Anktiva is one of the latest cancer treatments to get FDA approval. At the moment, it is approved in the USA for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). In Saudi Arabia, Anktiva is also approved for use in non-small cell lung cancer (NSCLC). 

While Anktiva is currently approved for these specific indications, it is also being studied in other cancers – suggesting its potential may extend beyond its current use.

In some cases, patients and their doctors may consider off-label prescribing based on emerging evidence, rather than waiting (often years) for approval from agencies like the U.S. Food and Drug Administration.

In this article, we explain what off-label use means, which indications may be considered based on current evidence, and how patients may be able to access Anktiva if prescribed off-label.

What is off label prescribing?

Off-label prescribing means using an approved medicine in a way that isn’t listed in its official approval – such as for a different condition, dose, or group of patients.

Once a medicine like Anktiva is approved, doctors can legally prescribe it off-label if they believe it’s appropriate. This is common in cancer care, where patients may need to explore all possible options within a limited timeframe.

For example, although Anktiva is approved for BCG-unresponsive NMIBC, a doctor might consider using it for other stages of bladder cancer or even different cancer types, based on early evidence and the patient’s specific situation.

Is off-label prescribing legal?

Yes, off-label prescribing is legal and common in medicine, including oncology. While medicines are approved for specific uses based on data submitted to agencies like the U.S. FDA, doctors are not limited to those uses.

In some cases, strong clinical trial data for a new indication may already exist, but the manufacturer has not yet submitted it for approval – or it is still under review. For example, although Anktiva is approved for BCG-unresponsive NMIBC, a doctor might consider using it for other stages of bladder cancer or even different cancer types, based on early evidence and the patient’s specific situation. 

As long as the decision is supported by sound medical judgment and scientific data, doctors can prescribe off-label. This flexibility allows them to tailor treatment to each patient – especially when standard options are limited or no longer effective.

When might your doctor consider Anktiva off-label use?

A doctor may consider using Anktiva off-label when there is a good scientific reason to believe it could help beyond its approved use – especially in conditions with few treatment options, where trying something new may be justified. This could be based on early clinical trial results, case reports, or how the medicine works.

For example, in non-small cell lung cancer (NSCLC), a doctor in the United States could technically prescribe Anktiva off-label even though it is not yet approved for this use there (while it may be used in countries like Saudi Arabia). In making that decision, the doctor might review available clinical trial data and, in some cases, consult with colleagues who already have experience using the treatment in that setting.

Can Anktiva be used off label in your country?

Most likely, yes. Off-label prescribing is widely accepted and practiced worldwide, with an EU survey indicating that between 3-95% of adult prescriptions in the region may be for off-label use.

With this in mind, if your doctor considers that Anktiva may be a suitable treatment for your condition and situation, in most cases they can legally prescribe it to you – regardless of whether it is locally approved.

Some countries may have stricter regulations for off-label use, requiring additional approvals or justifications. You can explore these specific requirements together with your treating doctor, if you want to consider Anktiva off-label use. 

For which conditions could Anktiva be prescribed off-label?

While Anktiva is approved for BCG-unresponsive NMIBC (and for NSCLC in Saudi Arabia), it is also being studied in other cancers. Doctors may technically prescribe it off-label for a number of indications, but in practice they are most likely to do so for indications where there is already solid clinical trial data or a clear scientific rationale.

Below are the Anktiva indications where research is most advanced, and where off-label use may be more realistically considered.

Non-Small Cell Lung Cancer (NSCLC)

Anktiva is already approved in Saudi Arabia for use in metastatic NSCLC. This increases the likelihood of a doctor elsewhere considering off-label use for this indication.

The approval was based on the Phase III study QUILT-3.055 in people with advanced lung cancer (NSCLC) who had already received treatment. It compared Anktiva® plus an immunotherapy drug to standard chemotherapy.

Key findings included:

• In studies including 151 patients, Anktiva helped restore immune function, which was linked to longer survival;
• Patients receiving Anktiva plus immunotherapy showed a clear increase in immune cell levels compared to immunotherapy alone;
• 77% of patients in later-line treatment maintained or regained healthy immune cell levels;
• Patients whose immune system improved lived longer: about 16.2 months vs 11.8 months;
• Those with stronger immune recovery lived even longer: about 21.1 months.

Pancreatic cancer

Anktiva is not yet approved anywhere for use in pancreatic cancer. However, the FDA granted it an RMAT (Regenerative Medicine Advanced Therapy) designation, recognizing it as a promising therapy in development, for which clinical data show restoring immune cells and longer survival across multiple cancers, including pancreatic cancer. As such, your doctor may consider off-label use for this indication.

One of the key studies currently focused on pancreatic cancer is the QUILT-88 trial, which combines Anktiva with chemotherapy, radiation, and NK cell therapy. 

Results presented at the American Society of Clinical Oncology 2025 meeting showed encouraging outcomes for this difficult-to-treat population:

• Patients treated in third-line or later settings lived about 5.7–6.2 months on average, which is relatively strong given the lack of standard options;
• Some patients experienced much longer responses, with a few remaining on treatment for up to ~2 years;
• Better outcomes were linked to higher immune cell levels (lymphocytes).

Glioblastoma

Glioblastoma is another area where Anktiva is being studied, particularly in patients whose disease has returned after standard treatments. One of the key studies is the QUILT-3.078 trial. Early Phase II results show encouraging signals in this difficult-to-treat cancer:

• Median survival has not yet been reached;
• Some patients have survived 12 months or longer after recurrence, which is notable compared to typical outcomes (6–9 months);
• Patients started with very low immune cell levels, but these increased quickly and remained stable during treatment.

These findings support the idea that restoring the immune system may help improve outcomes in glioblastoma, where treatment options are limited and prognosis is poor.

Because of this, doctors may consider Anktiva off-label in highly selected glioblastoma cases – particularly where there is a strong rationale based on immune recovery and emerging clinical data. 

Overview of Potential Anktiva Off-Label Indications

How to get Anktiva if you have an off-label prescription

If your doctor prescribes Anktiva off-label, how you access it will depend on where you live.

• If you are in a country where Anktiva is already approved (even for a different indication), it may be possible to obtain it locally with a valid prescription. However, reimbursement is not guaranteed – especially for off-label use – so patients may need to cover the cost themselves.
• If you are in a country where Anktiva is not yet approved at all, you can get the medicine through Named Patient Import (also called personal importation). This allows you to legally import a medicine for personal use.
  In some cases, even patients in countries like the United States may face challenges accessing Anktiva locally for off-label use. When that happens, sourcing the medicine from another country where it is approved can sometimes be a faster – and more affordable – option.

In all of these scenarios, having the right support is key. We helps patients and doctors access novel medicines before they’re available locally. We’ll handle the regulatory steps, sourcing, and delivery so you can focus on your treatment. To get started, simply request Anktiva.