Pomalidomide (Pomalyst®) approved by FDA for use in the United States

The United States (US) Food and Drugs Administration (FDA) has approved pomalidomide (brand name Pomalyst®) for use in myeloma patients with relapsed and refractory myeloma. This is the second new treatment to be approved in this setting by the FDA in the past 6 months, with Kyprolis™  approved for use in August last year.

Pomalyst is an immunomodulatory drug (IMiD) that works in a similar way to thalidomide and Revlimid®. It is specifically indicated for patients who have received at least two prior treatments, including Revlimid (lenalidomide) and Velcade® (bortezomib) and whose myeloma did not respond to treatment and progressed within 60 days of the last treatment.

Speaking about the announcement, Myeloma UK Chief Executive Eric Low said,

"The approval of Pomalyst by the FDA is an important milestone for myeloma patients as it ensures an additional treatment option for patients who have not responded to other drugs and whose myeloma is progressing. This is a clear area of unmet need and represents a significant development in the treatment of myeloma.

“We will now be working to ensure that Pomalyst is approved swiftly in Europe and made available for UK patients as soon as possible. Myeloma UK will also be ensuring early access to this treatment for relapsed, refractory myeloma patients through a clinical study due to be launched as part of the Myeloma UK Clinical Trial Network in spring 2013.”

The following Q&A provides more information about Pomalyst and the impact of the FDA decision for patients in the United Kingdom (UK).

Q&A

Why was Pomalyst approved by the FDA?

A licence for Pomalyst was granted by the FDA through its accelerated approval process on the strength of positive results from the ‘MM-003’ Phase III clinical study. As part of the accelerated approval process, the FDA licence can be updated to take into account new data on Pomalyst as more becomes available from ongoing studies.

The MM-003 study, involving 455 patients, compared the effectiveness of Pomalyst in combination with low-dose dexamethasone to high-dose dexamethasone alone in relapsed and refractory myeloma patients had previously been treated with both Revlimid and Velcade.

The results showed that Pomalyst in combination with low-dose dexamethasone significantly improved progression-free survival and had an overall survival advantage compared to high-dose dexamethasone.

The results of this study were presented at the ASH 2012 annual meeting. You can watch a video of Myeloma UK Chief Executive Eric Low discussing the central findings of this and other studies presented at the ASH 2012 annual meeting here. 

What are the benefits of Pomalyst?

As described above, Pomalyst is an immunomodulatory drug (IMiD) that has a similar mechanism of action to thalidomide and Revlimid – both are commonly used anti-myeloma treatments.

However, data from a number of different studies show that it is more potent and is effective even in relapsed and refractory patients who no longer respond to thalidomide, Revlimid and Velcade. The approval of Pomalyst is therefore very promising for this group of patients.

The MM-003 study also found that Pomalyst is associated with a better side-effect profile with fewer incidences of thrombocytopenia (low platelet count) and peripheral neuropathy than with Revlimid and thalidomide. There was, however, a higher incidence of neutropenia (low white blood cell count) in patients receiving Pomalyst compared to those receiving high-dose dexamethasone with levels comparable to Revlimid.

What does the FDA approval of Pomalyst mean for myeloma patients in the UK?

The FDA approval of Pomalyst does not apply to myeloma patients in the UK, as the FDA only covers decisions on new treatments for patients in the US. In Europe, new treatments must be approved by the European Medicines Agency (EMA) before they can become available. However, FDA approval is generally seen as positive news and normally sets a precedent for a treatment to be approved across Europe.

An application for a licence covering Pomalyst was submitted to the EMA in June 2012 and a decision is expected in the first quarter of 2013. Look out for an update on our website as soon as a decision has been reached.

In the interim, how can I access Pomalyst?

Whilst Pomalyst is not currently routinely available on the NHS, relapsed and/or refractory myeloma patients may be able to access it through clinical studies available in the UK.

The STRATUS study is looking at the efficacy of Pomalyst in combination with low-dose dexamethasone in patients with refractory or relapsed and refractory myeloma. To enter this study, patients must have received two previous lines of treatment that have included Velcade and Revlimid If you are interested in this study, speak to your haematologist in the first instance.

The Myeloma UK Clinical Trial Network is also setting up a Phase II clinical study for patients looking at Pomalyst in combination with dexamethasone. Look out for further information about this study on our website when it opens for recruitment this spring.

Where can I find more information about Pomalyst?

If you have any questions or comments about Pomalyst and the recent FDA approval, please contact the Myeloma Infoline on 0800 980 3332 or email kate.morgan@myeloma.org.uk