Here’s Celgene’s official press release about today’s FDA approval of pomalidomide.  I will review what the news means to me–and my fellow patients and caregivers–this weekend.

U.S. FOOD AND DRUG ADMINISTRATION APPROVES POMALYST® (POMALIDOMIDE) FOR THE TREATMENT OF PATIENTS WITH RELAPSED AND REFRACTORY MULTIPLE MYELOMA

Celgene logoSUMMIT, NJ – (Feb. 8, 2013) – Celgene Corporation (NASDAQ: CELG) today announced the U.S. Food and Drug Administration (FDA) has approved POMALYST® brand therapy (pomalidomide) for patients with multiple myeloma who have received at least two prior therapies including lenalidomide and bortezomib and have demonstrated disease progression on or within 60 days of completion of the last therapy.

Approval is based on response rate. Clinical benefit, such as improvement in survival or symptoms, has not been verified.

Supporting the approval were the results of MM-002, a phase II, randomized, open-label study evaluating pomalidomide (4 mg once daily on days 1-21 of each 28-day cycle) plus low-dose dexamethasone (40 mg per day given only on Days 1, 8, 15 and 22 of each 28-day cycle for patients 75 years or younger, or 20 mg per day given only on Days 1, 8, 15 and 22 of each 28-day cycle for patients greater than 75 years of age) versus pomalidomide (4 mg once daily on days 1-21 of each 28-day cycle) alone in patients with relapsed multiple myeloma who were refractory to their last myeloma therapy and had received lenalidomide and bortezomib.

Of the 221 patients that were evaluable for response, 29.2% (95% CI 21.0, 38.5) achieved a partial response or better in the pomalidomide plus low-dose dexamethasone arm compared to 7.4% (95% CI 3.3, 14.1) in the pomalidomide-alone arm. Overall Response Rate was based on responses assessed by the Independent Review Adjudication Committee (IRAC) based on the European Group for Blood and Marrow Transplantation (EMBT) criteria. The median duration of response for patients in the pomalidomide plus low-dose dexamethasone arm was 7.4 months (95% CI 5.1, 9.2) while the median has not yet been reached for the pomalidomide alone arm.

POMALYST is an analogue of thalidomide, is contraindicated in pregnancy and is only available through a restricted distribution program called POMALSYT REMS.TM Deep venous thrombosis (DVT) and pulmonary embolism (PE) occur in patients with multiple myeloma treated with POMALYST. Please see full Prescribing Information, including Boxed WARNINGS, CONTRAINDICATIONS, WARNINGS AND PRECAUTIONS, and ADVERSE REACTIONS.

In the study, 219 patients were evaluable for safety. The most common grade 3 or 4 adverse reactions (≥15%) in the pomalidomide plus low-dose dexamethasone arm versus pomalidomide alone respectively, were neutropenia (38% and 47%), anemia (21% and 22%), thrombocytopenia (19% and 22%), and pneumonia (23% and 16%).

POMALYST will only be available in the United States through POMALYST REMS,™ a restricted distribution program.

POMALYST® is a registered trademark of Celgene Corporation

About POMALYST®

POMALYST® oral therapy comprises pomalidomide, an IMiDs® compound. POMALYST and other IMiDs compounds continue to be evaluated in over 100 clinical trials.

POMALYST® (pomalidomide) is indicated for patients with multiple myeloma who have received at least two prior therapies including lenalidomide and bortezomib and have demonstrated disease progression on or within 60 days of completion of the last therapy. Approval is based on response rate. Clinical benefit, such as improvement in survival or symptoms, has not been verified.

It’s unclear why this particular study was featured.  There are others with much better numbers.  Note also that the FDA only approved pom to be used in a relatively small subset of patients, “who have received at least two prior therapies including lenalidomide and bortezomib and have demonstrated disease progression on or within 60 days of completion of the last therapy.”

Of course doctors can and will start using the drug “off label” immediately.  Don’t be surprised to start hearing about myeloma specialists that are prescribing the new “dream team” as a combination therapy: Kyprolis, POMALYST and dex.

More to follow, including links to important POM studies and anectodal evidence that supports the argument that pomalidomide may be the most effective multiple myeloma therapy agent developed to date–that is until Millennium’s oral proteasome inhibitor, MLN9708 is approved in a year or two.

Feel good, keep smiling and make sure you pay your insurance and Medicare premiums on time; Kyprolis and POM aren’t cheap!  Pat