Leukemia Options Begin To Improve For Elderly Patients
Should FDA approval of Mylotarg happen later this year - for the second time - the acute myeloid leukemia space will be in for something of a gold rush. The disease, which typically strikes the elderly, has seen woefully little innovation in decades, but the Pfizer (NYSE:PFE) drug is one of four novel agents being reviewed by regulators this year.
The first, Novartis' (NYSE:NVS) Rydapt, won a green light in the US in April, and the strong backing of an advisory committee for Mylotarg this week makes its return look likely. Considering the unmet need in this disease, Celgene (NASDAQ:CELG) and Jazz (NASDAQ:JAZZ) also have reasons for optimism with their candidates (see table below).
On Tuesday the FDA's oncology committee voted 6-1 in favor of the risk-benefit profile of Mylotarg, largely based on the Alfa-0701 trial. The phase III study, conducted by a French research group, was submitted along with re-analysed data from several other trials in an attempt to get the drug back on the market.
Concerns about toxicity and lack of benefit caused the drug to be pulled in 2010, after 10 years on the market, but Pfizer continued to support research in the interim. Generically known as gemtuzumab ozogamicin, the antibody-drug conjugate targets CD33, which is expressed on a large proportion of AML tumors.
Changes to the chemotherapy dose and scheduling allowed researchers to discover a less toxic way of administering Mylotarg, although Pfizer never managed to prove that it can extend survival, normally a must for a drug proposed for first-line use. Still, reviewers were apparently convinced by the use of event-free survival as the primary endpoint in the Alfa-0701 trial, and approval now looks likely.
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