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UroGen’s Zusduri wins FDA’s blessing as first therapy for certain bladder cancer patients, despite negative adcomm vote

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The FDA recently approved a new oral therapy, Zegfrovy (sunvozertinib) from Dizal (Jiangsu) Pharmaceutical Co., Ltd., for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations (exon20ins), as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy.

Zegfrovy is the only approved targeted oral treatment for NSCLC with EGFR exon20ins. Zegfrovy will compete with Rybrevant, an infused therapy, in this patient segment. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Roughly 2% of NSCLC patients have the genetic type, though it is the third most common type of EGFR mutation.

Dizal has not announced the cost of Zegfrovy.
The company has not announced details for logistics or distribution.

CLICK HERE to access prescribing information

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Dizal’s Zegfrovy (sunvozertinib) Receives FDA Accelerated Approval as the Only Targeted Oral Treatment for Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations

CLICK HERE to read the press release

The 2025 JD Power US Pharmacy Study reveals significant shifts in pharmacy customer satisfaction and industry trends that pose major concerns for brick-and-mortar pharmacies.

An important finding of the study is that chain drugstore customers are significantly more likely than those using supermarkets or mass-market retailers to consider switching pharmacies within the next year, revealing a growing openness to change. This trend is amplified by rising satisfaction scores at mail-order pharmacies.

Brick-and-mortar pharmacy closures have accelerated. In 2024, 2,275 more closures were reported in the first nine months, intensifying concerns about patient access to in-person pharmacy care. Recent news reports suggest many more closures are in the offing.

Despite these closures, the 2024 JD Power’s report found that brick-and-mortar pharmacies still led in customer satisfaction compared to mail-order, supermarket, and mass-market retail outlets. However, the market is shifting quickly with supermarkets such as Wegmans and mass-market retailers like Sam’s Club ahead of chain drugstores in the 2025 report.

As alternative pharmacy models gain popularity – and market share – the industry stands at a pivotal moment. Persistent declines in satisfaction and widespread drugstore closures signal a market in flux, suggesting a transformational change may soon be in play.

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Brick-and-Mortar Pharmacy Satisfaction Decreases Below Supermarkets, Mail Order

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The FDA recently approved an ORAL therapy, Ekterly (sebetralstat) from KalVista Pharmaceuticals, indicated as an oral treatment for acute hereditary angioedema (HAE) attacks in patients aged 12 and older, after a delay attributed to FDA resource constraints.

HAE is a rare genetic disorder causing C1 esterase inhibitor dysfunction, leading to potentially life-threatening swelling attacks. The approval of Ekterly represents a significant advancement in the management of HAE, offering patients a more convenient, effective, and timely treatment option. This can lead to better overall patient outcomes and an improved quality of life for those affected by this rare genetic disorder. The company is promoting the therapy as a Potential Foundational Treatment with this oral form clinically superior to the time delays associated with injectable alternatives.

CLICK HERE to access prescribing information

KalVista said the Ekterly will cost $16,720 per dose. The drug will be packaged in a portable blister card that contains two doses.

The company has since disclosed details for logistics and distribution announcing that CVS Specialty, Accredo, Optum, and Orsini have been selected as the specialty pharmacy partners for Ekterly (sebetralstat).

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KalVista bounces back from FDA delay with approval for oral rare disease med Ekterly

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