AstraZeneca, Merck finally grab a green light for rare-disease med Koselugo

FDA Building 2
The FDA has approved AstraZeneca's Koselugo for tumors associated with a rare genetic condition. (FDA)

It’s been a long road for AstraZeneca’s selumitinib, but one that’s finally led to a green light for the British drugmaker and its partner, Merck.

The FDA approved the drug, to be known commercially as Koselugo, to treat inoperable tumors in children two years and older with the rare neurological disorder neurofibromatosis type 1 (NF1).

The genetic condition affects an estimated 10,000 to 12,000 children in the U.S., and Koselugo will come with a rare-disease list price to match its niche indication.

Its wholesale acquisition cost averages out to about $12,500 per month, Dave Fredrickson, EVP of AstraZeneca’s oncology business unit, said, adding that the company’s goal is for “each eligible commercially insured patient to pay $0 out of pocket.”

Meanwhile, Koselugo’s OK marks a “huge step forward” in therapy options for NF1 sufferers, Fredrickson said, noting that treatment options in the past have been “limited to those that would manage symptoms.” Koselugo, meanwhile, demonstrated in trials that it could shrink tumors and improve children’s quality of life, a performance that snared it an FDA priority review last November.

RELATED: AstraZeneca's selumetinib moves past cancer failures to nab FDA priority review in neurofibromatosis

That win followed a string of stumbles for the med, which AZ grabbed from Array BioPharma—now part of Pfizer—in 2003. Over the years, it’s flopped trials in KRAS mutation-positive non-small cell lung cancer and thyroid cancer, among others, and found itself at the center of a legal battle centered on royalties, too.

Now, though, AstraZeneca and Merck’s eyes are on the future, and to rolling Koselugo out to pediatric patients as fast as possible. To do so, the companies will field a “dedicated small team” to launch the med, whose reps—new hires with experience in rare diseases and, in the case of some, pediatrics—will work only on Koselugo, Fredrickson said.

RELATED: AstraZeneca eyes multibillion-dollar sales bump for lung cancer med Tagrisso with postsurgery trial win

“This is a medicine that the NF1 community has been awaiting now for quite some time, and so our plan was ... in spite of the pandemic, to make sure that we launched as soon as we got FDA approval,” he said. “We know exactly who the important clinicians are to connect with, we know who the right advocacy groups are to connect with. It’s our job to get the news out on this as soon as possible.”

The approval capped a big Friday for AstraZeneca. Earlier in the day the company had revealed that its big-selling EGFR lung cancer med Tagrisso had scored a big win in postsurgery patients—an opportunity analysts say could be worth billions of dollars.

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