Pomalyst (pomalidomide) For Advanced Multiple Myeloma Approved By FDA

Editor's Choice
Main Category: Lymphoma / Leukemia / Myeloma
Also Included In: Blood / Hematology;  Regulatory Affairs / Drug Approvals
Article Date: 11 Feb 2013 - 0:00 PST


Pomalyst (pomalidomide) has been approved by the FDA for the treatment of patients with multiple myeloma whose cancer progressed after being treated with other medications.

Pomalyst, which is presented in capsule form, modulates the immune system so that the patient's own body destroys cancerous cells and undermines their growth. Pomalidomide is aimed at multiple myeloma patients who had been administered at least two previous therapies which did not stop the progression of the disease within two months (relapsed and refractory), including therapies with bortezomib and lenalidomide.

Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research, said:

"Pomalyst is the third drug in a class of immunomodulatory agents that includes lenalidomide and thalidomide, and is the second drug approved in the past year to treat multiple myeloma. Treatment for multiple myeloma is tailored to meet individual patient's needs, and today's approval provides an additional treatment option for patients who have not responded to other drugs."

Kyprolis (carfilzomib), another multiple myeloma drug, was approved by the FDA (Food and Drug Administration) in July 2012. Both Kyprolis and Pomalyst were approved under the FDA's accelerated approval program. In such cases, the drug is approved early and the company that makes and markets it has to carry out additional studies to confirm its clinical efficacy and safety.

Pomalyst was also granted orphan product designation because it is aimed at a rare disease/condition.


Pomalyst(R) brand therapy (Photo: Business Wire)

FDA assessors examined safety and effectiveness data on Pomalyst from a clinical trial involving 221 patients, all with relapsed or refractory multiple myeloma. The trial was designed to measure patients' objective response rate (ORR) - how many cancers disappeared completely or partially after treatment.

The patients were randomly selected into one of two groups:

• The Pomalyst alone group
• The Pomalyst with low-dose dexamethasone group. Dexamethasone is a corticosteroid

The study showed that:

• 7.4% of those in the Pomalyst alone group achieved ORR. There is no median duration of response yet for this group
• 29.2% of those in the Pomalyst plus low-dose dexamethasone group achieved ORR, with a 7.4 month median duration response

A Boxed Warning for Pomalyst (pomalidomide)

A Boxed Warning contained in the Pomalyst packaging alerts doctors and patients that the medication must not be given to pregnant mothers because of the serious risk of life-threatening birth defects (Pomalyst is an analogue of thalidomide), as well as increasing the possibility of blood clots.

The drug is only available through the Pomalyst Risk Evaluation and Mitigation Strategy (REMS) Program because of the potential risk to the unborn child (embryo-fetal risk). Only doctors who are certified with the Pomalyst REMS Program are allowed to prescribe this drug. The patient has to sign a Patient-Doctor agreement form and adhere to the REMS requirements.

If the patient is a non-pregnant woman who is of fertile age, she must comply with the pregnancy testing and contraception requirements. Male patients have to adhere to the contraception requirements.

All pharmacies that dispense Pomalyst need to be certified with the Pomalyst REMS Program - they are only allowed to dispense to patients who are authorized to receive the medication and must comply with REMS requirements. Thalidomide and lenalidomide both have similar REMS.

Side effects related to Pomalyst treatment include neutropenia (reduction in white blood cell count), fatigue/weakness, constipation, diarrhea, anemia (low red blood cell count), fever, back pain, upper respiratory tract infections, and thrombocytopenia (low blood platelet levels).

Pomalyst, lenalidomide and thalidomide are marketed by the Celgene Corporation, New Jersey.
Kyprolis is marketed by Onyx Pharmaceuticals, California.

About Multiple Myeloma

Multiple myeloma, also known as plasma cell myeloma or simply myeloma, is cancer of the plasma cells. Plasma cells are a type of white blood cells which are present in the bone marrow. Plasma cells make immunoglobulin, antibodies which fight off infections.

Myeloma cells (cancerous/malignant plasma cells) multiply and raise the number of plasma cells too excessive levels, this in turn leads to dangerously high levels of immunoglobulin.

Multiple myeloma affects the immune system, bones, red blood cell count, and the kidneys.

Multiple myeloma in the USA - The American Cancer Society says that multiple myeloma affects an estimated 1-to-4 per 100,000 people per year, making it a "relatively uncommon cancer". Over 21,700 new cases are diagnosed and 10,710 die from the disease each year.

The Multiple Myeloma Research Foundation says that the disease represents 1% of all cancers in Caucasian-Americans and 2% in African-Americans.

Multiple myeloma in the UK - Cancer Research UK estimates that nearly 4,000 people are diagnosed with multiple myeloma in the country each year, representing 1% of all UK cancers.

Researchers from Weill Cornell Medical College explained that timing is crucial in multiple myeloma therapy - the treatment needs to occur during a specific moment in the cell's cycle so that the cancer therapies may disable key survival genes, leading to cell death. In the journal Blood, the scientists said that two medications can be used in a "combination punch" (as in boxing) in a series. While PD 033299 (an experimental drug) delivers the first punch, which weakens the multiple myeloma defenses, bortezomib, the second medication, delivers the "knock-out punch".

Written by Christian Nordqvist


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