FDA approves first treatment for rare brain tumor in children

Published 06/08/2025, 20:14
FDA approves first treatment for rare brain tumor in children

DUBLIN - The U.S. Food and Drug Administration has granted accelerated approval for Modeyso (dordaviprone) to treat patients with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy, Jazz Pharmaceuticals (NASDAQ:JAZZ) announced Tuesday. The $6.4 billion pharmaceutical company, which maintains impressive gross profit margins of 92%, has received a "GREAT" financial health rating from InvestingPro analysts.

Modeyso is the first FDA-approved treatment for this ultra-rare and aggressive brain tumor that affects approximately 2,000 people annually in the U.S., primarily children and young adults. The oral medication, administered once weekly, is expected to be commercially available in the coming weeks. According to InvestingPro data, the stock appears undervalued based on its Fair Value analysis, with Wall Street analysts maintaining a strong buy consensus.

The FDA’s decision was based on an integrated efficacy analysis of 50 patients with recurrent H3 K27M-mutant diffuse midline glioma across five clinical studies. The overall response rate was 22%, with a median duration of response of 10.3 months. Among responders, 73% maintained their response for at least six months.

"For the first time, we have an FDA-approved therapy for patients with recurrent H3 K27M-mutant diffuse midline glioma," said Patrick Wen, Director of the Center for Neuro-Oncology at Dana-Farber Cancer Institute. "The objective responses observed with dordaviprone, including durable benefit in some patients, represent a meaningful advancement."

The most common adverse reactions in patients receiving Modeyso included fatigue, headache, vomiting, nausea and musculoskeletal pain. Serious adverse reactions occurred in 33% of patients.

The approval applies to adult and pediatric patients 1 year of age and older. Continued approval may be contingent upon verification of clinical benefit in the ongoing Phase 3 ACTION confirmatory trial.

Jazz Pharmaceuticals will host an investor webcast on August 27 to provide an overview of clinical data and commercialization strategy for Modeyso, according to the company’s press release statement. For deeper insights into Jazz Pharmaceuticals’ financial health and growth prospects, InvestingPro subscribers can access comprehensive Pro Research Reports, which include detailed analysis of the company’s valuation, financial metrics, and growth potential.

In other recent news, Jazz Pharmaceuticals reported its second-quarter earnings, which fell short of analyst expectations. The company posted an adjusted loss of $8.25 per share, missing the anticipated loss of $7.62 per share. Despite this, Jazz Pharmaceuticals slightly exceeded revenue estimates, reporting $1.05 billion in revenue compared to the consensus estimate of $1.04 billion. This figure represents a 2.1% increase from the same quarter last year. The earnings miss overshadowed the revenue beat, impacting investor sentiment. These developments reflect the company’s recent financial performance and have drawn attention from market analysts.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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