Month: November 2025

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.

CLICK HERE to read the full article