FDA

FDA approves Pomalyst for advanced multiple myeloma

The U.S. Food and Drug AdministrationThe U.S. Food and Drug Administration has approved Pomalyst (pomalidomide) to treat patients with multiple myeloma whose disease progressed after being treated with other cancer drugs. Multiple myeloma is a form of blood cancer that primarily affects older adults and arises from plasma cells in the bone marrow. According to the National Cancer Institute, approximately 21,700 Americans are diagnosed with multiple myeloma and 10,710 die yearly from the disease.

Pomalyst is a pill that modulates the body’s immune system to destroy cancerous cells and inhibit their growth. It is intended for patients who have received at least two prior therapies, including lenalidomide and bortezomib, and whose disease did not respond to treatment and progressed within 60 days of the last treatment (relapsed and refractory).

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

In July 2012, FDA approved Kyprolis (carfilzomib) to treat multiple myeloma. Similar to Kyprolis, Pomalyst is being approved under the agency's accelerated approval program, which provides patients earlier access to promising new drugs while the company conducts additional studies to confirm the drug's clinical benefit and safe use. The therapy was also granted orphan product designation because it is intended to treat a rare disease or condition.

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.

Pomalyst carries a Boxed Warning alerting patients and health care professionals that the drug should not be used in pregnant women because it can cause severe life-threatening birth defects, and that the drug can cause blood clots.

Because of Pomalyst's embryo-fetal risk, it is available only through the Pomalyst Risk Evaluation and Mitigation Strategy (REMS) Program. Prescribers must be certified with the Pomalyst REMS Program by enrolling and complying with the REMS requirements. Patients must sign a Patient-Physician agreement form and comply with the REMS requirements. In particular, female patients who are not pregnant but can become pregnant must comply with the pregnancy testing and contraception requirements, and males must comply with contraception requirements. Pharmacies must be certified with the Pomalyst REMS Program, must only dispense to patients who are authorized to receive the drug and must comply with REMS requirements. Both lenalidomide and thalidomide have similar REMS.

Common side effects include a decrease in infection-fighting white blood cells (neutropenia), fatigue and weakness, low red blood cell count (anemia), constipation, diarrhea, low levels of platelets in the blood (thrombocytopenia), upper respiratory tract infections, back pain and fever.

Pomalyst, lenalidomide and thalidomide are marketed by Celgene, based in Summit, N.J. Kyprolis is marketed by South San Francisco, Calif.-based Onyx Pharmaceuticals.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Most Popular Now

Pandemic influenza vaccine receives…

AstraZeneca and its global biologics research and development arm, MedImmune, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the Europe...

Read more

NIH launches large clinical trials …

Enrollment has begun in the first of two multinational clinical trials of an intravenously delivered investigational antibody for preventing HIV infection. Known as the A...

Read more

World's first public dengue immuniz…

Sanofi and its vaccines global business unit Sanofi Pasteur announced today that the first public dengue immunization program has started in the Philippines. At a major l...

Read more

Saxenda® demonstrated significant i…

Today, new data from the three-year part of the phase 3a SCALE™ (Satiety and Clinical Adiposity - Liraglutide Evidence) Obesity and Prediabetes trial were presented at th...

Read more

AstraZeneca and Eli Lilly and Compa…

AstraZeneca and Eli Lilly and Company today announced that AMARANTH, a Phase II/III study of AZD3293, an oral beta secretase cleaving enzyme (BACE) inhibitor currently in...

Read more

Vitamin D improves heart function, …

A daily dose of vitamin D3 improves heart function in people with chronic heart failure, a five-year University of Leeds research project has found. Dr Klaus Witte, from ...

Read more

Pfizer announces termination of pro…

Pfizer Inc. (NYSE: PFE) announced that the merger agreement between Pfizer and Allergan plc (NYSE: AGN) has been terminated by mutual agreement of the companies. The deci...

Read more

New analysis shows only minority of…

Boehringer Ingelheim has announced a new publication showing that levels of white blood cells called eosinophils can help identify patients with COPD who may and may not ...

Read more

Researchers open the way to new tre…

In a recent paper published in Nature Communications, a group of Case Western University School of Medicine researchers presented their discovery of the full-length struc...

Read more

Implantable device targets pancreat…

Pancreatic cancer is the third leading cause of cancer deaths in the United States, in part because it is very difficult for chemotherapy drugs to reach the pancreas, whi...

Read more

Physicians' knowledge about FDA app…

Since 2012, the FDA can designate a drug as a "breakthrough therapy" if preliminary clinical evidence - such as an improvement in a pharmacodynamic biomarker - suggests a...

Read more

Global spending on health is expect…

Global inequities in health spending are expected to persist and intensify over the next 25 years, according to a new study that estimates total health financing in count...

Read more

Digest World Pharma Newsletter

Subscribe to our weekly Digest World Pharma Newsletter and stay updated on the latest World Pharma News. Subscribe now, it's free!

Pharmaceutical Companies

[ A | B | C | D | E | F | G | H | I | J | K | L | M | N | O | P | Q | R | S | T | U | V | W | X | Z ]