Why Is Celgene’s POMALYST (Pomalidomide) FDA Approval For Advanced Myeloma Such A Big Deal?

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FDA approval of POMALYST, whats the big deal

The internet is rife with articles on the new drug, POMALYST’s FDA approval for advanced multiple myeloma.

The US Food and Drug Administration approved pomalidomide to treat patients with multiple myeloma who showed disease progression post treatment with other myeloma drugs. The drug will be marketed under the brand name POMALYST.

Pomalyst is the new addition to the armamentarium of immunomodulatory drugs used in treating myeloma. Pomalyst joins chemically similar Revlimid and Thalidomide and can be orally administered like them. It is designed to initiate the body’s immune system to destroy myeloma cells.

The FDA approval for Pomalyst came on the back of encouraging data from a phase II study (MM-002) on the candidate.

Pomalyst’s safety and effectiveness was evaluated in a clinical trial of 221 patients with relapsed or refractory multiple myeloma. The trial was designed to measure the number of patients whose cancer completely or partially disappeared after treatment (objective response rate, or ORR). Patients were randomly assigned to receive Pomalyst alone or Pomalyst with low-dose dexamethasone, a corticosteroid.

Results showed 7.4 percent of patients treated with Pomalyst alone achieved ORR. The median duration of response has not yet been reached in these patients. In patients treated with Pomalyst plus low-dose dexamethasone, 29.2 percent achieved ORR with a 7.4-month median duration of response.

The myeloma community is understandably excited; they have a new drug that may help them keep myeloma in check.

However, there are questions that pop up. Why is approval of Pomalyst such a big deal? Why was the drug approved based on a Phase II clinical trial study that showed only 29% partial response rate with a PFS rate of <4 months. Evidently, there are studies with better results…

Dr. Shaji K. Kumar, Professor of Medicine, Hematology, Mayo Clinic, Rochester explains the excitement shared by myeloma researchers and patients alike,

Pomalyst approval is a big deal due to several things,

it is effective in a proportion of patients where Revlimid stops working
it is oral and convenient
it provides another option for the patient who has exhausted other options
it has been approved through the accelerated approval for an unmet need. Hence, the data submitted to FDA represent that with the most refractory population, so it looks like it is worse than the other data out there.

But, what about the pricing, isn’t surviving myeloma becoming an expensive affair with recently approved Kyprolis (carfilzomib) priced at $10,000 per 28-day cycle and Pomalyst to be priced around $10,500 per 28-day cycle.

It is indeed sad that drug prices are where they are and is one of the root reasons for the expensive health care in this country. I see no reason for the high pricing for oncology drugs.

says Dr. Kumar.

Multiple myeloma affects 21,700 Americans every year and this new drug has instilled new hopes in the hearts of many myeloma patients.