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AstraZeneca and Merck drug wins approval for rare cancer after failing elsewhere

The Food and Drug Administration approved AstraZeneca and Merck & Co.’s drug selumetinib to treat neurofibromatosis type 1, or NF1, a rare and typically inherited disease that causes tumors to grow inside nerve sheaths.The drug, which will be sold as Koselugo, previously failed to improve survival in patients with lung cancer and melanoma that had spread to the eye. With this approval in NF1, announced Friday, AstraZeneca has won a rare pediatric disease voucher, which it can use to secure priority review for an experimental drug or sell to another company.Koselugo was among the pipeline projects AstraZeneca executives pointed to as future growth drivers when the company’s sales were shrinking following the loss of patent protection for key moneymakers. However, until now the drug has failed to live up to AstraZeneca’s hopes, unlike fellow cancer treatments Tagrisso and Lynparza.

NF1 affects an estimated one in 3,000 to 4,000 people in the U.S. Koselugo will be used to treat symptomatic, inoperable plexiform neurofibromas, which occur in 30% of patients. These are tumors that emerge from multiple nerves and form masses that involve skin and connective tissue.

After first trying to target uveal melanoma, KRAS-mutated non-small cell lung cancer and thyroid cancer, in which Koselugo failed, AstraZeneca and Merck finally got a win in NF1 when researchers behind the SPRINT trial reported that it helped shrink tumors in 33 of 50 patients.

That benefit was balanced by heart, eye, skin and intestinal side effects that occurred in at least 15% of patients in the trial.

Koselugo’s list price will be $12,500 per month for an average patient, although doses vary by weight, an AstraZeneca spokesperson told BioPharma Dive.

Merck joined AstraZeneca in developing and commercializing Koselugo as part of a deal that also included ovarian cancer drug Lynparza. That deal saw Merck pay $1.6 billion upfront and promise total payments of up to $8.5 billion.

Now that Koselugo is approved, AstraZeneca will book all sales and share half of gross profits with Merck.

With such small patient numbers in NF1, Koselugo isn’t likely to achieve the blockbuster numbers of its other new oncology drugs. However, a number of trials, if successful, could expand its uses.

Most significantly, investigator-led trials have paired Koselugo with Tagrisso in lung cancer with another mutation called EGFR, and head-to-head against chemotherapy in a type of brain cancer called a glioma.

The lung cancer trial, at Dana-Farber Cancer Institute, is in a 25-patient Phase 2 trial and might not be persuasive enough, if successful, to gain FDA approval. However, the brain cancer trial, sponsored by the National Cancer Institute, is a 200-patient Phase 3 trial that could be robust enough for the FDA to review.