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AstraZeneca's Koselugo Gets FDA Nod for Rare Genetic Disorder

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AstraZeneca plc (AZN - Free Report) along with partner Merck (MRK - Free Report) announced that the FDA has approved their MEK 1/2 inhibitor Koselugo (selumetinib) for the treatment of pediatric patients with neurofibromatosis type 1 (NF1)-related plexiform neurofibromas (PN), a rare and debilitating genetic condition.

Following this nod, Koselugo became the first FDA-approved medicine to address this rare and debilitating genetic condition.

The approval was based on positive data from the SPRINT Stratum 1 phase II study, testing Koselugoas, an oral monotherapy in pediatric patients aged two years or older with inoperable NF1-related PN.

Data from the study showed that treatment with Koselugo as a twice-daily oral monotherapy had an overall response rate (ORR) of 66% in the given patient population. Notably, ORR is the percentage of patients with confirmed complete or partial response of at least 20% reduction in tumor volume.

The study was sponsored by the National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP).

In November 2019, the FDA accepted and granted a priority review to the new drug application (NDA) for Koselugo.

A marketing authorization application seeking approval of Koselugo for the same indication is already submitted in Europe.

Notably, AstraZeneca and Merck are jointly developing and commercializing Koselugo globally for multiple cancer types, per a deal signed in July 2017. The company will now record product sales of Koselugo while Merck will book half the profits.

Notably, per the same deal, the companies also share development and commercialization of successful PARP inhibitor, Lynparza.

Shares of AstraZeneca have lost 10.4% so far this year compared with the industry’s decrease of 6.6%.


In a separate press release, AstraZeneca announced that the phase III ADAURA study evaluating its marketed drug Tagrisso (osimertinib) for the adjuvant treatment of early-stage EGFR-mutated non-small cell lung cancer (NSCLC) will be unblinded early. The decision taken by the company followed a recommendation from an Independent Data Monitoring Committee (IDMC) based on an overwhelming efficacy in the study.

The primary endpoint of the above-mentioned study was to evaluate disease-free survival (DFS). The study will continue to assess the secondary endpoint of overall survival. Tagrisso is presently marketed for first-line EGFR mutated advanced NSCLC.

Zacks Rank & Stocks to Consider

AstraZeneca currently carries a Zacks Rank #3 (Hold). Better-ranked stocks in the large cap pharma sector include AbbVie Inc. (ABBV - Free Report) and Bristol-Myers Squibb Company (BMY - Free Report) , both carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

AbbVie’s earnings estimates have moved 1.4% north for 2020 and 0.7% north for 2021 over the past 60 days.

Bristol-Myers’ earnings estimates have been revised 5.9% upward for 2020 and 0.4% upward for 2021 over the past 60 days.

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