Jazz Pharmaceuticals Secures Dual Regulatory Wins for Oncology Drug

Jazz Pharmaceuticals has achieved two significant regulatory milestones for its oncology therapeutic, Modeyso. The company obtained both an accelerated approval for the drug and a valuable Priority Review Voucher from the U.S. Food and Drug Administration (FDA) within a three-week period, marking a substantial achievement for its development pipeline.

The foundation for these successes stems from the company’s strategic acquisition of Chimerix, which was finalized on April 21. Jazz Pharmaceuticals completed the transaction for approximately $935 million in cash, equating to $8.55 per share. The acquisition was largely motivated by Chimerix’s lead clinical candidate, which was undergoing FDA Priority Review at the time and is now known as Modeyso.

First-Ever Approved Systemic Therapy for Rare Brain Cancer

On August 6, the FDA granted accelerated approval to Modeyso (formerly known as dordaviprone) for the treatment of adult and pediatric patients one year of age and older with diffuse midline glioma harboring an H3 K27M mutation. The indication is specifically for patients with progressive disease following prior radiation therapy.

This approval is particularly notable as it represents the first systemic therapy ever approved by the FDA for this aggressive and often fatal form of brain cancer. The regulatory decision was based on an integrated efficacy analysis encompassing 50 patients from five separate open-label clinical studies, which demonstrated:

• An Objective Response Rate (ORR) of 22%
• A median Duration of Response (DOR) of 10.3 months

Acquisition Yields Valuable and Tradable Asset

Should investors sell immediately? Or is it worth buying Chimerix?

Adding to this achievement, the FDA confirmed the issuance of a Rare Pediatric Disease Priority Review Voucher for Modeyso on August 27. These vouchers are highly coveted assets that entitle the holder to secure an expedited six-month review for a subsequent new drug application, as opposed to the standard ten-month review period.

Such vouchers are considered strategic, tradeable commodities within the pharmaceutical industry and have previously been sold to other companies for considerable sums, often exceeding $100 million. Securing this voucher provides Jazz Pharmaceuticals with additional flexibility for its future pipeline development.

Commercial Launch and Future Potential

With its accelerated approval secured, Jazz Pharmaceuticals is now preparing for the commercial launch of Modeyso, which is anticipated to occur in the second half of this year.

Looking ahead, the ongoing Phase 3 ACTION clinical trial has the potential to significantly expand the drug’s addressable market. The study is evaluating Modeyso in patients with newly diagnosed disease, which could lead to a broader label and increased commercial potential.