Clinical Trials and Efficacy of ODOMZO
Overview of ODOMZO
ODOMZO, also known as sonidegib, is an oral, selective smoothened (SMO) inhibitor approved by the FDA for the treatment of adult patients with locally advanced basal cell carcinoma (laBCC) that has recurred following surgery or radiation therapy, or those who are not candidates for these treatments[4].
Clinical Trial Design and Participants
The FDA approval of ODOMZO was based on evidence from a pivotal Phase II clinical trial known as the BOLT (Basal cell carcinoma Outcomes in LDE225 Trial). This trial was a multicenter, double-blind, randomized, two-arm, non-comparative study that enrolled 230 patients with either locally advanced basal cell carcinoma (laBCC) or metastatic basal cell carcinoma (mBCC). Patients were randomly assigned to receive either ODOMZO 200 mg or 800 mg daily[1][3][4].
Efficacy Outcomes
The primary efficacy endpoint of the trial was the objective response rate (ORR), determined by blinded central review according to the modified Response Evaluation Criteria in Solid Tumors (mRECIST). For patients with laBCC treated with ODOMZO 200 mg, the ORR was 58% (95% CI: 45, 70), consisting of 5% complete responses and 53% partial responses. The median duration of response was not reached at the time of the 12-month analysis, and at the 42-month analysis, the ORR was 56% (95% CI: 43%, 68%) with a median duration of response of 26.1 months[1][3][4].
Subgroup Analysis
The trial did not show significant differences in the efficacy of ODOMZO among different sex, race, and age groups, although the number of patients in some subgroups was small. Most patients in the trials were white, and differences in response among races could not be determined[1].
Safety Profile
The most frequent grade 3 and 4 laboratory abnormalities occurring in at least 5% of patients included serum creatine kinase (CK) elevation and lipase elevation. ODOMZO also carries significant safety warnings, particularly regarding its potential to cause severe birth defects or stillbirths, necessitating strict birth control measures for female patients[4].
Market Analysis and Projection for ODOMZO
Market Authorization and Regulatory Status
ODOMZO has been approved in several countries, including the United States and Switzerland, for the treatment of advanced basal cell carcinoma. The drug is part of Novartis's expanding portfolio of targeted treatments for skin cancer[4].
Patent and Exclusivity Status
The patent and exclusivity status of ODOMZO is crucial for understanding its market dynamics. According to DrugPatentWatch, the generic entry opportunity date for ODOMZO is estimated to be around 2024-2025, based on the expiration of compound-claiming patents and regulatory exclusivity protections. This timeline can be influenced by various factors, including patent challenges and regulatory approvals[2].
Global Sales and Financial Projections
While specific financial projections for ODOMZO are not publicly available without a subscription to certain databases, it is part of a broader trend of increasing expenditure on specialty medicines. Specialty medicines, including those for rare and ultra-rare diseases, have been the greatest drivers of drug expenditure in recent years. The approval and market entry of drugs like ODOMZO contribute to the growing budget impact on healthcare systems, particularly in regions like the EU where new orphan drugs are expected to have a significant budget impact by 2025[5].
Competitive Landscape
ODOMZO competes in the market for advanced basal cell carcinoma treatments, which includes other targeted therapies like Erivedge (vismodegib). The competitive landscape is characterized by high price premiums for these specialty drugs, with ODOMZO offering a non-invasive treatment option that is distinct from surgical and radiation therapies[5].
Clinical Trials Update
Ongoing and Completed Trials
The BOLT trial was the pivotal study that led to the FDA approval of ODOMZO. In addition to this trial, there have been other clinical trials and studies conducted to further evaluate the efficacy and safety of sonidegib. For example, recent clinical trials include Phase 2 studies sponsored by groups such as Gruppo Oncologico del Nord-Ovest and Melanoma Institute Australia[2].
Future Directions
ODOMZO is currently in clinical development for other indications, expanding its potential use beyond locally advanced basal cell carcinoma. The drug's mechanism as a SMO inhibitor makes it a candidate for treating other diseases where the hedgehog (Hh) signaling pathway is implicated[4].
Key Takeaways
- Efficacy: ODOMZO demonstrated a significant objective response rate in patients with locally advanced basal cell carcinoma, with durable responses.
- Safety: The drug has a notable safety profile, including potential birth defects, and requires strict birth control measures.
- Market Authorization: Approved in several countries, including the US and Switzerland.
- Patent and Exclusivity: Generic entry expected around 2024-2025.
- Financial Projections: Part of the growing expenditure on specialty medicines.
- Competitive Landscape: Competes with other targeted therapies for advanced basal cell carcinoma.
FAQs
What is ODOMZO used for?
ODOMZO (sonidegib) is used for the treatment of adult patients with locally advanced basal cell carcinoma (laBCC) that has recurred following surgery or radiation therapy, or those who are not candidates for these treatments.
What were the key findings of the BOLT clinical trial?
The BOLT trial showed an objective response rate (ORR) of 58% in patients with laBCC treated with ODOMZO 200 mg, with a median duration of response of 26.1 months at the 42-month analysis.
What are the significant safety concerns associated with ODOMZO?
ODOMZO can cause severe birth defects or stillbirths, and females who can become pregnant must use birth control during treatment and for at least 20 months after the final dose.
When is the generic entry for ODOMZO expected?
The generic entry opportunity date for ODOMZO is estimated to be around 2024-2025, based on the expiration of compound-claiming patents and regulatory exclusivity protections.
Is ODOMZO being explored for other indications?
Yes, ODOMZO is currently in clinical development for other diseases where the hedgehog (Hh) signaling pathway is implicated.
Sources
- FDA, "Drug Trials Snapshot: ODOMZO," August 19, 2015.
- DrugPatentWatch, "ODOMZO Drug Patent Profile."
- Health Canada, "Summary Basis of Decision for Odomzo."
- Novartis, "FDA approves Novartis drug Odomzo® (sonidegib) for locally advanced basal cell carcinoma," July 24, 2015.
- IQVIA, "Pricing and Market Access Outlook."
