Pfizer's Mylotarg resurrected for new chance at US market
- Pfizer’s cancer drug Mylotarg (gemtuzumab ozogamicin) got a positive vote from a panel of Food and Drug Administration experts on Tuesday.
- The Oncologic Drug Advisory Committee (ODAC) voted six to one that results from the ALFA-0701 study demonstrated a favorable risk-benefit profile for the drug in patients with newly-diagnosed CD33-positive acute myeloid leukemia (AML).
- The FDA has set a user fee goal date in September to decide about approval of the drug in combination with chemotherapy for both newly-diagnosed, CD33-positive AML patients, as well as a monotherapy for patients in first relapse who are eligible for cytotoxic chemo.
Pfizer’s Mylotarg has a storied past, one of great highs and even lower lows, but the recent vote from the ODAC could mean a new chance at a lucrative market.
Mylotarg was first approved by the FDA in 2000 under its accelerated approval program, which is meant to speed promising drugs to market. It was the first new treatment for AML to come to market in nearly 15 years and was touted as a potential miracle drug.
Accelerated approvals are often based on very strong mid-stage data and have the caveat of the drugmaker proving that very awesome data once again in a late-stage study after the treatment makes it to market. For Mylotarg, accelerated approval was based on three Phase 2 studies that included 142 patients.
As anyone who follows the drug industry knows, Phase 3 studies don’t always replicate their earlier counterparts and many a drug program has been blown up by one of these late-stage failures. Yet, drugs under the accelerated approval program are expected to be so solid that this isn’t supposed to happen.
Things did not work out that way for Mylotarg, which has long stood as a major black eye for Pfizer.
In 2010, Mylotarg was the first drug approved under the accelerated approval program to be pulled from the market. Pfizer voluntarily withdrew the cancer med after the confirmatory study failed to show that the drug improved survival — to make things worse, there was a much higher incidence of patient deaths in the Mylotarg arm of the study due to treatment-related toxicities.
Almost a decade later, Pfizer is seeking approval for the same indication its drug had been originally approved for. But this time, the big drugmaker has brought the FDA a new Phase 3 study with 280 patients, as well as another analysis of nearly 3,000 patients across five Phase 3 studies.
After nearly ten years of pouring over data and new research, researchers have found a more precise patient population that could be genetically predisposed to respond better to the drug. It has also reformulated the drug in a lower dose.
While the FDA is not required to follow the advice of its expert panels, it often does.
- Pfizer Statement
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