Quick Hits: FDA Decisions and FDA Approvals


FDA Decided That Mylotarg, a Leukemia Drug, Should be Made Available Again

An FDA advisory committee has decided that the leukemia drug Mylotarg, which was withdrawn from the market 7 years ago, should be made available again. In June 2010, Pfizer voluntarily withdrew Mylotarg (gemtuzumab ozogamicin) – which was granted accelerated approval by the agency – because a confirmatory clinical trial revealed there was no benefit in survival with the biologic compared to conventional cancer therapies and an increase in toxicities that led to some patient deaths. Under accelerated approval, a drugmaker can use a surrogate or secondary endpoint that is thought to predict benefit, such as a reduction in tumor size, to gain approval. But after approval, the company must conduct another trial to confirm that the drug does demonstrate a clinical benefit, such as longer survival. Since Mylotarg was withdrawn, Pfizer conducted another trial using a smaller dose of the biologic in combination with chemotherapy. Results showed that the combination led to improved event-free survival compared to chemotherapy alone. However, an improvement in overall survival with Mylotarg was not found to be statistically significant. Posted July 12, 2017. Via FDA.

Companies May be Able to Delay Reporting Dangerous Malfunctions to the FDA

Makers of cardiac defibrillators, insulin pumps, breast implants and other medical devices might be able to delay reporting dangerous malfunctions to the FDA under an agreement passed in the House. The provision is part of a larger bill that sets fees device makers have to pay to have their products reviewed by the FDA and also calls for the agency to speed up approvals. The provision would permit companies to report a malfunction within 3 months of being notified, rather than the current standard of within 30 days. It would also allow device makers to summarize a malfunction if it has happened before, rather than write a detailed report every time. However, malfunctions that result in serious injury or death would still have to be reported to the FDA immediately. Posted July 12, 2017. Via Star Tribune.

Endari, New Sickle Cell Disease Drug, Approved by the FDA

The first new drug for sickle cell disease in almost 20 years, Endari (L-glutamine oral powder), was approved by the FDA. Endari is for patients age 5 and older with the rare disease – it impacts about 100,000 patients in the US – to reduce severe complications associated with the blood disorder. The safety and efficacy of the drug were studied over 48 weeks in a randomized trial of patients between 5 and 58 years old with the disease. Patients who were treated with Endari experienced fewer hospital visits for pain and fewer hospitalizations compared to patients who received a placebo. Emmaus Life Sciences, Endari’s developer, said it hopes to have the drug available by the 4th quarter. Posted July 7, 2017. Via FDA.

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