FDA Approves Return of Previously Withdrawn Cancer Treatment

As expected, the FDA approves Pfizer’s (PFE) MYLOTARG (gemtuzumab ozogamicin) for the treatment of newly diagnosed adult acute myeloid leukemia (AML) patients whose tumors express the CD33 antigen. This week, the FDA approved a pioneering new Novartis AG leukemia treatment that costs $475,000 per patient, which some critics said was excessive but which the company defended as a responsible price in light of the treatment’s benefits.

Mylotarg got accelerated approval in 2000 as a standalone treatment for adult patients with CD33-positive AML who had experienced a relapse, but was voluntarily withdrawn as subsequent confirmatory studies failed to show clinical benefit and had safety concerns, including a high number of deaths. Today’s approval is highlighted by a lower recommended dose, an altered schedule as a stand-alone or in combination with chemotherapy, and a completely different patient population.

“Today is an important day for patients, their families and the entire AML community, as the approval of MYLOTARG brings forth a long-awaited treatment option that may lead to deeper, more durable remissions for patients with AML”, Jorge Cortes, MD, D. B. Lane cancer research distinguished professor for leukemia research at The University of Texas MD Anderson Cancer Center, said in the release.

Drugmakers have taken heat from politicians, patients and health insurers for charging high prices for new drugs and raising prices of older drugs in recent years. The American Cancer Society estimates there are about 21,380 new cases of AML annually in the USA, and about 10,590 deaths from the disease.

After being on the market for 10 years and withdrawn for 7 years, Pfizer’s Mylotarg is set to make its return. The first trial included 237 patients with newly diagnosed AML who could not tolerate or chose not to receive intensive chemotherapy. Mylotarg was evaluated as a stand-alone treatment in two separate trials, one involving newly diagnosed AML patients and one in patients who had experienced a relapse of disease. Patients who received gemtuzumab had a median OS of 4.9 months versus 3.6 months for best supportive care. For those with previous relapse, treatment with Mylotarg resulted in 26% of patients achieving complete remission which lasted a median 11.6 months.

The drug is not without risks, however.

Common side effects of the drug included fever, nausea, infection, vomiting, bleeding, thrombocytopenia, stomatitis, constipation, rash, headache, elevated liver function tests, and neutropenia. Severe side effects of gemtuzumab include low blood counts, infections, liver damage, hepatic veno-occlusive disease, infusion-related reactions, and hemorrhage. Accounting for average annual US medical-price inflation of about 3%, the 2010 price would be about $19,128 in today’s dollars, Pfizer said.

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