In a blood cancer win, AstraZeneca nabs FDA nod for rare leukemia drug Lumoxiti

The FDA has approved AstraZeneca's Lumoxiti, a novel antibody drug for relapsed or refractory hairy cell leukemia. (AstraZeneca)

About a year after getting Calquence approved in mantle cell lymphoma, AstraZeneca has pulled off its second blood cancer nod in an antibody drug with peak sales estimated at $500 million.

The FDA approved Lumoxiti, also known as moxetumomab pasudotox, for patients with the rare hairy cell leukemia (HCL) who have undergone at least two prior therapies. It marks the first medicine for the condition in more than 20 years, said AZ’s global head of oncology, Dave Fredrickson.

Prepping for a U.S. launch within this year, AZ is also in talks with the European Medicines Agency, which granted Lumoxiti an orphan drug designation in 2013, a company spokeswoman told FiercePharma.

A Jefferies analyst has forecast Lumoxiti could generate $500 million in peak sales for AstraZeneca. That represents a decent business opportunity, considering HCL accounts for only 3% of all adult leukemias, with about 1,000 new cases diagnosed in the U.S. and 1,600 in Europe each year. While most patients respond to purine analogs including Janssen’s Leustatin (cladribine) and Pfizer’s Nipent (pentostatin), about 30% to 40% would relapse five to 10 years after their first treatment, a population Lumoxiti could soon help.

Lumoxiti attacks the cancer in a novel, two-pronged approach: a CD22-targeting antibody fused with a bacterial toxin. After binding to the cancerous B lymphocyte, the toxin is released to inhibit protein synthesis, causing cell death.

In a phase 3 in 80 patients, Lumoxiti showed a 75% overall response rate and a 30% durable complete response rate, defined as normal blood count for over 180 days. Severe cases of capillary leak syndrome and hemolytic uremic syndrome observed in the study are now included in a boxed warning on the drug’s label.

RELATED: AbbVie, Johnson & Johnson grab another Imbruvica nod with early OK in rare blood cancer

AstraZeneca broke into the blood cancer scene last October, when BTK inhibitor Calquence faced off against Johnson & Johnson and AbbVie’s Imbruvica with an FDA green light in mantle cell lymphoma. The fight will likely spread to a rare disorder called Waldenstrom macroglobulinemia, and more importantly in the much larger market of chronic lymphocytic leukemia (CLL). AZ is now testing Calquence head-to-head against Imbruvica in CLL in a phase 3 that’s slated to report results next year. For the first half of 2018, it turned in $20 million.

Oncology in general represents a bright spot at AZ, thanks to Tagrisso and Lynparza. Sales of lung cancer drug Tagrisso skyrocketed 82% to reach $760 million in the first half of 2018, due to a new nod in the first-line, EGFR-mutated setting. PARP inhibitor Lynparza hauled in $269 million for H1 2018, and is closing in on a boost in first-line ovarian cancer use.